November 2020

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Amylyx Pharmaceuticals Announces FDA Granted Orphan Drug Designation for AMX0035 for the Treatment of Wolfram Syndrome

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AMX0035 for the treatment of Wolfram syndrome.

“There is a critical unmet need for people living with Wolfram syndrome, a rare genetic disease in which many patients die prematurely with severe neurological disabilities,” said Wolfram syndrome expert Fumihiko Urano, M.D., Ph.D., a Professor of Medicine and of Pathology and Immunology at Washington University School of Medicine in St. Louis. “Endoplasmic reticulum (ER) dysfunction is a critical component of Wolfram syndrome. AMX0035 is designed to target the ER stress, and preclinical data suggest that it may be a promising approach to halt the irreversible progression of optic nerve atrophy in patients with Wolfram syndrome.”

The FDA may grant orphan designation to drugs and biologics intended to treat a rare disease or condition affecting fewer than 200,000 persons in the U.S. Orphan designation qualifies a company for certain benefits, including financial incentives to support clinical development and the potential for seven years of market exclusivity in the U.S. upon regulatory approval.

“We are grateful to the advocacy groups, Dr. Urano and his team, and the dedicated parents who are working with us to determine if AMX0035 can help patients and families affected by Wolfram syndrome,” said Kent Leslie, Global Head of R&D and Chief Scientific Officer at Amylyx. “Orphan drug designation will support us as we explore AMX0035 in treating this rare and underserved population living with a life-threatening disease that has no approved therapies. This designation is an important milestone as we continue to develop AMX0035 for the potential treatment of neurodegenerative diseases. We look forward to continuing our evaluation of AMX0035 in animal models of Wolfram syndrome and, hopefully soon, in patients.”

AMX0035 is currently being investigated in people living with ALS and Alzheimer’s disease.

About Wolfram Syndrome

Wolfram syndrome is an autosomal recessive neurodegenerative disease characterized by childhood-onset diabetes, optic nerve atrophy, and neurodegeneration. Common manifestations of Wolfram syndrome include diabetes mellitus, optic nerve atrophy, central diabetes insipidus, sensorineural deafness, neurogenic bladder, and progressive neurologic difficulties. Genetic and experimental evidence suggest that endoplasmic reticulum (ER) dysfunction is a critical pathogenic component of Wolfram syndrome. The prognosis of Wolfram syndrome is poor, and many patients die prematurely with severe neurological disabilities.

To learn more about Wolfram syndrome, please visit: Ellie White Foundation , Eye Hope Foundation , Snow Foundation , and Wolfram Syndrome Research Alliance .

About AMX0035

AMX0035 is an investigational product designed to reduce neuronal death and dysfunction. AMX0035 targets endoplasmic reticulum and mitochondrial dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases.

About Amylyx Pharmaceuticals

Amylyx Pharmaceuticals, Inc. is a pharmaceutical company working on developing a novel therapeutic for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases. For more information, visit www.amylyx.com and follow us on LinkedIn and Twitter

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InCarda Therapeutics Presents Positive New Data from Phase 2 Study of InRhythm™ in Patients with Paroxysmal Atrial Fibrillation at American Heart Association Scientific Sessions 2020

– Data Demonstrate Achievement of Rapid Conversions from Atrial Fibrillation to Normal Sinus Rhythm
– Study Findings Provide Proof of Concept for First-of-its-Kind Inhaled Antiarrhythmic for Treatment of Paroxysmal Atrial Fibrillation (PAF)
– Provides Roadmap for Start of Pivotal Phase 3 in Medically Supervised Setting in 2021

San Francisco, CA – InCarda Therapeutics, Inc. (“InCarda”), a privately-held biopharmaceutical company developing first-of-their-kind inhaled therapies for cardiovascular diseases today announced the presentation of positive data from the open-label, dose-escalation Part A portion of the company’s multinational INSTANT Phase 2 clinical trial of InRhythm™ (flecainide for inhalation) in patients with recent-onset paroxysmal atrial fibrillation (PAF) at the American Heart Association’s (AHA) Scientific Sessions 2020. The positive study results reported today during the AHA conference provide the first proof of concept for inhaled flecainide as a potentially safe and effective therapeutic option for rapidly converting PAF to normal sinus rhythm (NSR).

InRhythm is a novel inhaled therapeutic candidate designed to rapidly deliver flecainide, a well-established antiarrhythmic agent, to the heart via the lungs to restore NSR and to relieve symptoms associated with acute episodes of PAF. The therapy is being developed initially for its use under medical supervision in a hospital, emergency room or physician office and subsequently as a portable treatment that can be self-administered by patients in a non-medically supervised setting (such as the home) to rapidly achieve conversion of PAF to NSR.

Dr. Harry J.G.M. Crijns, professor, chair of cardiology and board member of the Cardiovascular Research Institute Maastricht (CARIM) at Maastricht UMC+, and co-principal investigator of the INSTANT trial, presented data from Part A of the INSTANT trial. The results demonstrated the rapid achievement of therapeutic plasma levels (Cmax > 200 ng/mL) of flecainide via oral inhalation with InRhythm. Approximately 80% of patients who received the study’s highest administered dose (120 mg) achieved a flecainide Cmax > 200 ng/mL. For all study participants who reached these therapeutic plasma levels, nearly 50 percent achieved a successful conversion from PAF to NSR. Importantly, those successful conversions occurred rapidly, with a median time to conversion of 3.6 minutes after the end of the administration of InRhythm (total inhalation time for InRhythm administration is eight minutes). The treatment was shown to be safe and well-tolerated, and the majority of adverse events were transient, mild in severity and resolved without treatment.

“There is growing clinical and scientific evidence to support the importance of rhythm control in the overall management of atrial fibrillation,” said Jeremy N. Ruskin, M.D., professor of medicine at Harvard Medical School and founder and director emeritus of the Cardiac Arrhythmia Service at Massachusetts General Hospital and co-principal investigator of the INSTANT study. “Currently available treatment options for the acute conversion of atrial fibrillation to normal sinus rhythm are limited and do not fully address the unmet needs of improving patients’ symptoms and quality of life and reducing healthcare costs. This novel therapy for rapid restoration of normal rhythm, if successful in pivotal clinical trials, has the potential to play a significant role in addressing these unmet clinical needs.”

Based on these positive study results, InCarda has commenced enrollment of Part B of the INSTANT study which includes a confirmatory cohort using the selected optimal therapeutic dose (120 mg) and a pilot study referred to as the “Patient-led Sub-study.” In this latter study patients who have already experienced a safe cardioversion with InRhythm will receive training so that they can self-administer InRhythm under medical supervision when another (“recurrent”) episode of PAF occurs. In addition to current study sites in the Netherlands and Belgium, InCarda will start recruiting patients in the US later this year.

“The data presented today from the INSTANT study provide the first proof of concept of this novel strategy to deliver flecainide via oral inhalation to rapidly restore NSR in patients with symptomatic episodes of recent-onset PAF. These data will be used to finalize the design of a multinational, Phase 3 trial of InRhythm to support marketing approval for in-hospital, medically supervised use. We expect to initiate this study in the first half of 2021,” said Luiz Belardinelli, M.D., chief medical officer of InCarda.

“With six million atrial fibrillation patients in the US contributing to over $26B in annual healthcare expenditures, and more than 30 million atrial fibrillation patients worldwide, there is a tremendous opportunity for InCarda to uniquely address this important and growing need,” said Grace E. Colon, Ph.D., chief executive officer of InCarda. “We look forward to the next steps in the development of this important new therapy and reporting on several key company milestones over the next year.”

About Atrial Fibrillation (AF)

Atrial fibrillation (AF) is the most common sustained cardiac arrhythmia (abnormal heart rhythm) and is characterized by rapid and irregular heartbeats often resulting in palpitations and other symptoms that can be debilitating. A chronic, progressive condition, AF is estimated to affect six million people in the U.S., with that number expected to double by 20501. This expected increase is partially due to the correlation between AF prevalence and an aging population, with approximately 9% of those aged 65 and older affected by the condition1. AF is associated with significant morbidity and a substantial reduction in quality of life, with the condition potentially resulting in exercise intolerance, congestive heart failure, tachycardia-induced cardiomyopathy and stroke. The annual cost of AF to the U.S. healthcare system is estimated at more than $26 billion1.

Paroxysmal AF (PAF) is a type of AF in which episodes occur intermittently and resolve spontaneously in fewer than seven days. Approximately 25% of PAF patients progress to the permanent form of AF within five years2. Common symptoms of PAF can include racing heartbeat, chest pain or pressure, a fluttering feeling in the chest, weakness, fatigue, dizziness, sweating and lightheadedness. Current treatments for patients with PAF rely upon either chronic administration of oral antiarrhythmic drugs or acute hospital-based procedures such as intravenous drug administration and electrical cardioversion, neither of which fully address the unmet need of patients for a rapid-acting treatment that can be administered whenever an episode of PAF occurs. There are currently no approved treatments that can be patient self-administered whenever an episode of PAF occurs.

About InRhythm™

InRhythm (flecainide for inhalation) is a novel inhaled therapeutic candidate designed to rapidly deliver flecainide, a well-established antiarrhythmic agent, to the heart via the lungs, to restore normal sinus rhythm (NSR) and relieve the patient’s symptoms following the onset of an episode of PAF. InRhythm is intended to address the unmet need for a non-invasive, rapid-acting treatment that can be administered in a medically supervised setting (initial indication) and, ultimately, self-administered by patients anywhere they happen to be, whenever they experience an episode of PAF. Phase 1 clinical results in healthy volunteers demonstrated that InRhythm rapidly and safely delivered flecainide resulting in ECG changes consistent with the potential to restore NSR in patients with PAF. InCarda is currently conducting the INSTANT Phase 2 trial of InRhythm in patients with recent-onset PAF. InRhythm represents a first-in-class, multi-billion dollar global opportunity to address a significant unmet medical need.

About InCarda Therapeutics

InCarda Therapeutics, Inc. is a privately-held, clinical-stage biopharmaceutical company developing first-of-their-kind inhaled therapies for acute cardiovascular diseases and conditions. The company is leveraging the ability of inhaled therapy to deliver medicine in the “first pass” to cardiac tissue, presenting a small, but effective dose of drug directly to affected regions of the heart. This permits rapid-onset, lower off-target tissue exposure of the drug, lower exposure to cardiac tissue and, more importantly, has the potential to be patient self-administered in a non-medical setting (e.g., home). InCarda employs a de-risked approach by using approved drugs with a long history of efficacy and safety as candidates for the new dosing paradigm via inhalation. The company’s lead development product, InRhythm, is in Phase 2 development to treat acute episodes of PAF, a prevalent atrial arrhythmia. For more information, please visit: www.incardatherapeutics.com.

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United States Patent for Oximetry Probe with Electronically Selectable Tissue Depth Analysis Issued to Vioptix Inc

Alexandria–(Global IP News)–United States Patent for oximetry probe with electronically selectable tissue depth analysis has been issued to Vioptix Inc. This invention was developed by Bechtel Kate Leeann, Keating Jennifer Elizabeth and Coleridge Scott. The patent application number is 15/493,111.

Global Partnership to Introduce a Connected, Point-of-Care Diagnostic Platform for COVID-19 Antigen Testing in Africa

LONDON–(PRNewswire)– LumiraDx, the next generation point-of-care diagnostic company, announced today a $32 million global partnership with Africa Union, Africa Centres for Disease Control and Prevention (Africa CDC), Africa Medical Supplies Platform (AMSP), Bill and Melinda Gates Foundation (BMGF), and Clinton Health Access Initiative (CHAI) with the support of the COVID-19 Therapeutics Accelerator, The Mastercard Foundation and The Rockefeller Foundation to expand access to fast, accurate and equitable COVID-19 testing in Africa.

With Foundations’ support, LumiraDx is providing five thousand portable diagnostic instruments and related COVID-19 antigen tests, while AMSP – the single-source platform enabling faster, more transparent and cost-effective access to COVID-19-related critical diagnostics and medical equipment – will supply it equitably across 55 African Union member states, addressing both the immediate critical need for COVID-19 testing and building the foundation for long-term primary care infrastructure.

“LumiraDx is committed to meeting the global challenge of delivering fast, accurate, and equitable diagnostic testing for COVID-19 and beyond,” said LumiraDx CEO Ron Zwanziger. “We’re proud to work with global health NGOs and government leaders to make our high-sensitivity microfluidic based COVID-19 antigen test available across Africa.”

In studies, the LumiraDx SARS-CoV-2 antigen test demonstrated 97.6% positive agreement versus PCR in patients tested up to 12 days after the onset of symptoms, allowing patients in remote areas a wide window in which to get high sensitivity diagnostic test results. The LumiraDx Platform offers secure, cloud-based connectivity via cell phone, enabling real-time reporting and management of results. Together, the LumiraDx Platform offers a high-quality, connected COVID-19 testing solution while setting up capabilities for broader point-of-care testing in community-care settings in Africa.

“Testing technology that offers results in minutes – not days or weeks – is an important part of the African Union’s Partnership to Accelerate COVID-19 Testing (PACT) initiative,” said Africa CDC Executive Director Dr. John Nkengasong. “We look forward to working together with our global health partners to mobilize experts, community workers, supplies and other resources to minimize the impact of the pandemic on the African continent by testing, tracing, and treating COVID-19 cases in a timely manner.”

“Through this partnership, our African Union Member States will have access to high-sensitivity point-of-care COVID-19 antigen tests on the AMSP,” said African Union Special Envoy Strive Masiyiwa. “The platform was created to bring manufacturers and suppliers together to collectively address the quality, affordability and accessibility challenges facing our communities during the COVID-19 crisis.”

“We have to double down our efforts and make sure Africa has access to the latest and most performant technologies to protect our people and reopen our economies. The AMSP team has been working relentlessly to launch its COVID-19 antigen offering and we are proud to partner with LumiraDx, Bill & Melinda Gates Foundation, Mastercard Foundation and others to achieve this goal,” said Fatoumata Bâ, Founder & Executive Chair of Janngo and Managing Partner of Janngo Capital.

“We cannot fight COVID-19 without understanding where the disease is spreading. Access to diagnostics are vital in providing data to equip governments and health professionals to take measures to slow transmission. We are pleased to be working with partners to improve COVID-19 diagnostics and improve primary care capabilities in Africa,” said Gates Foundation President of Global Health, Trevor Mundel.

About LumiraDx

LumiraDx was founded in 2014 by a group of entrepreneurs: Ron Zwanziger, our Chairman and Chief Executive Officer; Dave Scott, Ph.D., our Chief Technology Officer; and Jerry McAleer, Ph.D., our Chief Scientist, who have a successful track record in building and scaling diagnostics businesses over three decades, including at companies such as Medisense, Inc., Inverness Medical Technology Inc. and Alere Inc. The company is supported by institutional and strategic investors including the Bill & Melinda Gates Foundation, Morningside Ventures and U.S. Boston Capital Corporation. Based in the UK and supported by its worldwide affiliates to provide access in all major markets, LumiraDx has over 750 employees worldwide.

LumiraDx’s Platform simplifies, scales down, and integrates techniques used in laboratory analyzers to provide lab-comparable diagnostic tests on a single point-of-care instrument that can be easily used in community care settings. It’s able to deliver a broad menu of tests on a single instrument and guarantees high-quality results at low cost, making it an ideal resource for developing communities as they establish local health care infrastructure.

Further information on LumiraDx and the LumiraDx Platform is available at lumiradx.com.

Apnimed Further Strengthens Leadership Team and Board of Directors

– Seasoned finance executive Michael Rogers joins as company’s Chief Financial Officer
– Former Biotech CEO and senior Merck executive James (Jay) Galeota joins Board of Directors

CAMBRIDGE, Mass. – Apnimed, a clinical-stage pharmaceutical company focused on developing medicines to treat sleep apnea and related disorders, today announced it has added two highly experienced executives to help lead the company. Michael Rogers joins Apnimed as Chief Financial Officer and brings more than 25 years of biotechnology experience in both private and public finance management. Additionally, Jay Galeota joins the company’s Board of Directors, and brings more than 30 years of commercial, operational, and company building experience in large pharmaceutical, specialty generics, and biotechnology companies to Apnimed’s Board of Directors.

“We are at an exciting inflection point in Apnimed’s development as we have initiated Phase 2 clinical studies with our lead product, AD109, for the treatment of Obstructive Sleep Apnea,” said Larry Miller, M.D., Chief Executive Officer of Apnimed. “Adding Mike to our leadership team and Jay to our Board will bring valuable expertise and insight as we continue to build a leading biotech company to develop the first pharmacologic, disease-modifying treatment for the estimated 25 million Americans who suffer from sleep disorders.”

Prior to joining Apnimed as Chief Financial Officer (CFO), Michael Rogers was CFO at Aerpio Pharmaceuticals, Inc., which was focused on advancing first-in-class compounds to treat ocular disease and complications of diabetes. Mr. Rogers has been CFO for numerous companies including Acorda Therapeutics, Inc., BG Medicine, Inc., Indevus Pharmaceuticals, Inc., Advanced Health Corporation, and Autoimmune Inc. Mr. Rogers has organized and led numerous successful financing rounds for both pre-commercial and commercial, and privately and publicly traded companies. He received his B.A. from Union College and his M.B.A. from Darden Graduate School of Business Administration at the University of Virginia.

“For years, patients with OSA have been limited to cumbersome or invasive treatment options,” said Michael Rogers, newly appointed Chief Financial Officer at Apnimed. “Apnimed is working on an elegant solution for patients with a once-daily, oral treatment. I look forward to working with the team to help complete a robust set of late-stage trials on an ambitious timeline to fully demonstrate the value of this potentially significant advance.”

Jay Galeota was the President and Chief Executive Officer of Inheris Biopharma, Inc., where he transformed the Necktar spinout, helping to build a new, independent pharmaceutical company in less than a year. Prior to Inheris, he was President of G&W Laboratories, Inc. where he oversaw business operations including research and development, commercial, manufacturing, business development, quality, supply chain, information technology, and corporate support functions. Mr. Galeota spent nearly 30 years with Merck & Co. where he held positions of increasing seniority and oversaw the global development and commercialization for multiple therapeutic areas, such as those in the Diabetes and Obesity Franchise. He was also the President of Merck’s Hospital and Specialty Care business line. He completed his career at Merck as Chief Strategy and Business Development officer and President of Emerging Business for the company. He received his B.S. in Biology from Villanova University, is a graduate of Harvard Business School’s Advanced Management Program, and completed Officer Field Training for the United States Air Force, Reserve Officer Training Corps. Mr. Galeota is also a member of Boards of Directors and Committees for multiple public and privately held companies.

Jay Galeota, newly appointed Apnimed Board Member, said “Throughout my career I have had the privilege to help develop and commercialize some highly innovative and life-changing medicines for patients. I believe Apnimed’s approach to disease-modifying therapies that address the underlying needs of patients with Obstructive Sleep Apnea and other sleep disorders, could be game changing for the sleep field. I am excited to bring my expertise in managing late-stage clinical and commercial development to the Board.”

About Apnimed’s Lead Product – AD109
Apnimed’s lead product candidate, AD109, facilitates the activation of the upper airway dilator muscles to maintain an open airway during sleep. AD109 is a first-in-class, oral pharmaceutical combination dosed once-daily at bedtime, designed to treat OSA patients across a broad spectrum of disease severity. There are currently no medicines to treat the underlying disordered breathing associated with OSA. The drug is designed to be safe, effective, and convenient, addressing the key limitations of the current devices used to treat OSA.

About Obstructive Sleep Apnea
Obstructive Sleep Apnea (OSA) is one of the most common and serious sleep disorders and is estimated to affect more than 25 million Americans, though underdiagnosis continues to be a serious problem. OSA is characterized by partial or complete upper airway closure that occurs during sleep, which often leads to poor sleep, and in the long-term, hypertension, diabetes, cardiovascular disease, strokes, and early mortality. The vast majority of diagnosed patients are prescribed positive air pressure therapy devices such as continuous positive airway pressure, or CPAP. Fewer than half of all CPAP patients are compliant long-term, leaving a significant population untreated, undertreated and at risk.

About Apnimed
Apnimed is a clinical-stage pharmaceutical company working to transform the treatment of sleep apnea based on a simple idea – patients with Obstructive Sleep Apnea will benefit from treatment with a safe and effective oral medication. Apnimed’s lead development program targets the neurologic control of upper airway muscles to maintain an open airway during sleep. The drug is delivered as a convenient once-daily at bedtime. Based in Cambridge, Mass., the company is developing a portfolio of novel pharmacologic therapies for sleep apnea and related disorders. Learn more at Apnimed.com

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Vigeo Therapeutics to Present New Phase 1/2 VT1021 Dose Escalation and Expansion Data at the Society for Immunotherapy of Cancer’s 2020 Annual Meeting

– 75% of Patients Who Achieved a Partial Response or Stable Disease Had High Expression of Both CD47 and CD36
– Dose Escalation Complete; Single-Agent VT1021 Demonstrating Favorable Safety Profile and Early Signals of Clinical Activity

CAMBRIDGE, Mass.–(PRNewswire)–Vigeo Therapeutics, a clinical-stage immuno-oncology company pioneering novel cancer therapies, today announced that interim clinical data from its ongoing Phase 1/2 study evaluating single-agent activities of VT1021 in patients with advanced solid tumors, will be presented in a poster session at the Society for Immunotherapy of Cancer’s (SITC) 2020 Annual Meeting, taking place from November 9-14, 2020.

VT1021 is a first-in-class, dual-modulating compound that blocks the CD47 immune checkpoint and activates CD36, stimulating cytotoxic T-cell functions, inducing apoptosis in tumor and endothelial cells, and increasing the phagocytosis of the tumor by M1 macrophages by stimulating the production of thrombospondin-1 (Tsp-1). Vigeo is developing VT1021 as a therapeutic agent across a range of cancers, with a current focus on solid tumors.

“The data being presented at SITC are important because they demonstrate that VT1021 as a single-agent has a favorable safety profile, and is showing early signals of clinical activity across a wide variety of solid tumors, including pancreatic cancer and glioblastoma,” said Dr. Devalingam Mahalingam, Associate Professor of Medicine at Northwestern University Feinberg School of Medicine, member of the Robert H. Lurie Comprehensive Cancer Center of Northwestern University and lead author of the study. “Notably, in the dose escalation cohort we observed a durable partial response rate in a patient with thymoma and a 43% disease control rate in subjects with tumors expressing both high CD47 and high CD36, which speaks to the potential of this dual-modulating agent in subsets of patients with advanced chemo-refractory solid tumors.”

Lou Vaickus, MD, FACP, interim Chief Medical Officer at Vigeo, stated, “Patients who have high levels of either CD36 or CD47 have a worse prognosis and a greater likelihood of developing resistance to conventional therapies, and currently there are no therapeutics that simultaneously target both receptors. VT1021 is unique because it stimulates the expression of Tsp-1 which then binds with high affinity to its natural receptors CD47 and CD36, blocking CD47 and the ‘do not eat me’ signal and activating CD36, leading to a cascade of beneficial changes to the TME that are believed to enhance anti-tumor effects. We are highly encouraged by this new data and look forward to further elucidating the clinical activity of VT1021 in the dose expansion portion of this study and in combination studies slated to begin during the first half of 2021.”

Key Findings

– VT1021 was found to be safe and well tolerated across all doses tested.
– PK analysis has demonstrated dose proportionality across all dose levels.
– Dose escalation cohort showed preliminary efficacy: Of 28 evaluable patients, one showed partial response (PR) and 11 others achieved stable disease (SD) in 9 different solid tumors with a median of 4.5 prior lines of therapy for a disease control rate of 43%.
– Of the 12 patients who achieved PR/SD’s in the dose escalation cohort, 9 (75%) have showed high CD36 and high CD47 expression as determined by an immunohistochemical (IHC) assay.
– The RP2D of VT1021 has been determined as 11.8mg/kg.

Details for the SITC 2020 Presentation

Title: A first-in-human Phase 1/2 open label trial evaluating the safety, pharmacology, and preliminary efficacy of VT1021 in subjects with advanced solid tumors
Presenter: Devalingam Mahalingam, Associate Professor of Medicine, Feinberg School of Medicine, Northwestern University
Session Type: Poster Session
Abstract Number: 374
Date and Time: Thursday, November 12 from 4:50–5:20 p.m. EST and Saturday, November 14 from 1–1:30 p.m. EST

About VT1021

Vigeo’s lead asset, VT1021, is a first-in-class dual modulating compound that blocks the CD47 immune checkpoint and activates CD36, which induces apoptosis and increases the M1:M2 macrophage ratio. VT1021 achieves this through stimulation of thrombospondin-1 (Tsp-1). The goal of these dual-modulating effects is conversion of immuno-suppressive, or “cold,” tumors that don’t respond to immuno-oncology agents, to immuno-stimulated, or “hot,” tumors that are potentially more receptive to immuno-oncology agents. Vigeo is developing VT1021 as a therapeutic agent across a range of cancers, with a current focus on solid tumors. Pre-clinical results have demonstrated that single-agent VT1021 causes tumor regression at both the primary and metastatic sites.

Single-agent VT1021 is currently being evaluated in a Phase 1/2, open label, multicenter trial (NCT03364400) that assesses the drug’s safety, tolerability, and preliminary anti-tumor efficacy. The trial’s dose escalation phase is now concluded, and the dose expansion phase has been initiated. The expansion phase is expected to enroll 75-100 `patients into one of three cohorts: pancreatic cancer, glioblastoma, and a basket cohort with cancers expressing both high CD47 and high CD36.

About Vigeo Therapeutics

Based in Cambridge, MA, Vigeo Therapeutics is a clinical-stage immuno-oncology company pioneering novel cancer therapies. The company is building a first-in-class drug development pipeline being led by VT1021, its dual-modulating compound that blocks the CD47 immune checkpoint and activates CD36. Single-agent VT1021 is currently being evaluated in a Phase 1/2 clinical trial, and the company expects to initiate combination studies during the first half of 2021. For more information visit vigeotherapeutics.com.

PhoreMost and Oxford Biomedica Enter Gene Therapy Discovery Collaboration

– Collaboration to develop next-generation CAR-T cell therapies with improved efficacy and durability
– PhoreMost’s SITESEEKER platform will identify active peptides to be deployed within Oxford Biomedica’s LentiVector delivery system

Cambridge and Oxford, UK — PhoreMost Limited, the UK-based biopharmaceutical company dedicated to developing drugs against intractable disease targets, and Oxford Biomedica (UK) Ltd plc (LSE:OXB) (“Oxford Biomedica”), a leading gene and cell therapy group, today announced that they have entered into a discovery collaboration to develop next generation CAR-T cell therapies. Financial details of the agreement are not disclosed.

PhoreMost will deploy its in-house expertise and next-generation phenotypic screening platform, SITESEEKER®, to identify therapeutic candidates for Oxford Biomedica’s LentiVector® gene therapy delivery system. The programme will initially focus on CAR-T therapy and aims to develop next generation cell therapies with significantly improved efficacy and durability.

“This collaboration with Oxford Biomedica, a global pioneer in cell and gene therapies, is further recognition of the power of SITESEEKER, offering an exciting opportunity to discover and accelerate the development of clinical stage products. The natural complementarity between SITESEEKER and LentiVector offers great promise for this and future collaborations between the two companies.” DR CHRIS TORRANCE, PHOREMOST CEO

“We are excited to apply this next-generation technology to our LentiVector platform. The collaboration has the potential to deliver more effective CAR-T therapies, and we look forward to working closely with the PhoreMost team.” JOHN DAWSON, OXFORD BIOMEDICA CEO

SITESEEKER exploits protein shape diversity to find functionally active peptides linked to any chosen disease setting, significantly enhancing the power of phenotypic screening and translation into therapeutic modalities. Based on proprietary protein interference, or ‘PROTEINi®’, technology, SITESEEKER is able to systematically probe the entire proteome in a live cell environment to identity and exploit novel drug targets.

Oxford Biomedica is a world-leading pioneer of cell and gene therapies. Its LentiVector platform enables the successful development of breakthrough gene and cell-based medicines, and through collaborations with pharmaceutical partners, has delivered the first FDA and EMA approved CAR-T cell therapy.

Amylyx Pharmaceuticals: Last Patient Wraps Up Final Study Visit in PEGASUS Trial of AMX0035 in Alzheimer’s Disease

– Topline Results Expected in the First Half of 2021

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, today announced that the last participant has completed the planned 24 weeks in the Phase II PEGASUS trial assessing the safety and biological activity of AMX0035 administration in people with Alzheimer’s disease.

“Alzheimer’s is a complex, progressive brain disease defined by amyloid and tau pathologies, but we now realize that there are multiple pathways driving the disease,” said Steven E. Arnold, M.D., Professor and Translational Neurology Head of the Interdisciplinary Brain Center at Massachusetts General Hospital and Harvard Medical School and the PEGASUS trial principal investigator. “The PEGASUS trial design and broad inclusion criteria will allow us to assess whether AMX0035 can prevent neurodegeneration in people with Alzheimer’s via MR imaging and fluid biomarkers. We are immensely thankful to the trial participants and their families for their time and commitment to this critical research effort.”

In June 2020, Amylyx announced the PEGASUS trial completed enrollment and dosed 96 participants. Following brief disruptions due to the coronavirus pandemic, including trial site closures, the trial team worked to prioritize patient safety and mitigate delays by capping trial enrollment four patients shy of the planned 100. Amylyx worked with site investigators and clinic teams to facilitate remote visits and expand visit windows when possible. Topline results are targeted for the first half of 2021.

The PEGASUS trial will provide data on the safety and tolerability profile of AMX0035 in people with Alzheimer’s disease, its biological activity on biomarkers related to disease processes and preliminary information on cognitive and functional effects of AMX0035.

Alzheimer’s disease affects memory, thinking and behavior. Symptoms eventually become severe enough to interfere with daily tasks. Alzheimer’s is a slow but relentlessly progressive disease. On average, a person with Alzheimer’s lives four to eight years after diagnosis, but can live up to 20 years, depending on other factors. Alzheimer’s disease is the sixth leading cause of death in the United States.

“Worldwide, 50 million people are awaiting new treatments for Alzheimer’s disease, one of the most critically unmet healthcare needs today,” said Dr. Rudolph Tanzi, Ph.D., Kennedy Professor of Neurology, Harvard Medical School, Vice-Chair of Neurology, Massachusetts General Hospital, Founding Chair of the Amylyx Scientific Advisory Board. “The PEGASUS trial will assess the impact of investigational product AMX0035 on biomarkers in people with Alzheimer’s and hopefully lead to a future treatment. We are greatly looking forward to the data in the first half of 2021.”

PEGASUS was supported by the Alzheimer’s Combination Therapy Opportunities (ACTO) program, a joint research funding initiative supported by the Alzheimer’s Association and the Alzheimer’s Drug Discovery Foundation, and Cure’s Alzheimer’s Fund.

“We feel tremendous responsibility to all those living with Alzheimer’s disease, their families and loved ones,” said Kent Leslie, Chief Scientific Officer at Amylyx. “Amylyx is committed to rigorous scientific research to improve people’s health, and we thank all patients, investigators, site teams, the Alzheimer’s Association, the Alzheimer’s Drug Discovery Foundation and Cure Alzheimer’s Fund for their contributions to the PEGASUS study milestone. We look forward to sharing the topline results of the trial in the first half of 2021.”

About PEGASUS

PEGASUS (NCT03533257) is a 3:2 randomized, double-blind, multi-center, placebo-controlled study evaluating the safety, tolerability and activity of AMX0035 in patients with late mild cognitive impairment or early dementia due to Alzheimer’s disease over 24 weeks. PEGASUS is designed to yield deep phenotyping insight for the purposes of demonstrating the effects of AMX0035 on mechanistic targets of engagement and disease biology. The study will evaluate diverse disease-relevant markers and produce an informative dataset that will allow for evaluation and correlation of imaging-based markers, neurobiological changes, functional measures, and cognitive outcomes.

About AMX0035

AMX0035 is an investigational product designed to reduce neuronal death and dysfunction. AMX0035 targets endoplasmic reticulum and mitochondrial dependent neuronal degeneration pathways in amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases.

About Amylyx Pharmaceuticals

Amylyx Pharmaceuticals, Inc. is a pharmaceutical company working on developing a novel therapeutic for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases. For more information, visit www.amylyx.com and follow us on LinkedIn and Twitter.

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Apellis to Present New Data Supporting the Efficacy and Safety of Pegcetacoplan in PNH at the American Society of Hematology Annual Meeting

– Eight abstracts accepted for presentation emphasize the potential of targeted C3 therapy to elevate the standard of care in paroxysmal nocturnal hemoglobinuria (PNH)

– New analyses from the Phase 3 PEGASUS head-to-head study demonstrate greater treatment response and quality-of-life improvements with pegcetacoplan versus eculizumab, a C5 inhibitor

– Using a matching-adjusted indirect comparison (MAIC), improvements in clinical, hematological and quality-of-life outcomes were demonstrated in patients treated with pegcetacoplan compared to ravulizumab, a long-acting C5 inhibitor

WALTHAM, Mass.–(GLOBE NEWSWIRE)–Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that eight abstracts were accepted for presentation at the virtual American Society of Hematology (ASH) Annual Meeting to be held December 5-8, 2020. Data support the positive efficacy and safety of pegcetacoplan, a targeted C3 therapy, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

Highlights include new analyses from the Phase 3 head-to-head PEGASUS study, which demonstrate a markedly greater proportion of patients achieved better hematological responses as well as quality-of-life improvements with pegcetacoplan versus eculizumab, a C5 inhibitor. Additionally, using a matching-adjusted indirect comparison (MAIC) methodology, patients treated with pegcetacoplan enrolled in the PEGASUS study experienced greater improvements in hemoglobin stabilization, transfusion avoidance and fatigue compared to patients treated with ravulizumab, a long-acting C5 inhibitor, using the data from the ALXN1210-PNH-302 published study. In the absence of a clinical head-to-head study, MAIC is a valid and accepted method for comparative effectiveness research used by health technology assessment bodies across the world.1,2

“We continue to see in the analyses that pegcetacoplan demonstrated a substantial improvement over C5 inhibition,” said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. “The breadth of data that we are presenting at ASH this year emphasizes the potential of pegcetacoplan to elevate the standard of care in PNH.”

Accepted abstracts regarding pegcetacoplan include:

– Results of the PEGASUS Phase 3 Randomized Trial Demonstrating Superiority of the C3 Inhibitor, Pegcetacoplan, Compared to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria – #2579 – December 7, 7:00 a.m. – 3:00 p.m. PT
– Categorized Hematologic Response to Pegcetacoplan Versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria: Post Hoc Analysis of Data from a Phase 3 Randomized Trial (PEGASUS) – #2588 – December 7, 7:00 a.m. – 3:00 p.m. PT
– Effect of Pegcetacoplan on Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria from the PEGASUS Phase 3 Trial Comparing Pegcetacoplan to Eculizumab – #764 – December 5, 7:00 a.m. – 3:30 p.m. PT
– Pegcetacoplan is Superior to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Regardless of Prior Transfusion Requirement – #1681 – December 6, 7:00 a.m. – 3:30 p.m. PT
– C3 Inhibition with Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria: Results from the PADDOCK and PALOMINO Trials – #753 – December 5, 7:00 a.m. – 3:30 p.m. PT
– Comparative Effectiveness of Pegcetacoplan Versus Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Previously Treated with Eculizumab: A Matching-Adjusted Indirect Comparison – #2581 – December 7, 7:00 a.m. – 3:00 p.m. PT

Accepted abstracts regarding additional data in the PNH population include:

– Real-World Eculizumab Dosing Patterns among Patients with Paroxysmal Nocturnal Hemoglobinuria in a US Population – #3412 – December 7, 7:00 a.m. – 3:00 p.m. PT
– Real-World Treatment Patterns and Healthcare Resource Utilization (HRU) of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Receiving Eculizumab in a US Population – #3415 – December 7, 7:00 a.m. – 3:00 p.m. PT

About the PEGASUS Study

The PEGASUS study (APL2-302; NCT03500549) is a multi-center, randomized, active-comparator controlled Phase 3 study in 80 adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of pegcetacoplan compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a hemoglobin level of <10.5 g/dL at the screening visit. During the four-week run-in, patients were dosed with 1080 mg of pegcetacoplan twice weekly (n=41) in addition to their current dose of eculizumab. During the 16-week randomized, controlled period, patients were randomized to receive either 1080 mg of pegcetacoplan twice weekly or their current dose of eculizumab (n=39). All participants completing the randomized controlled period entered the open-label pegcetacoplan treatment period. About the Matching-Adjusted Indirect Comparison Analysis

Using a matching-adjusted indirect comparison (MAIC) methodology, individual patient data from the PEGASUS study were compared to aggregate, published results from the ALXN1210-PNH-302 study, which compared ravulizumab and eculizumab among patients with PNH who previously were treated with eculizumab. To adjust for cross-study differences in baseline characteristics, propensity score weighting was used to balance demographic and clinical characteristics. Outcomes assessed from the PEGASUS study at week 16 and the ALXN1210-PNH-302 study at week 26 included transfusion avoidance, number of units transfused, hemoglobin stabilization and change in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score. As with other MAIC analyses, matching may not adjust for all confounding factors due to differences inherent in study design and entry criteria.

About Pegcetacoplan (APL-2)

Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and the treatment of geographic atrophy, and received orphan drug designation for the treatment of C3G by the FDA and European Medicines Agency. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials.

About Paroxysmal Nocturnal Hemoglobinuria (PNH)

PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, hemoglobinuria and difficulty breathing (dyspnea). A retrospective analysis shows that, even on eculizumab, approximately 72% of people with PNH have anemia, a key indicator of ongoing hemolysis.3 The analysis also finds that 36% of patients require one or more transfusions a year and 16% require three or more.3

About Apellis

Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

3

US Patent Issued to ViOptix for “Determining Tissue Oxygen Saturation with Melanin Correction” 

ALEXANDRIA, VA–(US Fed News)–United States Patent no. 10,820,863, issued on Nov. 3, was assigned to ViOptix Inc. (Newark, CA).

“Determining tissue oxygen saturation with melanin correction” was invented by Kate LeeAnn Bechtel (Pleasant Hill, Calif.), Kimberly Merritt Shultz (Mountain View, Calif.), Alex Michael Margiott (Fremont, Calif.) and George Edward Kechter (Peoria, Ill.). According to the abstract* released by the U.S. Patent & Trademark Office: “An oximeter probe that takes into account tissue color (e.g., skin color or melanin content) to improve accuracy when determining oxygen saturation of tissue. Light is transmitted from a light source into tissue having melanin (e.g., eumelanin or pheomelanin). Light reflected from the tissue is received by a detector. A compensation factor is determined to account for absorption due to the melanin. The oximeter uses this compensation factor and determines a melanin-corrected oxygen saturation value.”

The patent was filed on April 21, 2017, under Application No. 15/494,444.

October 2020

30

Atea, Riding Interest in COVID-19 Drugs, Pulls Off One of 2020′s Top Biotech IPOs

– Boston biotech Atea Pharmaceuticals, a developer of small molecule drugs for infectious diseases, has raised $300 million in an initial public offering, selling 12.5 million shares at $24 apiece.
– The new funding will help Atea advance an antiviral to treat or potentially prevent COVID-19, as well as a portfolio of other medicines for viruses like Dengue and respiratory syncytial virus. The IPO comes seven days after Roche paid the firm $350 million for partial rights to the COVID-19 antiviral AT-527, which is currently in mid-stage testing and could advance to multiple Phase 3 trials next year.
– Atea’s IPO is the fourth largest U.S. biotech stock offering of 2020 and the eighth biggest since the start of 2018, according to a database compiled by Biopharma Dive. Despite an initial slowdown at the beginning of the pandemic, biotech company IPOs are on a record-setting pace this year, reflecting excitement surrounding the industry’s role in developing coronavirus treatments and vaccines.

Infectious disease drugs haven’t generated nearly the type of interest from IPO investors in recent years as cancer drugs. Of the ten largest offerings in each of the last three years, for example, 14 of those 30 biotechs had an experimental or marketed cancer medicine as their lead program. Just one biotech — AlloVir, which raised $276 million in July — had an infectious disease drug at the forefront of its pipeline, according to Biopharma Dive’s IPO database.

The coronavirus pandemic, however, increased interest in both vaccines and infectious disease drugs as the industry scrambled to respond. Shares of Moderna and BioNTech, for instance, have surged to all-time highs because of their progress with experimental coronavirus vaccines. CureVac, another developer of a coronavirus shot, has the second highest market value of any biotech to go public in the U.S. this year. Newcomers AlloVir and Vaxcyte, meanwhile, are among the year’s better performers, climbing at least 54% from their offering price as of Thursday’s market close.

Atea, then, appears to have gone public at the right time. Formed in 2014, the company was originally developing AT-527 for hepatitis C infections. In May, it was cleared to begin testing the drug, an oral small molecule that interferes with the RNA of several viruses, against SARS-CoV-2.

Atea has since started a Phase 2 trial of 190 patients with moderate disease and one or more risk factors for poor outcomes, with results expected next year.

The pivot to coronavirus research has been lucrative for Atea. Since then, the biotech has raised $215 million in a private funding round, added $350 million in cash through the partnership with Roche and now another $300 million through its IPO.

Atea claims its antiviral is easy to manufacture and less burdensome to administer earlier in a patient’s disease course, when stopping the virus from spreading might help prevent the progression of COVID-19. Gilead’s approved COVID-19 drug Veklury, by comparison, must be infused over several days, making it more difficult to administer when it might be most effective.

Other, larger companies are also seeking to improve on Veklury. Merck & Co. has an antiviral pill it licensed from Ridgeback Therapeutics in Phase 2 testing. And Pfizer is advancing an intravenously administered antiviral drug.

Engineered antibody drugs from Regeneron, Eli Lilly and others could also play a role in treating COVID-19 patients before they need to be hospitalized.

The Roche partnership, announced just a week before Atea’s IPO, has given the company the backing to run a broad development program. Positive results in the ongoing mid-stage trial could lead to two Phase 3 tests in non-hospitalized patients and those newly exposed to infection.

In addition to the $350 million upfront, Roche could pay Atea another $650 million more under the agreement, with $330 million tied to regulatory development milestones. In return, Roche secured international rights to AT-527, and will help manufacture and distribute the drug overseas.

Cell therapy developer SQZ Biotechnologies also went public on Friday, raising $71 million. Sixty three biotechs — which Biopharma Dive defines as companies developing human medicines — have raised more than $50 million through a U.S. IPO this year.

www.biopharmadive.com

29

IM Therapeutics Launches Phase 1b Trial of Lead Drug IMT-002 in Autoimmunity; Announces Positive Safety Data of Phase 1a Study

– IMT-002 is the first oral genetically targeted drug candidate to be tested in type 1 diabetes patients

– Completed Phase 1a study showed no adverse events in all patient groups

– Therapeutic approach blocks activity of specific genes known to play a strong role in autoimmune disease development

WOBURN, Mass.–(BUSINESS WIRE)–ImmunoMolecular Therapeutics, Inc. (“IM Therapeutics”), a leading innovator in the field of precision medicine for autoimmune diseases, today announced it has launched a Phase 1b study as a multiple ascending dose (MAD) trial. The study is in patients who have type 1 diabetes (T1D), an as yet incurable autoimmune disorder that affects nearly 1.6 million Americans, and who also have a specific genetic trait known as HLA-DQ8, present in about 60% of T1D patients. Variants in certain genes called human leukocyte antigens (HLA) are known to play a strong role in activating an autoimmune response.

The Phase 1b trial will enroll 30 patients between the ages of 18 and 45 into four groups, each of which will have six subjects receiving varying doses of IMT-002 over a two-week duration. The remaining six subjects will receive placebo. The study will compare drug versus placebo for safety, tolerability and pharmacokinetic (PK) profiles over the two-week duration of dosing and a follow-up one week after dose completion. In addition, the study will assess pharmacodynamic (PD) activity with a blood-based assay that measures the ability of IMT-002 to block DQ8 presentation of “self-antigens” or the body’s own proteins, such as insulin or gluten, that can result in autoimmunity.

In conjunction with the Phase 1b study, IM Therapeutics announced the completion of a Phase 1a study of IMT-002 in healthy subjects. The study recruited 28 volunteers in four different groups, each of which had six subjects on a single ascending dose of IMT-002 and one subject on placebo. The study showed no adverse events in any of the groups. In addition, pharmacokinetic (PK) profiles followed over a 24-hour period after drug administration indicated an average drug half-life of about five hours and measurable levels in blood up to 12 hours. The completion of Phase 1a has helped inform frequency and levels of dosing in the Phase 1b study.

“We are encouraged by the favorable safety findings in our first study,” said Nandan Padukone, Ph.D., CEO of IM Therapeutics. “The PK profile of our lead drug candidate indicates a strong possibility of eventually having a once-daily oral therapy for patients with recent diagnosis of T1D, more than half of whom are pediatric patients,” he added. “We will continue to focus on expanding our pipeline of therapeutic candidates for patients impacted by autoimmune disease including celiac, lupus, and rare diseases.”

“We look forward to not only showing further safety and tolerability of our drug candidate, but also its potential as a selective inhibitor of DQ8-related autoimmunity,” said Sarah Bird, Ph.D., VP of Clinical Development at IM Therapeutics. “Tailored therapies against known genetic targets can significantly improve patient-centered care in autoimmune disease.”

About IMT-002

IMT-002 has been developed as a selective HLA-DQ8 blocker based on extensive computational work, in vitro and in vivo characterization. Previous studies of a tool drug, L-methyldopa, which is FDA-approved for treating hypertension, in a Phase 1b study, showed effective inhibition of DQ8 activity in new onset type 1 diabetes patients who had the DQ8 gene variant. Several in vivo IND studies indicate that IMT-002, which unlike the tool drug, is not metabolized physiologically, has more potency to block DQ8 activity and a favorable safety profile. Extensive preclinical studies of IMT-002 have been completed leading to IND clearance prior to initiation of human clinical trials.

About IM Therapeutics

IM Therapeutics (http://imtherapeutics.com/) is a clinical stage company developing personalized medicines for autoimmune diseases by building oral drug therapies against human leukocyte antigen (HLA) variants known to confer high risk of disease. The Company’s technology platform develops small molecule drugs using in silico docking of millions of compounds into pockets of an HLA protein where self-antigens may bind to trigger autoimmunity. Selected drug hits are then optimized using proprietary structure-based design and activity screening with cell-based assays for specificity of HLA inhibition. Lead drugs developed against an HLA variant have the ability to block a series of self-antigens and therefore the potential to treat several autoimmune indications related to a selected HLA. The Company is building a broad HLA-targeted pipeline in autoimmune disorders including celiac disease. In June 2019, IM Therapeutics raised $10 million in Series A financing led by Morningside Ventures and the JDRF T1D Fund.

Atea Pharmaceuticals Announces Pricing of Initial Public Offering

BOSTON, Oct. 29, 2020 (GLOBE NEWSWIRE) — Atea Pharmaceuticals, Inc. (Nasdaq: AVIR) (“Atea”), a clinical-stage biopharmaceutical company focused on discovering, developing and commercializing antiviral therapeutics to improve the lives of patients suffering from life-threatening viral infections, today announced the pricing of its initial public offering of 12,500,000 shares of its common stock at a price to the public of $24.00 per share. All of the shares of common stock are being offered by Atea. The gross proceeds from the offering, before deducting underwriting discounts and commissions and estimated offering expenses payable by Atea, are expected to be $300 million, excluding any exercise of the underwriters’ option to purchase additional shares. Atea’s common stock is expected to begin trading on the Nasdaq Global Select Market under the ticker symbol “AVIR” on October 30, 2020. The offering is expected to close on November 3, 2020, subject to satisfaction of customary closing conditions. In addition, Atea has granted the underwriters a 30-day option to purchase up to an additional 1,875,000 shares of Atea’s common stock at the initial public offering price less the underwriting discounts and commissions.

J.P. Morgan Securities LLC, Morgan Stanley & Co. LLC, Evercore Group L.L.C. and William Blair & Company, L.L.C. are acting as joint book-running managers of the offering.

A registration statement on Form S-1 (File No. 333-249404) relating to the offering has been filed with the Securities and Exchange Commission and became effective on October 29, 2020. The offering was made only by means of a prospectus. Copies of the final prospectus relating to the offering may be obtained from: J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: 866-803-9204; Morgan Stanley & Co. LLC, 180 Varick Street, 2nd Floor, New York, NY 10014, Attention: Prospectus Department, or by email at prospectus@morganstanley.com; Evercore Group, L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 35th Floor, New York, NY 10055, by telephone at (888) 474-0200, or by email at ecm.prospectus@evercore.com; or William Blair & Company, L.L.C., Attention: Prospectus Department, 150 North Riverside Plaza, Chicago, IL 60606, by telephone at (800) 621-0687 or by email at prospectus@williamblair.com.

This press release does not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

28

Gracell Biotechnologies Raises $100 Million in Series C Funding to Advance Next Generation CAR-T Cell Therapies

SHANGHAI and SUZHOU, China, Oct. 28, 2020 /PRNewswire/ — Gracell Biotechnologies Inc. (“Gracell”), a global clinical-stage biopharmaceutical company dedicated to developing highly efficacious and affordable cell therapies for the treatment of cancer, today announced it has secured $100 million in Series C funding. The round is led by Wellington Management Company, OrbiMed and Morningside Ventures, and joined by new investor Vivo Capital. Existing investors Temasek Holdings, Lilly Asia Ventures, OrbiMed and King Star Med LP are also participating.

“We are very pleased to expand our investor base with support from a high caliber consortium,” said Dr. William Wei Cao, founder, Chairman, and CEO of Gracell, “Our passion is to bring transformative CAR-T cell therapies to a broader group of patients by developing products that are efficacious and can be made widely available.”

Gracell was founded in 2017 with the mission to overcome the major industry challenges that persist for both autologous and allogeneic CAR-T cell therapy approaches. Gracell has developed two pioneering platforms—FasTCAR and TruUCAR. With FasTCAR, Gracell is able to deliver younger, less exhausted T cells for autologous cell therapies with greater potency and next-day manufacturing (22 to 36 hours). With TruUCAR, Gracell is able to derive T cells from non-HLA-matched healthy donors to generate allogeneic CAR-T cell therapies that are readily available off-the-shelf at lower cost for a broad patient base.

Leveraging its FasTCAR and TruUCAR platforms, Gracell is developing a pipeline of autologous and allogeneic cell therapy candidates with the potential to treat both hematologic malignancies and solid tumors. Currently, Gracell’s lead FasTCAR autologous product candidate, GC012F, is being studied in an ongoing investigator-initiated Phase 1 trial in China for the treatment of relapsed or refractory multiple myeloma (r/r MM). Its lead TruUCAR allogeneic product candidate, GC027, is being studied in an ongoing investigator-initiated Phase 1 trial in China for the treatment of relapsed or refractory T cell acute lymphoblastic leukemia (r/r T-ALL).

Proceeds from the round will be used to fund internal research and development and further advance current clinical programs. Jefferies and Cooley advised in the transaction.

About Gracell

Gracell Biotechnologies Inc. (“Gracell”) is a global clinical-stage biopharmaceutical company dedicated to discovering and developing breakthrough cell therapies. Leveraging its pioneering FasTCAR and TruUCAR technology platforms, Gracell is developing a rich clinical-stage pipeline of multiple autologous and allogeneic product candidates with the potential to overcome major industry challenges that persist with conventional CAR-T therapies, including lengthy manufacturing time, suboptimal production quality, high therapy cost and lack of effective CAR-T therapies for solid tumors.

27

Prilenia Enrolls First Patients into Its PROOF-HD Phase 3 Clinical Trial for Huntington’s Disease in the United States

– PROOF-HD is designed to replicate previous findings of pridopidine demonstrating maintenance of functional capacity in early Huntington’s Disease patients

– Conducted in collaboration with the Huntington Study Group, a world leader in clinical research for HD

– If successful, PROOF-HD could lead to the registration of pridopidine, one of the few oral drugs currently in clinical development for the treatment of HD.

[Naarden, NL, 27 October 2020] Prilenia Therapeutics B.V., a clinical stage biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders, today announces the enrollment of the first patients in the U.S. into PROOF-HD, a global phase 3 clinical trial for Huntington’s Disease (HD). The study is being conducted in collaboration with the Huntington Study Group (HSG).

​Pridopidine Outcome On Function in Huntington’s Disease (PROOF-HD) is a randomized, double-blind, placebo-controlled, phase 3 study evaluating the efficacy and safety of pridopidine 45mg bid in patients with early stage HD. The study will enroll 480 participants aged 25 or older with a clinical diagnosis of adult-onset HD at approximately 60 study centers in the U.S., Canada and Europe. The treatment period will last up to 78 weeks and there will be an optional open-label extension.

The study is designed to replicate previous findings that demonstrated pridopidine’s effect in maintaining functional capacity in patients with early HD. The trial treatment dose (45mg bid) has a favorable safety profile based on over 1,000 patient years in previous HD trials.

Andrew Feigin, MD, North American Principal Investigator for the PROOF-HD Phase 3 Trial, said: “Huntington’s Disease is a serious condition with no known treatments that slow functional decline. A successful result would be a major advance in our ability to treat HD. I look forward to working with the Prilenia team as we push forward with patient recruitment.”

​Michael R. Hayden, CEO of Prilenia and world-renowned scientist in Huntington’s Disease research, commented: “The design of the PROOF-HD study is based on strong scientific and clinical data, including in vivo target engagement for the selected dose, prior clinical efficacy results and extensive long-term safety data in our target population”. He added: “Enrolling our first patients is a significant milestone that brings hope to others suffering from this devastating disease.”

​Pridopidine is a first in class small molecule developed by Prilenia for the treatment of neurodegenerative disorders such as HD and Amyotrophic Lateral Sclerosis (ALS). Pridopidine is a highly selective Sigma-1 receptor (S1R) agonist. It binds and activates the S1R, a protein that is expressed at high levels within the brain and regulates key cellular pathways, commonly impaired in neurodegeneration.

https://huntingtonstudygroup.org/proof-hd/

23

Kezar Life Sciences Gets Orphan Drug Designations for KZR-616 for the Treatment of Polymyositis and Dermatomyositis

SAN FRANCISCO–(BUSINESS WIRE)– Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncologic disorders, today announced that the U.S. Food and Drug Administration has granted Orphan Drug Designations (ODD) for KZR-616 for the treatment of polymyositis (PM) and dermatomyositis (DM). Both orphan diseases are autoimmune inflammatory myopathies that are chronic and debilitating diseases characterized by marked morbidity and mortality. The estimated prevalence of PM and DM in the United States is up to 51,000 and 71,000, respectively.

KZR-616 is a first-in-class selective immunoproteasome inhibitor with the potential to impact multiple drivers of immune-mediated diseases and inflammation. PRESIDIO, a Phase 2 placebo-controlled cross-over clinical trial to evaluate KZR-616 for the treatment of PM and DM, is currently enrolling subjects.

“We are very pleased to have received orphan drug designations for both polymyositis and dermatomyositis. This recognition spotlights the significant unmet need for patients living with these autoimmune myopathies. KZR-616 has potential to truly modify the underlying pathophysiology of these two diseases,” said Noreen R. Henig, MD, Kezar’s Chief Medical Officer. “We will share more detail on the preclinical scientific rationale for the use of selective immunoproteasome inhibition to treat these rare and debilitating diseases during the 2020 American College of Rheumatology Conference next month.”

Orphan Drug Designation is intended to advance drug development for rare diseases. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States. Orphan Drug Designation can provide certain benefits and incentives for KZR-616, including a period of marketing exclusivity for the first marketing application, if regulatory approval is received for the designated indication, potential tax credits for qualified clinical testing and waiver of certain administrative fees.

About KZR-616

KZR-616 is a novel, first-in-class, selective immunoproteasome inhibitor with broad therapeutic potential across multiple autoimmune diseases. Preclinical research demonstrates that selective immunoproteasome inhibition results in a broad anti-inflammatory response in animal models of several autoimmune diseases, while avoiding immunosuppression. Data generated from Phase 1a and 1b trials provide evidence that KZR-616 exhibits a favorable safety and tolerability profile for development in chronic autoimmune diseases. Phase 2 trials are underway in severe autoimmune diseases.

About Polymyositis and Dermatomyositis

Polymyositis (PM) and Dermatomyositis (DM) are two of the five types of autoimmune myositis diseases. Both are chronic, debilitating, inflammatory autoimmune myopathies that are distinguished by inflammation of the muscles as well as the skin (in DM). Up to ~120,000 people in the United States are living with these severe and progressive inflammatory myopathies that are characterized by marked morbidity and associated mortality. While debilitating muscle weakness is the hallmark of these myopathies, including compromised muscles of respiration, other internal organ system dysfunctions can be equally disabling. The aim of treatment for these diseases is to suppress inflammation, increase muscle strength and prevent long-term damage to muscles and extramuscular organs; however, treatment options are limited for DM, and there are currently no approved treatments for PM.

About Kezar Life Sciences

Based in South San Francisco, Kezar Life Sciences is combining courage, conviction and cutting-edge science to develop breakthrough treatments for immune-mediated and oncologic disorders. The company is pioneering first-in-class, small-molecule therapies that harness master regulators of cellular function and inhibit multiple drivers of disease via a single target. KZR-616, a first-in-class selective immunoproteasome inhibitor, is being evaluated in severe and underserved autoimmune diseases. Additionally, KZR-261, the first clinical candidate for the treatment of cancer from the company’s protein secretion program targeting the Sec61 translocon, is undergoing IND-enabling activities.

Kezar Life Sciences Announces Orphan Drug Designations for KZR-616 for the Treatment of Polymyositis and Dermatomyositis

SAN FRANCISCO–(BUSINESS WIRE)– Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncologic disorders, today announced that the U.S. Food and Drug Administration has granted Orphan Drug Designations (ODD) for KZR-616 for the treatment of polymyositis (PM) and dermatomyositis (DM). Both orphan diseases are autoimmune inflammatory myopathies that are chronic and debilitating diseases characterized by marked morbidity and mortality. The estimated prevalence of PM and DM in the United States is up to 51,000 and 71,000, respectively.

KZR-616 is a first-in-class selective immunoproteasome inhibitor with the potential to impact multiple drivers of immune-mediated diseases and inflammation. PRESIDIO, a Phase 2 placebo-controlled cross-over clinical trial to evaluate KZR-616 for the treatment of PM and DM, is currently enrolling subjects.

“We are very pleased to have received orphan drug designations for both polymyositis and dermatomyositis. This recognition spotlights the significant unmet need for patients living with these autoimmune myopathies. KZR-616 has potential to truly modify the underlying pathophysiology of these two diseases,” said Noreen R. Henig, MD, Kezar’s Chief Medical Officer. “We will share more detail on the preclinical scientific rationale for the use of selective immunoproteasome inhibition to treat these rare and debilitating diseases during the 2020 American College of Rheumatology Conference next month.”

Orphan Drug Designation is intended to advance drug development for rare diseases. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States. Orphan Drug Designation can provide certain benefits and incentives for KZR-616, including a period of marketing exclusivity for the first marketing application, if regulatory approval is received for the designated indication, potential tax credits for qualified clinical testing and waiver of certain administrative fees.

About KZR-616

KZR-616 is a novel, first-in-class, selective immunoproteasome inhibitor with broad therapeutic potential across multiple autoimmune diseases. Preclinical research demonstrates that selective immunoproteasome inhibition results in a broad anti-inflammatory response in animal models of several autoimmune diseases, while avoiding immunosuppression. Data generated from Phase 1a and 1b trials provide evidence that KZR-616 exhibits a favorable safety and tolerability profile for development in chronic autoimmune diseases. Phase 2 trials are underway in severe autoimmune diseases.

About Polymyositis and Dermatomyositis

Polymyositis (PM) and Dermatomyositis (DM) are two of the five types of autoimmune myositis diseases. Both are chronic, debilitating, inflammatory autoimmune myopathies that are distinguished by inflammation of the muscles as well as the skin (in DM). Up to ~120,000 people in the United States are living with these severe and progressive inflammatory myopathies that are characterized by marked morbidity and associated mortality. While debilitating muscle weakness is the hallmark of these myopathies, including compromised muscles of respiration, other internal organ system dysfunctions can be equally disabling. The aim of treatment for these diseases is to suppress inflammation, increase muscle strength and prevent long-term damage to muscles and extramuscular organs; however, treatment options are limited for DM, and there are currently no approved treatments for PM.

About Kezar Life Sciences

Based in South San Francisco, Kezar Life Sciences is combining courage, conviction and cutting-edge science to develop breakthrough treatments for immune-mediated and oncologic disorders. The company is pioneering first-in-class, small-molecule therapies that harness master regulators of cellular function and inhibit multiple drivers of disease via a single target. KZR-616, a first-in-class selective immunoproteasome inhibitor, is being evaluated in severe and underserved autoimmune diseases. Additionally, KZR-261, the first clinical candidate for the treatment of cancer from the company’s protein secretion program targeting the Sec61 translocon, is undergoing IND-enabling activities.

Cautionary Note on Forward-looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “may,” “will,” “should,” “expect,” “believe” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Kezar’s expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties that could cause Kezar’s clinical development programs, future results or performance to differ materially from those expressed or implied by the forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to estimates of the prevalence of polymyositis and dermatomyositis, the potential of KZR-616 to modify the underlying pathophysiology of polymyositis and dermatomyositis, statements about the likelihood that pre-clinical data will support the scientific rationale to develop KZR-616 in polymyositis and dermatomyositis, the design, progress, timing, scope and results of clinical trials, the anticipated timing of disclosure of results of clinical trials and the likelihood and timing of obtaining regulatory approval of KZR-616. Orphan Drug Designation does not provide any assurance of regulatory approval or expedite regulatory review. Many factors may cause differences between current expectations and actual results, including the impacts of the COVID-19 pandemic on the company’s business, clinical trials and financial position, unexpected safety or efficacy data observed during preclinical or clinical studies, clinical trial site activation or enrollment rates that are lower than expected, changes in expected or existing competition, changes in the regulatory environment, the uncertainties and timing of the regulatory approval process, and unexpected litigation or other disputes. Other factors that may cause actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Kezar’s filings with the U.S. Securities and Exchange Commission, including the “Risk Factors” contained therein. Except as required by law, Kezar assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.

www.businesswire.com/news/home/20201023005106/en/

22

Atea Pharmaceuticals Announces Strategic Collaboration with Roche to Develop and Distribute AT-527 for Patients with COVID-19

Roche Obtains Exclusive Right to Develop and Distribute AT-527 Outside the United States

BOSTON, Mass., October 22, 2020 – Atea Pharmaceuticals, Inc., a clinical stage biopharmaceutical company focused on discovering, developing and commercializing antiviral therapeutics to improve the lives of patients suffering from life-threatening viral infections, today announces that the company has entered into an agreement with Roche (SIX: RO, ROG; OTCQX: RHHBY) for the exclusive rights to research, develop and distribute AT-527 as an oral antiviral treatment for COVID-19 in territories outside of the United States. Under the terms of the agreement, Atea will receive an upfront payment of $350 million in cash from Roche with the potential for future milestone payments and royalties.

“Roche shares our passion for delivering innovative new medicines to address great unmet medical needs. The COVID-19 pandemic has highlighted the urgent need for a novel, oral antiviral to treat this highly infectious and often deadly virus,” said Jean-Pierre Sommadossi, Ph.D., Chief Executive Officer and Founder of Atea Pharmaceuticals. “This collaboration with Roche enhances Atea’s efforts and underscores the potential for AT-527 to effectively address the COVID-19 crisis on a global scale. AT-527 is expected to be ideally suited to combat COVID-19 as it inhibits viral replication by interfering with viral RNA polymerase, a key component in the replication machinery of RNA viruses. Importantly, the manufacturing process for our small molecule direct-acting antiviral allows us to produce AT-527 quickly and at scale.”

“The ongoing complexities of COVID-19 require multiple lines of defence. By joining forces with Atea, we hope to offer an additional treatment option for hospitalised and non-hospitalised COVID-19 patients, and provide important relief for hospital infrastructures during a global pandemic.” said Bill Anderson, Chief Executive Officer of Roche Pharmaceuticals. “In jointly developing and manufacturing AT-527 at scale, we seek to make this treatment option available to as many people around the world as we possibly can.”

About AT-527

AT-527 is an orally administered, direct-acting antiviral agent derived from Atea’s purine nucleotide prodrug platform. At-527 is currently under evaluation as a treatment for patients with COVID-19. AT-527 is designed to inhibit viral replication by interfering with viral RNA polymerase, a key component in the replication machinery of RNA viruses, such as positive single-stranded human flaviviruses and human coronaviruses. AT-527 is currently in a global Phase 2 clinical study for hospitalized patients with moderate COVID-19 and has plans to initiate a global, registrational Phase 3 clinical trial in outpatients in the first half of 2021. Additionally, Atea is planning to study in a Phase 3 clinical trial the use of AT-527 in the post-exposure prophylaxis setting.

Advisors

Evercore served as exclusive financial advisor to Atea in connection with this transaction.

About Atea Pharmaceuticals

Atea Pharmaceuticals is a clinical stage biopharmaceutical company engaged in discovering and developing therapies to address the unmet medical needs of patients with severe viral diseases. Our lead programs are focused on the development of orally- administered direct acting antivirals for the treatment of patients with COVID-19 in the hospital and community settings, the treatment of patients with chronic hepatitis C infection, the treatment of patients with dengue, and the treatment of high-risk patients with severe respiratory syncytial virus infection. Our medicinal chemistry, virology, and pharmacology expertise, bolstered by our collective experience in drug development, enables us to pioneer new advancements in antiviral science. Leveraging the power of our purine nucleotide prodrug platform, our goal is to rapidly advance novel drug candidates with optimal therapeutic profiles for RNA virus targets. Founded by its Chairman and Chief Executive Officer, Jean-Pierre Sommadossi, PhD, Atea began operations in 2014 and is headquartered in Boston, MA. For more information about Atea and our pipeline of product candidates please visit our company website at www.ateapharma.com.

F2G Receives Second US FDA Breakthrough Therapy Designation for Olorofim

– In Phase 2b development for the treatment of life-threatening fungal infections
– First antifungal agent to receive Breakthrough Therapy Designation
– New second Breakthrough Therapy Designation for CNS Valley Fever (coccidioidomycosis)

MANCHESTER, UK / VIENNA, Austria – October 22, 2020 – F2G Ltd, a UK- and Austria-based biotech company developing novel therapies for life-threatening systemic fungal infections, announced today that the US Food and Drug Administration (FDA) has granted an additional Breakthrough Therapy Designation to its lead first-in-class candidate, olorofim, for the indication of ‘Treatment of Central Nervous System (CNS) coccidioidomycosis refractory or otherwise unable to be treated with standard of care therapy’. This is the second Breakthrough Therapy Designation for olorofim; a designation was granted previously on 11 November 2019 for ‘Treatment of invasive mold infections in patients with limited or no treatment options, including aspergillosis refractory or intolerant to currently available therapy, and infections due to Lomentospora prolificans, Scedosporium, and Scopulariopsis species’. Olorofim (formerly F901318) is the first antifungal agent to be granted Breakthrough Therapy Designation.

Olorofim has orphan drug designation in the United States for coccidioidomycosis, an endemic fungal infection with a geographic distribution focused mainly in the desert Southwest of the United States but also including Mexico, Central America, and South America. Infection begins by inhalation and can spread to any part of the body, even in otherwise healthy individuals. Spread to the brain is particularly feared as it produces a devastating illness that cannot always be controlled with any currently available agent.

Patients with coccidioidomycosis are being studied in an ongoing open-label single-arm Phase 2b study (ClinicalTrials.gov Identifier: NCT03583164) in patients with proven invasive fungal disease (IFD) or probable invasive aspergillosis (IA) and either refractory disease, resistance, or intolerance to available agents. Olorofim has been well tolerated across more than 30 years of cumulative patient dosing days with a median therapy duration of 12 weeks. Preliminary data from this study were provided to the FDA as part of the Breakthrough Therapy Designation submission.

Breakthrough Therapy Designation is an FDA process designed to expedite the development and review of drugs that are intended to treat a serious or life-threatening condition and is granted based on preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.

Breakthrough Therapy Designation conveys all the features of fast track designation, more intensive FDA guidance on an efficient drug development program, an organisational commitment by FDA to involve senior managers, and eligibility for rolling review and priority review.

Commenting on the news, Ian Nicholson, CEO of F2G Ltd, said: “The granting of a second FDA Breakthrough Therapy Designation will further support our goal of rapidly developing this novel treatment for patients suffering from serious and life-threatening fungal infections. Olorofim acts via a novel and differentiated mechanism to traditional antifungals, and preliminary data have indicated that it is efficacious in tackling life-threatening invasive fungal infections that cannot be managed with currently approved agents.

“Our Phase 2b programme is on track with over 85 patients recruited in Europe, Asia, and the US. We look forward to working closely with the US FDA to accelerate development of this therapy for patients having limited or no approved treatment options for an invasive mold infection.”

Professor George Thompson, UC Davis and Investigator for the Phase 2b study said: “This news is very exciting for clinicians caring for patients with Valley Fever (coccidioidomycosis) as spread to the brain is the most-feared complication of infection with this fungus. Unfortunately, available drugs are not curative, must be administered as life-long therapy, and are associated with substantial toxicity. The news that olorofim has encouraging preliminary clinical data that support Breakthrough Therapy Designation brings realistic hope that we can change the paradigm for managing this devastating infection.”

About Olorofim / Clinical trial
The Phase 2b study for olorofim (ClinicalTrials.gov Identifier: NCT03583164) is a global open-label study in patients who have limited treatment options for difficult-to-treat invasive fungal mold infections such as azole-resistant aspergillosis, scedosporiosis, lomentosporiosis, and other rare mold infections. More than 40 centres are currently open in nine countries (AU, BE, DE, ES, IS, NL, TH, UK, USA) and a further 10 will open in 2020/2021. Olorofim is being developed both as IV and oral formulations.

About F2G
F2G is a world-leading UK- and Austria-based biotech company (F2G Ltd and F2G Biotech GmbH) focused on the discovery and development of novel therapies to treat life-threatening invasive fungal infections. F2G has discovered and developed a completely new class of antifungal agents called the orotomides. The orotomides selectively target fungal dihydroorotate dehydrogenase (DHODH), a key enzyme in the de novo pyrimidine biosynthesis pathway. This is a completely different mechanism from that of the currently marketed antifungal agents and gives the orotomides fungicidal activity against a broad range of rare and resistant fungal mold infections. Olorofim (formerly, F901318) is F2G’s leading candidate from this class and is in a Phase 2b open-label study focussing on rare and resistant invasive fungal infections such as aspergillosis (including azole-resistant strains), scedosporiosis (including lomentosporiosis). Olorofim has received orphan drug status from the European Medicines Agency for the treatment of invasive aspergillosis and invasive scedosporiosis. Olorofim has received orphan drug status from the US FDA for the treatment of coccidioidomycosis, lomentosporiosis/scedosporiosis, and invasive aspergillosis. Olorofim has been granted Qualified Infectious Disease Product (QIDP) designation Invasive for aspergillosis, invasive scedosporiosis, invasive lomentosporiosis, coccidioidomycosis, invasive disease due to Scopulariopsis species, and invasive fusariosis. Olorofim is being developed both as IV and oral formulations. www.f2g.com

20

Atea Pharmaceuticals Announces IND Clearance of AT-527 for COVID-19 and $215 Million Financing

– Proceeds to support the clinical development of Atea’s oral, direct acting antiviral medicine for COVID-19, in addition to advancing its diverse pipeline of treatments for viral diseases
– Investigational new drug application cleared by U.S. Food and Drug Administration to start Phase 2 study of AT-527, Atea’s oral purine nucleotide prodrug for patients hospitalized with moderate COVID-19

BOSTON, Mass., May 20, 2020 – Atea Pharmaceuticals, Inc., a biopharmaceutical company
engaged in the discovery and development of next-generation therapeutics for severe human viral infections, today announced a $215 million Series D financing. The financing was led by Bain Capital Life Sciences and also included new investors RA Capital Management, Perceptive Advisors, Rock Springs Capital, Adage Capital Management, Redmile Group, Omega Funds, and funds and accounts managed by T. Rowe Price Associates, Inc. Existing Atea investors, including Morningside Ventures, Cormorant Asset Management, Ally Bridge Group, and Sectoral Asset Management, as well as other investors also participated in this financing.

Atea also announced today that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug application (IND) for AT-527, a novel, oral, purine nucleotide prodrug, for the treatment of adult patients hospitalized with moderate COVID-19 disease, with one or more risk factors for poor outcomes. A Phase 2 clinical trial, scheduled to begin shortly, will evaluate the safety and efficacy of AT-527 in this patient population.

AT-527 is a highly selective, orally administered direct acting antiviral, (DAA) designed to inhibit the RNA polymerase enzyme, a key element in the replication machinery of RNA viruses. Antiviral activity of AT-527 has been observed in vitro and in vivo against replication of multiple RNA viruses including, but not limited to, human coronaviruses and flaviviruses.

In addition to supporting its work to find a treatment for COVID-19, Atea expects to also apply proceeds from this financing towards advancing its diverse pipeline of highly selective DAAs that target other severe RNA viral infections. Atea’s pipeline currently includes investigative treatments for hepatitis C virus, dengue virus, and respiratory syncytial virus, in addition to its COVID-19 program.

“We are delighted to have the strong support of this group of blue-chip healthcare investors,” said Jean-Pierre Sommadossi, PhD, Atea’s Founder, Chairman, and Chief Executive Officer. “Atea’s portfolio is focused on developing novel, best-in-class, potent DAA’s and we have shifted all of our immediate resources and our team’s deep expertise in virology and pharmacology to help address the unmet needs in the fight against the COVID-19 pandemic. An oral treatment for COVID-19 patients should prevent progression of the disease and may help lessen the burden on critical inpatient resources. Atea is moving rapidly, in concert with regulatory authorities, to determine if our oral DAA is a safe and effective therapeutic against COVID-19.”

“Atea’s team has an outstanding track record in developing novel, potent DAAs, which we believe can contribute to the urgent fight against the COVID-19 pandemic and other RNA viruses,” said Andrew Hack, M.D., Ph.D., Managing Director of Bain Capital Life Sciences. “We are pleased to partner with Atea’s leadership team and an outstanding group of leading healthcare investors as Atea advances its diverse pipeline of transformative antiviral medicines”

About AT-527

AT-527 is an investigational, oral, purine nucleotide prodrug, which has demonstrated in vitro and in vivo antiviral activity against several enveloped single-stranded RNA viruses, including human flaviviruses and coronaviruses. This highly selective purine nucleotide prodrug was designed to uniquely inhibit viral RNA dependent RNA polymerase, an enzyme that is essential for the replication of RNA viruses. Antiviral activity and safety of AT-527 has been demonstrated in Phase 2 clinical studies of hepatitis C patients. AT-527 is not yet licensed or approved for any
indication in the U.S. or any other country.

About Atea Pharmaceuticals

Atea Pharmaceuticals is a clinical stage biopharmaceutical company engaged in discovering and developing best-in-class therapies to address the unmet medical needs of patients with severe viral diseases. Our lead programs are focused on the development of orally administered direct acting antivirals for the treatment of patients with mild to moderate COVID-19 in the hospital and community settings, the treatment of patients with chronic hepatitis C infection, the treatment of patients with dengue, and the treatment of high-risk patients with severe respiratory syncytial virus infection. Our medicinal chemistry, virology, and pharmacology expertise, bolstered by our collective experience in drug development, enables us to pioneer new advancements in antiviral science. Leveraging the power of our purine nucleotide prodrug platform, our goal is to rapidly advance novel drug candidates with optimal
therapeutic profiles for RNA virus targets. Founded by its Chairman and Chief Executive Officer, Jean-Pierre Sommadossi, PhD, Atea began operations in 2014 and is headquartered in Boston, MA. For more information about Atea and our pipeline of products please visit our company website at https://www.ateapharma.com/.

September 2020

28

Fierce Biotech Names Amylyx Pharmaceuticals as One of Its “Fierce 15″ Biotech Companies of 2020

Amylyx Pharmaceuticals, Inc.
Contify Life Science News
September 28, 2020

CAMBRIDGE, Massachusetts, Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, today announced that it has been named by Fierce Biotechas one of 2020′s Fierce 15 biotechnology companies, designating Amylyx as one of the most promising private biotechnology companies in the industry.

The 2020 Fierce 15 comes at a time when the world is focused on a pandemic, but even as coronavirus remains a threat, patients with cancer, rare diseases and other disorders still need treatment, Fierce Biotech Senior Editor Ben Adams said. “This year, we’ve chosen from a diverse range of those fighting COVID, as well as those fighting longer term plagues against our biology,” Adams said.

Amylyx’ lead product candidate, AMX0035, is an investigational neuroprotective therapy that seeks to prevent or reduce neuron death and dysfunction in ALS and other neurodegenerative diseases. Recently, Amylyx announced positive results from the pivotal CENTAUR trial evaluating AMX0035 in people with ALS, published in the New England Journal of Medicine. Amylyx continues to evaluate the long-term effects of AMX0035 in people with ALS in an ongoing open-label extension (OLE) of CENTAUR, and plans to publish the results from this study in Q4 2020. Additionally, AMX0035 is being evaluated for Alzheimer’s disease in the Phase 2 PEGASUS trial and expects topline results in Q1 2021.

“Amylyx is honored to be named one of Fierce Biotech’s Fierce 15 of 2020. This recognition highlights the tremendous responsibility our team has to continue working towards bringing a new potential treatment to those living with neurodegenerative diseases,” said Justin Klee, Co-CEO and Co-Founder of Amylyx.

An internationally recognized daily report reaching a network of over 285,000 biotech and pharma industry professionals, Fierce Biotech provides subscribers with an authoritative analysis of the day’s top stories. Every year Fierce Biotech evaluates hundreds of private companies from around the world for its annual Fierce 15 list, which is based on a variety of factors such as the strength of its technology, partnerships, venture backers and a competitive market position.

About Fierce Biotech

Fierce Biotech is the biotech industry’s daily monitor, an email newsletter and web resource providing the latest biotech news, articles, and resources related to clinical trials, drug discovery, FDA approval, FDA regulation, patent news, pharma news, biotech company news and more. More than 150,000 top biotech professionals rely on Fierce Biotech for an insider briefing on the day’s top stories. Signup is free at www.fiercebiotech.com/signup.

About Amylyx Pharmaceuticals

Amylyx Pharmaceuticals, Inc. is a pharmaceutical company working on developing a novel therapeutic for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases. For more information, visit www.amylyx.com and follow us on LinkedIn and Twitter.

24

Cognoa Moves Ahead on FDA Clearance of Digital Autism Diagnostic

Beg Bryant
BioWorld
September 24, 2020

Children with autism spectrum disorder (ASD) can face a lifetime of frustration because of challenges with communication, social behaviors and flexibility of thought. Early intervention can improve outcomes, but nailing a diagnosis of ASD often takes years. Cognoa Inc. wants to change that with its digital ASD Diagnostic and is on track to apply for U.S. FDA clearance before the end of the year.

“We’re submitting a package for de novo application in the fourth quarter of this year,” David Happel, Cognoa’s CEO, told BioWorld.

The Palo Alto, Calif.-based company snagged an FDA breakthrough device designation for the ASD Diagnostic device in October 2018, paving the way for a priority review. With such reviews averaging eight to nine months, Happel anticipates gaining FDA clearance early in the second half of 2021 and launching the product about eight weeks after that.

Need for earlier diagnosis

Approximately 1 in 54 children in the U.S. is diagnosed with ASD, according to the CDC. Getting to that diagnosis, however, can be laborious.

Once concerns have been raised about a child’s developmental progress, a pediatrician refers the parents to a specialist who puts the child through a battery of tests, assesses the results and makes a diagnosis. This can take anywhere from six to 18 months, Happel said, citing a lack of specialists who perform ASD evaluations. Although the condition typically start to present between 14 and 18 months, the average diagnosis is about four-and-a-half years of age.

By contrast, Cognoa’s prescription diagnostic leverages artificial intelligence (AI) and machine learning to quickly assess whether a child has autism or an autism spectrum disorder as soon as concerns arise.

The device has three modules. First, the parent or caregiver inputs data – a questionnaire and videos – via Cognoa’s parent/caregiver-facing mobile app. The one-to-two minute videos are filmed in the child’s natural environment to avoid creating stress and discomfort. Next, a pediatrician completes an assessment with the child, which they input into Cognoa’s platform. Cognoa’s AI then combines the parent/caregiver and pediatrician inputs to produce a result.

The videos are also forwarded to an expert in dealing with autism, trained and employed by Cognoa, for observation and assessment, who completes a questionnaire about their observations and feeds that into the AI as well.

The ASD Diagnostic can be downloaded to a hand-held device and performed in the primary care setting. The entire process takes anywhere from two to six weeks.

Pivotal study

In a pivotal study of 425 children ages 18 to 72 months whose parents had expressed concern about their development but had never undergone evaluation, Cognoa’s ASD Diagnostic surpassed its targeted benchmarks, the company said.

The multisite study, prospective, double-blind, active comparator study, which ran from July 2019 through May 2020, assessed the ASD Diagnostic’s ability to aid in the diagnosis of autism spectrum disorder by comparing diagnostic output with the clinical reference standard, which is a specialist clinician’s diagnosis, based on DSM-5 criteria and validated by one or more reviewing specialists. The goal was to determine how often the device correctly identified a patient with ASD and how often it concluded that a patient does not have the condition.

“It was exceptionally accurate to rule in or rule out autism to the degree that it easily surpassed the thresholds that we identified with the FDA for the point estimates,” Happel said. The results are being prepared for publication in a peer-reviewed journal.

He believes Cognoa’s device offers a real advancement in ASD diagnosis. It gives doctors a consistent and reliable diagnostic instrument that will accurately determine whether the child has autism that can be evaluated at the pediatrician level. And, perhaps equally important, it removes all bias when evaluating the child.

According to Happel, there are significant biases around how the condition is diagnosed, based on gender, race and other factors. For example, young Caucasian boys are most often considered to be at highest risk of having an autism diagnosis – four times more so than a girls of the same age.

“It’s not because boys are four times more likely to get autism. It’s the fact that girls present differently, as do individuals with different race, culture, ethnicity and socioeconomic status,” he said. “Our instrument is able to sort through that because it’s trained on hundreds of thousands of unique profiles, which allows it to remove the noise and agnostically make a diagnosis.”

Cognoa’s approach aligns with the most recent guidelines of the American Academy of Pediatrics, which is pushing for more ASD diagnoses to be made at the pediatrician level.

Robust pipeline

Cognoa is also developing a digital therapeutic for ASD. The company recently completed a 30-patient engagement study to determine the usability of hand-held devices for individuals living with autism and ASD.

Also accorded breakthrough status by the FDA, the ASD therapeutic was developed by Cognoa founder Dennis Wall, an associate professor of pediatrics and biomedical data science at Stanford University. “He’s been primarily working with wearable devices such as Google Glass and another program,” Happel said, adding the work has shown remarkable promise in helping people with autism recognize facial expressions, eye contact and emotions.

The company is working with Wall to put together a clinical trial next year to assess the value of the programs and when they might have the most benefit for patients.

“There’s an enormous amount of literature in the space that, particularly for medium to high functioning individuals that are autistic or on the spectrum, if you can get them diagnosed early enough, you can … effect these patterns in their developmental windows at a much earlier age, which will allow them either to, hopefully, reach a nondiagnosis at some point … or at least participate in a mainstream educational curricula and in system that can support that,” Happel said.

In addition to the ASD products, Cognoa is also developing a digital diagnostic for attention deficit hyperactivity disorder (ADHD) and speech and language. The ADHD tool may eventually be combined with the ASD Diagnostic, Happel said.

Other pipeline programs, still in the discovery stage, include an ADHD therapeutic and a diagnostic and therapeutic for anxiety.

Financing

To date, Cognoa has raised about $60 million to advance its behavioral health solutions. It is currently talking with potential investors for a follow-on to its series A financing, with plans to close at the beginning of 2021. Those funds should get it through FDA clearance and commercial launch of the ASD Diagnostic and at least one other pipeline project, Happel said.

The company also plans further enhancements for the ASD Diagnostic, including a Spanish version and eventually more languages to expand its use in OUS markets.

Cognoa will begin working on obtaining CE mark approval after it files its submission with the FDA, Happel said.

23

AI Tool to Diagnose Autism Could Give Concerned Parents a Fast Diagnosis – The startup Cognoa has submitted its app-based tool for FDA clearance

Megan Scudellari
IEEE Spectrum
September 23, 2020

This week, a California-based company announced it will seek FDA clearance for a first-of-its-kind autism spectrum disorder (ASD) diagnostic tool. Cognoa’s technology uses artificial intelligence to make an ASD diagnosis within weeks of signs of concern—far faster than the current standard of care. If cleared by the FDA, it would be the first tool enabling primary care pediatricians to diagnose autism.

The approach is “innovative,” says Robin Goin-Kochel, a clinical autism researcher at Baylor College of Medicine and associate director for research at Texas Children’s Hospital’s Autism Center, who is not affiliated with Cognoa. The field absolutely needs a way to “minimize the time between first concerns about development or behavior and eventual ASD diagnosis,” she adds.

Cognoa’s tool is the latest application of AI to healthcare, a fast-moving field we’ve been tracking at IEEE Spectrum. In many situations, AI tools seek to replace doctors in the prediction or diagnosis of a condition. In this case, however, the application of AI could enable more doctors to make a diagnosis of autism, thereby opening a critical bottleneck in children’s healthcare.

While most parents of children with autism notice developmental changes early on, within the first 1 to 3 years of life, the median diagnosis age in the United States is 4.3 years old. That’s because families often wait months, even years, to see a specialist and get a diagnosis. The time lost during that period is critical: Numerous studies show that early intervention, before the syndrome is fully manifest, can reduce the severity of ASD and improve a child’s brain and behavioral development.

Cognoa’s technology comes out of the lab of founder Dennis Wall, an associate professor of pediatrics at the Stanford University School of Medicine. “I went into this with the hope of objectively asking the question: Can we reduce the complexity of the autism diagnostic process without loss of accuracy?” says Wall.

By feeding electronic health record data into a set of algorithms, Wall’s team was able to distinguish particular features central to an ASD diagnosis, including social and emotional traits such as smiling in response to another person’s smile, joint attention at an object, creativity, and imagination.

The team’s ASD diagnostic seeks to capture those features with three modules: a parent survey, home videos, and a clinician questionnaire.

David Happel, CEO of Cognoa, explains how the tool works: When a parent expresses concern at a pediatrician appointment, or a child fails an ASD screening questionnaire, the pediatrician gives the parent a code to access Cognoa’s app on their smartphone. Once in the app, the parent answers a 15-minute questionnaire about their child’s behavioral patterns, then uploads two home videos of the child, 1 to 2 minutes in length, capturing the child’s behavior in a natural environment. The videos are sent to a trained Cognoa professional who reviews them and answers pertinent questions. Those answers are fed into Cognoa’s AI along with the parent answers and a short questionnaire filled out by the pediatrician. Then, the algorithm sends a result to the pediatrician, and the pediatrician renders a diagnosis.

The tool’s algorithms are trained on data from hundreds of real cases across genders, races and ethnic backgrounds, says Happel. “It has proven to not only accelerate the time of diagnosis, but also remove a lot of the biases that are inherently in place in the current system.” Today’s standard ASD diagnostic tools, Happel notes, were constructed with health data from young Caucasian boys, so girls and children of non-white backgrounds are not well recognized by the tools, contributing to delays in diagnosis for those groups.

In a Cognoa study published in March, which demonstrated an earlier version of the tool, the ASD diagnostic out-performed current screening tools for autism. Today’s screening tools include questionnaires answered by a parent, teacher, or clinician that flag some at-risk children.

More recently, the company completed a pivotal, double-blind clinical trial at 14 sites around the United States. The trial involved 425 participants, aged between 18 and 72 months, whose parents or doctors had expressed concern about their development but had not previously been evaluated for ASD, according to a press release. Each child was assessed twice: once using Cognoa’s tool, and once by a specialist clinician based on DSM-5 criteria, whose diagnosis was validated by a second specialist clinician.

The results of the pivotal trial are not yet published, so there is no specific data to report, but the company says the trial “surpassed its targeted benchmarks”—as agreed upon with the FDA—and was accurate across genders and races. Uniquely, the study ran from July 2019 through May 2020, so some of the children were evaluated remotely this spring during the pandemic via telemedicine. The tool performed equally as well when administered remotely, says Happel.

The company plans to submit the full study for publication in coming months. And formal submission to the FDA will occur shortly, says Happel. He hopes to receive approval in the second half of 2021, then be ready to launch the product into the hands of pediatricians two months after that.

If and when Cognoa’s technology or others become available, “it will be really important to understand the plan for how new tools and technologies will be implemented in primary pediatric care,” says Goin-Kochel. Doctors are often slow to adopt new models, especially for making diagnoses they may not feel comfortable making, she notes, and new technologies raise practical questions such as when they should be applied and whether insurance companies will pay.

“I’m very hopeful there is a near-term future where this product is available, covered by insurance, and made available to everybody as immediately as possible,” says Wall. He is now working on several technology-assisted therapies for ASD, including a project with Cognoa using Google Glass as part of a behavioral therapy to help children with autism recognize emotion.

17

Kezar Highlights Data from MISSION Phase 1b Study of KZR-616 During the Pan American Congress of Rheumatology

Contify Life Science News

SOUTH SAN FRANCISCO, California — Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncologic disorders, today highlighted data from the Phase 1b portion of the MISSION study demonstrating safety, tolerability and early efficacy signals of KZR-616 in patients with systemic lupus erythematosus (SLE) and lupus nephritis (LN) at the Pan-American Congress of Rheumatology (PANLAR 2020). The data were presented in a poster titled “Treatment of Systemic Lupus Erythematosus with the Immunoproteasome Inhibitor KZR-616: Results from the First 5 Cohorts of the MISSION Study, an Open-label Phase 1b Dose-Escalation Study” by study investigator Richard Furie, M.D., Chief, Division of Rheumatology, Northwell Health in New York. The poster can be found on Kezar’s corporate website under the “Science” section.

“Lupus and lupus nephritis are life-threatening diseases that disproportionately impact young Latina women in the prime of their life, and there is an urgent need for new treatment options that can target the full spectrum of their disease and don’t cause debilitating side effects that add to the disease burden,” said Dr. Furie. “These encouraging early positive data suggest that the novel mechanism of KZR-616 has the potential to address the underlying drivers of inflammation, resulting in improvements across organ systems in this disease.”

MISSION is a Phase 1b/2 study of KZR-616 in SLE patients with and without nephritis. The Phase 1b portion has completed enrollment in the final cohort, which is evaluating a 75 mg dose of KZR-616. The Phase 2 portion exclusively in LN is actively enrolling.

As of the May 4, 2020 data analysis, 39 patients were enrolled in the MISSION Phase 1b study across five dose cohorts evaluating 45 mg and step-up dosing to 60 mg weekly for 13 weeks. Patients are followed to week 25 and kept on stable background treatment. At this time point, 22 patients completed 13 weeks of treatment and are included in the exploratory efficacy measures reported below:

* Patients with increased DNA antibodies (serologic markers of SLE disease activity) at baseline that completed through week 25 of the study showed decreased titers following treatment.

As previously reported:

* Notably, two of two patients with active proliferative LN, despite being on stable background therapy, saw a greater than 50% decrease from baseline in proteinuria, a biomarker of disease severity. Both patients also experienced reductions in SLEDAI-2K and reductions in anti-dsDNA (double-stranded DNA) antibody levels.

* Among patients completing treatment, all seven measures of disease activity improved (decrease in score) in the majority of patients from Baseline to Week 13. Improvement in disease activity persisted following the end-of-treatment.

* Step-up dosing of KZR-616 improved overall tolerability. Most patients had mild (87.2%) or moderate (30.8%) TEAEs, which occurred early and diminished with later doses. The most common treatment emergent adverse events were transient injection site reactions.

* To date, no patients have discontinued treatment in Cohorts 2b and 2c, which utilize a lyophilized formulation of KZR-616.

About MISSION
MISSION (NCT03393013) is a Phase 1b/2 clinical trial evaluating KZR-616 in SLE patients with and without nephritis. The study consists of two parts. The Phase 1b portion is an open-label dose escalation study which is evaluating doses up to 75 mg of KZR-616 across 6 cohorts, which has completed enrollment. The primary objective of the Ph1b portion of MISSION is to assess safety and tolerability. Secondary objectives include evaluating pharmacokinetics (PK) and pharmacodynamics (PD) and selecting dose levels for the Phase 2 trials. Several exploratory efficacy measures are also being assessed: Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K), Cutaneous Lupus Erythematosus Severity Index-Activity (CLASI-A), Tender and Swollen Joint Counts (TJC/SJC), Physician Global Assessment (PhGA), Patient Global Assessment (PtGA) and Patient Assessment of Pain (PtP). The Phase 2 portion of the MISSION study evaluating KZR-616 in patients with LN is currently enrolling.

About KZR-616

KZR-616 is a novel, first-in-class, selective immunoproteasome inhibitor with broad therapeutic potential across multiple autoimmune diseases. Preclinical research demonstrates that selective immunoproteasome inhibition results in a broad anti-inflammatory response in animal models of several autoimmune diseases, while avoiding immunosuppression. Data generated from Phase 1a and 1b trials provide evidence that KZR-616 exhibits a favorable safety and tolerability profile for development in severe, chronic autoimmune diseases. Phase 2 trials are underway in severe autoimmune diseases.

About Kezar Life Sciences
Based in South San Francisco, Kezar Life Sciences is combining courage, conviction and cutting-edge science to develop breakthrough treatments for immune-mediated and oncologic disorders. The company is pioneering first-in-class, small-molecule therapies that harness master regulators of cellular function and inhibit multiple drivers of disease via a single target. KZR-616, a first-in-class selective immunoproteasome inhibitor, is being evaluated in severe and underserved autoimmune diseases. Additionally, KZR-261, the first clinical candidate for the treatment of cancer from the company’s protein secretion program targeting the Sec61 translocon, is undergoing IND-enabling activities. For more information, visit www.kezarlifesciences.com, and follow us on Twitter at @KezarBio, Facebook and LinkedIn.

Source: Kezar Life Sciences, Inc.

Apnimed Successfully Completes Phase 1 Study in Lead Program for Obstructive Sleep Apnea

– Data show favorable safety and pharmacokinetic profile for key component of AD109
– Lead program AD109 advancing into a Phase 2 study before year end

CAMBRIDGE, Mass. September 17, 2020 – (BUSINESSWIRE) – Apnimed, a clinical-stage pharmaceutical company focused on developing medicines to treat sleep apnea and related disorders, today announced the results of a Phase 1 study for a component of the company’s lead combination drug candidate, AD109, which is being explored as a pharmacologic treatment for Obstructive Sleep Apnea (OSA). In this healthy volunteer study, R-oxybutynin, a key component of AD109, demonstrated a favorable pharmacokinetic (PK) profile and was well tolerated with no adverse events related to the study drug.

“Obstructive Sleep Apnea represents a significant public health problem in the U.S. and around the globe and current treatment options do not meet the needs of patients,” said Larry Miller, M.D., chief executive officer of Apnimed. “We believe that AD109, an oral drug candidate dosed once-daily at bedtime, could be a significant breakthrough for these patients by giving them a simple, safe, and effective solution that does not require a CPAP device or surgery. The results from this study support our advancement of this program.”

Miller continued by saying, “we look forward to initiating a Phase 2 study with AD109 in Q4 of this year.”

About the study and results
AD109 consists of the norepinephrine reuptake inhibitor (NRI) atomoxetine combined with the antimuscarinic R-oxybutynin. The study, called APC-001, was a Phase 1, randomized, single-dose, open-label, two-way crossover study conducted to evaluate the safety, tolerability, and PK of 2.5 mg of R-oxybutynin compared to 5 mg of racemic (R,S) oxybutynin in 24 healthy adult volunteers. While S-oxybutynin has been studied as a bladder antispasmodic, most of the antimuscarinic activity of racemic oxybutynin necessary to activate the upper airway muscles and maintain an open airway during sleep is thought to be mediated by R-oxybutynin. By purifying the R-oxybutynin and removing the S-oxybutynin, unwanted bladder effects can potentially be reduced and a lower dose of R-oxybutynin alone, relative to racemic oxybutynin, may be effective to treat OSA.

Study participants received treatments in random order, administered as a single oral dose, with a washout period between treatments of at least 7 days. The results indicated that R-oxybutynin was well-tolerated and that there were no adverse events related to the study drug. The blood plasma concentrations of the 2.5 mg dose of R-oxybutynin, when administered alone, closely matched the profile of R-oxybutynin when dosed as 5 mg of racemic oxybutynin.

Additionally, no interconversion to S-oxybutynin was observed following administration of 2.5 mg R-oxybutynin.

About AD109
Apnimed’s lead product candidate, AD109, targets neurotransmitter levels in the central nervous system to activate upper airway muscles and maintain an open airway during sleep. AD109 is a first-in-class, oral pharmaceutical combination dosed once-daily at bedtime, designed to treat OSA patients across a broad spectrum of disease severity. The drug is designed to be safe, effective, and convenient, addressing the key limitations of the current standard of care treatments.

Apnimed has successfully completed its first Phase 1 study with AD109 and Phase 2 clinical trials are planned for 4Q 2020. Proof of concept for the AD109 program was demonstrated in Apnimed study APN-002, a Phase 2, parallel group dose-finding study of the combination of atomoxetine and racemic oxybutynin. That study provided evidence of safety and efficacy of a norepinephrine reuptake inhibitor (NRI) + antimuscarinic combination in 140 patients.

About Obstructive Sleep Apnea
Obstructive Sleep Apnea (OSA) is one of the most common and serious sleep disorders and is estimated to affect more than 25 million Americans, though underdiagnosis continues to be a serious problem. OSA is characterized by partial or complete upper airway closure that occurs during sleep, which often leads to poor sleep, and in the long-term, hypertension, diabetes, cardiovascular disease, strokes, and early mortality. The vast majority of diagnosed patients are prescribed positive air pressure therapy devices such as continuous positive airway pressure, or CPAP, but fewer than half are compliant long-term, leaving a significant population untreated, undertreated and at risk.

About Apnimed
Apnimed is a clinical-stage pharmaceutical company working to transform the treatment of sleep apnea based on a simple idea – patients with Obstructive Sleep Apnea will benefit from treatment with a safe and effective oral medication. Apnimed’s lead development program targets nighttime neurotransmitter levels in the central nervous system to activate upper airway muscles and maintain an open airway during sleep. The drug is delivered as a convenient once-daily at bedtime. Based in Cambridge, Mass., the company is developing a portfolio of novel pharmacologic therapies for sleep apnea and related disorders. Learn more at Apnimed.com

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Apic Bio Appoints Dr. Jorge Quiroz as Executive VP and Chief Medical Officer

Professional Services Close-Up
September 8, 2020

Apic Bio reported the appointment of Jorge A. Quiroz, MD, MBA, as Executive Vice President, Chief Medical Officer.

According to a media release, Dr. Quiroz brings over 20 years of scientific, clinical, and regulatory experience helping to develop and evolve gene therapies and small molecule drug programs from preclinical to regulatory filing. Most recently, Dr. Quiroz served as Chief Medical Officer of Solid Biosciences where he was responsible for leading the clinical advancement of its gene therapy program.

“We are excited to welcome Jorge to Apic during this critical period for the company and our pipeline. His extensive expertise in the clinical and regulatory development of gene therapies for rare diseases will support the advancement of both our SOD1 amyotrophic lateral sclerosis (ALS) and alpha-1 antitrypsin deficiency (Alpha-1) programs,” said John Reilly, MS, MBA, Co-founder and Chief Executive Officer of Apic. “As CMO, Jorge will also lead the development and build out of our early-stage gene therapy programs derived from our THRIVE platform. We look forward to working with Jorge during this next chapter for Apic as we rapidly advance our therapies into the clinic on behalf of patients and families in need.”

“I am delighted to join Apic during an exciting period of expansion as we prepare to submit an Investigational New Drug (IND) application for APB-102 for the treatment of patients with SOD1 ALS this year and enter the IND-enabling stage for APB-101 for the treatment of patients with alpha-1 antitrypsin deficiency,” said Dr. Jorge Quiroz, EVP and Chief Medical Officer of Apic. “The Company’s mission, deep scientific foundation, clinical approach, and manufacturing expertise puts us in an excellent position to bring new gene therapy treatments to patients living with rare and monogenic disorders.”

Dr. Quiroz previously served as the Head of Neurodevelopment & Psychiatry, Translational Medicine Neurosciences at F. Hoffmann-La Roche AG. He has also served as a Director at Johnson & Johnson Pharmaceutical Research & Development.

PhoreMost and XtalPi Sign AI-Based Pharmaceutical Drug Discovery Collaboration Agreement

PhoreMost Limited, UK
September 8, 2020

- Collaboration to rapidly develop high-quality leads from novel targets and sites identified by PhoreMost’s proprietary SITESEEKER platform

- XtalPi’s AI-based ID4 platform will explore ultra-large chemical spaces to discover novel inhibitors of protein-protein interaction sites

PhoreMost Limited, the UK-based biopharmaceutical company dedicated to drugging ‘undruggable’ disease targets, and XtalPi Inc., a leading algorithm-driven artificial intelligence (AI)-based pharmaceutical technology company, today announced they have entered into a drug discovery collaboration agreement. Financial details are not disclosed.

Under the terms of agreement, PhoreMost and XtalPi will rapidly identify and develop compounds to advance a drug discovery program against targets that epigenetically regulate tumour progression, and have been previously classified as ‘undruggable’. The companies will pursue this challenging goal with a unique combination of machine learning and physics-based computation methods, using XtalPi’s intelligent digital drug discovery and development (ID4) platform, to explore novel sites discovered by PhoreMost’s SITESEEKER® platform.

PhoreMost’s SITESEEKER platform exploits protein shape diversity to find new peptide targets, significantly enhancing the power of phenotypic screening and translation into therapeutic modalities. Based on proprietary protein interference, or ‘PROTEINi®’, technology, PhoreMost uses SITESEEKER to probe the entire proteome in a live cell environment for novel druggable targets linked to any chosen disease. This enables the systematic discovery of functionally active peptides which are directly linked to useful therapeutic applications.

Protein-protein interaction sites are traditionally considered ’undruggable’ by small-molecules due to their large flat surface and the absence of well-defined binding pockets. XtalPi’s ID4 platform integrates data-driven AI models with physics-based methods to quickly distil a small number of high-quality compounds from an AI-generated target-specific chemical space that includes millions of diverse molecules. Such diversity, combined with ID4’s drug property prediction algorithms’ speed and accuracy, allows scientists to unearth promising candidates, overcome the bottleneck of simultaneously optimising all pharmacological properties, and ensure a smooth progression into pre-clinical and clinical studies.

“XtalPi’s AI-based ID4 and PhoreMost’s SITESEEKER are highly complementary platforms, and we look forward to working together with the team. This alliance marks an exciting chapter in PhoreMost’s development, as we now seek to rapidly progress our internal portfolio of novel first-in-class targets into drug discovery.”
- Dr. Chris Torrance, CEO of PhoreMost

“Today, many diseases lack effective treatment because their corresponding targets are too challenging for traditional drug discovery methods. We are excited to combine PhoreMost’s target discovery technology with our ability to quickly identify lead compounds with desirable drug properties, and continuously translate high-quality ‘undruggable’ targets into first-in-class pipeline assets to address unmet medical needs.”
- Dr. Jian Ma, Co-Founder and CEO of XTALPI

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Amylyx Pharmaceuticals Announces New England Journal of Medicine Publication of Pivotal AMX0035 Data Demonstrating Statistically Significant Benefit in People with ALS

- Patients Retained Function Longer on AMX0035 Versus Placebo; Study Achieved Its Primary Outcome of a Difference on the Revised ALS Functional Rating Scale

- AMX0035 is the First Investigational Therapy to Demonstrate Statistically Significant Benefit on this Prespecified Primary Outcome in People with ALS Since Approved Therapy Edaravone

- AMX0035 Showed Numerical Benefits on Secondary Outcomes Including Measures of Muscle Strength, Breathing, and Hospitalizations

- AMX0035 Was Generally Well Tolerated with Similar Rates of Adverse Events Recorded in the AMX0035 and Placebo Groups

September 02, 2020

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, today announced the publication of results from the pivotal CENTAUR trial evaluating AMX0035 – an investigational neuroprotective therapy designed to reduce the death and dysfunction of motor neurons – in people with ALS in the New England Journal of Medicine (NEJM).

“CENTAUR met its prespecified primary outcome, showing a clinically meaningful and statistically significant treatment benefit on the Revised ALS Functional Rating Scale (ALSFRS-R), the most commonly used scale in clinics worldwide to measure function in ALS,” said Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR trial, investigator at the Sean M. Healey & AMG Center for ALS at Mass General and Assistant Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital. “These results represent a major milestone for the ALS community, and I am thrilled about the promise of this therapy for people with ALS.”

The CENTAUR trial of 137 individuals with ALS was conducted across 25 top medical centers in the U.S. through the Northeast ALS (NEALS) consortium. It demonstrated that treatment with AMX0035 was well tolerated and decreased the rate of decline in the ALSFRS-R compared to placebo in people with ALS.

“The data published today in the New England Journal of Medicine show that AMX0035 demonstrated a clinically meaningful benefit and a favorable safety profile for people living with ALS. This development is a breakthrough for the ALS community and we are working collaboratively and expeditiously with agencies worldwide to bring this potential new treatment option forward,” said Josh Cohen, Co-CEO, Chairman and Co-Founder at Amylyx.

“The data published today makes a clear and compelling case that AMX0035 should be made available to people with ALS as soon as possible,” said Calaneet Balas, President and CEO of The ALS Association. “We look forward to working with Amylyx, the FDA, and the entire community to help make that happen. We are grateful to all the Ice Bucket Challenge donors whose contributions helped make this trial possible.”

CENTAUR RESULTS:

Primary Outcomes:

After 24 weeks, patients treated with AMX0035 scored on average 2.32 points higher on the ALSFRS-R than the placebo group (p=0.03) using the study’s primary prespecified analysis. A change from baseline analysis was also conducted and indicated that the AMX0035 group scored 2.92 points higher at the end of 24 week follow up (p=0.01).
- The ALSFRS-R is a 48-point questionnaire measuring daily functions such as the ability to walk, dress independently, self-feed, speak and breathe.
- Just a 1-2 point change in the ALSFRS-R score can indicate a significant reduction in a patient’s ability to function independently. The ALSFRS-R measures many different daily functions so point differences can manifest differently in different patients. Some examples of a two point change on this scale include the difference between an individual eating successfully with some difficulty vs needing a feeding tube, or walking with assistance versus not walking at all.

Secondary Outcomes:

In line with the primary outcome, patients on AMX0035 also showed numerical benefits on secondary outcomes including measures of muscle strength, breathing and hospitalization frequency, although the study was not powered for secondary outcomes.
- Progression in lung function (percent predicted slow vital capacity) was numerically slower in patients taking AMX0035 (Least Squares Difference=5.11 points, p=0.08).
- Participants in the AMX0035 group were hospitalized numerically less often (Hazard Ratio = 0.54, p=0.09).
- Rate of decline in overall muscle strength (percent predicted ATLIS) was numerically slower in patients taking AMX0035 (Least Squares Difference=2.82 points, p=0.12). The effect of AMX0035 on progression was nominally statistically significant for the upper limbs measurements (Least Squares Difference=4.27 points, p=0.04).

Overall Safety:

- Nearly all participants (46 out of 48 patients in placebo (96%) vs 86 out of 89 patients in the AMX0035 group (97%)) reported one or more treatment-emergent adverse events (TEAEs) during the trial. Most were nonserious, did not lead to modification or interruption of study drug dosing, and were not considered related to the study.
- Overall, safety was comparable between the groups, with fewer serious adverse events in the active group as compared to the placebo group (9 out of 48 patients (19%) in placebo vs 11 out of 89 patients (12%) in the AMX0035 group).
- GI adverse events, which were generally characterized as mild by investigators, occurred more frequently in the active group in the first 3 weeks of the trial (28.1% vs 12.5% placebo) and returned to levels comparable to placebo thereafter.

Most CENTAUR participants (77%) were receiving an approved ALS therapy (riluzole, edaravone, or both) during and/or before trial entry. Sensitivity analyses accounting for the duration of treatment under riluzole, edaravone, or both confirmed that the treatment effect of AMX0035 was independent of background approved ALS therapies.

CENTAUR was the recipient of the ALS ACT grant, and is supported by The ALS Association, ALS Finding a Cure, a program of The Leandro P. Rizzuto Foundation, the Northeast ALS Consortium, Healey Center for ALS at Mass General, and was funded in part by the ALS Ice Bucket Challenge.

“I am so proud of the ALS community efforts that made this milestone possible,” said Dr. Merit Cudkowicz, M.D., Chief Medical Officer from ALS Finding a Cure®, Director of the Sean M. Healey & AMG Center for ALS, Chief of Neurology at Mass General, and the Julianne Dorn Professor of Neurology at Harvard Medical School. “The CENTAUR study was designed and run through NEALS, was supported by a partnership between The ALS Association and ALS Finding a Cure, and is a phenomenal example of what can happen when a community works closely together to accelerate ALS progress.”

AMX0035 Long-Term Survival and Extension Data, Future Plans

Participants who completed CENTAUR were given the option after the trial to enroll in an open-label extension study and receive AMX0035 long-term. 92% of eligible CENTAUR participants elected to enroll in the extension study. Interim data from the ongoing open-label extension study are being submitted to a peer-reviewed journal shortly and will be published in the coming months.

Long-term survival analysis for the patients in the AMX0035 and placebo groups has been conducted as well. These data will be submitted to a peer-reviewed journal in the near future.

“We are deeply grateful to all of the CENTAUR participants and our partners who have helped and continue to help develop this important therapy for those living with ALS,” said Justin Klee, Co-CEO and Co-Founder of Amylyx. “We also look forward in the coming months to sharing results from the CENTAUR open-label extension study and the long-term survival analysis, and we will continue to keep the community closely informed on next steps.”

About CENTAUR

CENTAUR was a 24-week, randomized, double-blind, placebo-controlled Phase 2/3 clinical trial that evaluated the safety and tolerability of AMX0035 and assessed the drug’s impact on disease progression as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) when compared to placebo. The trial also assessed the effects of AMX0035 on other measures that are critical to people with ALS, including muscle strength, lung vital capacity, and biomarkers of neuronal degeneration.

CENTAUR enrolled patients 18-80 years old with definite ALS and within 18 months of symptom onset. The trial did not restrict patients from receiving edaravone or riluzole. More information on the CENTAUR trial can be found at https://amylyx.com/trials/ or www.clinicaltrials.gov, NCT03127514.

About Amyotrophic Lateral Sclerosis (ALS)

ALS is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually death. The vast majority of patients with ALS (>90%) have sporadic disease, showing no clear family history. Approximately 6000 people are diagnosed with ALS in the United States every year with an approximately similar number of deaths every year.

About AMX0035

AMX0035 is an investigational neuroprotective therapy designed to reduce neuronal death and dysfunction. AMX0035 targets endoplasmic reticulum and mitochondrial dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases.

About Amylyx Pharmaceuticals

Amylyx Pharmaceuticals, Inc. is a pharmaceutical company working on developing a novel therapeutic for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases. For more information, visit www.amylyx.com and follow us on LinkedIn and Twitter.

1

ViOptix Inc Awarded Patent for Oximeter with Marking Feature

Global IP News – Medical Patent News
September 1, 2020

ViOptix Inc has been awarded a patent for oximeter with marking feature. This invention was developed by Heaton II Larry C and Lash Robert E. The patent application number is 15/904,299. The International Patent Classification codes are A61B 5/00 (20060101), A61B 5/1455 (20060101) and A61B 90/30 (20160101). Cooperative Patent Classification codes are A61B 5/7278 (20130101), A61B 5/14552 (20130101), A61B 5/742 (20130101), A61B 90/30 (20160201), A61B 5/7282 (20130101), A61B 2090/309 (20160201) and A61B 2090/308 (20160201).

The abstract released by U.S. Patent and Trademark Office states, “A medical device such as an oximeter includes a marking feature. In an implementation, a marking mechanism of the device marks tissue based on a location of where a measurement was taken by the device. In an implementation, the marking mechanism of the device marks tissue based on an oxygen saturation measurement obtained by the device.”

Archive

November 2020

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Amylyx Pharmaceuticals Announces FDA Granted Orphan Drug Designation for AMX0035 for the Treatment of Wolfram Syndrome

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AMX0035 for the treatment of Wolfram syndrome. "There is a critical unmet need for people living with Wolfram syndrome, a rare genetic disease in which many patients die prematurely with severe neurological disabilities," said Wolfram syndrome expert Fumihiko Urano, M.D., Ph.D., a Professor of Medicine and of Pathology and Immunology at Washington University School of Medicine in St. Louis. "Endoplasmic reticulum (ER) dysfunction is a critical component of Wolfram syndrome. AMX0035 is designed to target the ER stress, and preclinical data suggest that it may be a promising approach to halt the irreversible progression of optic nerve atrophy in patients with Wolfram syndrome." The FDA may grant orphan designation to drugs and biologics intended to treat a rare disease or condition affecting fewer than 200,000 persons in the U.S. Orphan designation qualifies a company for certain benefits, including financial incentives to support clinical development and the potential for seven years of market exclusivity in the U.S. upon regulatory approval. "We are grateful to the advocacy groups, Dr. Urano and his team, and the dedicated parents who are working with us to determine if AMX0035 can help patients and families affected by Wolfram syndrome," said Kent Leslie, Global Head of R&D and Chief Scientific Officer at Amylyx. "Orphan drug designation will support us as we explore AMX0035 in treating this rare and underserved population living with a life-threatening disease that has no approved therapies. This designation is an important milestone as we continue to develop AMX0035 for the potential treatment of neurodegenerative diseases. We look forward to continuing our evaluation of AMX0035 in animal models of Wolfram syndrome and, hopefully soon, in patients." AMX0035 is currently being investigated in people living with ALS and Alzheimer's disease. About Wolfram Syndrome Wolfram syndrome is an autosomal recessive neurodegenerative disease characterized by childhood-onset diabetes, optic nerve atrophy, and neurodegeneration. Common manifestations of Wolfram syndrome include diabetes mellitus, optic nerve atrophy, central diabetes insipidus, sensorineural deafness, neurogenic bladder, and progressive neurologic difficulties. Genetic and experimental evidence suggest that endoplasmic reticulum (ER) dysfunction is a critical pathogenic component of Wolfram syndrome. The prognosis of Wolfram syndrome is poor, and many patients die prematurely with severe neurological disabilities. To learn more about Wolfram syndrome, please visit: Ellie White Foundation , Eye Hope Foundation , Snow Foundation , and Wolfram Syndrome Research Alliance . About AMX0035 AMX0035 is an investigational product designed to reduce neuronal death and dysfunction. AMX0035 targets endoplasmic reticulum and mitochondrial dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases. About Amylyx Pharmaceuticals Amylyx Pharmaceuticals, Inc. is a pharmaceutical company working on developing a novel therapeutic for amyotrophic lateral sclerosis (ALS), Alzheimer's disease and other neurodegenerative diseases. For more information, visit www.amylyx.com and follow us on LinkedIn and Twitter

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InCarda Therapeutics Presents Positive New Data from Phase 2 Study of InRhythm™ in Patients with Paroxysmal Atrial Fibrillation at American Heart Association Scientific Sessions 2020

- Data Demonstrate Achievement of Rapid Conversions from Atrial Fibrillation to Normal Sinus Rhythm - Study Findings Provide Proof of Concept for First-of-its-Kind Inhaled Antiarrhythmic for Treatment of Paroxysmal Atrial Fibrillation (PAF) - Provides Roadmap for Start of Pivotal Phase 3 in Medically Supervised Setting in 2021 San Francisco, CA – InCarda Therapeutics, Inc. (“InCarda”), a privately-held biopharmaceutical company developing first-of-their-kind inhaled therapies for cardiovascular diseases today announced the presentation of positive data from the open-label, dose-escalation Part A portion of the company’s multinational INSTANT Phase 2 clinical trial of InRhythm™ (flecainide for inhalation) in patients with recent-onset paroxysmal atrial fibrillation (PAF) at the American Heart Association’s (AHA) Scientific Sessions 2020. The positive study results reported today during the AHA conference provide the first proof of concept for inhaled flecainide as a potentially safe and effective therapeutic option for rapidly converting PAF to normal sinus rhythm (NSR). InRhythm is a novel inhaled therapeutic candidate designed to rapidly deliver flecainide, a well-established antiarrhythmic agent, to the heart via the lungs to restore NSR and to relieve symptoms associated with acute episodes of PAF. The therapy is being developed initially for its use under medical supervision in a hospital, emergency room or physician office and subsequently as a portable treatment that can be self-administered by patients in a non-medically supervised setting (such as the home) to rapidly achieve conversion of PAF to NSR. Dr. Harry J.G.M. Crijns, professor, chair of cardiology and board member of the Cardiovascular Research Institute Maastricht (CARIM) at Maastricht UMC+, and co-principal investigator of the INSTANT trial, presented data from Part A of the INSTANT trial. The results demonstrated the rapid achievement of therapeutic plasma levels (Cmax > 200 ng/mL) of flecainide via oral inhalation with InRhythm. Approximately 80% of patients who received the study’s highest administered dose (120 mg) achieved a flecainide Cmax > 200 ng/mL. For all study participants who reached these therapeutic plasma levels, nearly 50 percent achieved a successful conversion from PAF to NSR. Importantly, those successful conversions occurred rapidly, with a median time to conversion of 3.6 minutes after the end of the administration of InRhythm (total inhalation time for InRhythm administration is eight minutes). The treatment was shown to be safe and well-tolerated, and the majority of adverse events were transient, mild in severity and resolved without treatment. “There is growing clinical and scientific evidence to support the importance of rhythm control in the overall management of atrial fibrillation,” said Jeremy N. Ruskin, M.D., professor of medicine at Harvard Medical School and founder and director emeritus of the Cardiac Arrhythmia Service at Massachusetts General Hospital and co-principal investigator of the INSTANT study. “Currently available treatment options for the acute conversion of atrial fibrillation to normal sinus rhythm are limited and do not fully address the unmet needs of improving patients’ symptoms and quality of life and reducing healthcare costs. This novel therapy for rapid restoration of normal rhythm, if successful in pivotal clinical trials, has the potential to play a significant role in addressing these unmet clinical needs.” Based on these positive study results, InCarda has commenced enrollment of Part B of the INSTANT study which includes a confirmatory cohort using the selected optimal therapeutic dose (120 mg) and a pilot study referred to as the “Patient-led Sub-study.” In this latter study patients who have already experienced a safe cardioversion with InRhythm will receive training so that they can self-administer InRhythm under medical supervision when another (“recurrent”) episode of PAF occurs. In addition to current study sites in the Netherlands and Belgium, InCarda will start recruiting patients in the US later this year. “The data presented today from the INSTANT study provide the first proof of concept of this novel strategy to deliver flecainide via oral inhalation to rapidly restore NSR in patients with symptomatic episodes of recent-onset PAF. These data will be used to finalize the design of a multinational, Phase 3 trial of InRhythm to support marketing approval for in-hospital, medically supervised use. We expect to initiate this study in the first half of 2021,” said Luiz Belardinelli, M.D., chief medical officer of InCarda. “With six million atrial fibrillation patients in the US contributing to over $26B in annual healthcare expenditures, and more than 30 million atrial fibrillation patients worldwide, there is a tremendous opportunity for InCarda to uniquely address this important and growing need,” said Grace E. Colon, Ph.D., chief executive officer of InCarda. “We look forward to the next steps in the development of this important new therapy and reporting on several key company milestones over the next year.” About Atrial Fibrillation (AF) Atrial fibrillation (AF) is the most common sustained cardiac arrhythmia (abnormal heart rhythm) and is characterized by rapid and irregular heartbeats often resulting in palpitations and other symptoms that can be debilitating. A chronic, progressive condition, AF is estimated to affect six million people in the U.S., with that number expected to double by 20501. This expected increase is partially due to the correlation between AF prevalence and an aging population, with approximately 9% of those aged 65 and older affected by the condition1. AF is associated with significant morbidity and a substantial reduction in quality of life, with the condition potentially resulting in exercise intolerance, congestive heart failure, tachycardia-induced cardiomyopathy and stroke. The annual cost of AF to the U.S. healthcare system is estimated at more than $26 billion1. Paroxysmal AF (PAF) is a type of AF in which episodes occur intermittently and resolve spontaneously in fewer than seven days. Approximately 25% of PAF patients progress to the permanent form of AF within five years2. Common symptoms of PAF can include racing heartbeat, chest pain or pressure, a fluttering feeling in the chest, weakness, fatigue, dizziness, sweating and lightheadedness. Current treatments for patients with PAF rely upon either chronic administration of oral antiarrhythmic drugs or acute hospital-based procedures such as intravenous drug administration and electrical cardioversion, neither of which fully address the unmet need of patients for a rapid-acting treatment that can be administered whenever an episode of PAF occurs. There are currently no approved treatments that can be patient self-administered whenever an episode of PAF occurs. About InRhythm™ InRhythm (flecainide for inhalation) is a novel inhaled therapeutic candidate designed to rapidly deliver flecainide, a well-established antiarrhythmic agent, to the heart via the lungs, to restore normal sinus rhythm (NSR) and relieve the patient’s symptoms following the onset of an episode of PAF. InRhythm is intended to address the unmet need for a non-invasive, rapid-acting treatment that can be administered in a medically supervised setting (initial indication) and, ultimately, self-administered by patients anywhere they happen to be, whenever they experience an episode of PAF. Phase 1 clinical results in healthy volunteers demonstrated that InRhythm rapidly and safely delivered flecainide resulting in ECG changes consistent with the potential to restore NSR in patients with PAF. InCarda is currently conducting the INSTANT Phase 2 trial of InRhythm in patients with recent-onset PAF. InRhythm represents a first-in-class, multi-billion dollar global opportunity to address a significant unmet medical need. About InCarda Therapeutics InCarda Therapeutics, Inc. is a privately-held, clinical-stage biopharmaceutical company developing first-of-their-kind inhaled therapies for acute cardiovascular diseases and conditions. The company is leveraging the ability of inhaled therapy to deliver medicine in the “first pass” to cardiac tissue, presenting a small, but effective dose of drug directly to affected regions of the heart. This permits rapid-onset, lower off-target tissue exposure of the drug, lower exposure to cardiac tissue and, more importantly, has the potential to be patient self-administered in a non-medical setting (e.g., home). InCarda employs a de-risked approach by using approved drugs with a long history of efficacy and safety as candidates for the new dosing paradigm via inhalation. The company’s lead development product, InRhythm, is in Phase 2 development to treat acute episodes of PAF, a prevalent atrial arrhythmia. For more information, please visit: www.incardatherapeutics.com.

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United States Patent for Oximetry Probe with Electronically Selectable Tissue Depth Analysis Issued to Vioptix Inc

Alexandria--(Global IP News)--United States Patent for oximetry probe with electronically selectable tissue depth analysis has been issued to Vioptix Inc. This invention was developed by Bechtel Kate Leeann, Keating Jennifer Elizabeth and Coleridge Scott. The patent application number is 15/493,111.

10

Global Partnership to Introduce a Connected, Point-of-Care Diagnostic Platform for COVID-19 Antigen Testing in Africa

LONDON--(PRNewswire)-- LumiraDx, the next generation point-of-care diagnostic company, announced today a $32 million global partnership with Africa Union, Africa Centres for Disease Control and Prevention (Africa CDC), Africa Medical Supplies Platform (AMSP), Bill and Melinda Gates Foundation (BMGF), and Clinton Health Access Initiative (CHAI) with the support of the COVID-19 Therapeutics Accelerator, The Mastercard Foundation and The Rockefeller Foundation to expand access to fast, accurate and equitable COVID-19 testing in Africa. With Foundations' support, LumiraDx is providing five thousand portable diagnostic instruments and related COVID-19 antigen tests, while AMSP – the single-source platform enabling faster, more transparent and cost-effective access to COVID-19-related critical diagnostics and medical equipment – will supply it equitably across 55 African Union member states, addressing both the immediate critical need for COVID-19 testing and building the foundation for long-term primary care infrastructure. "LumiraDx is committed to meeting the global challenge of delivering fast, accurate, and equitable diagnostic testing for COVID-19 and beyond," said LumiraDx CEO Ron Zwanziger. "We're proud to work with global health NGOs and government leaders to make our high-sensitivity microfluidic based COVID-19 antigen test available across Africa." In studies, the LumiraDx SARS-CoV-2 antigen test demonstrated 97.6% positive agreement versus PCR in patients tested up to 12 days after the onset of symptoms, allowing patients in remote areas a wide window in which to get high sensitivity diagnostic test results. The LumiraDx Platform offers secure, cloud-based connectivity via cell phone, enabling real-time reporting and management of results. Together, the LumiraDx Platform offers a high-quality, connected COVID-19 testing solution while setting up capabilities for broader point-of-care testing in community-care settings in Africa. "Testing technology that offers results in minutes – not days or weeks – is an important part of the African Union's Partnership to Accelerate COVID-19 Testing (PACT) initiative," said Africa CDC Executive Director Dr. John Nkengasong. "We look forward to working together with our global health partners to mobilize experts, community workers, supplies and other resources to minimize the impact of the pandemic on the African continent by testing, tracing, and treating COVID-19 cases in a timely manner." "Through this partnership, our African Union Member States will have access to high-sensitivity point-of-care COVID-19 antigen tests on the AMSP," said African Union Special Envoy Strive Masiyiwa. "The platform was created to bring manufacturers and suppliers together to collectively address the quality, affordability and accessibility challenges facing our communities during the COVID-19 crisis." "We have to double down our efforts and make sure Africa has access to the latest and most performant technologies to protect our people and reopen our economies. The AMSP team has been working relentlessly to launch its COVID-19 antigen offering and we are proud to partner with LumiraDx, Bill & Melinda Gates Foundation, Mastercard Foundation and others to achieve this goal," said Fatoumata Bâ, Founder & Executive Chair of Janngo and Managing Partner of Janngo Capital. "We cannot fight COVID-19 without understanding where the disease is spreading. Access to diagnostics are vital in providing data to equip governments and health professionals to take measures to slow transmission. We are pleased to be working with partners to improve COVID-19 diagnostics and improve primary care capabilities in Africa," said Gates Foundation President of Global Health, Trevor Mundel. About LumiraDx LumiraDx was founded in 2014 by a group of entrepreneurs: Ron Zwanziger, our Chairman and Chief Executive Officer; Dave Scott, Ph.D., our Chief Technology Officer; and Jerry McAleer, Ph.D., our Chief Scientist, who have a successful track record in building and scaling diagnostics businesses over three decades, including at companies such as Medisense, Inc., Inverness Medical Technology Inc. and Alere Inc. The company is supported by institutional and strategic investors including the Bill & Melinda Gates Foundation, Morningside Ventures and U.S. Boston Capital Corporation. Based in the UK and supported by its worldwide affiliates to provide access in all major markets, LumiraDx has over 750 employees worldwide. LumiraDx's Platform simplifies, scales down, and integrates techniques used in laboratory analyzers to provide lab-comparable diagnostic tests on a single point-of-care instrument that can be easily used in community care settings. It's able to deliver a broad menu of tests on a single instrument and guarantees high-quality results at low cost, making it an ideal resource for developing communities as they establish local health care infrastructure. Further information on LumiraDx and the LumiraDx Platform is available at lumiradx.com.

10

Apnimed Further Strengthens Leadership Team and Board of Directors

– Seasoned finance executive Michael Rogers joins as company’s Chief Financial Officer – Former Biotech CEO and senior Merck executive James (Jay) Galeota joins Board of Directors CAMBRIDGE, Mass. – Apnimed, a clinical-stage pharmaceutical company focused on developing medicines to treat sleep apnea and related disorders, today announced it has added two highly experienced executives to help lead the company. Michael Rogers joins Apnimed as Chief Financial Officer and brings more than 25 years of biotechnology experience in both private and public finance management. Additionally, Jay Galeota joins the company’s Board of Directors, and brings more than 30 years of commercial, operational, and company building experience in large pharmaceutical, specialty generics, and biotechnology companies to Apnimed’s Board of Directors. “We are at an exciting inflection point in Apnimed’s development as we have initiated Phase 2 clinical studies with our lead product, AD109, for the treatment of Obstructive Sleep Apnea,” said Larry Miller, M.D., Chief Executive Officer of Apnimed. “Adding Mike to our leadership team and Jay to our Board will bring valuable expertise and insight as we continue to build a leading biotech company to develop the first pharmacologic, disease-modifying treatment for the estimated 25 million Americans who suffer from sleep disorders.” Prior to joining Apnimed as Chief Financial Officer (CFO), Michael Rogers was CFO at Aerpio Pharmaceuticals, Inc., which was focused on advancing first-in-class compounds to treat ocular disease and complications of diabetes. Mr. Rogers has been CFO for numerous companies including Acorda Therapeutics, Inc., BG Medicine, Inc., Indevus Pharmaceuticals, Inc., Advanced Health Corporation, and Autoimmune Inc. Mr. Rogers has organized and led numerous successful financing rounds for both pre-commercial and commercial, and privately and publicly traded companies. He received his B.A. from Union College and his M.B.A. from Darden Graduate School of Business Administration at the University of Virginia. “For years, patients with OSA have been limited to cumbersome or invasive treatment options,” said Michael Rogers, newly appointed Chief Financial Officer at Apnimed. “Apnimed is working on an elegant solution for patients with a once-daily, oral treatment. I look forward to working with the team to help complete a robust set of late-stage trials on an ambitious timeline to fully demonstrate the value of this potentially significant advance.” Jay Galeota was the President and Chief Executive Officer of Inheris Biopharma, Inc., where he transformed the Necktar spinout, helping to build a new, independent pharmaceutical company in less than a year. Prior to Inheris, he was President of G&W Laboratories, Inc. where he oversaw business operations including research and development, commercial, manufacturing, business development, quality, supply chain, information technology, and corporate support functions. Mr. Galeota spent nearly 30 years with Merck & Co. where he held positions of increasing seniority and oversaw the global development and commercialization for multiple therapeutic areas, such as those in the Diabetes and Obesity Franchise. He was also the President of Merck’s Hospital and Specialty Care business line. He completed his career at Merck as Chief Strategy and Business Development officer and President of Emerging Business for the company. He received his B.S. in Biology from Villanova University, is a graduate of Harvard Business School’s Advanced Management Program, and completed Officer Field Training for the United States Air Force, Reserve Officer Training Corps. Mr. Galeota is also a member of Boards of Directors and Committees for multiple public and privately held companies. Jay Galeota, newly appointed Apnimed Board Member, said “Throughout my career I have had the privilege to help develop and commercialize some highly innovative and life-changing medicines for patients. I believe Apnimed’s approach to disease-modifying therapies that address the underlying needs of patients with Obstructive Sleep Apnea and other sleep disorders, could be game changing for the sleep field. I am excited to bring my expertise in managing late-stage clinical and commercial development to the Board.” About Apnimed’s Lead Product – AD109 Apnimed’s lead product candidate, AD109, facilitates the activation of the upper airway dilator muscles to maintain an open airway during sleep. AD109 is a first-in-class, oral pharmaceutical combination dosed once-daily at bedtime, designed to treat OSA patients across a broad spectrum of disease severity. There are currently no medicines to treat the underlying disordered breathing associated with OSA. The drug is designed to be safe, effective, and convenient, addressing the key limitations of the current devices used to treat OSA. About Obstructive Sleep Apnea Obstructive Sleep Apnea (OSA) is one of the most common and serious sleep disorders and is estimated to affect more than 25 million Americans, though underdiagnosis continues to be a serious problem. OSA is characterized by partial or complete upper airway closure that occurs during sleep, which often leads to poor sleep, and in the long-term, hypertension, diabetes, cardiovascular disease, strokes, and early mortality. The vast majority of diagnosed patients are prescribed positive air pressure therapy devices such as continuous positive airway pressure, or CPAP. Fewer than half of all CPAP patients are compliant long-term, leaving a significant population untreated, undertreated and at risk. About Apnimed Apnimed is a clinical-stage pharmaceutical company working to transform the treatment of sleep apnea based on a simple idea – patients with Obstructive Sleep Apnea will benefit from treatment with a safe and effective oral medication. Apnimed’s lead development program targets the neurologic control of upper airway muscles to maintain an open airway during sleep. The drug is delivered as a convenient once-daily at bedtime. Based in Cambridge, Mass., the company is developing a portfolio of novel pharmacologic therapies for sleep apnea and related disorders. Learn more at Apnimed.com

09

Vigeo Therapeutics to Present New Phase 1/2 VT1021 Dose Escalation and Expansion Data at the Society for Immunotherapy of Cancer's 2020 Annual Meeting

- 75% of Patients Who Achieved a Partial Response or Stable Disease Had High Expression of Both CD47 and CD36 - Dose Escalation Complete; Single-Agent VT1021 Demonstrating Favorable Safety Profile and Early Signals of Clinical Activity CAMBRIDGE, Mass.--(PRNewswire)--Vigeo Therapeutics, a clinical-stage immuno-oncology company pioneering novel cancer therapies, today announced that interim clinical data from its ongoing Phase 1/2 study evaluating single-agent activities of VT1021 in patients with advanced solid tumors, will be presented in a poster session at the Society for Immunotherapy of Cancer's (SITC) 2020 Annual Meeting, taking place from November 9-14, 2020. VT1021 is a first-in-class, dual-modulating compound that blocks the CD47 immune checkpoint and activates CD36, stimulating cytotoxic T-cell functions, inducing apoptosis in tumor and endothelial cells, and increasing the phagocytosis of the tumor by M1 macrophages by stimulating the production of thrombospondin-1 (Tsp-1). Vigeo is developing VT1021 as a therapeutic agent across a range of cancers, with a current focus on solid tumors. "The data being presented at SITC are important because they demonstrate that VT1021 as a single-agent has a favorable safety profile, and is showing early signals of clinical activity across a wide variety of solid tumors, including pancreatic cancer and glioblastoma," said Dr. Devalingam Mahalingam, Associate Professor of Medicine at Northwestern University Feinberg School of Medicine, member of the Robert H. Lurie Comprehensive Cancer Center of Northwestern University and lead author of the study. "Notably, in the dose escalation cohort we observed a durable partial response rate in a patient with thymoma and a 43% disease control rate in subjects with tumors expressing both high CD47 and high CD36, which speaks to the potential of this dual-modulating agent in subsets of patients with advanced chemo-refractory solid tumors." Lou Vaickus, MD, FACP, interim Chief Medical Officer at Vigeo, stated, "Patients who have high levels of either CD36 or CD47 have a worse prognosis and a greater likelihood of developing resistance to conventional therapies, and currently there are no therapeutics that simultaneously target both receptors. VT1021 is unique because it stimulates the expression of Tsp-1 which then binds with high affinity to its natural receptors CD47 and CD36, blocking CD47 and the 'do not eat me' signal and activating CD36, leading to a cascade of beneficial changes to the TME that are believed to enhance anti-tumor effects. We are highly encouraged by this new data and look forward to further elucidating the clinical activity of VT1021 in the dose expansion portion of this study and in combination studies slated to begin during the first half of 2021." Key Findings - VT1021 was found to be safe and well tolerated across all doses tested. - PK analysis has demonstrated dose proportionality across all dose levels. - Dose escalation cohort showed preliminary efficacy: Of 28 evaluable patients, one showed partial response (PR) and 11 others achieved stable disease (SD) in 9 different solid tumors with a median of 4.5 prior lines of therapy for a disease control rate of 43%. - Of the 12 patients who achieved PR/SD's in the dose escalation cohort, 9 (75%) have showed high CD36 and high CD47 expression as determined by an immunohistochemical (IHC) assay. - The RP2D of VT1021 has been determined as 11.8mg/kg. Details for the SITC 2020 Presentation Title: A first-in-human Phase 1/2 open label trial evaluating the safety, pharmacology, and preliminary efficacy of VT1021 in subjects with advanced solid tumors Presenter: Devalingam Mahalingam, Associate Professor of Medicine, Feinberg School of Medicine, Northwestern University Session Type: Poster Session Abstract Number: 374 Date and Time: Thursday, November 12 from 4:50–5:20 p.m. EST and Saturday, November 14 from 1–1:30 p.m. EST About VT1021 Vigeo's lead asset, VT1021, is a first-in-class dual modulating compound that blocks the CD47 immune checkpoint and activates CD36, which induces apoptosis and increases the M1:M2 macrophage ratio. VT1021 achieves this through stimulation of thrombospondin-1 (Tsp-1). The goal of these dual-modulating effects is conversion of immuno-suppressive, or "cold," tumors that don't respond to immuno-oncology agents, to immuno-stimulated, or "hot," tumors that are potentially more receptive to immuno-oncology agents. Vigeo is developing VT1021 as a therapeutic agent across a range of cancers, with a current focus on solid tumors. Pre-clinical results have demonstrated that single-agent VT1021 causes tumor regression at both the primary and metastatic sites. Single-agent VT1021 is currently being evaluated in a Phase 1/2, open label, multicenter trial (NCT03364400) that assesses the drug's safety, tolerability, and preliminary anti-tumor efficacy. The trial's dose escalation phase is now concluded, and the dose expansion phase has been initiated. The expansion phase is expected to enroll 75-100 `patients into one of three cohorts: pancreatic cancer, glioblastoma, and a basket cohort with cancers expressing both high CD47 and high CD36. About Vigeo Therapeutics Based in Cambridge, MA, Vigeo Therapeutics is a clinical-stage immuno-oncology company pioneering novel cancer therapies. The company is building a first-in-class drug development pipeline being led by VT1021, its dual-modulating compound that blocks the CD47 immune checkpoint and activates CD36. Single-agent VT1021 is currently being evaluated in a Phase 1/2 clinical trial, and the company expects to initiate combination studies during the first half of 2021. For more information visit vigeotherapeutics.com.

09

PhoreMost and Oxford Biomedica Enter Gene Therapy Discovery Collaboration

- Collaboration to develop next-generation CAR-T cell therapies with improved efficacy and durability - PhoreMost’s SITESEEKER platform will identify active peptides to be deployed within Oxford Biomedica’s LentiVector delivery system Cambridge and Oxford, UK -- PhoreMost Limited, the UK-based biopharmaceutical company dedicated to developing drugs against intractable disease targets, and Oxford Biomedica (UK) Ltd plc (LSE:OXB) (“Oxford Biomedica”), a leading gene and cell therapy group, today announced that they have entered into a discovery collaboration to develop next generation CAR-T cell therapies. Financial details of the agreement are not disclosed. PhoreMost will deploy its in-house expertise and next-generation phenotypic screening platform, SITESEEKER®, to identify therapeutic candidates for Oxford Biomedica’s LentiVector® gene therapy delivery system. The programme will initially focus on CAR-T therapy and aims to develop next generation cell therapies with significantly improved efficacy and durability. "This collaboration with Oxford Biomedica, a global pioneer in cell and gene therapies, is further recognition of the power of SITESEEKER, offering an exciting opportunity to discover and accelerate the development of clinical stage products. The natural complementarity between SITESEEKER and LentiVector offers great promise for this and future collaborations between the two companies." DR CHRIS TORRANCE, PHOREMOST CEO "We are excited to apply this next-generation technology to our LentiVector platform. The collaboration has the potential to deliver more effective CAR-T therapies, and we look forward to working closely with the PhoreMost team." JOHN DAWSON, OXFORD BIOMEDICA CEO SITESEEKER exploits protein shape diversity to find functionally active peptides linked to any chosen disease setting, significantly enhancing the power of phenotypic screening and translation into therapeutic modalities. Based on proprietary protein interference, or ‘PROTEINi®’, technology, SITESEEKER is able to systematically probe the entire proteome in a live cell environment to identity and exploit novel drug targets. Oxford Biomedica is a world-leading pioneer of cell and gene therapies. Its LentiVector platform enables the successful development of breakthrough gene and cell-based medicines, and through collaborations with pharmaceutical partners, has delivered the first FDA and EMA approved CAR-T cell therapy.

09

Amylyx Pharmaceuticals: Last Patient Wraps Up Final Study Visit in PEGASUS Trial of AMX0035 in Alzheimer's Disease

- Topline Results Expected in the First Half of 2021 CAMBRIDGE, Mass.–(BUSINESS WIRE)–Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, today announced that the last participant has completed the planned 24 weeks in the Phase II PEGASUS trial assessing the safety and biological activity of AMX0035 administration in people with Alzheimer’s disease. “Alzheimer’s is a complex, progressive brain disease defined by amyloid and tau pathologies, but we now realize that there are multiple pathways driving the disease,” said Steven E. Arnold, M.D., Professor and Translational Neurology Head of the Interdisciplinary Brain Center at Massachusetts General Hospital and Harvard Medical School and the PEGASUS trial principal investigator. “The PEGASUS trial design and broad inclusion criteria will allow us to assess whether AMX0035 can prevent neurodegeneration in people with Alzheimer’s via MR imaging and fluid biomarkers. We are immensely thankful to the trial participants and their families for their time and commitment to this critical research effort.” In June 2020, Amylyx announced the PEGASUS trial completed enrollment and dosed 96 participants. Following brief disruptions due to the coronavirus pandemic, including trial site closures, the trial team worked to prioritize patient safety and mitigate delays by capping trial enrollment four patients shy of the planned 100. Amylyx worked with site investigators and clinic teams to facilitate remote visits and expand visit windows when possible. Topline results are targeted for the first half of 2021. The PEGASUS trial will provide data on the safety and tolerability profile of AMX0035 in people with Alzheimer’s disease, its biological activity on biomarkers related to disease processes and preliminary information on cognitive and functional effects of AMX0035. Alzheimer’s disease affects memory, thinking and behavior. Symptoms eventually become severe enough to interfere with daily tasks. Alzheimer’s is a slow but relentlessly progressive disease. On average, a person with Alzheimer’s lives four to eight years after diagnosis, but can live up to 20 years, depending on other factors. Alzheimer’s disease is the sixth leading cause of death in the United States. “Worldwide, 50 million people are awaiting new treatments for Alzheimer’s disease, one of the most critically unmet healthcare needs today,” said Dr. Rudolph Tanzi, Ph.D., Kennedy Professor of Neurology, Harvard Medical School, Vice-Chair of Neurology, Massachusetts General Hospital, Founding Chair of the Amylyx Scientific Advisory Board. “The PEGASUS trial will assess the impact of investigational product AMX0035 on biomarkers in people with Alzheimer’s and hopefully lead to a future treatment. We are greatly looking forward to the data in the first half of 2021.” PEGASUS was supported by the Alzheimer’s Combination Therapy Opportunities (ACTO) program, a joint research funding initiative supported by the Alzheimer’s Association and the Alzheimer’s Drug Discovery Foundation, and Cure’s Alzheimer’s Fund. “We feel tremendous responsibility to all those living with Alzheimer’s disease, their families and loved ones,” said Kent Leslie, Chief Scientific Officer at Amylyx. “Amylyx is committed to rigorous scientific research to improve people’s health, and we thank all patients, investigators, site teams, the Alzheimer’s Association, the Alzheimer’s Drug Discovery Foundation and Cure Alzheimer’s Fund for their contributions to the PEGASUS study milestone. We look forward to sharing the topline results of the trial in the first half of 2021.” About PEGASUS PEGASUS (NCT03533257) is a 3:2 randomized, double-blind, multi-center, placebo-controlled study evaluating the safety, tolerability and activity of AMX0035 in patients with late mild cognitive impairment or early dementia due to Alzheimer’s disease over 24 weeks. PEGASUS is designed to yield deep phenotyping insight for the purposes of demonstrating the effects of AMX0035 on mechanistic targets of engagement and disease biology. The study will evaluate diverse disease-relevant markers and produce an informative dataset that will allow for evaluation and correlation of imaging-based markers, neurobiological changes, functional measures, and cognitive outcomes. About AMX0035 AMX0035 is an investigational product designed to reduce neuronal death and dysfunction. AMX0035 targets endoplasmic reticulum and mitochondrial dependent neuronal degeneration pathways in amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases. About Amylyx Pharmaceuticals Amylyx Pharmaceuticals, Inc. is a pharmaceutical company working on developing a novel therapeutic for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases. For more information, visit www.amylyx.com and follow us on LinkedIn and Twitter.

05

Apellis to Present New Data Supporting the Efficacy and Safety of Pegcetacoplan in PNH at the American Society of Hematology Annual Meeting

- Eight abstracts accepted for presentation emphasize the potential of targeted C3 therapy to elevate the standard of care in paroxysmal nocturnal hemoglobinuria (PNH) - New analyses from the Phase 3 PEGASUS head-to-head study demonstrate greater treatment response and quality-of-life improvements with pegcetacoplan versus eculizumab, a C5 inhibitor - Using a matching-adjusted indirect comparison (MAIC), improvements in clinical, hematological and quality-of-life outcomes were demonstrated in patients treated with pegcetacoplan compared to ravulizumab, a long-acting C5 inhibitor WALTHAM, Mass.--(GLOBE NEWSWIRE)--Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that eight abstracts were accepted for presentation at the virtual American Society of Hematology (ASH) Annual Meeting to be held December 5-8, 2020. Data support the positive efficacy and safety of pegcetacoplan, a targeted C3 therapy, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Highlights include new analyses from the Phase 3 head-to-head PEGASUS study, which demonstrate a markedly greater proportion of patients achieved better hematological responses as well as quality-of-life improvements with pegcetacoplan versus eculizumab, a C5 inhibitor. Additionally, using a matching-adjusted indirect comparison (MAIC) methodology, patients treated with pegcetacoplan enrolled in the PEGASUS study experienced greater improvements in hemoglobin stabilization, transfusion avoidance and fatigue compared to patients treated with ravulizumab, a long-acting C5 inhibitor, using the data from the ALXN1210-PNH-302 published study. In the absence of a clinical head-to-head study, MAIC is a valid and accepted method for comparative effectiveness research used by health technology assessment bodies across the world.1,2 “We continue to see in the analyses that pegcetacoplan demonstrated a substantial improvement over C5 inhibition,” said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. “The breadth of data that we are presenting at ASH this year emphasizes the potential of pegcetacoplan to elevate the standard of care in PNH.” Accepted abstracts regarding pegcetacoplan include: - Results of the PEGASUS Phase 3 Randomized Trial Demonstrating Superiority of the C3 Inhibitor, Pegcetacoplan, Compared to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria – #2579 – December 7, 7:00 a.m. – 3:00 p.m. PT - Categorized Hematologic Response to Pegcetacoplan Versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria: Post Hoc Analysis of Data from a Phase 3 Randomized Trial (PEGASUS) – #2588 – December 7, 7:00 a.m. – 3:00 p.m. PT - Effect of Pegcetacoplan on Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria from the PEGASUS Phase 3 Trial Comparing Pegcetacoplan to Eculizumab – #764 – December 5, 7:00 a.m. – 3:30 p.m. PT - Pegcetacoplan is Superior to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Regardless of Prior Transfusion Requirement – #1681 – December 6, 7:00 a.m. – 3:30 p.m. PT - C3 Inhibition with Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria: Results from the PADDOCK and PALOMINO Trials – #753 – December 5, 7:00 a.m. – 3:30 p.m. PT - Comparative Effectiveness of Pegcetacoplan Versus Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Previously Treated with Eculizumab: A Matching-Adjusted Indirect Comparison – #2581 – December 7, 7:00 a.m. – 3:00 p.m. PT Accepted abstracts regarding additional data in the PNH population include: - Real-World Eculizumab Dosing Patterns among Patients with Paroxysmal Nocturnal Hemoglobinuria in a US Population – #3412 – December 7, 7:00 a.m. – 3:00 p.m. PT - Real-World Treatment Patterns and Healthcare Resource Utilization (HRU) of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Receiving Eculizumab in a US Population – #3415 – December 7, 7:00 a.m. – 3:00 p.m. PT About the PEGASUS Study The PEGASUS study (APL2-302; NCT03500549) is a multi-center, randomized, active-comparator controlled Phase 3 study in 80 adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of pegcetacoplan compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a hemoglobin level of <10.5 g/dL at the screening visit. During the four-week run-in, patients were dosed with 1080 mg of pegcetacoplan twice weekly (n=41) in addition to their current dose of eculizumab. During the 16-week randomized, controlled period, patients were randomized to receive either 1080 mg of pegcetacoplan twice weekly or their current dose of eculizumab (n=39). All participants completing the randomized controlled period entered the open-label pegcetacoplan treatment period. About the Matching-Adjusted Indirect Comparison Analysis Using a matching-adjusted indirect comparison (MAIC) methodology, individual patient data from the PEGASUS study were compared to aggregate, published results from the ALXN1210-PNH-302 study, which compared ravulizumab and eculizumab among patients with PNH who previously were treated with eculizumab. To adjust for cross-study differences in baseline characteristics, propensity score weighting was used to balance demographic and clinical characteristics. Outcomes assessed from the PEGASUS study at week 16 and the ALXN1210-PNH-302 study at week 26 included transfusion avoidance, number of units transfused, hemoglobin stabilization and change in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score. As with other MAIC analyses, matching may not adjust for all confounding factors due to differences inherent in study design and entry criteria. About Pegcetacoplan (APL-2) Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and the treatment of geographic atrophy, and received orphan drug designation for the treatment of C3G by the FDA and European Medicines Agency. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials. About Paroxysmal Nocturnal Hemoglobinuria (PNH) PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, hemoglobinuria and difficulty breathing (dyspnea). A retrospective analysis shows that, even on eculizumab, approximately 72% of people with PNH have anemia, a key indicator of ongoing hemolysis.3 The analysis also finds that 36% of patients require one or more transfusions a year and 16% require three or more.3 About Apellis Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

03

US Patent Issued to ViOptix for "Determining Tissue Oxygen Saturation with Melanin Correction" 

ALEXANDRIA, VA--(US Fed News)--United States Patent no. 10,820,863, issued on Nov. 3, was assigned to ViOptix Inc. (Newark, CA). "Determining tissue oxygen saturation with melanin correction" was invented by Kate LeeAnn Bechtel (Pleasant Hill, Calif.), Kimberly Merritt Shultz (Mountain View, Calif.), Alex Michael Margiott (Fremont, Calif.) and George Edward Kechter (Peoria, Ill.). According to the abstract* released by the U.S. Patent & Trademark Office: "An oximeter probe that takes into account tissue color (e.g., skin color or melanin content) to improve accuracy when determining oxygen saturation of tissue. Light is transmitted from a light source into tissue having melanin (e.g., eumelanin or pheomelanin). Light reflected from the tissue is received by a detector. A compensation factor is determined to account for absorption due to the melanin. The oximeter uses this compensation factor and determines a melanin-corrected oxygen saturation value." The patent was filed on April 21, 2017, under Application No. 15/494,444.

03

Atea Pharmaceuticals Announces Closing of Initial Public Offering

Full Exercise of the Underwriters’ Overallotment Brings Additional $45 Million BOSTON, Nov. 03, 2020 (GLOBE NEWSWIRE) -- Atea Pharmaceuticals, Inc. (Nasdaq: AVIR) (“Atea”), a clinical-stage biopharmaceutical company focused on discovering, developing and commercializing antiviral therapeutics to improve the lives of patients suffering from life-threatening viral infections, today announced the closing of its initial public offering of 14,375,000 shares of common stock, including the exercise in full by the underwriters of their option to purchase up to 1,875,000 additional shares of common stock, at a public offering price of $24.00 per share. The aggregate gross proceeds to Atea from the offering were $345 million, before deducting underwriting discounts and commissions and other offering expenses. All of the shares in the offering were offered by Atea Pharmaceuticals. Atea’s common stock began trading on the Nasdaq Global Select Market under the ticker symbol “AVIR” on October 30, 2020. J.P. Morgan Securities LLC, Morgan Stanley & Co. LLC, Evercore Group L.L.C. and William Blair & Company, L.L.C. acted as joint book-running managers of the offering. A registration statement on Form S-1 (File No. 333-249404) relating to the offering has been filed with the Securities and Exchange Commission and became effective on October 29, 2020. The offering was made only by means of a prospectus. Copies of the final prospectus relating to the offering may be obtained from: J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: 866-803-9204; Morgan Stanley & Co. LLC, 180 Varick Street, 2nd Floor, New York, NY 10014, Attention: Prospectus Department, or by email at prospectus@morganstanley.com; Evercore Group, L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 35th Floor, New York, NY 10055, by telephone at (888) 474-0200, or by email at ecm.prospectus@evercore.com; or William Blair & Company, L.L.C., Attention: Prospectus Department, 150 North Riverside Plaza, Chicago, IL 60606, by telephone at (800) 621-0687 or by email at prospectus@williamblair.com.

October 2020

30

Atea, Riding Interest in COVID-19 Drugs, Pulls Off One of 2020's Top Biotech IPOs

- Boston biotech Atea Pharmaceuticals, a developer of small molecule drugs for infectious diseases, has raised $300 million in an initial public offering, selling 12.5 million shares at $24 apiece. - The new funding will help Atea advance an antiviral to treat or potentially prevent COVID-19, as well as a portfolio of other medicines for viruses like Dengue and respiratory syncytial virus. The IPO comes seven days after Roche paid the firm $350 million for partial rights to the COVID-19 antiviral AT-527, which is currently in mid-stage testing and could advance to multiple Phase 3 trials next year. - Atea's IPO is the fourth largest U.S. biotech stock offering of 2020 and the eighth biggest since the start of 2018, according to a database compiled by Biopharma Dive. Despite an initial slowdown at the beginning of the pandemic, biotech company IPOs are on a record-setting pace this year, reflecting excitement surrounding the industry's role in developing coronavirus treatments and vaccines. Infectious disease drugs haven't generated nearly the type of interest from IPO investors in recent years as cancer drugs. Of the ten largest offerings in each of the last three years, for example, 14 of those 30 biotechs had an experimental or marketed cancer medicine as their lead program. Just one biotech — AlloVir, which raised $276 million in July — had an infectious disease drug at the forefront of its pipeline, according to Biopharma Dive's IPO database. The coronavirus pandemic, however, increased interest in both vaccines and infectious disease drugs as the industry scrambled to respond. Shares of Moderna and BioNTech, for instance, have surged to all-time highs because of their progress with experimental coronavirus vaccines. CureVac, another developer of a coronavirus shot, has the second highest market value of any biotech to go public in the U.S. this year. Newcomers AlloVir and Vaxcyte, meanwhile, are among the year's better performers, climbing at least 54% from their offering price as of Thursday's market close. Atea, then, appears to have gone public at the right time. Formed in 2014, the company was originally developing AT-527 for hepatitis C infections. In May, it was cleared to begin testing the drug, an oral small molecule that interferes with the RNA of several viruses, against SARS-CoV-2. Atea has since started a Phase 2 trial of 190 patients with moderate disease and one or more risk factors for poor outcomes, with results expected next year. The pivot to coronavirus research has been lucrative for Atea. Since then, the biotech has raised $215 million in a private funding round, added $350 million in cash through the partnership with Roche and now another $300 million through its IPO. Atea claims its antiviral is easy to manufacture and less burdensome to administer earlier in a patient's disease course, when stopping the virus from spreading might help prevent the progression of COVID-19. Gilead's approved COVID-19 drug Veklury, by comparison, must be infused over several days, making it more difficult to administer when it might be most effective. Other, larger companies are also seeking to improve on Veklury. Merck & Co. has an antiviral pill it licensed from Ridgeback Therapeutics in Phase 2 testing. And Pfizer is advancing an intravenously administered antiviral drug. Engineered antibody drugs from Regeneron, Eli Lilly and others could also play a role in treating COVID-19 patients before they need to be hospitalized. The Roche partnership, announced just a week before Atea's IPO, has given the company the backing to run a broad development program. Positive results in the ongoing mid-stage trial could lead to two Phase 3 tests in non-hospitalized patients and those newly exposed to infection. In addition to the $350 million upfront, Roche could pay Atea another $650 million more under the agreement, with $330 million tied to regulatory development milestones. In return, Roche secured international rights to AT-527, and will help manufacture and distribute the drug overseas. Cell therapy developer SQZ Biotechnologies also went public on Friday, raising $71 million. Sixty three biotechs — which Biopharma Dive defines as companies developing human medicines — have raised more than $50 million through a U.S. IPO this year. www.biopharmadive.com

29

IM Therapeutics Launches Phase 1b Trial of Lead Drug IMT-002 in Autoimmunity; Announces Positive Safety Data of Phase 1a Study

- IMT-002 is the first oral genetically targeted drug candidate to be tested in type 1 diabetes patients - Completed Phase 1a study showed no adverse events in all patient groups - Therapeutic approach blocks activity of specific genes known to play a strong role in autoimmune disease development WOBURN, Mass.--(BUSINESS WIRE)--ImmunoMolecular Therapeutics, Inc. (“IM Therapeutics”), a leading innovator in the field of precision medicine for autoimmune diseases, today announced it has launched a Phase 1b study as a multiple ascending dose (MAD) trial. The study is in patients who have type 1 diabetes (T1D), an as yet incurable autoimmune disorder that affects nearly 1.6 million Americans, and who also have a specific genetic trait known as HLA-DQ8, present in about 60% of T1D patients. Variants in certain genes called human leukocyte antigens (HLA) are known to play a strong role in activating an autoimmune response. The Phase 1b trial will enroll 30 patients between the ages of 18 and 45 into four groups, each of which will have six subjects receiving varying doses of IMT-002 over a two-week duration. The remaining six subjects will receive placebo. The study will compare drug versus placebo for safety, tolerability and pharmacokinetic (PK) profiles over the two-week duration of dosing and a follow-up one week after dose completion. In addition, the study will assess pharmacodynamic (PD) activity with a blood-based assay that measures the ability of IMT-002 to block DQ8 presentation of “self-antigens” or the body’s own proteins, such as insulin or gluten, that can result in autoimmunity. In conjunction with the Phase 1b study, IM Therapeutics announced the completion of a Phase 1a study of IMT-002 in healthy subjects. The study recruited 28 volunteers in four different groups, each of which had six subjects on a single ascending dose of IMT-002 and one subject on placebo. The study showed no adverse events in any of the groups. In addition, pharmacokinetic (PK) profiles followed over a 24-hour period after drug administration indicated an average drug half-life of about five hours and measurable levels in blood up to 12 hours. The completion of Phase 1a has helped inform frequency and levels of dosing in the Phase 1b study. “We are encouraged by the favorable safety findings in our first study,” said Nandan Padukone, Ph.D., CEO of IM Therapeutics. “The PK profile of our lead drug candidate indicates a strong possibility of eventually having a once-daily oral therapy for patients with recent diagnosis of T1D, more than half of whom are pediatric patients,” he added. “We will continue to focus on expanding our pipeline of therapeutic candidates for patients impacted by autoimmune disease including celiac, lupus, and rare diseases.” “We look forward to not only showing further safety and tolerability of our drug candidate, but also its potential as a selective inhibitor of DQ8-related autoimmunity,” said Sarah Bird, Ph.D., VP of Clinical Development at IM Therapeutics. “Tailored therapies against known genetic targets can significantly improve patient-centered care in autoimmune disease.” About IMT-002 IMT-002 has been developed as a selective HLA-DQ8 blocker based on extensive computational work, in vitro and in vivo characterization. Previous studies of a tool drug, L-methyldopa, which is FDA-approved for treating hypertension, in a Phase 1b study, showed effective inhibition of DQ8 activity in new onset type 1 diabetes patients who had the DQ8 gene variant. Several in vivo IND studies indicate that IMT-002, which unlike the tool drug, is not metabolized physiologically, has more potency to block DQ8 activity and a favorable safety profile. Extensive preclinical studies of IMT-002 have been completed leading to IND clearance prior to initiation of human clinical trials. About IM Therapeutics IM Therapeutics (http://imtherapeutics.com/) is a clinical stage company developing personalized medicines for autoimmune diseases by building oral drug therapies against human leukocyte antigen (HLA) variants known to confer high risk of disease. The Company’s technology platform develops small molecule drugs using in silico docking of millions of compounds into pockets of an HLA protein where self-antigens may bind to trigger autoimmunity. Selected drug hits are then optimized using proprietary structure-based design and activity screening with cell-based assays for specificity of HLA inhibition. Lead drugs developed against an HLA variant have the ability to block a series of self-antigens and therefore the potential to treat several autoimmune indications related to a selected HLA. The Company is building a broad HLA-targeted pipeline in autoimmune disorders including celiac disease. In June 2019, IM Therapeutics raised $10 million in Series A financing led by Morningside Ventures and the JDRF T1D Fund.

29

Atea Pharmaceuticals Announces Pricing of Initial Public Offering

BOSTON, Oct. 29, 2020 (GLOBE NEWSWIRE) -- Atea Pharmaceuticals, Inc. (Nasdaq: AVIR) (“Atea”), a clinical-stage biopharmaceutical company focused on discovering, developing and commercializing antiviral therapeutics to improve the lives of patients suffering from life-threatening viral infections, today announced the pricing of its initial public offering of 12,500,000 shares of its common stock at a price to the public of $24.00 per share. All of the shares of common stock are being offered by Atea. The gross proceeds from the offering, before deducting underwriting discounts and commissions and estimated offering expenses payable by Atea, are expected to be $300 million, excluding any exercise of the underwriters’ option to purchase additional shares. Atea’s common stock is expected to begin trading on the Nasdaq Global Select Market under the ticker symbol “AVIR” on October 30, 2020. The offering is expected to close on November 3, 2020, subject to satisfaction of customary closing conditions. In addition, Atea has granted the underwriters a 30-day option to purchase up to an additional 1,875,000 shares of Atea’s common stock at the initial public offering price less the underwriting discounts and commissions. J.P. Morgan Securities LLC, Morgan Stanley & Co. LLC, Evercore Group L.L.C. and William Blair & Company, L.L.C. are acting as joint book-running managers of the offering. A registration statement on Form S-1 (File No. 333-249404) relating to the offering has been filed with the Securities and Exchange Commission and became effective on October 29, 2020. The offering was made only by means of a prospectus. Copies of the final prospectus relating to the offering may be obtained from: J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: 866-803-9204; Morgan Stanley & Co. LLC, 180 Varick Street, 2nd Floor, New York, NY 10014, Attention: Prospectus Department, or by email at prospectus@morganstanley.com; Evercore Group, L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 35th Floor, New York, NY 10055, by telephone at (888) 474-0200, or by email at ecm.prospectus@evercore.com; or William Blair & Company, L.L.C., Attention: Prospectus Department, 150 North Riverside Plaza, Chicago, IL 60606, by telephone at (800) 621-0687 or by email at prospectus@williamblair.com. This press release does not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

28

Gracell Biotechnologies Raises $100 Million in Series C Funding to Advance Next Generation CAR-T Cell Therapies

SHANGHAI and SUZHOU, China, Oct. 28, 2020 /PRNewswire/ -- Gracell Biotechnologies Inc. ("Gracell"), a global clinical-stage biopharmaceutical company dedicated to developing highly efficacious and affordable cell therapies for the treatment of cancer, today announced it has secured $100 million in Series C funding. The round is led by Wellington Management Company, OrbiMed and Morningside Ventures, and joined by new investor Vivo Capital. Existing investors Temasek Holdings, Lilly Asia Ventures, OrbiMed and King Star Med LP are also participating. "We are very pleased to expand our investor base with support from a high caliber consortium," said Dr. William Wei Cao, founder, Chairman, and CEO of Gracell, "Our passion is to bring transformative CAR-T cell therapies to a broader group of patients by developing products that are efficacious and can be made widely available." Gracell was founded in 2017 with the mission to overcome the major industry challenges that persist for both autologous and allogeneic CAR-T cell therapy approaches. Gracell has developed two pioneering platforms—FasTCAR and TruUCAR. With FasTCAR, Gracell is able to deliver younger, less exhausted T cells for autologous cell therapies with greater potency and next-day manufacturing (22 to 36 hours). With TruUCAR, Gracell is able to derive T cells from non-HLA-matched healthy donors to generate allogeneic CAR-T cell therapies that are readily available off-the-shelf at lower cost for a broad patient base. Leveraging its FasTCAR and TruUCAR platforms, Gracell is developing a pipeline of autologous and allogeneic cell therapy candidates with the potential to treat both hematologic malignancies and solid tumors. Currently, Gracell's lead FasTCAR autologous product candidate, GC012F, is being studied in an ongoing investigator-initiated Phase 1 trial in China for the treatment of relapsed or refractory multiple myeloma (r/r MM). Its lead TruUCAR allogeneic product candidate, GC027, is being studied in an ongoing investigator-initiated Phase 1 trial in China for the treatment of relapsed or refractory T cell acute lymphoblastic leukemia (r/r T-ALL). Proceeds from the round will be used to fund internal research and development and further advance current clinical programs. Jefferies and Cooley advised in the transaction. About Gracell Gracell Biotechnologies Inc. ("Gracell") is a global clinical-stage biopharmaceutical company dedicated to discovering and developing breakthrough cell therapies. Leveraging its pioneering FasTCAR and TruUCAR technology platforms, Gracell is developing a rich clinical-stage pipeline of multiple autologous and allogeneic product candidates with the potential to overcome major industry challenges that persist with conventional CAR-T therapies, including lengthy manufacturing time, suboptimal production quality, high therapy cost and lack of effective CAR-T therapies for solid tumors.

27

Prilenia Enrolls First Patients into Its PROOF-HD Phase 3 Clinical Trial for Huntington’s Disease in the United States

- PROOF-HD is designed to replicate previous findings of pridopidine demonstrating maintenance of functional capacity in early Huntington’s Disease patients - Conducted in collaboration with the Huntington Study Group, a world leader in clinical research for HD - If successful, PROOF-HD could lead to the registration of pridopidine, one of the few oral drugs currently in clinical development for the treatment of HD. [Naarden, NL, 27 October 2020] Prilenia Therapeutics B.V., a clinical stage biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders, today announces the enrollment of the first patients in the U.S. into PROOF-HD, a global phase 3 clinical trial for Huntington’s Disease (HD). The study is being conducted in collaboration with the Huntington Study Group (HSG). ​Pridopidine Outcome On Function in Huntington’s Disease (PROOF-HD) is a randomized, double-blind, placebo-controlled, phase 3 study evaluating the efficacy and safety of pridopidine 45mg bid in patients with early stage HD. The study will enroll 480 participants aged 25 or older with a clinical diagnosis of adult-onset HD at approximately 60 study centers in the U.S., Canada and Europe. The treatment period will last up to 78 weeks and there will be an optional open-label extension. The study is designed to replicate previous findings that demonstrated pridopidine’s effect in maintaining functional capacity in patients with early HD. The trial treatment dose (45mg bid) has a favorable safety profile based on over 1,000 patient years in previous HD trials. Andrew Feigin, MD, North American Principal Investigator for the PROOF-HD Phase 3 Trial, said: “Huntington’s Disease is a serious condition with no known treatments that slow functional decline. A successful result would be a major advance in our ability to treat HD. I look forward to working with the Prilenia team as we push forward with patient recruitment.” ​Michael R. Hayden, CEO of Prilenia and world-renowned scientist in Huntington’s Disease research, commented: “The design of the PROOF-HD study is based on strong scientific and clinical data, including in vivo target engagement for the selected dose, prior clinical efficacy results and extensive long-term safety data in our target population”. He added: "Enrolling our first patients is a significant milestone that brings hope to others suffering from this devastating disease.” ​Pridopidine is a first in class small molecule developed by Prilenia for the treatment of neurodegenerative disorders such as HD and Amyotrophic Lateral Sclerosis (ALS). Pridopidine is a highly selective Sigma-1 receptor (S1R) agonist. It binds and activates the S1R, a protein that is expressed at high levels within the brain and regulates key cellular pathways, commonly impaired in neurodegeneration. ​ https://huntingtonstudygroup.org/proof-hd/ ​

23

Kezar Life Sciences Gets Orphan Drug Designations for KZR-616 for the Treatment of Polymyositis and Dermatomyositis

SAN FRANCISCO--(BUSINESS WIRE)-- Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncologic disorders, today announced that the U.S. Food and Drug Administration has granted Orphan Drug Designations (ODD) for KZR-616 for the treatment of polymyositis (PM) and dermatomyositis (DM). Both orphan diseases are autoimmune inflammatory myopathies that are chronic and debilitating diseases characterized by marked morbidity and mortality. The estimated prevalence of PM and DM in the United States is up to 51,000 and 71,000, respectively. KZR-616 is a first-in-class selective immunoproteasome inhibitor with the potential to impact multiple drivers of immune-mediated diseases and inflammation. PRESIDIO, a Phase 2 placebo-controlled cross-over clinical trial to evaluate KZR-616 for the treatment of PM and DM, is currently enrolling subjects. “We are very pleased to have received orphan drug designations for both polymyositis and dermatomyositis. This recognition spotlights the significant unmet need for patients living with these autoimmune myopathies. KZR-616 has potential to truly modify the underlying pathophysiology of these two diseases,” said Noreen R. Henig, MD, Kezar’s Chief Medical Officer. “We will share more detail on the preclinical scientific rationale for the use of selective immunoproteasome inhibition to treat these rare and debilitating diseases during the 2020 American College of Rheumatology Conference next month.” Orphan Drug Designation is intended to advance drug development for rare diseases. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States. Orphan Drug Designation can provide certain benefits and incentives for KZR-616, including a period of marketing exclusivity for the first marketing application, if regulatory approval is received for the designated indication, potential tax credits for qualified clinical testing and waiver of certain administrative fees. About KZR-616 KZR-616 is a novel, first-in-class, selective immunoproteasome inhibitor with broad therapeutic potential across multiple autoimmune diseases. Preclinical research demonstrates that selective immunoproteasome inhibition results in a broad anti-inflammatory response in animal models of several autoimmune diseases, while avoiding immunosuppression. Data generated from Phase 1a and 1b trials provide evidence that KZR-616 exhibits a favorable safety and tolerability profile for development in chronic autoimmune diseases. Phase 2 trials are underway in severe autoimmune diseases. About Polymyositis and Dermatomyositis Polymyositis (PM) and Dermatomyositis (DM) are two of the five types of autoimmune myositis diseases. Both are chronic, debilitating, inflammatory autoimmune myopathies that are distinguished by inflammation of the muscles as well as the skin (in DM). Up to ~120,000 people in the United States are living with these severe and progressive inflammatory myopathies that are characterized by marked morbidity and associated mortality. While debilitating muscle weakness is the hallmark of these myopathies, including compromised muscles of respiration, other internal organ system dysfunctions can be equally disabling. The aim of treatment for these diseases is to suppress inflammation, increase muscle strength and prevent long-term damage to muscles and extramuscular organs; however, treatment options are limited for DM, and there are currently no approved treatments for PM. About Kezar Life Sciences Based in South San Francisco, Kezar Life Sciences is combining courage, conviction and cutting-edge science to develop breakthrough treatments for immune-mediated and oncologic disorders. The company is pioneering first-in-class, small-molecule therapies that harness master regulators of cellular function and inhibit multiple drivers of disease via a single target. KZR-616, a first-in-class selective immunoproteasome inhibitor, is being evaluated in severe and underserved autoimmune diseases. Additionally, KZR-261, the first clinical candidate for the treatment of cancer from the company's protein secretion program targeting the Sec61 translocon, is undergoing IND-enabling activities.

23

Kezar Life Sciences Announces Orphan Drug Designations for KZR-616 for the Treatment of Polymyositis and Dermatomyositis

SAN FRANCISCO--(BUSINESS WIRE)-- Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncologic disorders, today announced that the U.S. Food and Drug Administration has granted Orphan Drug Designations (ODD) for KZR-616 for the treatment of polymyositis (PM) and dermatomyositis (DM). Both orphan diseases are autoimmune inflammatory myopathies that are chronic and debilitating diseases characterized by marked morbidity and mortality. The estimated prevalence of PM and DM in the United States is up to 51,000 and 71,000, respectively. KZR-616 is a first-in-class selective immunoproteasome inhibitor with the potential to impact multiple drivers of immune-mediated diseases and inflammation. PRESIDIO, a Phase 2 placebo-controlled cross-over clinical trial to evaluate KZR-616 for the treatment of PM and DM, is currently enrolling subjects. “We are very pleased to have received orphan drug designations for both polymyositis and dermatomyositis. This recognition spotlights the significant unmet need for patients living with these autoimmune myopathies. KZR-616 has potential to truly modify the underlying pathophysiology of these two diseases,” said Noreen R. Henig, MD, Kezar’s Chief Medical Officer. “We will share more detail on the preclinical scientific rationale for the use of selective immunoproteasome inhibition to treat these rare and debilitating diseases during the 2020 American College of Rheumatology Conference next month.” Orphan Drug Designation is intended to advance drug development for rare diseases. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States. Orphan Drug Designation can provide certain benefits and incentives for KZR-616, including a period of marketing exclusivity for the first marketing application, if regulatory approval is received for the designated indication, potential tax credits for qualified clinical testing and waiver of certain administrative fees. About KZR-616 KZR-616 is a novel, first-in-class, selective immunoproteasome inhibitor with broad therapeutic potential across multiple autoimmune diseases. Preclinical research demonstrates that selective immunoproteasome inhibition results in a broad anti-inflammatory response in animal models of several autoimmune diseases, while avoiding immunosuppression. Data generated from Phase 1a and 1b trials provide evidence that KZR-616 exhibits a favorable safety and tolerability profile for development in chronic autoimmune diseases. Phase 2 trials are underway in severe autoimmune diseases. About Polymyositis and Dermatomyositis Polymyositis (PM) and Dermatomyositis (DM) are two of the five types of autoimmune myositis diseases. Both are chronic, debilitating, inflammatory autoimmune myopathies that are distinguished by inflammation of the muscles as well as the skin (in DM). Up to ~120,000 people in the United States are living with these severe and progressive inflammatory myopathies that are characterized by marked morbidity and associated mortality. While debilitating muscle weakness is the hallmark of these myopathies, including compromised muscles of respiration, other internal organ system dysfunctions can be equally disabling. The aim of treatment for these diseases is to suppress inflammation, increase muscle strength and prevent long-term damage to muscles and extramuscular organs; however, treatment options are limited for DM, and there are currently no approved treatments for PM. About Kezar Life Sciences Based in South San Francisco, Kezar Life Sciences is combining courage, conviction and cutting-edge science to develop breakthrough treatments for immune-mediated and oncologic disorders. The company is pioneering first-in-class, small-molecule therapies that harness master regulators of cellular function and inhibit multiple drivers of disease via a single target. KZR-616, a first-in-class selective immunoproteasome inhibitor, is being evaluated in severe and underserved autoimmune diseases. Additionally, KZR-261, the first clinical candidate for the treatment of cancer from the company's protein secretion program targeting the Sec61 translocon, is undergoing IND-enabling activities. Cautionary Note on Forward-looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “may,” “will,” “should,” “expect,” “believe” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Kezar’s expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties that could cause Kezar’s clinical development programs, future results or performance to differ materially from those expressed or implied by the forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to estimates of the prevalence of polymyositis and dermatomyositis, the potential of KZR-616 to modify the underlying pathophysiology of polymyositis and dermatomyositis, statements about the likelihood that pre-clinical data will support the scientific rationale to develop KZR-616 in polymyositis and dermatomyositis, the design, progress, timing, scope and results of clinical trials, the anticipated timing of disclosure of results of clinical trials and the likelihood and timing of obtaining regulatory approval of KZR-616. Orphan Drug Designation does not provide any assurance of regulatory approval or expedite regulatory review. Many factors may cause differences between current expectations and actual results, including the impacts of the COVID-19 pandemic on the company’s business, clinical trials and financial position, unexpected safety or efficacy data observed during preclinical or clinical studies, clinical trial site activation or enrollment rates that are lower than expected, changes in expected or existing competition, changes in the regulatory environment, the uncertainties and timing of the regulatory approval process, and unexpected litigation or other disputes. Other factors that may cause actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Kezar’s filings with the U.S. Securities and Exchange Commission, including the “Risk Factors” contained therein. Except as required by law, Kezar assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available. www.businesswire.com/news/home/20201023005106/en/

22

Atea Pharmaceuticals Announces Strategic Collaboration with Roche to Develop and Distribute AT-527 for Patients with COVID-19

Roche Obtains Exclusive Right to Develop and Distribute AT-527 Outside the United States BOSTON, Mass., October 22, 2020 – Atea Pharmaceuticals, Inc., a clinical stage biopharmaceutical company focused on discovering, developing and commercializing antiviral therapeutics to improve the lives of patients suffering from life-threatening viral infections, today announces that the company has entered into an agreement with Roche (SIX: RO, ROG; OTCQX: RHHBY) for the exclusive rights to research, develop and distribute AT-527 as an oral antiviral treatment for COVID-19 in territories outside of the United States. Under the terms of the agreement, Atea will receive an upfront payment of $350 million in cash from Roche with the potential for future milestone payments and royalties. “Roche shares our passion for delivering innovative new medicines to address great unmet medical needs. The COVID-19 pandemic has highlighted the urgent need for a novel, oral antiviral to treat this highly infectious and often deadly virus,” said Jean-Pierre Sommadossi, Ph.D., Chief Executive Officer and Founder of Atea Pharmaceuticals. “This collaboration with Roche enhances Atea’s efforts and underscores the potential for AT-527 to effectively address the COVID-19 crisis on a global scale. AT-527 is expected to be ideally suited to combat COVID-19 as it inhibits viral replication by interfering with viral RNA polymerase, a key component in the replication machinery of RNA viruses. Importantly, the manufacturing process for our small molecule direct-acting antiviral allows us to produce AT-527 quickly and at scale.” “The ongoing complexities of COVID-19 require multiple lines of defence. By joining forces with Atea, we hope to offer an additional treatment option for hospitalised and non-hospitalised COVID-19 patients, and provide important relief for hospital infrastructures during a global pandemic.” said Bill Anderson, Chief Executive Officer of Roche Pharmaceuticals. “In jointly developing and manufacturing AT-527 at scale, we seek to make this treatment option available to as many people around the world as we possibly can.” About AT-527 AT-527 is an orally administered, direct-acting antiviral agent derived from Atea’s purine nucleotide prodrug platform. At-527 is currently under evaluation as a treatment for patients with COVID-19. AT-527 is designed to inhibit viral replication by interfering with viral RNA polymerase, a key component in the replication machinery of RNA viruses, such as positive single-stranded human flaviviruses and human coronaviruses. AT-527 is currently in a global Phase 2 clinical study for hospitalized patients with moderate COVID-19 and has plans to initiate a global, registrational Phase 3 clinical trial in outpatients in the first half of 2021. Additionally, Atea is planning to study in a Phase 3 clinical trial the use of AT-527 in the post-exposure prophylaxis setting. Advisors Evercore served as exclusive financial advisor to Atea in connection with this transaction. About Atea Pharmaceuticals Atea Pharmaceuticals is a clinical stage biopharmaceutical company engaged in discovering and developing therapies to address the unmet medical needs of patients with severe viral diseases. Our lead programs are focused on the development of orally- administered direct acting antivirals for the treatment of patients with COVID-19 in the hospital and community settings, the treatment of patients with chronic hepatitis C infection, the treatment of patients with dengue, and the treatment of high-risk patients with severe respiratory syncytial virus infection. Our medicinal chemistry, virology, and pharmacology expertise, bolstered by our collective experience in drug development, enables us to pioneer new advancements in antiviral science. Leveraging the power of our purine nucleotide prodrug platform, our goal is to rapidly advance novel drug candidates with optimal therapeutic profiles for RNA virus targets. Founded by its Chairman and Chief Executive Officer, Jean-Pierre Sommadossi, PhD, Atea began operations in 2014 and is headquartered in Boston, MA. For more information about Atea and our pipeline of product candidates please visit our company website at www.ateapharma.com.

22

F2G Receives Second US FDA Breakthrough Therapy Designation for Olorofim

- In Phase 2b development for the treatment of life-threatening fungal infections - First antifungal agent to receive Breakthrough Therapy Designation - New second Breakthrough Therapy Designation for CNS Valley Fever (coccidioidomycosis) MANCHESTER, UK / VIENNA, Austria – October 22, 2020 – F2G Ltd, a UK- and Austria-based biotech company developing novel therapies for life-threatening systemic fungal infections, announced today that the US Food and Drug Administration (FDA) has granted an additional Breakthrough Therapy Designation to its lead first-in-class candidate, olorofim, for the indication of ‘Treatment of Central Nervous System (CNS) coccidioidomycosis refractory or otherwise unable to be treated with standard of care therapy’. This is the second Breakthrough Therapy Designation for olorofim; a designation was granted previously on 11 November 2019 for ‘Treatment of invasive mold infections in patients with limited or no treatment options, including aspergillosis refractory or intolerant to currently available therapy, and infections due to Lomentospora prolificans, Scedosporium, and Scopulariopsis species’. Olorofim (formerly F901318) is the first antifungal agent to be granted Breakthrough Therapy Designation. Olorofim has orphan drug designation in the United States for coccidioidomycosis, an endemic fungal infection with a geographic distribution focused mainly in the desert Southwest of the United States but also including Mexico, Central America, and South America. Infection begins by inhalation and can spread to any part of the body, even in otherwise healthy individuals. Spread to the brain is particularly feared as it produces a devastating illness that cannot always be controlled with any currently available agent. Patients with coccidioidomycosis are being studied in an ongoing open-label single-arm Phase 2b study (ClinicalTrials.gov Identifier: NCT03583164) in patients with proven invasive fungal disease (IFD) or probable invasive aspergillosis (IA) and either refractory disease, resistance, or intolerance to available agents. Olorofim has been well tolerated across more than 30 years of cumulative patient dosing days with a median therapy duration of 12 weeks. Preliminary data from this study were provided to the FDA as part of the Breakthrough Therapy Designation submission. Breakthrough Therapy Designation is an FDA process designed to expedite the development and review of drugs that are intended to treat a serious or life-threatening condition and is granted based on preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. Breakthrough Therapy Designation conveys all the features of fast track designation, more intensive FDA guidance on an efficient drug development program, an organisational commitment by FDA to involve senior managers, and eligibility for rolling review and priority review. Commenting on the news, Ian Nicholson, CEO of F2G Ltd, said: “The granting of a second FDA Breakthrough Therapy Designation will further support our goal of rapidly developing this novel treatment for patients suffering from serious and life-threatening fungal infections. Olorofim acts via a novel and differentiated mechanism to traditional antifungals, and preliminary data have indicated that it is efficacious in tackling life-threatening invasive fungal infections that cannot be managed with currently approved agents. “Our Phase 2b programme is on track with over 85 patients recruited in Europe, Asia, and the US. We look forward to working closely with the US FDA to accelerate development of this therapy for patients having limited or no approved treatment options for an invasive mold infection.” Professor George Thompson, UC Davis and Investigator for the Phase 2b study said: “This news is very exciting for clinicians caring for patients with Valley Fever (coccidioidomycosis) as spread to the brain is the most-feared complication of infection with this fungus. Unfortunately, available drugs are not curative, must be administered as life-long therapy, and are associated with substantial toxicity. The news that olorofim has encouraging preliminary clinical data that support Breakthrough Therapy Designation brings realistic hope that we can change the paradigm for managing this devastating infection.” About Olorofim / Clinical trial The Phase 2b study for olorofim (ClinicalTrials.gov Identifier: NCT03583164) is a global open-label study in patients who have limited treatment options for difficult-to-treat invasive fungal mold infections such as azole-resistant aspergillosis, scedosporiosis, lomentosporiosis, and other rare mold infections. More than 40 centres are currently open in nine countries (AU, BE, DE, ES, IS, NL, TH, UK, USA) and a further 10 will open in 2020/2021. Olorofim is being developed both as IV and oral formulations. About F2G F2G is a world-leading UK- and Austria-based biotech company (F2G Ltd and F2G Biotech GmbH) focused on the discovery and development of novel therapies to treat life-threatening invasive fungal infections. F2G has discovered and developed a completely new class of antifungal agents called the orotomides. The orotomides selectively target fungal dihydroorotate dehydrogenase (DHODH), a key enzyme in the de novo pyrimidine biosynthesis pathway. This is a completely different mechanism from that of the currently marketed antifungal agents and gives the orotomides fungicidal activity against a broad range of rare and resistant fungal mold infections. Olorofim (formerly, F901318) is F2G’s leading candidate from this class and is in a Phase 2b open-label study focussing on rare and resistant invasive fungal infections such as aspergillosis (including azole-resistant strains), scedosporiosis (including lomentosporiosis). Olorofim has received orphan drug status from the European Medicines Agency for the treatment of invasive aspergillosis and invasive scedosporiosis. Olorofim has received orphan drug status from the US FDA for the treatment of coccidioidomycosis, lomentosporiosis/scedosporiosis, and invasive aspergillosis. Olorofim has been granted Qualified Infectious Disease Product (QIDP) designation Invasive for aspergillosis, invasive scedosporiosis, invasive lomentosporiosis, coccidioidomycosis, invasive disease due to Scopulariopsis species, and invasive fusariosis. Olorofim is being developed both as IV and oral formulations. www.f2g.com

20

Atea Pharmaceuticals Announces IND Clearance of AT-527 for COVID-19 and $215 Million Financing

- Proceeds to support the clinical development of Atea’s oral, direct acting antiviral medicine for COVID-19, in addition to advancing its diverse pipeline of treatments for viral diseases - Investigational new drug application cleared by U.S. Food and Drug Administration to start Phase 2 study of AT-527, Atea’s oral purine nucleotide prodrug for patients hospitalized with moderate COVID-19 BOSTON, Mass., May 20, 2020 – Atea Pharmaceuticals, Inc., a biopharmaceutical company engaged in the discovery and development of next-generation therapeutics for severe human viral infections, today announced a $215 million Series D financing. The financing was led by Bain Capital Life Sciences and also included new investors RA Capital Management, Perceptive Advisors, Rock Springs Capital, Adage Capital Management, Redmile Group, Omega Funds, and funds and accounts managed by T. Rowe Price Associates, Inc. Existing Atea investors, including Morningside Ventures, Cormorant Asset Management, Ally Bridge Group, and Sectoral Asset Management, as well as other investors also participated in this financing. Atea also announced today that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug application (IND) for AT-527, a novel, oral, purine nucleotide prodrug, for the treatment of adult patients hospitalized with moderate COVID-19 disease, with one or more risk factors for poor outcomes. A Phase 2 clinical trial, scheduled to begin shortly, will evaluate the safety and efficacy of AT-527 in this patient population. AT-527 is a highly selective, orally administered direct acting antiviral, (DAA) designed to inhibit the RNA polymerase enzyme, a key element in the replication machinery of RNA viruses. Antiviral activity of AT-527 has been observed in vitro and in vivo against replication of multiple RNA viruses including, but not limited to, human coronaviruses and flaviviruses. In addition to supporting its work to find a treatment for COVID-19, Atea expects to also apply proceeds from this financing towards advancing its diverse pipeline of highly selective DAAs that target other severe RNA viral infections. Atea’s pipeline currently includes investigative treatments for hepatitis C virus, dengue virus, and respiratory syncytial virus, in addition to its COVID-19 program. “We are delighted to have the strong support of this group of blue-chip healthcare investors,” said Jean-Pierre Sommadossi, PhD, Atea’s Founder, Chairman, and Chief Executive Officer. “Atea’s portfolio is focused on developing novel, best-in-class, potent DAA’s and we have shifted all of our immediate resources and our team’s deep expertise in virology and pharmacology to help address the unmet needs in the fight against the COVID-19 pandemic. An oral treatment for COVID-19 patients should prevent progression of the disease and may help lessen the burden on critical inpatient resources. Atea is moving rapidly, in concert with regulatory authorities, to determine if our oral DAA is a safe and effective therapeutic against COVID-19.” “Atea’s team has an outstanding track record in developing novel, potent DAAs, which we believe can contribute to the urgent fight against the COVID-19 pandemic and other RNA viruses,” said Andrew Hack, M.D., Ph.D., Managing Director of Bain Capital Life Sciences. “We are pleased to partner with Atea’s leadership team and an outstanding group of leading healthcare investors as Atea advances its diverse pipeline of transformative antiviral medicines” About AT-527 AT-527 is an investigational, oral, purine nucleotide prodrug, which has demonstrated in vitro and in vivo antiviral activity against several enveloped single-stranded RNA viruses, including human flaviviruses and coronaviruses. This highly selective purine nucleotide prodrug was designed to uniquely inhibit viral RNA dependent RNA polymerase, an enzyme that is essential for the replication of RNA viruses. Antiviral activity and safety of AT-527 has been demonstrated in Phase 2 clinical studies of hepatitis C patients. AT-527 is not yet licensed or approved for any indication in the U.S. or any other country. About Atea Pharmaceuticals Atea Pharmaceuticals is a clinical stage biopharmaceutical company engaged in discovering and developing best-in-class therapies to address the unmet medical needs of patients with severe viral diseases. Our lead programs are focused on the development of orally administered direct acting antivirals for the treatment of patients with mild to moderate COVID-19 in the hospital and community settings, the treatment of patients with chronic hepatitis C infection, the treatment of patients with dengue, and the treatment of high-risk patients with severe respiratory syncytial virus infection. Our medicinal chemistry, virology, and pharmacology expertise, bolstered by our collective experience in drug development, enables us to pioneer new advancements in antiviral science. Leveraging the power of our purine nucleotide prodrug platform, our goal is to rapidly advance novel drug candidates with optimal therapeutic profiles for RNA virus targets. Founded by its Chairman and Chief Executive Officer, Jean-Pierre Sommadossi, PhD, Atea began operations in 2014 and is headquartered in Boston, MA. For more information about Atea and our pipeline of products please visit our company website at https://www.ateapharma.com/.

20

Gracell Biotechnologies Raises $120M in Series C Funding to Advance Novel CAR-T Platform Technologies

SHANGHAI and SUZHOU, China, October, 2020 -- Gracell Biotechnologies Co., Ltd. (“Gracell”), a global clinical-stage biopharmaceutical company dedicated to developing highly efficacious and affordable cell therapies for the treatment of cancer, today announced it has secured [$120] million in Series C funding. The round is led by Wellington Management Company, OrbiMed Advisors LLC and Morningside Venture Capital, and joined by new investors Vivo Capital, [Capital Group and Logos Capital]. Existing investors Temasek Holdings, Lilly Asia Ventures and Kington Capital are also participating. “We are very pleased to expand our investor base with support from a high caliber consortium,” said Dr. Wei (William) Cao, founder, Chairman, and CEO of Gracell. “Our passion is to bring transformative CAR-T cell therapies to a broader group of patients by developing products that are efficacious and can be made widely available.” Gracell was founded in 2017 with the aim to overcome the major challenges that persist with conventional CAR-T cell therapies. Gracell has developed pioneering platforms, FasTCART™ and TruUCART™. FasTACRT™ is able to deliver younger, less exhausted T cells for autologous cell therapies with greater potency and next-day manufacturing (22 to 36 hours); while TruUCART™ derives T cells from non-HLA matched healthy donors to generate “off-the-shelf” allogenic CAR-T cell therapies at lower cost for a broad patient base. Both platform technologies can also be implemented for other targets of hematological malignancies and solid tumors. Gracell is currently evaluating four product candidates in ongoing investigator-initiated Phase 1 trials, including FasTCART™-enabled autologous product candidate GC012F for relapsed or refractory multiple myeloma (r/r MM) and TruUCART™-enabled allogeneic product candidate GC027 for relapsed or refractory T cell acute lymphoblastic leukemia (r/r T-ALL). Proceeds from the round will be used to fund internal research and development and further advance current clinical programs. Jefferies and Cooley advised in the transaction. About Gracell Gracell Biotechnologies Co., Ltd. ("Gracell") is a global clinical-stage biopharmaceutical company dedicated to developing highly efficacious and reliable cell therapies for leukemia, lymphoma, myeloma and solid tumors. Utilizing its pioneering FasTCART™ and TruUCART™ platform technologies, Gracell’s innovative cell therapies address several of the major challenges that persist with conventional CAR-T therapies, such as lengthy manufacturing time, high production costs, lack of off-the-shelf products, and products for solid tumors. www.gracellbio.com

19

Apellis Announces 18-Month Data from Phase 1b Study of Pegcetacoplan in Patients with Geographic Atrophy (GA)

- First data for 18 months of treatment with intravitreal pegcetacoplan demonstrates continued slowing of GA lesion growth beyond 12 months - Post hoc analysis demonstrated a 52% decrease in mean lesion growth in seven patients with bilateral GA comparing treated eye vs. untreated fellow eye at 18 months (p=0.01) - Top-line data from Phase 3 DERBY and OAKS studies expected in Q3 2021 WALTHAM, Mass., Oct. 19, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced 18-month data from the Phase 1b APL2-103 study of pegcetacoplan (APL-2) in patients with advanced geographic atrophy (GA) secondary to age-related macular degeneration (AMD) and low vision. GA is a complement-driven eye disease1,2 that can cause blindness, affects approximately five million people globally3,4 and has no approved treatment. The study, which enrolled 12 patients with bilateral GA (disease in both eyes), was initiated to assess the safety of the Phase 3 formulation of pegcetacoplan (15mg/0.1mL). Patients were dosed monthly with pegcetacoplan in one eye using the fellow eye as an untreated control. Apellis previously reported results at Month 12 in nine patients demonstrating a trend in reduced GA lesion growth in treated eyes versus untreated fellow eyes. The current post hoc analysis reports on the seven Phase 1b study patients for whom data were available for at least 18 months. In this population, the growth rate of GA lesions in the treated eye was on average 52% (mean square root) slower than the opposite untreated eye (p=0.01). It has been shown that lesions in both eyes tend to grow at the same rate in patients with bilateral GA.5 Of the 12 enrolled patients, there were no reported cases of inflammation and, as previously reported, one patient (8%) developed new-onset exudation at Month 12. “It is exciting to see data for the efficacy of intravitreal pegcetacoplan at 18 months among patients with GA,” said Charles Wykoff, M.D., Ph.D., Director of Research, Retina Consultants of Houston, and Investigator of Apellis’ Phase 1b and Phase 3 GA trials. “These data align with the Phase 2 FILLY results, where patients with bilateral GA in the monthly treated population had a significant reduction in growth relative to their untreated fellow eye in a post hoc analysis at 12 months. Finally, while this is a limited number of patients, I am encouraged to see that the benefit of pegcetacoplan in slowing GA growth seems to be maintained through Month 18. I believe that a 52% reduction in GA lesion growth at Month 18 is likely to be highly clinically meaningful.” Post hoc analysis at 18 months Figure 1. Mean (± SE) change from baseline in square root GA lesion measured by fundus autofluorescence in the study eye (SE) and fellow eye (FE). Percentage difference and p value represents the comparison in GA growth between the study and fellow eye at each timepoint. The ongoing pegcetacoplan development program in GA includes the Phase 1b APL2-103 study and the Phase 3 DERBY and OAKS studies. The patient population enrolled in the Phase 1b study is similar to DERBY and OAKS but allowed for more advanced disease with a wider range of baseline lesion size and lower baseline visual acuity. The DERBY and OAKS studies were initiated in 2019 with the pegcetacoplan formulation tested in this Phase 1b study and top-line data are expected in the third quarter of 2021. About the APL2-103 study The APL2-103 study is a 12-patient Phase 1b, multicenter, open label, single arm, 24-month clinical trial to assess the safety of monthly intravitreal (IVT) injections of pegcetacoplan in patients diagnosed with advanced geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The primary outcome measures include incidence and severity of ocular and systemic treatment-emergent adverse events (TEAEs). About the FILLY study The FILLY study was a 246-patient, Phase 2, multicenter, randomized, single-masked, sham-controlled clinical trial evaluating pegcetacoplan in patients with GA secondary to AMD conducted at over 40 clinical sites in the United States, Australia and New Zealand. Pegcetacoplan was administered as an intravitreal injection monthly or every other month (EOM) for 12 months, followed by six months of monitoring after the end of treatment. The primary efficacy endpoint was the change in GA lesion area from baseline to Month 12 in pegcetacoplan-treated patients compared to sham. About the DERBY and OAKS studies The DERBY and OAKS studies are 600-patient prospective, international, multicenter, randomized, double-masked, sham-injection controlled Phase 3 studies assessing the efficacy and safety of multiple IVT injections of pegcetacoplan in patients with GA secondary to AMD. For more information, please visit https://gastudy.com/. About Pegcetacoplan (APL-2) Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and the treatment of geographic atrophy, and received orphan drug designation for the treatment of C3 glomerulopathy by the FDA and European Medicines Agency. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials. About Geographic Atrophy (GA) GA is an advanced form of age-related macular degeneration (AMD), a leading cause of blindness. Excessive complement activation drives irreversible lesion growth in GA,6 and C3 is the only target to precisely control complement overactivation. Pegcetacoplan, studied in early and late-stage trials comprising a total of approximately 1,500 patients, is the only targeted C3 inhibitor being evaluated in late-stage trials to control lesion growth in GA.7 GA lesions affect the central portion of the retina, known as the macula, which is responsible for central vision. GA is progressive and irreversible, leading to central visual impairment and permanent loss of vision. Based on published studies, approximately one million people have GA in the United States and five million people have GA globally.1,2 There are currently no approved treatments for GA. About Apellis Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

15

Apellis Announces New Data Demonstrating Correlation Between Complement Activation and COVID-19 Severity

October 15, 2020 - Observational study in 41 patients hospitalized with COVID-19 found that nearly all patients had elevated systemic levels of C3a, a marker for C3 activation; median C3a levels were 3.7 times the upper limit of normal - In a Phase 1/2 interventional study, preliminary open-label safety results in first six patients support advancement of APL-9, an investigational targeted C3 therapy, for severe COVID-19; additional 60-patient randomized, double-blind, controlled study cohort is currently enrolling WALTHAM, Mass., Oct. 15, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced observational study results that found a correlation between COVID-19 severity and complement overactivation, which is a key immune response. Additionally, preliminary open-label safety data from six patients in a Phase 1/2 study support advancement of APL-9, an investigational targeted C3 therapy designed for acute interventions, for severe COVID-19. These results also showed that key markers of inflammation were within or near normal range at the end of the APL-9 treatment period. Observational Study Results In the 41-patient observational study, data from 40 patients with evaluable blood samples demonstrated that increased complement activation showed a correlation with disease severity. C3a, C4a, Bb and terminal complement complex (TCC) levels each correlated statistically with COVID-19 severity as quantified by the National Early Warning Score (NEWS), a widely used assessment for the need for critical care. A similar correlation was observed with lactate dehydrogenase (LDH), C-reactive protein (CRP) and cytokine interleukin 6 (IL-6). In addition, 39 (97.5%) patients had substantially elevated systemic levels of C3a, a marker for C3 activation, with median C3a levels 3.7 times the upper limit of normal. Multiple complement pathways were activated as evidenced by systemic elevations of C4a, Bb and TCC. Complement overactivation is known to worsen respiratory diseases by killing healthy cells and causing further complications such as blood clots and multiorgan failure, making it difficult for patients to recover. “These study results demonstrate that multiple complement pathways are going into overdrive in severe cases of COVID-19, reinforcing the critical need for therapies that can regulate overactive complement and improve patient outcomes in COVID-19,” said Lukas Scheibler, Ph.D., Chief Innovation Officer of Apellis. “Targeting C3 has the potential to control complement activation across all three major pathways due to its central location, so we believe it is a strong target to continue exploring.” Preliminary Results from the Phase 1/2 Study Apellis also announced preliminary results from the first part of the interventional Phase 1/2 study of APL-9 in patients with severe COVID-19. Of the six patients enrolled, all patients had elevated C3a and CRP levels and five of the six patients had elevated LDH levels at baseline. Five patients completed the study and were discharged from the hospital. Additionally, in these patients, C3a, LDH and CRP levels were within the normal reference range by the end of the APL-9 treatment period. Three patients experienced four treatment-emergent adverse events (TEAEs), which were all considered unrelated to study treatment. One serious adverse event of respiratory failure, which was considered unrelated to the study treatment, led to death from failed intubation. Based on the results, an independent data monitoring committee recommended continuing the investigation of APL-9 in this study. Apellis is currently enrolling an additional 60 patients with COVID-19 and respiratory failure who require oxygen supplementation or mechanical ventilation into the randomized, double-blind, controlled phase of the study to evaluate the safety and efficacy of APL-9 (as add-on to standard care for up to 21 days). Detailed results from both studies will be submitted for future scientific publication. About the Observational Study The observational exploratory study evaluated the relationship between markers of complement activation and disease severity in 41 patients with COVID-19. Patients in the observational study had blood drawn at baseline and at subsequent visits for up to six weeks. The primary objective of the observational study was to quantify systemic concentrations of the complement activation products Bb, C3a, C4a and terminal complement complex (TCC) and correlate them to COVID-19 severity. Secondary objectives were to quantify concentrations of C3 and C4 in serum and proinflammatory T-cell populations (CD4, CD8 and TH17) in whole blood and correlate them to COVID-19 severity. The exploratory objectives were to quantify the concentrations of interleukin 6 (IL-6) and C-reactive protein (CRP) in plasma and serum, respectively, and correlate them to COVID-19 severity. Additional detail and study results can be found on the Events and Presentations section of the Apellis website. About the Interventional Phase 1/2 Study The Phase 1/2 study (NCT04402060) is designed to evaluate the safety and feasibility of targeting C3 with APL-9 as an add-on therapy to standard of care in 66 patients with severe COVID-19. Patients included in the study have respiratory failure requiring oxygen supplementation or invasive or noninvasive mechanical ventilation. Patients are treated with APL-9 via intravenous (IV) infusion with an initial dose of 540 mg in the first 24 hours of dosing, followed by 360 mg/day on all subsequent days. The primary objective of the study is to assess the safety of APL-9 as an add-on to the current standard of care. Secondary objectives include evaluating length of stay in the hospital, overall survival, time on oxygen therapy or mechanical ventilation, and markers of complement activation, organ failure and coagulation (blood clotting). In the preliminary phase, six patients received open-label APL-9 treatment either until the end of study drug administration at day 7 or resolution of acute respiratory distress syndrome (ARDS). Based on the results, an independent data monitoring committee recommended continuing the investigation of APL-9 in this study. Apellis is enrolling an additional 60 patients into the randomized, double-blind, controlled phase of the study to evaluate the safety and efficacy of APL-9 (as add-on to standard care for up to 21 days). Additional detail and preliminary results can be found on the Events and Presentations section of the Apellis website. About APL-9 APL-9 is an investigational drug designed to control the complement cascade centrally at C3 and may have the potential to treat a range of diseases caused by excessive or uncontrolled activation of complement. APL-9 leverages the same mechanism of action as Apellis’ lead compound, pegcetacoplan (APL-2), but has a lower molecular weight and shorter half-life. APL-9 is designed to be intravenously administered for acute use. About Apellis Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

05

Chinook Therapeutics Closes Merger with Aduro Biotech and Completes $115M Private Placement Financing

- Combined Company Will Have Over $275 Million in Operating Capital and Trade on Nasdaq under the Ticker Symbol “KDNY” VANCOUVER, British Columbia and SEATTLE, Oct. 05, 2020 (GLOBE NEWSWIRE) -- Chinook Therapeutics, Inc. (NASDAQ: KDNY), a clinical-stage biotechnology company focused on the discovery, development and commercialization of precision medicines for kidney diseases, today announced the closing of its merger with Aduro Biotech, Inc. and $115 million private placement financing. The combined company, now known as Chinook Therapeutics, will commence trading October 6, 2020 on the Nasdaq Global Select Market under the trading symbol “KDNY.” As previously announced, the $115 million private placement financing includes participation from new investors EcoR1 Capital, OrbiMed Advisors, funds managed by Rock Springs Capital, Fidelity Management and Research Company LLC, Avidity Partners, Surveyor Capital (a Citadel company), Ally Bridge Group, Monashee Investment Management LLC, Northleaf Capital Partners, Janus Henderson Investors, Sphera Biotech and other top-tier healthcare investors. As part of the financing, Chinook’s existing investors, Versant Ventures, Apple Tree Partners and Samsara BioCapital, purchased $25 million in Chinook common stock on the same terms as the new investors. Effective as of the closing of the merger, Chinook has over $275 million in operating capital to advance its kidney disease programs. “Chinook’s merger with Aduro and entry into the public market is a transformative event that will propel the development of our atrasentan, BION-1301 and CHK-336 programs, and drive forward our research and discovery programs for other rare, severe chronic kidney diseases with large unmet medical needs,” said Eric Dobmeier, president and chief executive officer of Chinook Therapeutics. “With a strong cash position, a promising pipeline and our dedication to treating patients with debilitating kidney diseases, we are well positioned to achieve value-generating milestones and build a leading company in the kidney disease space.” Chinook will focus on advancing its product candidates for kidney disease, including: - Planned Phase 3 and Phase 2 trials of atrasentan, an investigational selective endothelin receptor antagonist, in development for the treatment of IgA nephropathy and other primary glomerular diseases; - An ongoing Phase 1b and future clinical trials of BION-1301, an investigational humanized monoclonal antibody that blocks APRIL binding to both the BCMA and TACI receptors, in development for the treatment of IgA nephropathy; - A planned Phase 1 trial of CHK-336, an investigational small molecule, in preclinical development for the treatment of an ultra-rare orphan kidney disease; and - Advancement of additional research and discovery programs focused on the treatment of rare, severe chronic kidney diseases. In connection with the closing of the merger, Aduro effected a 1:5 reverse split of its common stock. Post-merger and post-reverse split, Chinook has approximately 42 million shares of common stock outstanding. Prior Chinook stockholders collectively own approximately 39.5% of the combined company, prior Aduro stockholders collectively own approximately 39.9% of the combined company and investors in the Chinook private placement financing collectively own approximately 20.6% of the combined company. Effective as of the closing of the merger, the board of directors of Chinook will be comprised of seven directors: Eric Dobmeier, president and chief executive officer of Chinook Therapeutics; Jerel Davis, Ph.D., managing director at Versant Ventures; Srini Akkaraju, M.D., Ph.D., managing general partner at Samsara BioCapital; William M. Greenman, president and chief executive officer of Cerus Corporation; Ross Haghighat, founder, chairman and managing partner of Triton Systems, Inc.; Michelle Griffin, director and audit committee chair for Adaptive Biotechnologies, Acer Therapeutics and HTG Molecular Diagnostics, Inc.; and Dolca Thomas, M.D., chief medical officer of Principia Biopharma, Inc. MTS Health Partners acted as exclusive financial advisor to Chinook and Fenwick & West LLP served as legal counsel to Chinook for the merger. SVB Leerink acted as exclusive financial advisor to Aduro and Latham & Watkins LLP served as legal counsel to Aduro for the merger. SVB Leerink acted as lead placement agent and Evercore Group L.L.C. and William Blair acted as co-placement agents for the private placement financing. About Chinook Therapeutics, Inc. Chinook Therapeutics, Inc. is a clinical-stage biotechnology company developing precision medicines for kidney diseases. Chinook’s product candidates are being investigated in rare, severe chronic kidney disorders with opportunities for well-defined clinical pathways. Chinook’s lead program is atrasentan, an investigational Phase 3-ready endothelin receptor antagonist for the treatment of IgA nephropathy and other primary glomerular diseases. BION-1301, an investigational anti-APRIL monoclonal antibody is being evaluated in a Phase 1b trial for IgA nephropathy. In addition, Chinook is advancing advance CHK-336, a preclinical development candidate for an undisclosed ultra-orphan kidney disease, as well as research programs for other rare, severe chronic kidney diseases, including polycystic kidney disease. Chinook seeks to build its pipeline by leveraging insights in kidney single cell RNA sequencing, human-derived organoids and new translational models, to discover and develop therapeutics with differentiating mechanisms of action against key kidney disease pathways. To learn more, visit www.chinooktx.com.

03

Apellis Announces New Analysis Demonstrating Targeted C3 Therapy Pegcetacoplan Slows Progression of Early Disease in Patients with Geographic Atrophy (GA)

October 3, 2020 - Post hoc analysis of Phase 2 FILLY study shows 39% reduction in rate of progression from nascent GA, an earlier form of disease, to GA in patients treated with pegcetacoplan monthly vs. sham - First ever observation of slowed nascent GA progression in a Phase 2 study signals potential benefit of earlier intervention with pegcetacoplan in patients with GA - Data support hypothesis that targeting C3 with pegcetacoplan addresses an underlying cause of disease, excessive complement activation, as evidenced by slowed progression of nascent GA to GA WALTHAM, Mass., Oct. 03, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced the results of a post hoc analysis of the Phase 2 FILLY study investigating intravitreal pegcetacoplan (APL-2) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The post hoc analysis found that monthly treatment with pegcetacoplan significantly reduced the rate of progression from nascent GA to GA in areas of the retina outside of existing GA lesions. The data were presented today in a late-breaking oral session at the European Society of Retina Specialists 2020 Virtual (EURETINA). Pegcetacoplan is the only targeted C3 therapy in Phase 3 clinical trials for GA, a complement-driven eye disease1,2 that causes blindness, affects approximately five million people globally3,4 and has no approved treatment. - In the post hoc analysis, progression from nascent GA to GA was observed in 50.0% of the pegcetacoplan-treated group and 81.8% of the sham-treated group (p=0.02). - The 39% reduction in progression from nascent GA to GA indicates that pegcetacoplan slows the rate of progression of AMD in areas of the retina outside of GA lesions. - Progression from large drusen (deposits in the retina that are a hallmark of AMD) to nascent GA or GA at Month 12 was observed in 22.6% of patients in the pegcetacoplan-treated group and 33.3% of patients in the sham-treated group (p=0.31). “Our findings from this post-hoc analysis demonstrate that intravitreal pegcetacoplan, a targeted C3 therapy, significantly lowers the rate of progression from nascent GA to GA in patients when compared to sham controls,” said SriniVas Sadda, M.D., President & Chief Scientific Officer of the Doheny Eye Institute and lead investigator. “This study provides exciting evidence to support further exploration of the potential of pegcetacoplan for earlier intervention in the course of GA.” The post hoc analysis, a collaboration with the Doheny Image Reading Research Lab, included FILLY patients from the monthly pegcetacoplan-treated group (n=42) and sham-treated group (n=69) who completed the Month 12 study visit and who did not develop exudative or neovascular AMD. The objective of the analysis was to investigate the effects of pegcetacoplan on complement-driven progression of AMD outside of GA lesions. “Patients with geographic atrophy experience changes in the retina that progress from the earlier stages of AMD to the beginning of atrophy and ultimately irreversible vision loss,” said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. “This post hoc analysis of the FILLY study demonstrates that pegcetacoplan slows early disease progression and may have the potential to delay the onset of GA and vision loss in patients.” Earlier this year, Apellis announced the completion of patient enrollment in the ongoing Phase 3 DERBY and OAKS studies investigating pegcetacoplan in patients with GA secondary to AMD. Top-line results from these pivotal trials are expected in Q3 2021. About Pegcetacoplan (APL-2) Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies including paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (GA), cold agglutinin disease (CAD), and C3 glomerulopathy (C3G). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of PNH and the treatment of GA. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials. About Geographic Atrophy (GA) GA is an advanced form of age-related macular degeneration (AMD), a leading cause of blindness. Excessive complement activation drives irreversible lesion growth in GA5, and C3 is the only target to precisely control complement overactivation. Pegcetacoplan, studied in early and late-stage trials comprising a total of approximately 1,500 patients, is the only targeted C3 inhibitor being evaluated in late-stage trials to control lesion growth in GA.6 GA lesions affect the central portion of the retina, known as the macula, which is responsible for central vision. GA is progressive and irreversible, leading to central visual impairment and permanent loss of vision. Based on published studies, approximately one million people have GA in the United States and 5 million people have GA globally.1,2 There are currently no approved treatments for GA. About FILLY The FILLY study was a 246-patient, Phase 2, multicenter, randomized, single-masked, sham-controlled clinical trial evaluating pegcetacoplan in patients with GA secondary to AMD conducted at over 40 clinical sites in the United States, Australia and New Zealand. Pegcetacoplan was administered as an intravitreal injection monthly or every other month (EOM) for 12 months, followed by six months of monitoring after the end of treatment. The primary efficacy endpoint was the change in GA lesion area from baseline to Month 12 in pegcetacoplan-treated patients compared to sham. About DERBY and OAKS DERBY (621 patients enrolled) and OAKS (638 patients enrolled) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies to compare the efficacy and safety of intravitreal pegcetacoplan with sham injections in patients with GA secondary to AMD. The primary objective of the studies is to evaluate the efficacy of pegcetacoplan compared to sham injection in patients with GA secondary to AMD assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence (FAF). About Apellis Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology. For more information, please visit http://apellis.com.

September 2020

28

Fierce Biotech Names Amylyx Pharmaceuticals as One of Its "Fierce 15" Biotech Companies of 2020

Amylyx Pharmaceuticals, Inc. Contify Life Science News September 28, 2020 CAMBRIDGE, Massachusetts, Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer's disease and other neurodegenerative diseases, today announced that it has been named by Fierce Biotechas one of 2020's Fierce 15 biotechnology companies, designating Amylyx as one of the most promising private biotechnology companies in the industry. The 2020 Fierce 15 comes at a time when the world is focused on a pandemic, but even as coronavirus remains a threat, patients with cancer, rare diseases and other disorders still need treatment, Fierce Biotech Senior Editor Ben Adams said. "This year, we've chosen from a diverse range of those fighting COVID, as well as those fighting longer term plagues against our biology," Adams said. Amylyx' lead product candidate, AMX0035, is an investigational neuroprotective therapy that seeks to prevent or reduce neuron death and dysfunction in ALS and other neurodegenerative diseases. Recently, Amylyx announced positive results from the pivotal CENTAUR trial evaluating AMX0035 in people with ALS, published in the New England Journal of Medicine. Amylyx continues to evaluate the long-term effects of AMX0035 in people with ALS in an ongoing open-label extension (OLE) of CENTAUR, and plans to publish the results from this study in Q4 2020. Additionally, AMX0035 is being evaluated for Alzheimer's disease in the Phase 2 PEGASUS trial and expects topline results in Q1 2021. "Amylyx is honored to be named one of Fierce Biotech's Fierce 15 of 2020. This recognition highlights the tremendous responsibility our team has to continue working towards bringing a new potential treatment to those living with neurodegenerative diseases," said Justin Klee, Co-CEO and Co-Founder of Amylyx. An internationally recognized daily report reaching a network of over 285,000 biotech and pharma industry professionals, Fierce Biotech provides subscribers with an authoritative analysis of the day's top stories. Every year Fierce Biotech evaluates hundreds of private companies from around the world for its annual Fierce 15 list, which is based on a variety of factors such as the strength of its technology, partnerships, venture backers and a competitive market position. About Fierce Biotech Fierce Biotech is the biotech industry's daily monitor, an email newsletter and web resource providing the latest biotech news, articles, and resources related to clinical trials, drug discovery, FDA approval, FDA regulation, patent news, pharma news, biotech company news and more. More than 150,000 top biotech professionals rely on Fierce Biotech for an insider briefing on the day's top stories. Signup is free at www.fiercebiotech.com/signup. About Amylyx Pharmaceuticals Amylyx Pharmaceuticals, Inc. is a pharmaceutical company working on developing a novel therapeutic for amyotrophic lateral sclerosis (ALS), Alzheimer's disease and other neurodegenerative diseases. For more information, visit www.amylyx.com and follow us on LinkedIn and Twitter.

24

Cognoa Moves Ahead on FDA Clearance of Digital Autism Diagnostic

Beg Bryant BioWorld September 24, 2020 Children with autism spectrum disorder (ASD) can face a lifetime of frustration because of challenges with communication, social behaviors and flexibility of thought. Early intervention can improve outcomes, but nailing a diagnosis of ASD often takes years. Cognoa Inc. wants to change that with its digital ASD Diagnostic and is on track to apply for U.S. FDA clearance before the end of the year. “We’re submitting a package for de novo application in the fourth quarter of this year,” David Happel, Cognoa’s CEO, told BioWorld. The Palo Alto, Calif.-based company snagged an FDA breakthrough device designation for the ASD Diagnostic device in October 2018, paving the way for a priority review. With such reviews averaging eight to nine months, Happel anticipates gaining FDA clearance early in the second half of 2021 and launching the product about eight weeks after that. Need for earlier diagnosis Approximately 1 in 54 children in the U.S. is diagnosed with ASD, according to the CDC. Getting to that diagnosis, however, can be laborious. Once concerns have been raised about a child’s developmental progress, a pediatrician refers the parents to a specialist who puts the child through a battery of tests, assesses the results and makes a diagnosis. This can take anywhere from six to 18 months, Happel said, citing a lack of specialists who perform ASD evaluations. Although the condition typically start to present between 14 and 18 months, the average diagnosis is about four-and-a-half years of age. By contrast, Cognoa’s prescription diagnostic leverages artificial intelligence (AI) and machine learning to quickly assess whether a child has autism or an autism spectrum disorder as soon as concerns arise. The device has three modules. First, the parent or caregiver inputs data – a questionnaire and videos – via Cognoa’s parent/caregiver-facing mobile app. The one-to-two minute videos are filmed in the child’s natural environment to avoid creating stress and discomfort. Next, a pediatrician completes an assessment with the child, which they input into Cognoa’s platform. Cognoa’s AI then combines the parent/caregiver and pediatrician inputs to produce a result. The videos are also forwarded to an expert in dealing with autism, trained and employed by Cognoa, for observation and assessment, who completes a questionnaire about their observations and feeds that into the AI as well. The ASD Diagnostic can be downloaded to a hand-held device and performed in the primary care setting. The entire process takes anywhere from two to six weeks. Pivotal study In a pivotal study of 425 children ages 18 to 72 months whose parents had expressed concern about their development but had never undergone evaluation, Cognoa’s ASD Diagnostic surpassed its targeted benchmarks, the company said. The multisite study, prospective, double-blind, active comparator study, which ran from July 2019 through May 2020, assessed the ASD Diagnostic’s ability to aid in the diagnosis of autism spectrum disorder by comparing diagnostic output with the clinical reference standard, which is a specialist clinician’s diagnosis, based on DSM-5 criteria and validated by one or more reviewing specialists. The goal was to determine how often the device correctly identified a patient with ASD and how often it concluded that a patient does not have the condition. “It was exceptionally accurate to rule in or rule out autism to the degree that it easily surpassed the thresholds that we identified with the FDA for the point estimates,” Happel said. The results are being prepared for publication in a peer-reviewed journal. He believes Cognoa’s device offers a real advancement in ASD diagnosis. It gives doctors a consistent and reliable diagnostic instrument that will accurately determine whether the child has autism that can be evaluated at the pediatrician level. And, perhaps equally important, it removes all bias when evaluating the child. According to Happel, there are significant biases around how the condition is diagnosed, based on gender, race and other factors. For example, young Caucasian boys are most often considered to be at highest risk of having an autism diagnosis – four times more so than a girls of the same age. “It’s not because boys are four times more likely to get autism. It’s the fact that girls present differently, as do individuals with different race, culture, ethnicity and socioeconomic status,” he said. “Our instrument is able to sort through that because it’s trained on hundreds of thousands of unique profiles, which allows it to remove the noise and agnostically make a diagnosis.” Cognoa’s approach aligns with the most recent guidelines of the American Academy of Pediatrics, which is pushing for more ASD diagnoses to be made at the pediatrician level. Robust pipeline Cognoa is also developing a digital therapeutic for ASD. The company recently completed a 30-patient engagement study to determine the usability of hand-held devices for individuals living with autism and ASD. Also accorded breakthrough status by the FDA, the ASD therapeutic was developed by Cognoa founder Dennis Wall, an associate professor of pediatrics and biomedical data science at Stanford University. “He’s been primarily working with wearable devices such as Google Glass and another program,” Happel said, adding the work has shown remarkable promise in helping people with autism recognize facial expressions, eye contact and emotions. The company is working with Wall to put together a clinical trial next year to assess the value of the programs and when they might have the most benefit for patients. “There’s an enormous amount of literature in the space that, particularly for medium to high functioning individuals that are autistic or on the spectrum, if you can get them diagnosed early enough, you can … effect these patterns in their developmental windows at a much earlier age, which will allow them either to, hopefully, reach a nondiagnosis at some point … or at least participate in a mainstream educational curricula and in system that can support that,” Happel said. In addition to the ASD products, Cognoa is also developing a digital diagnostic for attention deficit hyperactivity disorder (ADHD) and speech and language. The ADHD tool may eventually be combined with the ASD Diagnostic, Happel said. Other pipeline programs, still in the discovery stage, include an ADHD therapeutic and a diagnostic and therapeutic for anxiety. Financing To date, Cognoa has raised about $60 million to advance its behavioral health solutions. It is currently talking with potential investors for a follow-on to its series A financing, with plans to close at the beginning of 2021. Those funds should get it through FDA clearance and commercial launch of the ASD Diagnostic and at least one other pipeline project, Happel said. The company also plans further enhancements for the ASD Diagnostic, including a Spanish version and eventually more languages to expand its use in OUS markets. Cognoa will begin working on obtaining CE mark approval after it files its submission with the FDA, Happel said.

23

AI Tool to Diagnose Autism Could Give Concerned Parents a Fast Diagnosis - The startup Cognoa has submitted its app-based tool for FDA clearance

Megan Scudellari IEEE Spectrum September 23, 2020 This week, a California-based company announced it will seek FDA clearance for a first-of-its-kind autism spectrum disorder (ASD) diagnostic tool. Cognoa’s technology uses artificial intelligence to make an ASD diagnosis within weeks of signs of concern—far faster than the current standard of care. If cleared by the FDA, it would be the first tool enabling primary care pediatricians to diagnose autism. The approach is “innovative,” says Robin Goin-Kochel, a clinical autism researcher at Baylor College of Medicine and associate director for research at Texas Children’s Hospital’s Autism Center, who is not affiliated with Cognoa. The field absolutely needs a way to “minimize the time between first concerns about development or behavior and eventual ASD diagnosis,” she adds. Cognoa’s tool is the latest application of AI to healthcare, a fast-moving field we’ve been tracking at IEEE Spectrum. In many situations, AI tools seek to replace doctors in the prediction or diagnosis of a condition. In this case, however, the application of AI could enable more doctors to make a diagnosis of autism, thereby opening a critical bottleneck in children’s healthcare. While most parents of children with autism notice developmental changes early on, within the first 1 to 3 years of life, the median diagnosis age in the United States is 4.3 years old. That’s because families often wait months, even years, to see a specialist and get a diagnosis. The time lost during that period is critical: Numerous studies show that early intervention, before the syndrome is fully manifest, can reduce the severity of ASD and improve a child’s brain and behavioral development. Cognoa’s technology comes out of the lab of founder Dennis Wall, an associate professor of pediatrics at the Stanford University School of Medicine. “I went into this with the hope of objectively asking the question: Can we reduce the complexity of the autism diagnostic process without loss of accuracy?” says Wall. By feeding electronic health record data into a set of algorithms, Wall’s team was able to distinguish particular features central to an ASD diagnosis, including social and emotional traits such as smiling in response to another person’s smile, joint attention at an object, creativity, and imagination. The team’s ASD diagnostic seeks to capture those features with three modules: a parent survey, home videos, and a clinician questionnaire. David Happel, CEO of Cognoa, explains how the tool works: When a parent expresses concern at a pediatrician appointment, or a child fails an ASD screening questionnaire, the pediatrician gives the parent a code to access Cognoa’s app on their smartphone. Once in the app, the parent answers a 15-minute questionnaire about their child’s behavioral patterns, then uploads two home videos of the child, 1 to 2 minutes in length, capturing the child’s behavior in a natural environment. The videos are sent to a trained Cognoa professional who reviews them and answers pertinent questions. Those answers are fed into Cognoa’s AI along with the parent answers and a short questionnaire filled out by the pediatrician. Then, the algorithm sends a result to the pediatrician, and the pediatrician renders a diagnosis. The tool’s algorithms are trained on data from hundreds of real cases across genders, races and ethnic backgrounds, says Happel. “It has proven to not only accelerate the time of diagnosis, but also remove a lot of the biases that are inherently in place in the current system.” Today’s standard ASD diagnostic tools, Happel notes, were constructed with health data from young Caucasian boys, so girls and children of non-white backgrounds are not well recognized by the tools, contributing to delays in diagnosis for those groups. In a Cognoa study published in March, which demonstrated an earlier version of the tool, the ASD diagnostic out-performed current screening tools for autism. Today's screening tools include questionnaires answered by a parent, teacher, or clinician that flag some at-risk children. More recently, the company completed a pivotal, double-blind clinical trial at 14 sites around the United States. The trial involved 425 participants, aged between 18 and 72 months, whose parents or doctors had expressed concern about their development but had not previously been evaluated for ASD, according to a press release. Each child was assessed twice: once using Cognoa’s tool, and once by a specialist clinician based on DSM-5 criteria, whose diagnosis was validated by a second specialist clinician. The results of the pivotal trial are not yet published, so there is no specific data to report, but the company says the trial “surpassed its targeted benchmarks”—as agreed upon with the FDA—and was accurate across genders and races. Uniquely, the study ran from July 2019 through May 2020, so some of the children were evaluated remotely this spring during the pandemic via telemedicine. The tool performed equally as well when administered remotely, says Happel. The company plans to submit the full study for publication in coming months. And formal submission to the FDA will occur shortly, says Happel. He hopes to receive approval in the second half of 2021, then be ready to launch the product into the hands of pediatricians two months after that. If and when Cognoa’s technology or others become available, “it will be really important to understand the plan for how new tools and technologies will be implemented in primary pediatric care,” says Goin-Kochel. Doctors are often slow to adopt new models, especially for making diagnoses they may not feel comfortable making, she notes, and new technologies raise practical questions such as when they should be applied and whether insurance companies will pay. “I’m very hopeful there is a near-term future where this product is available, covered by insurance, and made available to everybody as immediately as possible,” says Wall. He is now working on several technology-assisted therapies for ASD, including a project with Cognoa using Google Glass as part of a behavioral therapy to help children with autism recognize emotion.

17

Kezar Highlights Data from MISSION Phase 1b Study of KZR-616 During the Pan American Congress of Rheumatology

Contify Life Science News SOUTH SAN FRANCISCO, California -- Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncologic disorders, today highlighted data from the Phase 1b portion of the MISSION study demonstrating safety, tolerability and early efficacy signals of KZR-616 in patients with systemic lupus erythematosus (SLE) and lupus nephritis (LN) at the Pan-American Congress of Rheumatology (PANLAR 2020). The data were presented in a poster titled "Treatment of Systemic Lupus Erythematosus with the Immunoproteasome Inhibitor KZR-616: Results from the First 5 Cohorts of the MISSION Study, an Open-label Phase 1b Dose-Escalation Study" by study investigator Richard Furie, M.D., Chief, Division of Rheumatology, Northwell Health in New York. The poster can be found on Kezar's corporate website under the "Science" section. "Lupus and lupus nephritis are life-threatening diseases that disproportionately impact young Latina women in the prime of their life, and there is an urgent need for new treatment options that can target the full spectrum of their disease and don't cause debilitating side effects that add to the disease burden," said Dr. Furie. "These encouraging early positive data suggest that the novel mechanism of KZR-616 has the potential to address the underlying drivers of inflammation, resulting in improvements across organ systems in this disease." MISSION is a Phase 1b/2 study of KZR-616 in SLE patients with and without nephritis. The Phase 1b portion has completed enrollment in the final cohort, which is evaluating a 75 mg dose of KZR-616. The Phase 2 portion exclusively in LN is actively enrolling. As of the May 4, 2020 data analysis, 39 patients were enrolled in the MISSION Phase 1b study across five dose cohorts evaluating 45 mg and step-up dosing to 60 mg weekly for 13 weeks. Patients are followed to week 25 and kept on stable background treatment. At this time point, 22 patients completed 13 weeks of treatment and are included in the exploratory efficacy measures reported below: * Patients with increased DNA antibodies (serologic markers of SLE disease activity) at baseline that completed through week 25 of the study showed decreased titers following treatment. As previously reported: * Notably, two of two patients with active proliferative LN, despite being on stable background therapy, saw a greater than 50% decrease from baseline in proteinuria, a biomarker of disease severity. Both patients also experienced reductions in SLEDAI-2K and reductions in anti-dsDNA (double-stranded DNA) antibody levels. * Among patients completing treatment, all seven measures of disease activity improved (decrease in score) in the majority of patients from Baseline to Week 13. Improvement in disease activity persisted following the end-of-treatment. * Step-up dosing of KZR-616 improved overall tolerability. Most patients had mild (87.2%) or moderate (30.8%) TEAEs, which occurred early and diminished with later doses. The most common treatment emergent adverse events were transient injection site reactions. * To date, no patients have discontinued treatment in Cohorts 2b and 2c, which utilize a lyophilized formulation of KZR-616. About MISSION MISSION (NCT03393013) is a Phase 1b/2 clinical trial evaluating KZR-616 in SLE patients with and without nephritis. The study consists of two parts. The Phase 1b portion is an open-label dose escalation study which is evaluating doses up to 75 mg of KZR-616 across 6 cohorts, which has completed enrollment. The primary objective of the Ph1b portion of MISSION is to assess safety and tolerability. Secondary objectives include evaluating pharmacokinetics (PK) and pharmacodynamics (PD) and selecting dose levels for the Phase 2 trials. Several exploratory efficacy measures are also being assessed: Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K), Cutaneous Lupus Erythematosus Severity Index-Activity (CLASI-A), Tender and Swollen Joint Counts (TJC/SJC), Physician Global Assessment (PhGA), Patient Global Assessment (PtGA) and Patient Assessment of Pain (PtP). The Phase 2 portion of the MISSION study evaluating KZR-616 in patients with LN is currently enrolling. About KZR-616 KZR-616 is a novel, first-in-class, selective immunoproteasome inhibitor with broad therapeutic potential across multiple autoimmune diseases. Preclinical research demonstrates that selective immunoproteasome inhibition results in a broad anti-inflammatory response in animal models of several autoimmune diseases, while avoiding immunosuppression. Data generated from Phase 1a and 1b trials provide evidence that KZR-616 exhibits a favorable safety and tolerability profile for development in severe, chronic autoimmune diseases. Phase 2 trials are underway in severe autoimmune diseases. About Kezar Life Sciences Based in South San Francisco, Kezar Life Sciences is combining courage, conviction and cutting-edge science to develop breakthrough treatments for immune-mediated and oncologic disorders. The company is pioneering first-in-class, small-molecule therapies that harness master regulators of cellular function and inhibit multiple drivers of disease via a single target. KZR-616, a first-in-class selective immunoproteasome inhibitor, is being evaluated in severe and underserved autoimmune diseases. Additionally, KZR-261, the first clinical candidate for the treatment of cancer from the company's protein secretion program targeting the Sec61 translocon, is undergoing IND-enabling activities. For more information, visit www.kezarlifesciences.com, and follow us on Twitter at @KezarBio, Facebook and LinkedIn. Source: Kezar Life Sciences, Inc.

17

Apnimed Successfully Completes Phase 1 Study in Lead Program for Obstructive Sleep Apnea

– Data show favorable safety and pharmacokinetic profile for key component of AD109 – Lead program AD109 advancing into a Phase 2 study before year end CAMBRIDGE, Mass. September 17, 2020 – (BUSINESSWIRE) – Apnimed, a clinical-stage pharmaceutical company focused on developing medicines to treat sleep apnea and related disorders, today announced the results of a Phase 1 study for a component of the company’s lead combination drug candidate, AD109, which is being explored as a pharmacologic treatment for Obstructive Sleep Apnea (OSA). In this healthy volunteer study, R-oxybutynin, a key component of AD109, demonstrated a favorable pharmacokinetic (PK) profile and was well tolerated with no adverse events related to the study drug. “Obstructive Sleep Apnea represents a significant public health problem in the U.S. and around the globe and current treatment options do not meet the needs of patients,” said Larry Miller, M.D., chief executive officer of Apnimed. “We believe that AD109, an oral drug candidate dosed once-daily at bedtime, could be a significant breakthrough for these patients by giving them a simple, safe, and effective solution that does not require a CPAP device or surgery. The results from this study support our advancement of this program.” Miller continued by saying, “we look forward to initiating a Phase 2 study with AD109 in Q4 of this year.” About the study and results AD109 consists of the norepinephrine reuptake inhibitor (NRI) atomoxetine combined with the antimuscarinic R-oxybutynin. The study, called APC-001, was a Phase 1, randomized, single-dose, open-label, two-way crossover study conducted to evaluate the safety, tolerability, and PK of 2.5 mg of R-oxybutynin compared to 5 mg of racemic (R,S) oxybutynin in 24 healthy adult volunteers. While S-oxybutynin has been studied as a bladder antispasmodic, most of the antimuscarinic activity of racemic oxybutynin necessary to activate the upper airway muscles and maintain an open airway during sleep is thought to be mediated by R-oxybutynin. By purifying the R-oxybutynin and removing the S-oxybutynin, unwanted bladder effects can potentially be reduced and a lower dose of R-oxybutynin alone, relative to racemic oxybutynin, may be effective to treat OSA. Study participants received treatments in random order, administered as a single oral dose, with a washout period between treatments of at least 7 days. The results indicated that R-oxybutynin was well-tolerated and that there were no adverse events related to the study drug. The blood plasma concentrations of the 2.5 mg dose of R-oxybutynin, when administered alone, closely matched the profile of R-oxybutynin when dosed as 5 mg of racemic oxybutynin. Additionally, no interconversion to S-oxybutynin was observed following administration of 2.5 mg R-oxybutynin. About AD109 Apnimed’s lead product candidate, AD109, targets neurotransmitter levels in the central nervous system to activate upper airway muscles and maintain an open airway during sleep. AD109 is a first-in-class, oral pharmaceutical combination dosed once-daily at bedtime, designed to treat OSA patients across a broad spectrum of disease severity. The drug is designed to be safe, effective, and convenient, addressing the key limitations of the current standard of care treatments. Apnimed has successfully completed its first Phase 1 study with AD109 and Phase 2 clinical trials are planned for 4Q 2020. Proof of concept for the AD109 program was demonstrated in Apnimed study APN-002, a Phase 2, parallel group dose-finding study of the combination of atomoxetine and racemic oxybutynin. That study provided evidence of safety and efficacy of a norepinephrine reuptake inhibitor (NRI) + antimuscarinic combination in 140 patients. About Obstructive Sleep Apnea Obstructive Sleep Apnea (OSA) is one of the most common and serious sleep disorders and is estimated to affect more than 25 million Americans, though underdiagnosis continues to be a serious problem. OSA is characterized by partial or complete upper airway closure that occurs during sleep, which often leads to poor sleep, and in the long-term, hypertension, diabetes, cardiovascular disease, strokes, and early mortality. The vast majority of diagnosed patients are prescribed positive air pressure therapy devices such as continuous positive airway pressure, or CPAP, but fewer than half are compliant long-term, leaving a significant population untreated, undertreated and at risk. About Apnimed Apnimed is a clinical-stage pharmaceutical company working to transform the treatment of sleep apnea based on a simple idea – patients with Obstructive Sleep Apnea will benefit from treatment with a safe and effective oral medication. Apnimed’s lead development program targets nighttime neurotransmitter levels in the central nervous system to activate upper airway muscles and maintain an open airway during sleep. The drug is delivered as a convenient once-daily at bedtime. Based in Cambridge, Mass., the company is developing a portfolio of novel pharmacologic therapies for sleep apnea and related disorders. Learn more at Apnimed.com

08

Apic Bio Appoints Dr. Jorge Quiroz as Executive VP and Chief Medical Officer

Professional Services Close-Up September 8, 2020 Apic Bio reported the appointment of Jorge A. Quiroz, MD, MBA, as Executive Vice President, Chief Medical Officer. According to a media release, Dr. Quiroz brings over 20 years of scientific, clinical, and regulatory experience helping to develop and evolve gene therapies and small molecule drug programs from preclinical to regulatory filing. Most recently, Dr. Quiroz served as Chief Medical Officer of Solid Biosciences where he was responsible for leading the clinical advancement of its gene therapy program. "We are excited to welcome Jorge to Apic during this critical period for the company and our pipeline. His extensive expertise in the clinical and regulatory development of gene therapies for rare diseases will support the advancement of both our SOD1 amyotrophic lateral sclerosis (ALS) and alpha-1 antitrypsin deficiency (Alpha-1) programs," said John Reilly, MS, MBA, Co-founder and Chief Executive Officer of Apic. "As CMO, Jorge will also lead the development and build out of our early-stage gene therapy programs derived from our THRIVE platform. We look forward to working with Jorge during this next chapter for Apic as we rapidly advance our therapies into the clinic on behalf of patients and families in need." "I am delighted to join Apic during an exciting period of expansion as we prepare to submit an Investigational New Drug (IND) application for APB-102 for the treatment of patients with SOD1 ALS this year and enter the IND-enabling stage for APB-101 for the treatment of patients with alpha-1 antitrypsin deficiency," said Dr. Jorge Quiroz, EVP and Chief Medical Officer of Apic. "The Company's mission, deep scientific foundation, clinical approach, and manufacturing expertise puts us in an excellent position to bring new gene therapy treatments to patients living with rare and monogenic disorders." Dr. Quiroz previously served as the Head of Neurodevelopment & Psychiatry, Translational Medicine Neurosciences at F. Hoffmann-La Roche AG. He has also served as a Director at Johnson & Johnson Pharmaceutical Research & Development.

08

PhoreMost and XtalPi Sign AI-Based Pharmaceutical Drug Discovery Collaboration Agreement

PhoreMost Limited, UK September 8, 2020 - Collaboration to rapidly develop high-quality leads from novel targets and sites identified by PhoreMost’s proprietary SITESEEKER platform - XtalPi’s AI-based ID4 platform will explore ultra-large chemical spaces to discover novel inhibitors of protein-protein interaction sites PhoreMost Limited, the UK-based biopharmaceutical company dedicated to drugging ‘undruggable’ disease targets, and XtalPi Inc., a leading algorithm-driven artificial intelligence (AI)-based pharmaceutical technology company, today announced they have entered into a drug discovery collaboration agreement. Financial details are not disclosed. Under the terms of agreement, PhoreMost and XtalPi will rapidly identify and develop compounds to advance a drug discovery program against targets that epigenetically regulate tumour progression, and have been previously classified as ‘undruggable’. The companies will pursue this challenging goal with a unique combination of machine learning and physics-based computation methods, using XtalPi’s intelligent digital drug discovery and development (ID4) platform, to explore novel sites discovered by PhoreMost’s SITESEEKER® platform. PhoreMost’s SITESEEKER platform exploits protein shape diversity to find new peptide targets, significantly enhancing the power of phenotypic screening and translation into therapeutic modalities. Based on proprietary protein interference, or ‘PROTEINi®’, technology, PhoreMost uses SITESEEKER to probe the entire proteome in a live cell environment for novel druggable targets linked to any chosen disease. This enables the systematic discovery of functionally active peptides which are directly linked to useful therapeutic applications. Protein-protein interaction sites are traditionally considered ’undruggable’ by small-molecules due to their large flat surface and the absence of well-defined binding pockets. XtalPi’s ID4 platform integrates data-driven AI models with physics-based methods to quickly distil a small number of high-quality compounds from an AI-generated target-specific chemical space that includes millions of diverse molecules. Such diversity, combined with ID4’s drug property prediction algorithms’ speed and accuracy, allows scientists to unearth promising candidates, overcome the bottleneck of simultaneously optimising all pharmacological properties, and ensure a smooth progression into pre-clinical and clinical studies. "XtalPi’s AI-based ID4 and PhoreMost’s SITESEEKER are highly complementary platforms, and we look forward to working together with the team. This alliance marks an exciting chapter in PhoreMost’s development, as we now seek to rapidly progress our internal portfolio of novel first-in-class targets into drug discovery." - Dr. Chris Torrance, CEO of PhoreMost "Today, many diseases lack effective treatment because their corresponding targets are too challenging for traditional drug discovery methods. We are excited to combine PhoreMost’s target discovery technology with our ability to quickly identify lead compounds with desirable drug properties, and continuously translate high-quality ‘undruggable’ targets into first-in-class pipeline assets to address unmet medical needs." - Dr. Jian Ma, Co-Founder and CEO of XTALPI

03

Amylyx Pharmaceuticals Announces New England Journal of Medicine Publication of Pivotal AMX0035 Data Demonstrating Statistically Significant Benefit in People with ALS

- Patients Retained Function Longer on AMX0035 Versus Placebo; Study Achieved Its Primary Outcome of a Difference on the Revised ALS Functional Rating Scale - AMX0035 is the First Investigational Therapy to Demonstrate Statistically Significant Benefit on this Prespecified Primary Outcome in People with ALS Since Approved Therapy Edaravone - AMX0035 Showed Numerical Benefits on Secondary Outcomes Including Measures of Muscle Strength, Breathing, and Hospitalizations - AMX0035 Was Generally Well Tolerated with Similar Rates of Adverse Events Recorded in the AMX0035 and Placebo Groups September 02, 2020 CAMBRIDGE, Mass.--(BUSINESS WIRE)--Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, today announced the publication of results from the pivotal CENTAUR trial evaluating AMX0035 – an investigational neuroprotective therapy designed to reduce the death and dysfunction of motor neurons – in people with ALS in the New England Journal of Medicine (NEJM). “CENTAUR met its prespecified primary outcome, showing a clinically meaningful and statistically significant treatment benefit on the Revised ALS Functional Rating Scale (ALSFRS-R), the most commonly used scale in clinics worldwide to measure function in ALS,” said Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR trial, investigator at the Sean M. Healey & AMG Center for ALS at Mass General and Assistant Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital. “These results represent a major milestone for the ALS community, and I am thrilled about the promise of this therapy for people with ALS.” The CENTAUR trial of 137 individuals with ALS was conducted across 25 top medical centers in the U.S. through the Northeast ALS (NEALS) consortium. It demonstrated that treatment with AMX0035 was well tolerated and decreased the rate of decline in the ALSFRS-R compared to placebo in people with ALS. “The data published today in the New England Journal of Medicine show that AMX0035 demonstrated a clinically meaningful benefit and a favorable safety profile for people living with ALS. This development is a breakthrough for the ALS community and we are working collaboratively and expeditiously with agencies worldwide to bring this potential new treatment option forward,” said Josh Cohen, Co-CEO, Chairman and Co-Founder at Amylyx. “The data published today makes a clear and compelling case that AMX0035 should be made available to people with ALS as soon as possible,” said Calaneet Balas, President and CEO of The ALS Association. “We look forward to working with Amylyx, the FDA, and the entire community to help make that happen. We are grateful to all the Ice Bucket Challenge donors whose contributions helped make this trial possible.” CENTAUR RESULTS: Primary Outcomes: After 24 weeks, patients treated with AMX0035 scored on average 2.32 points higher on the ALSFRS-R than the placebo group (p=0.03) using the study’s primary prespecified analysis. A change from baseline analysis was also conducted and indicated that the AMX0035 group scored 2.92 points higher at the end of 24 week follow up (p=0.01). - The ALSFRS-R is a 48-point questionnaire measuring daily functions such as the ability to walk, dress independently, self-feed, speak and breathe. - Just a 1-2 point change in the ALSFRS-R score can indicate a significant reduction in a patient’s ability to function independently. The ALSFRS-R measures many different daily functions so point differences can manifest differently in different patients. Some examples of a two point change on this scale include the difference between an individual eating successfully with some difficulty vs needing a feeding tube, or walking with assistance versus not walking at all. Secondary Outcomes: In line with the primary outcome, patients on AMX0035 also showed numerical benefits on secondary outcomes including measures of muscle strength, breathing and hospitalization frequency, although the study was not powered for secondary outcomes. - Progression in lung function (percent predicted slow vital capacity) was numerically slower in patients taking AMX0035 (Least Squares Difference=5.11 points, p=0.08). - Participants in the AMX0035 group were hospitalized numerically less often (Hazard Ratio = 0.54, p=0.09). - Rate of decline in overall muscle strength (percent predicted ATLIS) was numerically slower in patients taking AMX0035 (Least Squares Difference=2.82 points, p=0.12). The effect of AMX0035 on progression was nominally statistically significant for the upper limbs measurements (Least Squares Difference=4.27 points, p=0.04). Overall Safety: - Nearly all participants (46 out of 48 patients in placebo (96%) vs 86 out of 89 patients in the AMX0035 group (97%)) reported one or more treatment-emergent adverse events (TEAEs) during the trial. Most were nonserious, did not lead to modification or interruption of study drug dosing, and were not considered related to the study. - Overall, safety was comparable between the groups, with fewer serious adverse events in the active group as compared to the placebo group (9 out of 48 patients (19%) in placebo vs 11 out of 89 patients (12%) in the AMX0035 group). - GI adverse events, which were generally characterized as mild by investigators, occurred more frequently in the active group in the first 3 weeks of the trial (28.1% vs 12.5% placebo) and returned to levels comparable to placebo thereafter. Most CENTAUR participants (77%) were receiving an approved ALS therapy (riluzole, edaravone, or both) during and/or before trial entry. Sensitivity analyses accounting for the duration of treatment under riluzole, edaravone, or both confirmed that the treatment effect of AMX0035 was independent of background approved ALS therapies. CENTAUR was the recipient of the ALS ACT grant, and is supported by The ALS Association, ALS Finding a Cure, a program of The Leandro P. Rizzuto Foundation, the Northeast ALS Consortium, Healey Center for ALS at Mass General, and was funded in part by the ALS Ice Bucket Challenge. “I am so proud of the ALS community efforts that made this milestone possible,” said Dr. Merit Cudkowicz, M.D., Chief Medical Officer from ALS Finding a Cure®, Director of the Sean M. Healey & AMG Center for ALS, Chief of Neurology at Mass General, and the Julianne Dorn Professor of Neurology at Harvard Medical School. “The CENTAUR study was designed and run through NEALS, was supported by a partnership between The ALS Association and ALS Finding a Cure, and is a phenomenal example of what can happen when a community works closely together to accelerate ALS progress.” AMX0035 Long-Term Survival and Extension Data, Future Plans Participants who completed CENTAUR were given the option after the trial to enroll in an open-label extension study and receive AMX0035 long-term. 92% of eligible CENTAUR participants elected to enroll in the extension study. Interim data from the ongoing open-label extension study are being submitted to a peer-reviewed journal shortly and will be published in the coming months. Long-term survival analysis for the patients in the AMX0035 and placebo groups has been conducted as well. These data will be submitted to a peer-reviewed journal in the near future. “We are deeply grateful to all of the CENTAUR participants and our partners who have helped and continue to help develop this important therapy for those living with ALS,” said Justin Klee, Co-CEO and Co-Founder of Amylyx. “We also look forward in the coming months to sharing results from the CENTAUR open-label extension study and the long-term survival analysis, and we will continue to keep the community closely informed on next steps.” About CENTAUR CENTAUR was a 24-week, randomized, double-blind, placebo-controlled Phase 2/3 clinical trial that evaluated the safety and tolerability of AMX0035 and assessed the drug’s impact on disease progression as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) when compared to placebo. The trial also assessed the effects of AMX0035 on other measures that are critical to people with ALS, including muscle strength, lung vital capacity, and biomarkers of neuronal degeneration. CENTAUR enrolled patients 18-80 years old with definite ALS and within 18 months of symptom onset. The trial did not restrict patients from receiving edaravone or riluzole. More information on the CENTAUR trial can be found at https://amylyx.com/trials/ or www.clinicaltrials.gov, NCT03127514. About Amyotrophic Lateral Sclerosis (ALS) ALS is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually death. The vast majority of patients with ALS (>90%) have sporadic disease, showing no clear family history. Approximately 6000 people are diagnosed with ALS in the United States every year with an approximately similar number of deaths every year. About AMX0035 AMX0035 is an investigational neuroprotective therapy designed to reduce neuronal death and dysfunction. AMX0035 targets endoplasmic reticulum and mitochondrial dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases. About Amylyx Pharmaceuticals Amylyx Pharmaceuticals, Inc. is a pharmaceutical company working on developing a novel therapeutic for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases. For more information, visit www.amylyx.com and follow us on LinkedIn and Twitter.

01

ViOptix Inc Awarded Patent for Oximeter with Marking Feature

Global IP News - Medical Patent News September 1, 2020 ViOptix Inc has been awarded a patent for oximeter with marking feature. This invention was developed by Heaton II Larry C and Lash Robert E. The patent application number is 15/904,299. The International Patent Classification codes are A61B 5/00 (20060101), A61B 5/1455 (20060101) and A61B 90/30 (20160101). Cooperative Patent Classification codes are A61B 5/7278 (20130101), A61B 5/14552 (20130101), A61B 5/742 (20130101), A61B 90/30 (20160201), A61B 5/7282 (20130101), A61B 2090/309 (20160201) and A61B 2090/308 (20160201). The abstract released by U.S. Patent and Trademark Office states, "A medical device such as an oximeter includes a marking feature. In an implementation, a marking mechanism of the device marks tissue based on a location of where a measurement was taken by the device. In an implementation, the marking mechanism of the device marks tissue based on an oxygen saturation measurement obtained by the device."

August 2020

26

Apnimed Strengthens its Scientific Advisory Board with Experts in Sleep Disorders

Businesswire CAMBRIDGE, Mass. Apnimed, a clinical-stage pharmaceutical company focused on advancing medicines to treat sleep apnea and related disorders, today announced its Scientific Advisory Board (SAB). The SAB will help guide the advancement of the company’s pipeline of small molecule solutions for the treatment of Obstructive Sleep Apnea, or OSA. “OSA represents a significant health problem for more than 25 million Americans, with some estimates significantly higher, and hundreds of millions more around the globe,” said Larry Miller, M.D. chief executive officer of Apnimed. “We are honored to have a group of highly esteemed leaders join our Scientific Advisory Board as we advance multiple pharmaceutical programs to address the significant unmet needs of patients suffering from and at risk of serious complications due to OSA.” Members of the Apnimed Scientific Advisory Board: - John Cronin, M.D., Chief Medical Officer, Sleep and Respiratory Care, Philips Healthcare - Danny Eckert, Ph.D., Director, Adelaide Institute for Sleep Health, Deputy Director Flinders Health and Medical Research Institute and Matthew Flinders Professor, College of Medicine and Public Health at Flinders University - Diane Lim, M.D., Assistant Professor of Medicine at the Hospital of the University of Pennsylvania and the Veteran’s Administration Medical Center - Reena Mehra, M.D., M.S., Professor of Medicine, Cleveland Clinic Lerner College of Medicine and Director, Sleep Disorders Research, Neurologic Institute - Carlos M. Nunez, M.D., Chief Medical Officer at ResMed - Naresh Punjabi, M.D. Ph.D., Chief, Division of Pulmonary, Allergy, Critical Care, and Sleep Medicine at University of Miami Health System, Jackson Memorial Hospital - Susan Redline, M.D., M.P.H., Director, Sleep Medicine Epidemiology, Physician, Brigham and Women’s Hospital, and Professor of Medicine, Harvard Medical School - Alan Schwartz, M.D., Professor of Medicine, ret., Johns Hopkins University and Adjunct Professor, Perelman School of Medicine, University of Pennsylvania - Andrew Wellman, M.D, Ph.D., Director, Sleep Disordered Breathing Lab at Brigham and Women’s Hospital and Associate Professor, Harvard Medical School About Obstructive Sleep Apnea Obstructive Sleep Apnea (OSA) is one of the most common and serious sleep disorders and is estimated to affect more than 25 million Americans, though underdiagnosis continues to be a serious problem. OSA is characterized by partial or complete upper airway closure that occurs during sleep, which often leads to poor sleep, and in the long-term, hypertension, diabetes, cardiovascular disease, strokes, and early mortality. The vast majority of diagnosed patients are prescribed positive air pressure therapy devices such as continuous positive airway pressure, or CPAP, but fewer than half are compliant long-term, leaving a significant population untreated and at risk. About Apnimed Apnimed is a clinical-stage pharmaceutical company working to transform the treatment of sleep apnea based on a simple idea – patients with obstructive sleep apnea will benefit from treatment with a safe and effective oral medication. Apnimed’s lead development program targets nighttime neurotransmitter levels in the central nervous system to activate upper airway muscles and maintain an open airway during sleep. The drug is delivered as a convenient once-a-day dose before bed. Based in Cambridge, Mass., the company is developing a portfolio of novel pharmacologic therapies for sleep apnea and related disorders. Learn more at Apnimed.com

24

Atea Pharmaceuticals Strengthens Management Team with Appointment of Clinical and Regulatory Leadership

Contify Life Science News Atea Pharmaceuticals, Inc. issued the following news release: - Executive Vice President of Clinical Sciences and Vice President of Regulatory Bring Decades of Clinical and Regulatory Experience Atea Pharmaceuticals, Inc., a biopharmaceutical company engaged in the discovery and development of next-generation therapeutics for severe human viral infections, today announced the appointments of Arantxa Horga, M.D., as Executive Vice President of Clinical Sciences and Mihaela MacNair, Ph.D., as Vice President of Regulatory. "These new additions to our leadership team will provide critical guidance as we approach late-stage clinical development of AT-527 as a treatment for COVID-19," said Jean-Pierre Sommadossi, Ph.D., Founder, Chairman and Chief Executive Officer of Atea. "Collectively, Arantxa and Mihaela's experience and clinical expertise will be invaluable as we both advance our current programs and expand our pipeline of oral direct acting antivirals to combat severe viral infections." Dr. Horga is an expert in infectious diseases drug development with over 15 years of broad biopharmaceutical experience including clinical development program leadership, clinical operations and pharmacovigilance. She joins Atea from Biohaven Pharmaceuticals, where she served as Vice President, Pharmacovigilance and Medical Affairs. Before that, she was at Hoffmann-La Roche where she served as Vice President, Global Head of Clinical Program Execution, Site Head of the Roche NY Innovation Center and as Global Head of Translational Medicine, Infectious Diseases. Dr. Horga began her career in drug discovery and development at Bristol-Myers Squibb where she rose through a series of clinical leadership positions to become Group Director, Global Clinical Research, Virology. She holds an M.D. from the Santander School of Medicine, and completed her residency in Pediatrics and fellowship in Pediatric Infectious Diseases at the Mount Sinai School of Medicine, where she remained as faculty. During her academic tenure, her NIH-funded laboratory research focused on mechanisms of pathogenesis of respiratory viruses. Dr. MacNair joins Atea from Biogen, where she served as Senior Director of Global Regulatory Affairs and Regulatory Therapeutic Area Head covering neurodegenerative and neuromuscular diseases. Having started her career in the pharmaceutical industry in the EU, Dr. MacNair has pursued opportunities in the US where she held positions of increasing global responsibility with pharmaceutical companies including Vertex Pharmaceuticals, Alkermes, and Shire (now Takeda Pharmaceuticals). Dr. MacNair holds an M.Sc. in Organic Chemistry from the Polytechnical Institute of Bucharest, Romania, and an M.Sc. in Pharmaceutical Products Development and International Registration from Universit Paris-Sud, France. Dr. MacNair holds a Ph.D. in Analytical Spectrochemistry from Universit Paris 7, Denis Diderot, France. About Atea Pharmaceuticals Atea Pharmaceuticals is a clinical stage biopharmaceutical company engaged in discovering and developing therapies to address the unmet medical needs of patients with severe viral diseases. Our lead programs are focused on the development of orally-administered direct acting antivirals for the treatment of patients with COVID-19 in the hospital and community settings, the treatment of patients with chronic hepatitis C infection, the treatment of patients with dengue, and the treatment of high-risk patients with severe respiratory syncytial virus infection. Our medicinal chemistry, virology, and pharmacology expertise, bolstered by our collective experience in drug development, enables us to pioneer new advancements in antiviral science. Leveraging the power of our purine nucleotide prodrug platform, our goal is to rapidly advance novel drug candidates with optimal therapeutic profiles for RNA virus targets. Founded by its Chairman and Chief Executive Officer, Jean-Pierre Sommadossi, PhD, Atea began operations in 2014 and is headquartered in Boston, MA. For more information about Atea and our pipeline of product candidates please visit our company website at ateapharma.com.

20

LumiraDx Receives FDA Emergency Use Authorization for Point of Care COVID-19 Antigen Test

LONDON, Aug. 20, 2020 /CNW/ -- LumiraDx, the next-generation point of care diagnostic company, announced today that it has received Emergency Use Authorization (EUA) from the U.S. Food and Drug Administration (FDA) for the LumiraDx SARS-CoV-2 antigen test, which will help meet the global challenge of delivering fast and cost-efficient COVID-19 tests in community care settings. The test detects antigen nucleocapsid protein from a nasal swab with results in under 12 minutes from sample application in symptomatic patients. In clinical studies, the LumiraDx SARS-CoV-2 antigen test demonstrated 97.6% positive agreement versus PCR in patients tested within 12 days of the onset of symptoms, making it the fastest, most sensitive antigen point of care test currently commercially available. "Actionable diagnostic results at the point of care lead to better health outcomes," said Ron Zwanziger, LumiraDx CEO. "Now that the FDA EUA has been received, we are working with health systems, major retail clinics, and employers to get our Platform to healthcare providers quickly to utilize in their testing programs." The company plans to begin shipping COVID-19 antigen tests by the end of the month, expects to produce 2 million tests in September, and will ramp to 10 million tests produced in December. LumiraDx High Sensitivity Point of Care Platform The LumiraDx Platform simplifies, scales down, and integrates techniques used in laboratory analyzers to provide lab-comparable diagnostic tests on a single point-of-care instrument that can be easily used in community care settings. The platform consists of a small, portable instrument; microfluidic test strip; simple, standardized workflow; and seamless, secure digital connectivity to the cloud and hospital IT systems. It is designed to offer: * Lab comparable performance at point of care in minutes – Each test is developed and validated against its respective lab reference standard, which we believe gives healthcare providers the benefit of both lab comparable performance and real-time results. * Broad menu of tests on a single instrument – The platform offers an INR (International Normalized Ratio) test for the management of anticoagulation patients in Europe under CE Mark. The company has a pipeline of over 30 assays across cardiovascular, infectious disease, diabetes and coagulation disorders with capability to run immunoassay, chemistry, molecular and other technologies, as well as multiple sample types using a single platform and workflow. * Low cost of ownership – The LumiraDx instrument aims to lower the cost per reportable result by offering a variety of low-cost test strips on a single instrument and associated savings including reduced cost of training, maintenance and supplies. * High quality assurance – The Platform performs more than 30 quality checks during its operation, with performance referenced to standard laboratory methods. LumiraDx COVID-19 Antigen and Antibody Tests on a Single Platform In addition to the antigen test, the LumiraDx SARS-CoV-2 antibody test, for use with the LumiraDx Instrument, will shortly be submitted to the FDA for EUA review. LumiraDx EUA for Fast Lab Solution LumiraDx recently also received an EUA for its molecular lab reagent kit LumiraDx SARS-CoV-2 RNA STAR, a Fast Lab Solution that utilizes LumiraDx's innovative qSTAR amplification technology in an accessible high-throughput format with open molecular systems to improve efficiency and speed. LumiraDx SARS-CoV-2 RNA STAR reduces the amplification step of the PCR process from approximately one hour down to 12 minutes. EUA Status of LumiraDx SARS-CoV-2 Ag test and LumiraDx SARS-CoV-2 RNA STAR The LumiraDx SARS-CoV-2 Ag test and the LumiraDx SARS-CoV-2 RNA STAR have not been cleared or approved by FDA. The LumiraDx SARS-CoV-2 Ag test has been authorized by FDA under an EUA only for the detection of SARS-CoV-2 nucleocapsid protein. LumiraDx SARS-CoV-2 RNA STAR has been authorized by FDA under an EUA only for the detection of nucleic acid from SARS-CoV-2. They have not been authorized for use to detect any other viruses or pathogens. The tests are authorized in the United States for the duration of the declaration that circumstances exist justifying the authorization of emergency use of in vitro diagnostic tests for detection and/or diagnosis of COVID-19 under Section 564(b)(1) of the Act, 21 U.S.C. § 360bbb-3(b)(1), unless the authorization is terminated or revoked sooner. About LumiraDx LumiraDx was founded in 2014 by a group of entrepreneurs: Ron Zwanziger, our Chairman and Chief Executive Officer; Dave Scott, Ph.D., our Chief Technology Officer; and Jerry McAleer, Ph.D., our Chief Scientist, who have a successful track record in building and scaling diagnostics businesses over three decades, including at companies such as Medisense, Inc., Inverness Medical Technology Inc. and Alere Inc. The company has raised approximately $600 million through debt and equity from institutional and strategic investors including the Bill & Melinda Gates Foundation, Morningside Ventures and U.S. Boston Capital Corporation. Based in the UK, with primary R&D and manufacturing operations in Stirling, Scotland, and supported by its worldwide affiliates to provide access in all major markets, LumiraDx has over 600 employees worldwide. LumiraDx develops, manufactures and commercializes an innovative point-of-care diagnostic Platform. The LumiraDx Platform is designed to deliver lab comparable diagnostic results at the point of care in minutes. It is designed to be affordable and accessible for healthcare providers globally, and to strengthen community-based healthcare. Further information on LumiraDx and the LumiraDx Platform is available at lumiradx.com.

12

F2G Closes US$60.8 Million Financing to Fund Late Stage Development of Novel Mechanism Antifungal Agent

PR Newswire Europe Financing round led by Cowen Healthcare Investments with strong support from existing investors Novo Holdings, Morningside Ventures, Brace Pharma Capital and Advent Life Sciences F2G Ltd, a UK- and Austria-based biotech developing novel therapies for life-threatening systemic fungal infections, announced today that it has secured US $60.8 million in new financing from new and existing investors. The financing round is led by Cowen Healthcare Investments and includes strong participation from existing investors Novo Holdings, Morningside Ventures, Brace Pharma Capital and Advent Life Sciences. Proceeds from the financing will be used to fund F2G's late-stage clinical programs for their novel antifungal agent olorofim and organisational scale-up in preparation for commercialisation. Olorofim (formerly F901318) is F2G's lead candidate and is in a Phase 2b open-label study focussing on rare and resistant life-threatening invasive fungal infections, such as invasive aspergillosis (including azole-resistant strains), scedosporiosis, lomentosporiosis, fusariosis, scopulariopsosis, and coccidioidomycosis (Valley Fever). Olorofim was granted Breakthrough Therapy designation (BTD) for the indication of 'Treatment of invasive mold infections in patients with limited or no treatment options, including aspergillosis refractory or intolerant to currently available therapy, and infections due toLomentospora prolificans,Scedosporium, andScopulariopsisspecies' in November 2019 by the US Food and Drug Administration (FDA), the only antifungal agent ever to have been awarded this status. Olorofim has the potential to be the first truly novel mechanism antifungal therapy for nearly twenty years and represents a very significant market opportunity in an area of high unmet clinical need. Commenting on the news, Ian Nicholson, CEO of F2G Ltd, said: "Following a successful year during which F2G has received FDA Breakthrough Therapy designation for olorofim, as well as FDA Orphan Drug Designation for Coccidioidomycosis (Valley Fever), and QIDP designation in multiple fungal infections, today's announcement is a significant milestone. We are delighted to welcome Cowen Healthcare Investments to F2G, and I would like to thank our existing investors for their continued support. This financing marks the continued commitment of our shareholders and paves the way for the advanced development and potential approval of the first new antifungal treatment in 20 years, offering hope for patients with very limited treatment options and a high medical need." Tim Anderson, Managing Director at Cowen Healthcare Investments said: "The necessity for the discovery and development of treatments to tackle infectious diseases is today more apparent than ever. F2G's antifungal candidate demonstrates significant promise in terms of safety, tolerability, and efficacy. With our focus on supporting transformational science that can deliver real clinical outcomes, we are pleased to work with this proven management team and group of renowned investors to build on F2G's significant scientific and commercial potential." Joining the F2G Board in conjunction with the financing will be Tim Anderson, Managing Director at CHI, Will West, Investment Advisor at Morningside Ventures and Naveed Siddiqi, Partner at Novo Ventures. Naveed takes over from Martin Edwards who is retiring from the Board. Ian Nicholson added: "We welcome Tim Anderson, Will West and Naveed Siddiqi who replaces Martin Edwards as the representative of Novo Holdings on the Board of Directors. On behalf of the Board we would like to thank Martin for his contribution to F2G. His industry expertise and counsel have been instrumental in guiding F2G through recent developmental and financing milestones, and we wish him the very best." About F2G F2G is a world-leading UK- and Austria-based biotech company (F2G Ltd and F2G Biotech GmbH) focused on the discovery and development of novel therapies to treat life-threatening invasive fungal infections. F2G has discovered and developed a completely new class of antifungal agents called the orotomides. The orotomides target dihydroorotate dehydrogenase (DHODH), a key enzyme in the de novo pyrimidine biosynthesis pathway. This is a completely different mechanism from that of the currently marketed antifungal agents and gives the orotomides fungicidal activity against a broad range of rare and resistant fungal mould infections. Olorofim (formerly, F901318) is F2G's leading candidate from this class.http://www.f2g.com About Olorofim Olorofim is currently being investigated in an open-label single-arm Phase 2b study (ClinicalTrials.gov Identifier: NCT03583164) in patients with proven invasive fungal disease (IFD) or probable invasive aspergillosis (IA) with limited treatment options (refractory disease, resistance, or intolerance to available agents). On 7 November 2019, olorofim was granted Breakthrough Therapy designation (BTD) by the US Food and Drug Administration (FDA), the only antifungal agent ever to have been awarded this status.

07

Vigeo's Tumor Microenvironment Platform Expands Indications and Combo Therapy Possibilities

Gail Dutton BioSpace Vigeo Therapeutics is expanding indications for its tumor microenvironment (TME)-targeting oncology platform, which inhibits tumor growth by reprogramming normal cells that were recruited and coopted by a tumor. Dose expansion for its Phase Ib/II study is ongoing for multiple solid tumors, and combination studies with immune checkpoint inhibitors and with standard-of-care chemotherapy are being planned for the latter half of this year. “The company was founded on the idea that targeting the tumor microenvironment (TME) is best strategy for treating cancer,” Jing Watnick, Ph.D., CEO, said during a presentation at the LifeSci Partners Summer Symposium on August 5. Vigeo’s platform approach “reprograms myeloid-derived suppressor cells in IO-cold patients’ TME, activates cytotoxic T lymphocyte (CTL) cells and M1 Tumor-associated macrophages (TAMs) in that microenvironment, and converts the environment from immuno-suppressive to immuno-stimulated,” she explained. Within the industry, therapeutic programs that target the tumor microenvironment have been beset by challenges “related to the size of the protein, delivery and stability,” she said. “Our program lets us overcome those burdens.” VT1021, Vigeo’s lead compound, is a first-in-class CD36/CD47 dual-targeting compound. “VT1021 is a five amino acid cyclic peptide derived from prosaposin (Psap). Psap is a precursor of saposin A, B, C and D, and functions as a lipid hydrolase activator,” Watnick said. The compound binds to myeloid-derived suppressor cells in the tumor microenvironment and induces the production of thrombospondin-1 (TSP-1) within the tumor. Then TSP-1 binds both to CD36 and CD47 to induce antitumor effects and therefore suppress the growth of tumors that have high expression of CD36 or CD47. “That is a very important aspect of the compound,” Watnick noted. “By binding to CD36, CD47, and other cell surface molecules, TSP-1 induces apoptosis in tumor and endothelial cells; regulates cell adhesion, migration, invasion, and proliferation; and modulates the immune and inflammatory responses. TSP-1 is fully activated in the TME.” Breaking down the interactions by binding site, she pointed out that when TSP-1 binds to CD36, it induces apoptosis in tumor and endothelial cells and increases the M1:M2 macrophage ratio, which affects cell adhesion and survival. By binding to CD47, TSP-1 stimulates CD8+ T cell functions, and also blocks what she called “do not eat me” signals, allowing the macrophages to kill the tumor cells. It also increases activity of CTL cells, allowing better targeting of tumor cells. Compared to magrolimab, an anti-CD47 therapy, Watnick said, “VT1021 has 1,000 times more affinity than magrolimab, so it avoids the toxicity of some of the anti-CD47 programs. It also has the benefit of binding to CD36,” which makes it more potent than anti-CD47 therapies. In preclinical studies, VT1021 showed strong single-agent efficacy, exhibited by tumor regression and inhibition of metastasis in mouse models of ovarian, pancreatic, breast, glioblastoma, prostate, lung and melanoma solid tumors. “What’s most interesting,” Watnick said, “is that in the ovarian tumor model, we stopped treatment at day 50 and then re-challenged the model one month later. Within one week of re-administration, VT1021 again triggered antitumor activity and again caused tumor regression.” In the pancreatic tumor model, the tumor size was reduced by half between day 25, when the treatment was administered, and day 45 when the models were assessed. At that time, she continued, “The animals also had slightly higher M1:M2 ratios, which indicated that the treatment was reprogramming the microenvironment.” As Watnick noted, “Resistance to various chemotherapies strongly correlates to upregulation of CD47, which sensitizes cells to TSP-1-mediated killing. When combined with anti-PD-1 therapy, VT1021 alleviates several sources of tumor suppression.” Vigeo expects to begin combination trials by year’s end. In pancreatic cancer studies, combining VT1021 and an anti-PD-1 monoclonal antibody (mAb) resulted in synergistic effects that improved survival and regressed the tumor. Watnick showed data from a study by Sui, et al, scheduled for Nature later this year. In that chart, the longest survivability for subjects receiving the anti-PD-1 mAb was 35 days after the initial treatment; for those receiving VT1021, the longest survival was 40 days; and for those receiving the combination therapy the longest survival was 75 days. Data from Vigeo Therapeutics’ dose escalation study also appeared promising. Of the 28 patients who could be evaluated, 43% achieved a partial response or disease stability. One (with thymoma, a tumor originating from the epithelial cells of the thymus) had a partial response that probably exhibited high expression levels of CD36 and CD47. “I said ‘probably’ because the size of the tumor was too small to biopsy, but a biomarker array analysis concluded it most likely had high expression levels of DC36 and CD47. “The 11 who achieved disease stability had very striking binding, which made us very excited,” she continued. Based on those indications, “High CD36 and CD47 expression with TSP-1 induction could be a very strong panel of indicators of clinical response.” That possibility is being evaluated in the clinic now. Vigeo Therapeutics’ clinical trial development plan for VT1021 calls for completing the expansion studies for multiple indications, followed by an FDA meeting in 2021, leading to a monotherapy trial for select indications. Watnick said she is hoping for accelerated approval in 2023 and the onset of a confirmatory trial in 2024. For combination trials, the company plans to stagger the launches for four indications. If they launch as scheduled, completion of the last trial is anticipated by the end of 2022. The Cambridge, Massachusetts company raised a total of $27.5 million in Series A and Series B rounds through Morningside Venture Investments in 2015 and 2018. Now it is raising Series C funding to support continued clinical development. “We’re thinking of going out aggressively, presenting our data to potential partners,” Watnick added. “We’re open to discussions.”

July 2020

11

UMass Medical School Announces ALS Breakthrough

Grant Welker, Worcester Business Journal July 8, 2020 Researchers at UMass Medical School and Massachusetts General Hospital have announced a major breakthrough in ALS research: a therapy that suppresses a harmful gene that causes the nervous system disease. The advancement, announced by researchers Wednesday and set to appear in the New England Journal of Medicine, is the latest made by UMass Medical School researchers, who've also identified critical genes that influence the development of ALS. A gene called SOD1 carries a genetic defect causing ALS, or amyotrophic lateral sclerosis, which gradually harms a victim's ability to move. New work by UMass Medical School researchers has been able to reverse that damage using something called a vector. In a lab, researchers have been able to transform a harmless virus, replacing its DNA with a synthetic RNA, or ribonucleic acid, that's specifically designed to attack the SOD1 gene. In a study, that altered virus was injected into participants' spinal fluid, delivering the lab-modified DNA. Far less harmful proteins were created by the gene after the injection, researchers found. The researchers are the first to safely treat two research participants with a synthetic microRNA, the medical school said. The hope is that by targeting the SOD1 gene, the breakthrough can slow down or even reverse the progression of that type of ALS. The study was led by two UMass Medical School researchers: Dr. Robert Brown Jr., a professor of neurology and director of the school's neurotherapeutics program, and Dr. Christian Mueller, an associate professor of pediatrics. They worked in consultation with two doctors at Mass. General: Dr. Merit Cudkowicz, the hospital's chief of neurology, and Dr. James Berry, the chief of the hospital's division of ALS and motor neuron diseases. "The biggest takeaway from this study is that we delivered a new class of silencing gene therapy to patients and suppressed levels of the ALS gene SOD1 quite effectively," Dr. Mueller said in a statement. Apic Bio, a Cambridge company founded by Brown and Mueller, has licensed the technology from the medical school and is leading the next phase of clinical trials. Dr. Brown has long been a leader in ALS research. In 1993, a team he led discovered the first gene linked to hereditary ALS, which is now known as SOD1. Among other ALS research done at UMass Medical School, Dr. John Landers, a neurology professor, has also discovered new genes related to ALS that could help researchers find a cure. Some of the research has been funded by proceeds from the Ice Bucket Challenge, a popular fundraising initiative in 2014. ALS, also known as Lou Gehrig's disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. The disease gradually makes it more difficult for a victim to speak, swallow, walk or even breathe. More than 5,000 people are diagnosed with it each year, and face a life expectancy of two to five years, according to the ALS Association. About 5% to 10% of ALS cases are inherited. Toxic mutations in the SOD1 gene account for about 20% of inherited ALS cases, according to UMass Medical School.

May 2020

20

Atea Pharmaceuticals Announces IND Clearance of AT-527 for COVID-19 and $215 Million Financing

May 20, 2020 BOSTON, May 20, 2020 (GLOBE NEWSWIRE) -- Atea Pharmaceuticals, Inc., a biopharmaceutical company engaged in the discovery and development of next-generation therapeutics for severe human viral infections, today announced a $215 million Series D financing. The financing was led by Bain Capital Life Sciences and also included new investors RA Capital Management, Perceptive Advisors, Rock Springs Capital, Adage Capital Management, funds and accounts managed by T. Rowe Price Associates, Inc., Redmile Group, and Omega Funds. Existing Atea investors, including Morningside Ventures, Cormorant Asset Management, Ally Bridge Group, and Sectoral Asset Management, as well as other investors also participated in this financing. Atea also announced today that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug application (IND) for AT-527, a novel, oral, purine nucleotide prodrug, for the treatment of adult patients hospitalized with moderate COVID-19 disease, with one or more risk factors for poor outcomes. A Phase 2 clinical trial, scheduled to begin shortly, will evaluate the safety and efficacy of AT-527 in this patient population. AT-527 is a highly selective, orally administered direct acting antiviral, (DAA) designed to inhibit the RNA polymerase enzyme, a key element in the replication machinery of RNA viruses. Antiviral activity of AT-527 has been observed in vitro and in vivo against replication of multiple RNA viruses including, but not limited to, human coronaviruses and flaviviruses. In addition to supporting its work to find a treatment for COVID-19, Atea expects to also apply proceeds from this financing towards advancing its diverse pipeline of highly selective DAAs that target other severe RNA viral infections. Atea's pipeline currently includes investigative treatments for hepatitis C virus, dengue virus, and respiratory syncytial virus, in addition to its COVID-19 program. "We are delighted to have the strong support of this group of blue-chip healthcare investors," said Jean-Pierre Sommadossi, PhD, Atea's Founder, Chairman, and Chief Executive Officer. "Atea's portfolio is focused on developing novel, best-in-class, potent DAA's and we have shifted all of our immediate resources and our team's deep expertise in virology and pharmacology to help address the unmet needs in the fight against the COVID-19 pandemic. An oral treatment for COVID-19 patients should prevent progression of the disease and may help lessen the burden on critical inpatient resources. Atea is moving rapidly, in concert with regulatory authorities, to determine if our oral DAA is a safe and effective therapeutic against COVID-19." "Atea's team has an outstanding track record in developing novel, potent DAAs, which we believe can contribute to the urgent fight against the COVID-19 pandemic and other RNA viruses," said Andrew Hack, M.D., Ph.D., Managing Director of Bain Capital Life Sciences. "We are pleased to partner with Atea's leadership team and an outstanding group of leading healthcare investors as Atea advances its diverse pipeline of transformative antiviral medicines" About AT-527 AT-527 is an investigational, oral, purine nucleotide prodrug, which has demonstrated in vitro and in vivo antiviral activity against several enveloped single-stranded RNA viruses, including human flaviviruses and coronaviruses. This highly selective purine nucleotide prodrug was designed to uniquely inhibit viral RNA dependent RNA polymerase, an enzyme that is essential for the replication of RNA viruses. Antiviral activity and safety of AT-527 has been demonstrated in Phase 2 clinical studies of hepatitis C patients. AT-527 is not yet licensed or approved for any indication in the U.S. or any other country. About Atea Pharmaceuticals Atea Pharmaceuticals is a clinical stage biopharmaceutical company engaged in discovering and developing best-in-class therapies to address the unmet medical needs of patients with severe viral diseases. Our lead programs are focused on the development of orally-administered direct acting antivirals for the treatment of patients with mild to moderate COVID-19 in the hospital and community settings, the treatment of patients with chronic hepatitis C infection, the treatment of patients with dengue, and the treatment of high-risk patients with severe respiratory syncytial virus infection. Our medicinal chemistry, virology, and pharmacology expertise, bolstered by our collective experience in drug development, enables us to pioneer new advancements in antiviral science. Leveraging the power of our purine nucleotide prodrug platform, our goal is to rapidly advance novel drug candidates with optimal therapeutic profiles for RNA virus targets. Founded by its Chairman and Chief Executive Officer, Jean-Pierre Sommadossi, PhD, Atea began operations in 2014 and is headquartered in Boston, MA. For more information about Atea and our pipeline of products please visit our company website at https://www.ateapharma.com/.

January 2020

21

Inotrem Secures Strategic Licensing Agreement for a Companion Diagnostics Test in Septic Shock

PARIS, January 21, 2020 (BUSINESS WIRE) -- Inotrem S.A., a biotechnology company specialized in the development of immunotherapies targeting the TREM-1 pathway with potential applications for acute and chronic inflammatory syndromes, announced today that it has entered a worldwide licensing agreement with Roche Diagnostics for the commercialization of a mechanism-based companion diagnostic test using a soluble plasma protein (sTREM-1).

This agreement consolidates existing ties between Roche Diagnostics and Inotrem who are jointly developing since 2017 an in vitro assay for measurement of sTREM-1 in plasma samples of septic shock patients. Measurement of sTREM-1 in blood provides a valuable indicator for the severity and outcome prediction of septic shock patients.

One of the main issues with septic shock is the heterogeneity of the patient population. The companion diagnostic test will allow a stratification of patients to identify those who are more likely to respond to Inotrem’s treatment. Septic shock is the ultimate complication of sepsis. The incidence of septic shock continuously raises, and mortality remains elevated (35%) in developed countries. There is currently no specific mechanism-based therapy approved for this indication besides antibiotics and symptomatic treatment. Inotrem’s therapeutic solution has the potential to become the first targeted treatment for septic shock.

“We are delighted to extend our partnership with Inotrem to make the sTREM-1 test, which we are co-developing at Roche, available for patients globally. Through the development and commercialization of this novel companion diagnostic test we are committed to delivering a solution that enables much needed, better decisions for sepsis patients”, said Ann Costello, Global Head Centralised and Point of Care Solutions, Roche Diagnostics.

“Roche Diagnostics’ licensing agreement is an important milestone for us: it is a strong endorsement of Inotrem’s innovative approach targeting the TREM-1 pathway, and it allows us to further focus on our core mission: the development of a nangibotide-based septic shock treatment” added Dr. Jean-Jacques Garaud, CEO of Inotrem.

Inotrem has recently initiated its Phase IIb study in septic shock patients (ASTONISH trial) and enrolled its first patient. The study aims at demonstrating efficacy of its lead compound, nangibotide, and bring a clinically relevant proof of clinical activity in septic shock patients. This study also intends to confirm the value of soluble TREM-1 as a potential companion diagnostic test to identify patients more likely to benefit from nangibotide treatment.

21

PhoreMost Enters Multi-Project Drug Discovery Collaboration With Otsuka Pharmaceutical

CAMBRIDGE, England, January 21, 2020 (BUSINESS WIRE) --PhoreMost Limited, the UK-based biopharmaceutical company dedicated to drugging ‘undruggable’ disease targets, today announced it has entered into a multi-project collaboration with Otsuka Pharmaceutical Co., the world-leading pharmaceutical company dedicated to the research and development of highly-innovative drugs and diagnostics. Financial details of the agreement are not disclosed. PhoreMost will deploy its in-house expertise and next-generation phenotypic screening platform, SITESEEKER®, towards disease relevant pathways nominated by Otsuka. Novel targets identified will be further validated and characterised by Otsuka as part of its internal development pipeline, with an initial focus on gene therapy applications of identified targets.

PhoreMost’s SITESEEKER platform exploits protein shape diversity to find functionally active peptides, significantly enhancing the power of phenotypic screening and translation into therapeutic modalities. Based on proprietary protein interference, or ‘PROTEINi®’, technology PhoreMost uses SITESEEKER to probe the entire proteome in a live cell environment for novel druggable targets linked to any chosen disease. This enables the systematic discovery of functionally active peptides which are directly linked to useful therapeutic applications.

Dr Chris Torrance, CEO of PhoreMost, said“This collaboration with Otsuka is further recognition of the power of the SITESEEKER approach to drive the identification of novel, druggable targets. We are particularly excited to be exploring not only small molecule therapeutics but also gene therapy applications of our platform.”

15

Alyce Named Leader in the G2 Winter 2020 Account-Based Execution Software Grid Report

BOSTON, MA, January 15, 2020 (BUSINESS WIRE) -- Alyce, the AI-powered gifting platform that’s redefining sales and marketing outreach, today announced its position in the Leader quadrant in G2’s Winter 2020 Grid Report for Account-Based Execution. Leaders are determined by their high levels of customer satisfaction and likeliness to recommend ratings from real users on G2, the world’s leading business solutions review website. As part of this Winter 2020 report, Alyce also scored highest in G2's Relationship Index for the Account-Based Execution category (with a score of 9.59 out of 10), which is measured based on ease of doing business, likely to recommend, and quality of customer support.

Alyce also scored highest in the same Account-Based Execution category (9.21 out of 10) in G2's Usability Index, ranking companies based on ease of administration, ease of use, and meeting other key requirements.

“Enterprise companies are turning to Alyce to transform their approach to growing their business, looking to create personal experiences and authentic connections that resonate with buyers and customers,” said Sean MacPherson, Head of Customer Success. “Alyce's commitment to creating lasting and sustained relationships with our customers and helping them see success is resonating with the market and we are thrilled by G2’s recognition as a category leader. This is truly a testament to the dedication, spirit, and strength of our customer success team.”

“Rankings on G2 reports are based on data provided to us by real users of the products,” said Michael Fauscette, Chief Research Officer at G2. “Technology buyers use the ranking on G2 to find the best software products based on the real-world voice of the user, accelerating their digital transformation projects and gaining competitive advantage.”

Alyce has received more than 125 G2 Crowd reviews. Review highlights include:

  • “Alyce is the best door opener - companies that I've been working for months, not responding whatsoever, finally answering me because it's a unique AND personal way to reach someone." ~Allison F.
  • “Creative and personalized way to engage prospects. Alyce's is a great way to engage with prospects on a personal level. The AI will recommend gifts to send to prospects based on a variety of factors - all of which increase your chance of getting a response from them. Giving the option to exchange gifts or donate the value of the gift to charity is also an awesome aspect of Alyce - I am really a big fan of the charity donation.” ~William R.

Since its inception, Alyce has been focused on creating real personal experiences that help businesses grow with the only platform that gives sales, marketing, and customer teams the ability to create individual personal gifts at scale. Fully focusing on the recipient and customers’ interests, rather than sending everyone the same generic item, Alyce bridges the physical and digital marketing worlds to help companies create breakthrough connections engaging people on their own terms. Providing the power of choice, recipients can select the personal gift recommended, pick something else from the Alyce gift marketplace, or even choose to donate the value of a gift to a charity of their interest.

“We’ve had a huge year at Alyce with 1,200% growth, closing out the year with some of the largest enterprise brands, including 6Sense, Bandwidth, Citrix, Dialpad and ServiceNow selecting our platform, and more importantly, investing in our unique personal experience business approach to help them scale and grow more effectively,” said Greg Segall, founder, and CEO of Alyce. “And now being recognized by G2 is an amazing milestone honor and testament to our focus on making our customers incredibly successful, with them accomplishing tremendous ROI - leading to greater expansion, adoption, and utilization of the Alyce platform throughout their organizations.”

15

Evonetix and Imec develop MEMS IC to scale up production of gene synthesis system

By David Manners January 15, 2020 | ElectronicsWeekly.com -- Evonetix’s technology controls the synthesis of DNA at many thousands of independently controlled reaction sites or ‘pixels’ on the chip’s  surface in a highly parallel fashion. Following synthesis, strands are assembled on-chip into double-strand DNA in a process that identifies and removes errors, enabling accuracy, scale and speed that is several orders of magnitude better than conventional approaches. Under the terms of the collaboration, Imec will work with Evonetix to scale up manufacturing of the MEMS technology on 8-inch silicon wafers, enabling Evonetix to supply customers in volume. Imec is able to leverage its experience in manufacturing silicon for life sciences applications to transfer the novel Evonetix process to their foundries and to manage further expansion in volume. “Evonetix has developed an innovative approach that integrates physics and biology to enable the production of high-fidelity long DNA in a highly parallel fashion,” says Imec’s Peter Peumans.

15

Freight Farms goes to school

The shipping containers are used for growing food — and for teaching vital lessons By Andrea Pyenson January 15, 2020 | The Boston Globe Inside the big white shipping container parked behind a classroom building on the campus of the Rivers School in Weston, it smells like a verdant field on a warm spring day, with a degree of humidity that is completely at odds with the cold, dry air outside. A variety of lettuces, herbs, and a smattering of other vegetables grow on vertical towers in adjustable rows. The sixth-grade students who maintain the school's Freight Farm cycle through in groups of four to reap the bounty of work they started at the beginning of the 2019-2020 academic year. The first harvest day was in late October. "They all love to come in here," says Emily Poland, who teaches eighth-grade science and is the farm director at this independent school for grades 6 through 12. The Freight Farm and related projects are built into the sixth-grade curriculum, incorporating humanities, social justice, and science, among other subjects. Students spend time there once a week planting, cleaning, and harvesting. Farming is a club activity for the school's high school students, who can go in during their free time. Based in Boston, Freight Farms manufactures technologically advanced hydroponic farming systems. In 320-square-foot, climate-controlled shipping containers, users can grow up to 13,000 plants at a time, vertically, without soil. The company was founded in 2010 by Brad McNamara and Jon Friedman. Several area schools, among them Rivers, Boston Latin School, and Worcester State University, are using the farms to grow food for their own communities, for their neighbors, and as educational tools. For Poland, managing the farm was a natural extension of her teaching. "I like to create curriculum. I care about food. I like to be outside," she says. One of the sixth-graders' annual activities, which combines academics with community service, is cooking a meal for the Natick Open Door at St. Paul's Episcopal Church. These are hosted every week and attended primarily by seniors. Poland explains that planning the meal incorporates math skills because the students have to scale recipes to feed up to 45 people. And naturally they use their own greens in the salads. The students run a farmers market in the spring. And this year they are maintaining a (very micro) CSA, which one parent won in an auction. The school's chef, Michael Clancy, also gets involved, using the students' produce in the dining room, and helping them cook with what they grow — so far this year they have made pesto and herb vinaigrette. "Their pride is really amazing," he says. Boston Latin, a public exam school for grades seven through 12, acquired its farm in 2013 after students in the Youth Climate Action Network won the $75,000 prize in the Global Green Schools Makeover Competition. Farming is a student-run after-school activity here, under the guidance of eighth-grade history and civics teacher Cate Arnold, whose evident affection for her students appears to be reciprocal. Though roughly 70 students have signed up as student farmers, there is a smaller core group that farms regularly, with an even smaller leadership team that is trained by Freight Farms. At the beginning of each academic year the leaders meet to plan what they will grow, who will work to train new students, and organize schedules. They keep track of chores on a whiteboard in the farm. Addy Krom, a junior, notes of the farm, "You can come in, it's a whole different environment. All the stress from school [goes] away." Adds sophomore Azalea Thompson, "This makes locally grown food more accessible to the city." The students give the food they grow to faculty members, bring some home, and are working to create a CSA. With Arnold's help they are also trying to reestablish a more consistent connection to a food pantry in Jamaica Plain, where a former Boston Latin parent, recently deceased, used to deliver their greens. At Worcester State, Mark Murphy, associate director of dining services, oversees the Freight Farm, which sits outside of Sheehan Hall, the school's newest dormitory and site of its main cafeteria. Rich Perna, former director of dining, made the decision to purchase the farm five years ago, says Murphy, "to bring hyperlocal produce to the campus." Murphy has been responsible for the farm for the last two years. An employee of Chartwells, which has the contract for all of the school's food services, he grows almost all of the greens for the cafeteria, as well as for alumni catering events, and the salad bar in the food court in the building next door. "I'm learning from trial and error," he says. At full capacity, Murphy explains, the farm produces about two acres' worth of crops. He is constantly looking for different varieties of lettuce that will appeal to the students and is currently "trying to figure out a gourmet mix." In addition to three varieties of lettuce, he grows kale, rainbow Swiss chard, parsley, and basil. He coordinates with the cafeteria's cooks, telling them what he is growing so they can plan menus to incorporate the farm's production. Though WSU students are not currently working in the farm Murphy says he promotes the fact that most of the greens in the cafeteria are grown right outside the door. And, he says, "We're trying to get the word out to get students involved." Through a partnership with the Worcester Public Schools and its program that helps young adults with differences transition from school to the workforce Murphy has three part-time helper/trainees. Once a week three students, who have completed high school with a certificate, come (often with a job coach from the program) to seed, plant, harvest, and clean. Murphy is in the process of hiring one of the students, who has aged out of the program. She "has a lot of passion for the farm," he says. "I never thought we'd be growing food inside a container," Murphy says. "I think it may become a necessity someday."  

13

BPS and 2020 On-site Launch ‘Vision for Boston’ Pilot Program

BOSTON, MA, January 13, 2020 -- The Boston Public Schools (BPS), 2020 On-site and eyewear brand Warby Parker have partnered to launch "Vision for Boston," a pilot program offering BPS students free, comprehensive eye examinations and corrective eyewear. The program brings the eye doctor to students in their schools. 2020 On-site licensed optometrists and specialists will visit 13 BPS schools over 20 days beginning January 6. All students in participating schools are eligible to receive free eye exams, and any form of health insurance will be accepted. Students without health insurance will not be asked to pay. Any student found to need eyeglasses can choose a pair at no cost. “The Boston Public Schools is committed to providing our students with the resources they need to thrive academically and throughout their lives,” said BPS Superintendent Brenda Cassellius. “A healthy body means a healthy mind, and this all hands on deck community support will allow our young people to become more engaged in their education, further preparing them for success." 2020 On-site, a leading provider of on-site vision care for businesses and schools, founded in Boston, previously partnered with individual BPS schools beginning in 2015. In recent years, 2020 On-site has visited over 30 schools in the Boston area. Other organizations, such as the New England College of Optometry (NECO), Boston Medical Center, and Prevent Blindness have also partnered with BPS Health Services to assist with state-mandated vision screenings for many years. BPS Health Services met with these organizations to combine and expand efforts, with the goal of creating a district-wide vision initiative. “It’s fantastic that we’re kicking off 2020 by making sure our students’ vision needs are being met inside their own schools,” said Boston School Committee Chairperson Michael Loconto. “I am grateful to 2020 On-site and all the partner organizations that help us to provide essential vision screenings and optometry services for students, easing the financial burden on our families.” There are several mandated health screenings for students required by state law. All BPS students in grades K0-5, 7, and 10 must receive a vision screening in school. Students are screened by BPS nurses and health paraprofessionals. In addition, NECO students and instructors also visit schools to assist with screenings. All students who do not pass their vision screenings are flagged as requiring a full eye examination. Families were asked to complete consent forms to permit students to participate in the pilot program. 2020 On-site will provide students in grades 4-12 a complete, comprehensive eye exam. Any student found to need corrective eyewear will have the opportunity to choose a pair at no cost. Students in grades K0-3 can also choose eyeglasses, for no cost, if the school has a copy of a recent prescription, written within the last year. Through the pilot program, Warby Parker will provide free prescription eyeglasses to students that need them. The company has designed a line of glasses specifically for students that are part of this program and Warby Parker’s Pupils Project initiative. "Warby Parker opened its first store in Boston nearly seven years ago. We've loved being part of the city's rich, historic community and look forward to making an impact on its students by providing access to free glasses through this public-private partnership," says co-founder and co-CEO Neil Blumenthal. Vision exams will take place both inside schools and onboard 2020 On-site’s mobile vision center, an eye clinic on-the-go. 2020 On-site optometrists and vision specialists will conduct pre-testing, including capturing a high-definition image of the retina and an eye pressure test for glaucoma detection, before students receive a comprehensive eye exam using fully-digital, top-of-the-line equipment. “So much of learning is visual. While we know that long-term educational performance is closely linked to a child's ability to see clearly in class, many students lack access to routine eye exams," said Alexis McLaughlin, CEO of 2020 On-site. "2020 On-site is incredibly proud to once again partner with Boston Public Schools to bring free eye exams and glasses to students who need them. Expanding access to vision care for all is central to 2020 On-site's mission, and we can't think of a better place to channel our passion than in our own community.” Thirteen BPS schools are participating in the pilot. Schools were recommended by Boston Public Schools Health Services Department due to their high need of vision support, while school administration and school nurses advocated for their school communities to participate. The participating schools are: Blackstone Elementary, BTU Pilot K-8, Conley Elementary, Curley K-8, East Boston High, Henderson Upper, Hernandez K-8, Irving Middle, JFK Elementary, King K-8, McCormack Middle, Sumner Elementary and Umana Academy.  

13

Apellis Pharmaceuticals Announces Closing of Public Offering of Common Stock and Full Exercise of Option to Purchase Additional Shares

WALTHAM Mass., Jan. 13, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq:APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced the closing of its previously announced underwritten public offering of common stock, including the exercise in full by the underwriters of their option to purchase an additional 1,425,000 shares at the public offering price of $37.00 per share. The exercise of the option to purchase additional shares brought the total number of shares of common stock sold by Apellis to 10,925,000 shares and increased the amount of gross proceeds, before deducting underwriting discounts and commissions and expenses payable by Apellis, to approximately $404.2 million.

Citigroup, J.P. Morgan and Evercore ISI acted as joint book-running managers for the offering. Cantor Fitzgerald & Co. and Baird acted as lead managers for the offering.  

10

Meissa Vaccines Receives U.S. FDA Fast Track Designation for Respiratory Syncytial Virus Vaccine, MV-012-968

SOUTH SAN FRANCISCO, Calif., Jan. 10, 2020 (BUSINESS WIRE) -- Meissa Vaccines (“Meissa”), a biotechnology company developing vaccines to prevent viral respiratory infections, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to MV-012-968, an investigational, live attenuated vaccine for protection against respiratory syncytial virus (RSV) infection. The FDA’s Fast Track program expedites the development and review of drugs and biologic agents demonstrating potential to fill an unmet medical need in the treatment or prevention of serious conditions.

With Fast Track Designation, Meissa is eligible for early and frequent interactions with FDA reviewers to discuss all aspects of the clinical development plan for MV-012-968, ensuring that the appropriate data are collected to support an application for vaccine licensure. The frequency of communication granted by Fast Track Designation assures that questions and issues are resolved quickly, often leading to earlier approval and access by patients.

Additionally, Fast Track Designation may allow Meissa to submit data for the MV-012-968 Biologics License Application (BLA) before the full application is complete (“Rolling Review”) and receive BLA review under an expedited time frame (“Priority Review”), if relevant criteria are met. A Phase 1 clinical trial evaluating the safety and immunogenicity of MV-012-968 is presently recruiting healthy adult volunteers.

“Fast Track Designation of MV-012-968 accelerates our ability to develop a much-needed RSV vaccine,” said Martin Moore, Ph.D., cofounder and CEO of Meissa. “A safe and effective RSV vaccine is a significant global health priority, and if we are successful, we can save thousands of lives and help millions of patients around the world.”

RSV is the most common cause of acute lower respiratory tract infections in young children and infects nearly all children by two years of age. The virus is responsible for more than 30 million acute lower respiratory infections and nearly 60,000 deaths per year among children under five worldwide.

09

Elysium Health™ Announces Clinical Trial to Study Correlations Between Epigenetic Aging and Nicotinamide Adenine Dinucleotide (NAD+) Levels in Healthy Adults

NEW YORKJan. 9, 2020 (PRNewswire) -- Elysium Health, Inc.™, a life sciences company developing clinically validated health products based on advancements in scientific research, today announced the initiation of a clinical trial at Duke Clinical Research Institute to study correlations between biological age and circulating NAD+ levels in healthy adults, in addition to other clinical biomarkers that have been implicated in aging. As opposed to marking calendar years, biological age is the average age an individual's body is expected to function and is a powerful measure of overall health and wellness. Biological age will be calculated using Elysium Health's next-generation epigenetic platform, Algorithmic Platform for Epigenetic Examination (APEX), which the company developed to support the 2019 launch of its at-home epigenetic test Index.

Published biological age applications such as DNAm PhenoAge typically look at hundreds of sites on the genome. To ensure greater accuracy at the individual level, Elysium Health head of bioinformatics and Yale School of Medicine professor Dr. Morgan Levine led the development of a proprietary measure that examines global methylation patterns across more than 100,000 sites in collaboration with Elysium Health bioinformatics director Dr. Tina Hu-Seliger. A key consideration for developing the methodology that underlies APEX and Index was the need to refine the technology to address the inherent variability of earlier epigenetic age predictors. Later this year, Elysium Health intends to publish the data that supports its methodology, and which demonstrates that Index is a better predictor of biological age than earlier measures.

As an essential coenzyme involved in hundreds of metabolic processes, NAD+ is critical to cellular metabolism; and its reduced form, NADH, is required for mitochondrial metabolism and the resulting synthesis of ATP for cellular energy creation. NAD+ is also utilized by sirtuins, often referred to as the "guardians of the genome." NAD+ levels have been shown to decline with age in humans in circulating blood as well as skin and brain tissue. Although the underlying causes for deteriorating NAD+ levels have not yet been fully determined, understanding correlations between epigenetic aging and NAD+ levels may help elucidate the benefits of maintaining NAD+ levels with age.

"Over the past decade, epigenetic tests have evolved to measure biological age with an increasing level of accuracy," said Elysium Health chief scientist and director of the Glenn Center for Biology of Aging Research at MIT Dr. Leonard Guarente. "Having studied aging for more than 30 years, it's my belief that the field is at a tipping point. The newfound ability to measure biological age allows us to study whether interventions can have an impact, thus unlocking the potential to realize the benefits of aging research in our lifetime."

This study is intended to further the understanding of the links between NAD+ levels and other markers of biological age. In the study, NAD+ levels in whole blood, interleukins, inflammatory cytokines, growth factors, omega-3 polyunsaturated fatty acids, and patterns in DNA methylation will be measured and compared in age-matched individuals to determine whether NAD+ levels are associated with biological age.

One hundred and seventy healthy adults ages 25–80 will be enrolled in the study, which is scheduled to complete in May 2020. More information can be found on www.clinicaltrials.gov under the identifier NCT04220658.

08

Allysta Pharmaceuticals, Inc. Doses First Patient with ALY688 Ophthalmic Solution in Phase 1/2a Dry Eye Study

SAN MATEO, CA, January 8, 2020 (ACCESSWIRE) -- Allysta Pharmaceuticals, Inc. (Allysta) today announced dosing of the first patient in its Phase 1/2a trial (ALY688-201) of ALY688 Ophthalmic Solution for the treatment of dry eye disease. Dry eye is a very common condition affecting millions of people in the US and causes eye symptoms (e.g., burning, foreign body sensation, pain) which can limit daily activities such as computer use, reading and driving. In advanced cases, significant inflammation and even scarring of the eye surface can occur. ALY688 is a novel peptide agonist that binds to and activates adiponectin receptors which are widely distributed on the ocular surface. Following receptor binding, it acts to reduce inflammation and promote healing of injured cells lining the ocular surface. In animal models of dry eye disease, this resulted in significant improvement in corneal damage, tear integrity, and tear volume associated with reductions in inflammatory cells and cytokines in the eye. "This marks a significant milestone for Allysta, as we now progress from a preclinical into a clinical stage company. We look forward to completing this trial by mid-year and reporting data in the second half of 2020," said Henry Hsu, M.D., Chief Executive Officer and President of Allysta.

08

Apellis Pharmaceuticals Announces Pricing of Public Offering of Common Stock

WALTHAM, Mass., Jan. 08, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq:APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced the pricing of its underwritten public offering of 9.5 million shares of its common stock at a public offering price of $37.00 per share, for total gross proceeds of $351.5 million, before deducting underwriting discounts and commissions and expenses payable by Apellis. All of the shares in the offering are being sold by Apellis. In addition, Apellis has granted the underwriters a 30-day option to purchase up to 1.425 million additional shares of its common stock at the public offering price, less the underwriting discount and commissions. The offering is expected to close on January 13, 2020, subject to customary closing conditions.

Citigroup, J.P. Morgan and Evercore ISI are acting as joint book-running managers for the offering. Cantor Fitzgerald & Co. and Baird are acting as lead managers for the offering.

07

PhoreMost and Boehringer Ingelheim enter multi-project drug discovery collaboration

CAMBRIDGE, England, January 7, 2020--(BUSINESS WIRE)--PhoreMost Limited, the UK-based biopharmaceutical company dedicated to drugging ‘undruggable’ disease targets, today announced it has entered into a multi-project drug discovery collaboration with Boehringer Ingelheim, one of the world-leading pharmaceutical companies developing innovative therapies for diseases with unsatisfactory treatments. Under the terms of the agreement, PhoreMost will receive an upfront payment and research funding together with downstream success-based milestones. Further financial terms are not disclosed. PhoreMost will deploy its in-house expertise and next-generation phenotypic screening platform, SITESEEKER®, towards disease relevant pathways nominated by Boehringer Ingelheim. Novel targets identified will be further validated and characterised by Boehringer Ingelheim as part of its internal Discovery Research pipeline. Boehringer Ingelheim’s Research programme is active in the fields of immunology and respiratory diseases, cardiometabolic diseases, oncology research and immuno-oncology, as well as diseases of the central nervous system. The SITESEEKER platform is based on PhoreMost’s core proprietary protein interference, or ‘PROTEINi’, technology. Using SITESEEKER, PhoreMost probes the entire proteome in a live cell environment for novel druggable targets linked to any chosen disease, using the vast 3-D shape diversity of natural protein fragment (sub-domain) libraries. This enables the systematic unmasking of cryptic druggable sites, directly linking them to useful therapeutic functions. Dr Chris Torrance, CEO of PhoreMost, said: “We are delighted that Boehringer Ingelheim has chosen to work with PhoreMost to enhance its drug discovery pipeline with attractive biological starting points. The collaboration is further recognition of the ability of PROTEINi and SITESEEKER to identify novel targets, and we look forward to working with the Boehringer Ingelheim team on these projects.”

07

Apellis Reports Positive Top-line Results from Phase 3 Head-to-Head Study of Pegcetacoplan (APL-2) Compared to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

WALTHAM, Mass., Jan. 07, 2020 (GLOBE NEWSWIRE) --  Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced positive results from the Phase 3 PEGASUS study evaluating pegcetacoplan (APL-2) in adults with paroxysmal nocturnal hemoglobinuria (PNH). Top-line data show that pegcetacoplan met the study’s primary efficacy endpoint, demonstrating superiority to eculizumab with a statistically significant improvement in adjusted means of 3.8 g/dL of hemoglobin at week 16 (p<0.0001). At week 16, pegcetacoplan-treated patients (n=41) had an adjusted mean hemoglobin increase of 2.4 g/dL from a baseline of 8.7 g/dL, compared to eculizumab-treated patients (n=39) who had a change of -1.5 g/dL from a baseline of 8.7 g/dL.

Additionally, pegcetacoplan showed promising results in key secondary endpoints. Pegcetacoplan met non-inferiority on transfusion avoidance and absolute reticulocyte count. Pegcetacoplan also showed positive trends on lactate dehydrogenase (LDH) and fatigue as measured by the Functional Assessment of Chronic Illness Therapy (FACIT)-fatigue score.

Key Secondary Endpoints Analysis "Pegcetacoplan is the first and only investigational therapy to demonstrate superiority compared to eculizumab on hemoglobin levels. We are also excited to see that 85% of patients treated with pegcetacoplan were transfusion free,” said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. “At Apellis, we are focused on developing groundbreaking therapies, and these results show that pegcetacoplan has the potential to transform the lives of people with PNH. We look forward to meeting with regulators in the first half of the year to discuss next steps.” “The majority of patients with PNH currently receiving treatment with eculizumab have continuing anemia,” said Peter Hillmen, M.B., Ch.B., Ph.D., Professor of Experimental Hematology at the University of Leeds and an investigator in the PEGASUS study. “The PEGASUS results show that pegcetacoplan has the potential to become a new standard of care for patients with PNH.” In this study, the safety profile of pegcetacoplan was comparable to eculizumab. Seven of 41 patients (17.1%) in the pegcetacoplan group experienced a serious adverse event (SAE), and 6 of 39 patients (15.4%) in the eculizumab group experienced SAEs. No cases of meningitis and no deaths were reported in either treatment group. The most common adverse events reported during the 16-week, randomized, controlled treatment period in the pegcetacoplan and eculizumab groups, respectively, were injection site reactions (36.6% vs. 2.6%), diarrhea (22.0% vs. 0%), headache (7.3% vs. 20.5%) and fatigue (4.9% vs. 15.4%). Another common adverse event was hemolysis, which was reported in four patients in the pegcetacoplan group (9.8%) and nine patients in the eculizumab group (23.1%). This led to the three discontinuations in pegcetacoplan group. All patients who completed the randomization period in both groups (77/80) entered the 32-week open-label pegcetacoplan treatment period. “Going into the study, our most optimistic expectation was to see a 2 g/dL or more change in hemoglobin and a trend on the key secondary endpoints. Needless to say, we are thrilled with these results,” said Cedric Francois, M.D., Ph.D., Co-Founder and Chief Executive Officer of Apellis. “These data give us strong confidence in the further development of pegcetacoplan as a targeted C3 inhibitor in geographic atrophy and other serious complement-driven diseases.” Detailed results from the PEGASUS study will be presented at a future scientific meeting.

December 2019

18

Cognoa Wins 2019 Fierce Innovation Life Science Award

Palo Alto, Calif., Dec. 18, 2019 (GLOBE NEWSWIRE) -- Cognoa, a company at the forefront of pediatric behavioral health, has won the Fierce Innovation Life Science Award for Technology Innovation. Cognoa’s digital therapeutics will advance the standard of care by giving every child the opportunity to receive treatments earlier when they can have the greatest impact.

Currently, diagnosis and treatment of behavioral conditions relies on a limited and decreasing number of specialists, resulting in delayed access to treatments and potential long-term ramifications. Cognoa is developing what is expected to be the first diagnostic for autism spectrum disorder (ASD) that enables pediatricians and primary care physicians to diagnose ASD so that children can receive earlier treatment. Cognoa has launched a pivotal trial of its ASD diagnostic and intends to submit the results as basis of its premarket submission to the FDA in 2020. The company will receive priority review by the FDA for the diagnostic and for its first digital therapeutic for children with autism, both of which have received FDA Breakthrough designation.

“All of us at Cognoa are proud of this prestigious recognition of our mission and work to improve lifelong outcomes with earlier diagnosis and treatment,” said Brent Vaughan, CEO and co-founder of Cognoa. “In 2020, we will be taking important steps forward for both children and pediatricians as we anticipate bringing our first prescription digital medicine to market and advancing our prescription digital therapeutic for autism. In order to fundamentally change the standard of care and make the benefits of early intervention available to every child, we must empower the trusted family pediatricians who are the first line of care and know the child best.”

Cognoa’s solutions will provide a continuum of care from identifying at-risk children to empowering early diagnosis and treatment of behavioral health conditions. By targeting the critical, early neurodevelopmental windows, Cognoa’s digital therapeutics promote neuromodulation of specific brain networks, improving functional connectivity to create lifelong gains.

With its commercialization partner EVERSANA, Cognoa will enable its digital medicines to be prescribed, dispensed and reimbursed through the same healthcare infrastructure utilized by payers and providers for other medicines, so that these solutions can be available to every pediatrician and child.

“It’s an incredibly exciting time for pediatric behavioral healthcare. Earlier this week, the American Academy of Pediatrics (AAP) updated, for the first time in over a decade, its guidelines for the identification, evaluation and management of children with autism,” explains Dr. Colleen Kraft, the 2018 Past President of the AAP and Professor of Pediatrics, Keck School of Medicine at the University of Southern California. “The guidelines emphasize the importance of early diagnosis and treatment and support pediatricians’ potential to diagnose autism consistent with DSM-V criteria. I’m confident that Cognoa will play a critical role in pediatricians’ ability to facilitate earlier treatments with its clinically validated, practical products.”

17

Amylyx Pharmaceuticals Announces AMX0035 Demonstrated Statistically Significant Treatment Benefit for People with ALS in the CENTAUR Trial

CAMBRIDGE, Mass., December 17, 2019--(BUSINESS WIRE)--Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, and the Sean M. Healey & AMG Center for ALS at Mass General today announced that AMX0035 demonstrated a significant treatment benefit for people with ALS in the CENTAUR study. In the study, participants taking AMX0035 had a statistically significant slowing of ALS disease progression as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) compared to placebo (p<0.05), the primary outcome of the trial. Detailed results from CENTAUR will be submitted for peer-reviewed publication and presentation at a future medical congress. “Today marks a significant step forward in the fight to develop new treatments for ALS,” said Dr. Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR study, investigator at the Healey Center for ALS at Mass General and Assistant Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital. “The study results highlight AMX0035 as a potentially beneficial new treatment for people with ALS, and the design and execution of the CENTAUR trial are a testament to true collaboration across the many stakeholders in this fight. Thank you to everyone who made this possible, and I look forward to presenting the full study results in the coming months.” “We are honored and humbled to have reached this milestone after working nearly seven years on the development of AMX0035,” said Joshua Cohen, CEO, Chairman, and Co-Founder of Amylyx. “Thank you to each and every participant, family, physician, nurse, coordinator, vendor, and advisor who has and continues to work with us to better the lives of people with ALS.” “With these results, Amylyx now has a responsibility to move ahead as efficiently as possible, as people living with ALS don’t have time to wait,” said Justin Klee, President and Co-Founder of Amylyx. “We will work closely with the FDA and the ALS community, including the wonderful Northeast ALS Consortium leadership and member sites that conducted the CENTAUR trial, to decide next steps and will continue to keep everyone informed.” Participants in CENTAUR were given the option after the trial to enroll in an open-label extension study to receive treatment with AMX0035. Nearly 90 percent of participants who completed CENTAUR elected to enroll in the extension study. Interim data from the ongoing extension study will be presented in 2020. In addition, the company will provide an update on regulatory plans and further details on expanded access plans in early 2020. “ALS Finding a Cure is proud to have catalyzed and supported the CENTAUR study, and I am encouraged by what the results mean for people living with ALS. Our team at the Mass General Neurological Clinical Research Institute is proud of this collaboration with Amylyx and our colleagues in the Northeast ALS Consortium on this important study,” commented Dr. Merit Cudkowicz, Chief Medical Officer from ALS Finding a Cure®, Director of the Healey Center for ALS, Chief of Neurology at Mass General, and the Julieanne Dorn Professor of Neurology at Harvard Medical School. “We look forward to advancing this research and what it might mean for those living with ALS.” Dr. Neil Thakur, executive vice president for mission strategy at The ALS Association, added, “We are proud to have supported AMX0035 and Amylyx from an early stage and are very excited about what AMX0035 may accomplish for people with ALS. This company and study team have focused on the patient perspective during the design and conduct of this study and we are happy to work with and innovate together with them. We are excited to continue to collaborate on this therapy in the future.” Dr. Rudolph Tanzi, Ph.D., Kennedy Professor of Neurology, Massachusetts General Hospital, chair of the Cure Alzheimer’s Fund Research Leadership Group and chair of the Amylyx SAB, shared, “The positive results from the CENTAUR ALS study demonstrate that the novel mechanism of AMX0035 may represent a new treatment approach for not only ALS, but for Alzheimer’s disease. I am very excited about the demonstrated benefit of AMX0035 in people with ALS, and look forward to the results from the ongoing PEGASUS trial for people with Alzheimer’s disease.” ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. The progressive neurodegeneration in ALS eventually leads to the death of motor neurons and loss of the ability of the brain to initiate and control muscle movement. With muscle action progressively affected, patients in the later stages of the disease may become paralyzed and unable to breathe normally.

17

ALS Drug Works in Study, Researchers Say

by Jonathan D. Rockoff December 17, 2019 | Wall Street Journal An experimental drug slowed the paralyzing march of amyotrophic lateral sclerosis, or Lou Gehrig’s disease, in a clinical trial, according to researchers who say the results are a fresh sign that recent insights into the condition may soon bring new medicines. The drug’s maker, closely held Amylyx Pharmaceuticals Inc., is releasing only the barest outlines of the mid-stage trial for now, as researchers continue to analyze the results. The drug slowed the rate of ALS’s progression in patients as measured by a widely used scale, said the researchers and the company’s founders. “We’re just another step closer to hopefully stopping this illness,” said Merit Cudkowicz, who heads the Healey Center for ALS at Massachusetts General Hospital in Boston and helped design and coordinate the study. If the drug is ultimately approved, it would add a much-needed weapon to a relatively limited arsenal of ALS treatments and cap an improbable drug-discovery effort by former Brown University students who began the project in a dorm room. More than 16,000 Americans have ALS, which kills off the nerve cells that control muscle movements. As these motor neurons die, patients lose the ability to eat, speak, move and breathe. Within three to five years of symptoms, most patients die. Only two drugs have been approved to treat the underlying cause of the disease, and neither provides a cure. About 20 drugs are in clinical testing, doctors and scientists say, after research furnished new insights in recent years about the roots and course of the disease and thereby provided drug researchers promising targets for experimental medicines. At least five medicines are now in the late stages of development, Dr. Cudkowicz said. Among them is an agent from Biogen Inc. attacking a genetic mutation that plays a role in the inherited form of the disease. ALS doctors said they are intrigued by Amylyx’s drug, but would need to wait for the full results from the study before they could say whether its drug might make a difference for patients. Raymond Roos, director of the ALS treatment center at the University of Chicago Medical Center, said he can’t be certain of the real-world benefit from Amylyx’s medicine without seeing how much it slowed ALS’s progression and the drug’s impact on specific functions, especially breathing. And the trial, which studied patients for six months, wasn’t long enough to answer the key question whether the drug would prolong the lives of ALS patients, said Dr. Roos, who wasn’t involved in the study. Sabrina Paganoni, an assistant professor at Harvard Medical School who led the study, said researchers were still reviewing all the results from the trial, and planned to publish them in a medical journal and present them at a medical meeting early next year. “Most trials fail to see a positive result,” Dr. Paganoni said in an interview, making the top-line findings of Amylyx’s study “really incredible.” Amylyx’s drug, a powder that has been going by the designation AMX0035, combines two separate medicines. Each strikes a different line of attack that ALS takes to destroy the motor neurons in patients. Justin Klee, Amylyx president and co-founder, said the company’s drug appears to have a bigger impact on the disease’s progression than giving each of its components individually. The synergistic effect, he said, suggests ALS treatment might require a cocktail of drugs. Mr. Klee said the company will reach out to the U.S. Food and Drug Administration about holding a meeting to discuss whether the trial results would be enough to seek the drug’s approval. In the trial, AMX0035 was tested in patients who were diagnosed within 18 months of symptoms appearing and had developed problems like trouble speaking, difficulty walking and arm weakness. The 137 subjects in the trial took either AMX0035 or a placebo for six months in addition to any other ALS drugs they were taking. Then researchers, following a standardized questionnaire widely used by doctors and scientists, asked the subjects to rate their ability to do things like dress, feed themselves and write on a five-point scale. “I believe my ALS slowed down somewhat during the trial,” said Kristina Golji, 34, from Grafton, Mass., who was diagnosed in March 2017. Ms. Golji said her ability to talk had been gradually declining before the trial, but steadied while in the study. After it ended, she lost fine motor skills and had more trouble walking and swallowing until she began taking the drug again under an extension of the study. The experimental drug was well tolerated by study subjects and safe, said Dr. Paganoni, the trial’s principal investigator. The idea for the drug came to Amylyx Chief Executive Josh Cohen, he said, while he was a Brown University junior in 2012 and 2013 majoring in biomedical engineering and reading scientific papers on how neurons die. Mr. Cohen told Mr. Klee, whom he had first met playing club tennis in college. Mr. Klee, a neuroscience major, spent the following night reading up on his friend’s idea. “We did what most people in our generation do” when trying to learn about a topic, Mr. Klee said. “We went to the Internet. We Googled it.” The research shed light on some molecular routes that neurodegeneration follows, which Mr. Cohen said sparked his interest in combining drugs that attacked two important pathways. The problem was, each drug alone hadn’t worked in studies. Unfamiliar with both drug research and the industry, Messrs. Cohen and Klee sounded out experts, including Dr. Cudkowicz, to learn how to test their hypothesis in a laboratory, start a company and conduct testing in patients. Their project took off after the pair scraped together $6,000 from personal savings and family donations to pay contract researchers in Finland, who found their combination drug worked in a petri dish. Mr. Cohen took all his courses during his final year of college on Thursdays so he could devote the rest of the week to Amylyx. Mr. Klee, who had moved to Cambridge, Mass., after graduating, took odd jobs coaching swimming, working as a research technician and participating in medical-research studies to earn money for the fledgling startup then based in his apartment. The company, based in Cambridge, had three employees last March and seven today, but plans to add 100 employees next year.

05

Meissa Vaccines Receives FDA Clearance of IND Application for a Phase 1 Clinical Trial of MV-012-968 for Respiratory Syncytial Virus

SOUTH SAN FRANCISCO, Calif, December 5, 2019 – Meissa Vaccines (“Meissa”), a biotechnology company developing vaccines to prevent viral respiratory infections, announced today that the U.S. Food and Drug Administration (FDA) has accepted the company’s Investigational New Drug (IND) application to proceed with a U.S. clinical trial of MV-012-968, an investigational vaccine for respiratory syncytial virus (RSV). The company plans to initiate a Phase 1 clinical trial in adults early in 2020 to assess tolerability and immunogenicity of MV-012-968. 
 
RSV is a common respiratory virus that usually causes mild, cold-like symptoms, but RSV infections can be serious, especially for infants and older adults. In children younger than one year of age, RSV is the most common cause of bronchiolitis and pneumonia. In children under five, RSV causes more than 30 million new acute lower respiratory infections, resulting in more than three million hospital admissions from serious infections around the world.
 
“FDA clearance of this IND for a live attenuated RSV vaccine is a significant milestone in the global mission to prevent RSV in at-risk populations,” said Martin Moore, Ph.D., cofounder and CEO of Meissa. “We look forward to beginning our clinical studies to develop a safe and effective RSV vaccine for infants and the elderly.”
 
Meissa’s technology platform leverages synthetic biology and genetic engineering for the rational design of vaccines that solve suboptimal immune responses, vaccine stability, and manufacturing. Meissa’s proprietary technologies of reverse genetics and human codon deoptimization allow for rapid generation of live attenuated vaccine candidates that may be safer yet more immunogenic than the natural pathogens.

November 2019

24

DNAtrix Presents Positive Results from the Phase 2 CAPTIVE/KEYNOTE-192 Study of DNX-2401 in Combination with Pembrolizumab for Glioblastoma at the 2019 SNO Annual Meeting

HOUSTON, Texas, Nov. 24, 2019 (PRNewswire) -- DNAtrix, a leader in the development of oncolytic viruses for cancer therapy, today presented updated safety and efficacy data from the fully-enrolled Phase 2 CAPTIVE / KEYNOTE-192 study of DNX-2401 (tasadenoturev), DNAtrix's oncolytic adenovirus, followed by pembrolizumab for patients with recurrent glioblastoma. The data demonstrate that the therapy elicits durable clinical activity and has a favorable safety profile. These results are being presented at the Society for Neuro-Oncology (SNO) Annual Meeting held from November 22-24, 2019 in Phoenix, Arizona.

A total of 48 patients with recurrent glioblastoma were treated at 15 participating clinical sites with the regimen of DNX-2401, followed by pembrolizumab. The majority of patients experienced clinical benefit following treatment, including durable complete and partial responses. Interim median overall survival for patients is currently 12.3 months. In addition, the interim safety analysis demonstrates that the therapy has a positive benefit-risk profile, there were no unanticipated adverse events and patients remained on pembrolizumab for a median of 5.5 months with a maximum of 34 cycles.

"The data hold great promise for patients afflicted with glioblastoma, the most common form of adult brain cancer. Glioblastoma is a disease associated with near uniform fatality. The results are striking in this context," said Clark Chen, MD, PhD, Professor, Lyle French Chair in Neurosurgery and Department Head at the University of Minnesota, presenting author for the CAPTIVE / KEYNOTE-192 study. "The results suggest that the combination of DNX-2401 and pembrolizumab has the potential to revolutionize the care of glioblastoma patients. I look forward to the rigorous studies aimed to validate the efficacy of DNX-2401."

"These interim data from our CAPTIVE trial are a significant milestone for us in the development of DNX-2401 as an innovative treatment for glioblastoma," said Frank Tufaro, PhD, CEO of DNAtrix. "Importantly, these data suggest that DNX-2401 has a superior clinical benefit and safety profile compared to currently approved chemotherapy for recurrent glioblastoma, and we are particularly pleased that, together with pembrolizumab, DNX-2401 elicited a number of robust and durable antitumor responses."

21

Inotrem Announces Enrollment of First Patient in its Phase IIb ASTONISH Trial for Nangibotide in the Treatment of Septic Shock

PARIS, France, November 21, 2019 --  Inotrem S.A., a biotechnology company specializing in immunotherapy for acute inflammatory syndromes, through its knowledge of the TREM-1 pathway biology, announced today it has initiated its Phase IIb ASTONISH trial to evaluate nangibotide in the treatment of septic shock and has enrolled the first patient in the trial. Nangibotide is a TREM-1 inhibitor peptide with the potential to restore appropriate inflammatory response, vascular function, and improve post septic shock survival. Septic shock is the ultimate complication of sepsis. The incidence of septic shock continuously raises and mortality remains elevated (35%) in developed countries. There is currently no specific therapy approved for this indication besides antibiotics and supportive treatment. Inotrem’s therapeutic solution has the potential to become the first mechanism-based treatment for septic shock. Nangibotide in septic shock has been granted the fast track status in September 2019 by the FDA and the PRIME status in 2017 by the EMA. The Phase IIb ASTONISH study aims to demonstrate the efficacy of nangibotide and bring a medically relevant proof of clinical activity in septic shock patients. In addition, this global multicentric study intends to validate a personalized medicine approach using soluble TREM-1 as potential companion diagnostic test to identify patients more likely to benefit from nangibotide treatment. The study will be conducted in 48 clinical sites across 5 European countries and the United States and will enroll a total number of 450 patients. Jean-Jacques Garaud, CEO of Inotrem, said: “The ASTONISH trial is a Phase III enabling trial and we expect that it will generate important insights about nangibotide’s clinical activity and our personalized medicine approach in septic shock. We are enthusiastic about this study and Inotrem’s capacity to bring a first in class product in an area with major unmet medical need and to patients who today have no access to any approved treatment”. With nangibotide, Inotrem has developed a novel approach of immunomodulation targeting the TREM-1 pathway which has the potential to address, beyond septic shock, several others acute inflammatory syndromes for which there is a major and today unsatisfied therapeutic need. Based on its its extensive knowledge of the TREM-1 pathway biology, Inotrem has aso launched a new program to expand its TREM-1 franchise into chronic inflammatory diseases.

19

'A Clever Bit of Chemistry': NuCana CEO Speaks on Company's Biliary Tract Cancer Drug

November 19, 2019 | BioSpace.com -- Hugh Griffith, founder and chief executive officer of NuCana, has been excited about what he called a “clever bit of chemistry.” And that excitement was finally revealed days ago when the U.S. Food and Drug Administration cleared the path for a Phase III study of a potential new biliary tract cancer treatment. The FDA provided NuCana with clearance for its Investigational New Drug Application for Acelarin, what Griffith described as a transformation of Eli Lilly’s old drug Gemzar. In the Phase III trial, Acelarin will be studied in combination with cisplatin for patients with previously untreated locally advanced or metastatic biliary tract cancer. If successful, Griffith told BioSpace in an exclusive interview that Acelarin will break the glass ceiling as the first FDA-approved treatment for biliary tract cancer. “No drug has been approved for this cancer but there are more than 40,000 people with this disease. The standard of care is two chemotherapy drugs and that manages to buy patients on average a year of survival,” Griffith said. He is hoping that Acelarin will change that. He said the drug, internally called NUC-1031, has the potential to significantly improve the survival outcomes of patients with advanced biliary tract cancer. In a Phase Ib study, the company saw an approximate doubling of the response rate with Acelarin plus cisplatin compared to previously reported data for gemcitabine plus cisplatin. “Our goal is to establish Acelarin in combination with cisplatin as the global standard of care for the first-line treatment of patients with advanced biliary tract cancer,” he said. Looking at the oncology landscape, Griffith said it’s exciting to be part of an “era of biology and understanding cancer cells” that leads to new treatments being developed for cancer patients. Griffith said it’s important to realize that chemotherapy will still be the cornerstone of cancer treatment but added that the advent of IO drugs are significantly improving outcomes in many patients. Their belief is that chemo will always be important but cannot always be relied upon to hit certain targets and sometimes comes with its own toxicity concerns. “Chemo is the workhorse of the treatment, but what we’ve got here (meaning Acelarin) is an approach than can make this less efficacious and less safe medicine more efficacious and more safe,” he said. With the Phase III trial, the primary endpoints will be overall survival and objective response rate. NuCana believes that a statistically significant improvement in ORR at either of the first two planned interim analyses supported by positive trends in other endpoints, could potentially allow for an accelerated approval of a New Drug Application for Acelarin. There are other companies going after biliary tract cancer but, as of yet, none have been successful. Most recently, Singapore’s ASLAN Pharmaceuticals revealed its biliary tract cancer drug failed to meet the primary endpoints of progression-free survival and overall response rate. Other companies developing therapies for biliary tract cancer include Agios Pharmaceuticals, Imbrium Therapeutics, Ability Pharmaceuticals and Puma Biotechnology. In addition to the biliary tract study, Acelarin is currently being evaluated a Phase II study for patients with ovarian cancer and a Phase III study for patients with pancreatic cancer. In addition to the biliary tract cancer drug, Griffith said he is also excited about the company’s second ProTide NUC-3373. Griffith said they are making strides in the administration of this intravenous medication, which is a transformation of the colorectal cancer drug 5FU. The standard drug has to be given intravenously over the course of two days and Griffith said it’s “deeply flawed” because the body breaks much of it down before it gets to the target, which can cause some toxicity issues. However, by applying their chemistry to this product, Griffith said they are seeing big advantages. Not only can it be administered in only a few hours, they’re not seeing the toxicities.

11

F2G Receives US FDA Breakthrough Therapy Designation for Olorofim

MANCHESTER, England and VIENNA, November 11, 2019 (PRNewswire) -- F2G Ltd, a UK- and Austria-based biotech company developing novel therapies for life-threatening systemic fungal infections, announced today that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to its lead first-in-class candidate, olorofim (formerly F901318) for the indication of 'Treatment of invasive mold infections in patients with limited or no treatment options, including aspergillosis refractory or intolerant to currently available therapy, and infections due to Lomentospora prolificans, Scedosporium, and Scopulariopsis species'. Olorofim is the first antifungal agent to be granted Breakthrough Therapy designation. Olorofim is currently being investigated in an open-label single-arm Phase 2b study (ClinicalTrials.gov Identifier: NCT03583164) in patients with proven invasive fungal disease (IFD) or probable invasive aspergillosis (IA) and either refractory disease, resistance, or intolerance to available agents. Olorofim has been well tolerated across more than 10 years of patient dosing days with a median therapy duration of 12 weeks. Preliminary data from this study were provided to the FDA as part of the Breakthrough Therapy designation submission. Breakthrough Therapy designation is an FDA process designed to expedite the development and review of drugs that are intended to treat a serious or life-threatening condition and is granted based on preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. Breakthrough Therapy designation conveys all the features of fast track designation, more intensive FDA guidance on an efficient drug development program, an organisational commitment by FDA to involve senior managers, and eligibility for rolling review and priority review. Commenting on the news, Ian Nicholson, CEO of F2G Ltd, said: "The granting of FDA Breakthrough Therapy designation is a truly transformational step for our company and will support our goal of rapidly developing this novel treatment for patients suffering from serious and life-threatening fungal infections. Olorofim acts via a novel and differentiated mechanism to traditional antifungals, and preliminary data have indicated that it is efficacious in tackling life-threatening invasive fungal infections that cannot be managed with currently approved agents. "Our Phase 2b programme is on track with over 40 patients recruited in Europe, Australia and the US. We look forward to working closely with the FDA to accelerate development of this therapy for patients having limited or no approved treatment options for an invasive mold infection." Professor Sharon Chen, Westmead Hospital Sydney and Principal Investigator for the Phase 2b study said: "This news is very exciting for clinicians caring for patients with these very serious, and devastating mold infections. We have had limited treatment options for many years and now the news about olorofim brings realistic hope that we can cure these previously treatment–refractory infections."

07

TransThera Biosciences Lead Product TT-00420 Granted Orphan Drug Designation from FDA to Treat Cholangiocarcinoma

NANJING, China, Nov. 7, 2019 (PRNewswire) -- TransThera Biosciences Co. Ltd, announced today that company received Orphan Drug Designation from US Food and Drug Administration (FDA) for TT-00420, a clinical stage investigational drug, for the treatment of cholangiocarcinoma. Dr. Frank Wu, Founder and CEO of TransThera, commented: "Cholangiocarcinoma lacks effective therapies and remains a huge unmet medical need around the world. TT-00420 has demonstrated great potential in multiple experiments. We believe that today's orphan drug designation will help accelerate the development of this potential product in the clinical trials and bring meaningful benefit to cholangiocarcinoma patients. We are very excited about this news, which expands the horizon of our lead product in development. In addition to triple negative breast cancer, cholangiocarcinoma is identified by our scientists to be second indication for TT-00420." TT-00420 is in global Phase I trial both in the US and China. Dose expansion Phase Ib/II trials in cholangiocarcinoma and triple negative breast cancer are planned to start in 2020.

07

Viken Unveils the Osprey Integrated Checkpoint SolutionTM for High Throughput Passenger & Commercial Vehicle Scanning at US Border Crossings

BURLINGTON, Mass., November 7, 2019 -- (BUSINESS WIRE)--Viken Detection, pioneer of handheld x-ray imaging and analytical devices, today announced an award to install four Osprey-UVXTM under vehicle x-ray inspection systems at select U.S. Customs and Border Protection (CBP) land points of entry. The Osprey-UVX, in conjunction with Viken’s widely deployed handheld imagers, provides U.S. CBP and other security agencies with a comprehensive, practical, affordable and safe non-intrusive inspection (NII) solution for high-throughput vehicle scanning at border, critical infrastructure and other security and military checkpoints. “Together, Viken’s HBI-120 and Osprey-UVX represent a powerful combination in the fight against drug and human trafficking, and terrorism that targets critical infrastructure,” said Viken CEO, Jim Ryan. “Instead of solely relying on camera-based systems or canine teams, law enforcement and security professionals can actually ‘see through’ an entire vehicle in under a minute to safely make fast and accurate threat assessments.” The Osprey-UVX is a fixed “in-the-ground” system that provides real-time undercarriage and lower vehicle imaging for security officers. Passengers can safely stay in the vehicle while under vehicle images are created using Viken’s X-ray imaging technology. Both a passenger and commercial versions are available, and mobile configurations are in the works. Viken is also launching its Osprey-EVXTM portal with demonstrations in the first half of 2020. The Osprey-EVX is a complementary solution to already installed Osprey-UVX units and is specifically targeted for locations with the highest traffic. With nearly 1,000 units deployed, the HBI-120 is currently in use by law enforcement agencies around the country and with CBP at the southern U.S. border. The HBI-120 is responsible for significant seizures of cash, drugs and weapons concealed in vehicles. The addition of the Broadwing-LAD (large-area detector) accessory provides for wider and deeper (and thus faster) scanning, without an increase in x-ray intensity. Viken Detection is the market leader in handheld x-ray imaging and in handheld lead detection, helping officials in each of these areas keep the public safe. Viken manufactured the first ever handheld device capable of imaging vehicles for drug interdiction. For more information, please visit us at www.VikenDetection.com

04

Elysium Health™ Announces Head of Bioinformatics and the Launch of INDEX by Elysium Health

NEW YORK, Nov. 4, 2019 (PRNewswire) -- Elysium Health, Inc.,TM a life sciences company developing clinically validated health products based on aging research, today announced the launch of INDEX by Elysium Health, an at-home test that enables customers to measure their cumulative rate of aging and their biological age. The company also announced the addition of Yale School of Medicine professor and aging researcher Morgan Levine, Ph.D., who joined as head of bioinformatics to lead the development of Index using the latest generation of an epigenetic biomarker called DNAm PhenoAge. Unlike earlier epigenetic clocks, which were developed to support population-level research, the Elysium Health team worked with Levine to further improve the testing methodology to make its application accurate and informative at the individual level. Since its founding, Elysium Health has been best known for the company's cellular health supplement Basis. Index marks the beginning of an expansion of the brand's product portfolio to include aging tests as well as interventions that target fundamental processes of aging.

"Scientists in the field of aging research have long explored two fundamental questions: Can aging itself be measured, and can we slow, stop, or reverse the processes associated with aging?" said Elysium Health CEO Eric Marcotulli. "With aging as the single greatest risk factor for all major chronic conditions—and a force that affects everyone even before birth—these questions are focused on supporting our collective ability to not only improve lifespan but, more importantly, healthspan. With the launch of Index, we hope to continue the scientific exploration of these two questions by extending the conversation to the consumer market and allowing everyone to benefit from the ability to determine their cumulative rate of aging and, with it, to see if lifestyle and other changes can impact how they age in the future."
Levine helped to identify specific DNA methylation sites along the epigenome that are highly correlated with nine clinical chemistry biomarkers of biological age and chronological age in the NHANES study. Since the development of the first epigenetic clock in 2011, the methodology has been further refined and validated in multiple large-scale data sets across diverse populations and tissues. A challenge to developing Index—and a key differentiator—was ensuring its accuracy and repeatability at the individual level. While the published applications of the biological age measure typically look at hundreds of sites on the genome, with the custom Elysium chip, Index examines global patterns across more than 100,000 sites, which solves for issues related to technical replicates—an incredibly important consideration for making this technology available to consumers and researchers. "Historically, chronological age has served as the best estimation of health and aging. However, not all individuals age at the same rate—based on genetics, demographics, and lifestyle factors—and therefore, people of the same chronological age don't necessarily share the same current health status or future outlook," said Levine. "Index provides a better picture of where an individual is in his/her aging process, which is why I'm excited to make this aging measure and the technology that supports it directly available to consumers. It shows incredible promise when it comes to monitoring overall health and wellness and there is potential for even more diverse applications in the future. In clinical research, tools like this also enable faster evaluation of intervention efficacy, avoiding the need for decades of follow-up. It may also inform basic and population research, by shedding light on factors that alter the pace of aging and facilitating the identification of therapeutic targets to slow the process. For people in their everyday lives, while there are no guarantees, understanding one's cumulative rate of aging and the factors that are reflected on the epigenome may help them prioritize behaviors and lifestyle choices to proactively optimize their health and wellness today and in the future." While the technology required for epigenome and genome mapping has historically made comprehensive testing cost prohibitive, with help from Illumina— a global leader in DNA sequencing and array-based technologies —Elysium Health has developed the custom Elysium chip to make the initial release of Index as accessible as possible. Elysium Health is committed to continuing to innovate the testing technology to make the powerful health measure of biological age available to everyone. During the first month of the product's initial limited release, Index will be available for purchase to existing Basis subscribers. New Elysium Health customers will have the option to purchase Basis semi-annual or annual subscriptions to become eligible to purchase Index in advance of the full public release in 2020. In addition to early access, Basis subscribers also have the benefit of preferred pricing during the limited release period.

04

PhoreMost and Sentinel Oncology Expand Collaboration to Jointly Accelerate Novel Glioma Therapeutic Through Preclinical Development

CAMBRIDGE, England, November 4, 2019 (BUSINESS WIRE)--PhoreMost Limited, the UK-based biopharmaceutical company dedicated to drugging ‘undruggable’ disease targets, and Sentinel Oncology, today announce an expansion of their collaboration to accelerate the progression of SOL686, a novel allosteric Polo-like kinase 1 (PLK1) inhibitor through preclinical development and IND enabling studies for the treatment of Glioma. Mitotic PLKs are widely recognised as playing crucial roles in disease causing pathways, including K-Ras mutant cancers. Traditional approaches to drugging PLK enzymes have focused on targeting their active site; however this tactic has been hindered by toxicity-associated adverse events. Sentinel Oncology’s allosteric PLK1 inhibitor takes the novel approach of targeting the Polo-box domain (PBD) of the PLKs, thereby aiming to mitigate adverse events seen by active site inhibitors. The programme has demonstrated a promising combination of specific drug-like properties, mode of action and target validation data obtained so far. Originating from the laboratory of Prof Ashok Venkitaraman at the University of Cambridge, PhoreMost subsequently developed the lead chemical series. Sentinel Oncology then optimised drug-like properties for the series and guided therapeutic positioning. Both PhoreMost and Sentinel Oncology received funding from Innovate UK for the drug discovery programme. Dr Chris Torrance, CEO of PhoreMost, said: “We are delighted to deepen our long-standing association with Sentinel Oncology, and excited to be progressing this drug discovery programme towards the clinic. This lead compound exemplifies the value of PhoreMost’s strategy to use functional protein-protein interactions to drive the development of novel therapies, and to capitalise on its SITESEEKER® platform to change the model of drug discovery through innovation, strategic partnerships and collaboration.” Robert Boyle, CEO of Sentinel Oncology, commented: “We are very excited about the prospects for this programme, and to be collaborating with PhoreMost to advance our allosteric PLK1 inhibitor. The programme adds to our NeuroOncology pipeline, has started formal preclinical studies and is well positioned to enter clinical development as a glioma treatment by 2021.”

October 2019

31

Cambridge Startup Targets Alzheimer's With A New Approach To An Old Idea

By Callum Borchers October 31, 2019 | WBUR.org --

Recent efforts to treat Alzheimer's disease have been marked by high hopes and major letdowns. Now, a Cambridge biotechnology startup called Pinteon is again raising hope — and millions of dollars — with what it touts as a new approach to an old idea.

The idea is that a protein called tau can become tangled and harm brain function in Alzheimer's patients. This "tau hypothesis" is popular among Alzheimer's researchers, but it hasn't yielded an effective treatment.

 

"There's a well-documented large body of work previously and, in the main, the outcomes of that work have been disappointing to both patients and sponsors," said Pinteon Chief Executive Marty Jefson.

His company aims to rewrite the narrative with a highly targeted therapy that entered its first clinical trial this month. Pinteon's prospective treatment zeroes in on a specific form of tau protein that "really is not present, in any great abundance, in normal, healthy individuals," according to Jefson.

"It really is essentially a hallmark of a pathologic state," he said. "So, we're hopeful that precisely targeting is going to be key to success for our program."

It will be years before Pinteon's therapy is available to patients — if it ever becomes available, at all. Many promising drugs flame out in trials, including a tau-centered treatment that the pharmaceutical giant AbbVie abandoned just a few months ago.

AbbVie had been testing the drug's potential to treat a brain disease called progressive supranuclear palsy; it remains a possible treatment for Alzheimer's according to the company.

Earlier this year, Roche walked away from two Alzheimer's treatment trials. Biotech industry publication BioSpace reported at the time that there had been about 150 failed attempts to develop Alzheimer's drugs in the past two decades.

Cambridge-based Biogen scuttled an Alzheimer's drug trial in March, then surprised many observers when it revived the prospective treatment last week. Biogen now says it plans to seek FDA approval next year.

The Biogen and Roche therapies are based on the "amyloid hypothesis," another leading idea about the cause of Alzheimer's, which focuses on the buildups of amyloid proteins in the brain.

Though Alzheimer's has stubbornly resisted treatment under the amyloid and tau hypotheses, the Hong Kong-based venture capital firm Morningside Ventures is optimistic enough about Pinteon's approach to invest $17 million in the startup.

"We know neurodegeneration is a tough field, but it's also a field with tremendous unmet need, and we, as a society, must keep pushing for new approaches," Morningside investment adviser Jason Dinges said in a statement. "We're proud to invest in Pinteon's novel approach to targeting toxic tau, and we're hopeful that future clinical studies will demonstrate its efficacy."

Pinteon was founded in 2014, when Morningside purchased a license to technology developed by Kun Ping Lu at Beth Israel Deaconess Medical Center, according to Jefson. The company has operated quietly for five years, building toward this month's launch of a clinical trial.

 

31

Pinteon Therapeutics Launches Phase 1 Trial of Novel Tau Antibody that Targets a Potent Driver of Neurodegenerative Disease

CAMBRIDGE, Mass., Oct. 31, 2019 – Pinteon Therapeutics today announced the launch of a Phase 1 trial of PNT001, a novel tau antibody that uniquely targets a toxic epitope known to drive neurodegenerative disease. Pinteon is funded by a $17 million Series A from Morningside Ventures and led by an experienced team of executives with strong track records in CNS drug discovery and development. The company’s lead asset, PNT001, targets a highly neurotoxic conformation of tau, known as cis-pT231 tau, that promotes the abnormal aggregation of tau proteins in neurons. This aggregation creates neurofibrillary tangles that cause neuronal death, block communication and impair memory and learning. PNT001 aims to disrupt the spread of this toxic form of tau by precisely targeting and neutralizing pathologic tau containing the cis-pT231 epitope. “We believe that PNT001 can intervene at a critical point in disease progression to interrupt the transmission of pathological tau through neural circuits,” said Martin Jefson, Ph.D., Pinteon’s chief executive officer. “By halting the spread of pathologic tau, we hope to protect and preserve brain function in patients living with tauopathies. It’s a critically important goal, and I’m proud to have such an experienced team focused single-mindedly on this mission.” PNT001 is the only antibody in development that targets cis-pT231 tau, which has been identified in multiple preclinical studies as a potent driver of neurodegenerative disease. Pinteon has initiated a Phase 1 trial of PNT001 in healthy volunteers that is designed to evaluate five single escalating dose levels. The primary endpoints will be safety and tolerability; Pinteon will also assess the pharmacokinetics of PNT001. The company plans to move next into one or more Phase 1 trials of multiple ascending doses in patients with traumatic brain injury, progressive supranuclear palsy or Alzheimer’s disease. Tau pathology is a central component in each condition. “PNT001 targets a potential pathogenic mechanism that other monoclonal antibodies in clinical development don’t target,” said Lawrence I. Golbe, M.D., emeritus professor of neurology at Rutgers University’s Robert Wood Johnson Medical School and a member of Pinteon’s scientific advisory board. “Of course, we hope that this mechanism is relevant in mTBI, PSP and Alzheimer’s, that PNT001 adequately engages its target in humans and above all, that patients benefit. The upcoming trials are designed to answer these important questions.”

30

Standard International and Elysium Health™ Announce Science-Backed Wellness Partnership

NEW YORK, Oct. 30, 2019 (PRNewswire) -- Elysium Health, Inc.™, a life sciences company developing clinically validated health products based on aging research, and Standard International, known for its boutique properties on New York's High Line and London's King Cross, today announced an ambitious partnership to better serve the needs of health-conscious travelers by introducing science-backed wellness to the hospitality experience. Expanding on partnerships with the Massachusetts Institute of Technology, Mayo Clinic, and University of Oxford, Elysium Health is teaming up with The Standard to launch a travel size version of its cellular health supplement Basis, as well as a range of wellness programming rooted in scientific research. Both initiatives will begin rolling out across The Standard properties in November 2019.

The partnership will bolster each brand's commitment to disrupting their respective industries to better serve the needs of consumers, and will provide travelers who are seeking a restorative, health-focused experience with scientifically supported products and services. "People are investing in wellness travel more than ever," said Standard International CEO Amar Lalvani, "and The Standard is known for unexpected, genuine experiences. With Elysium Health, our goal is to harness life science innovations to enable guests to thrive during their travels and when they return to their everyday lives."
At launch, the partners will introduce a co-branded, travel-sized jar of Elysium Health's cellular health supplement Basis in The Standard U.S. hotel rooms. In addition to in-room travel-sized jars, full size jars of Basis will also be available for purchase in The Standard U.S. hotel shops and vitrines. Basis is designed to increase NAD+ levels and activate sirtuins, which because of their importance in supporting overall health, scientists often refer to as "longevity genes." As an essential coenzyme, NAD+ plays a critical role in energy creation, circadian rhythm regulation, and DNA repair. Given the role of NAD+ in supporting a healthy sleep/wake cycle and that it naturally declines with age, increasing NAD+ levels may be able to improve the ability of travelers to adapt to jet lag. "The global wellness industry grew to $4.2 trillion in 2017, and that market will continue to grow as people explore new ways to prioritize their health," said Elysium Health CEO Eric Marcotulli. "Very few brands in this market, however, test their products or services to demonstrate effectiveness. People think that they're making healthier choices to benefit their long-term health but those decisions are often based on marketing tactics, not research. With Standard International, our goal is to make the scientific innovations coming out of aging research more accessible, better understood, and more impactful by creating meaningful experiences. In doing so, I hope that we will inspire other brands to test their products and services for safety and efficacy, and help people make more informed health purchases." The partners will also explore the use of emerging technologies like epigenetics to further improve the travel experience in novel ways. This includes collaborative content that will cover breakthrough advancements in aging research through a travel lens and leverage the authority and influence of their individual editorial properties. With live events that are grounded in science, the partnership will broaden the Standard Hotels celebrated cultural programming – covering a range of topics from fundamental processes of aging and sleep to women's health and epigenetics. As a core tenet of the Elysium Health brand, the activations will provide guests direct access to scientific research and the opportunity to engage with Elysium Health's renowned scientific advisory board members and research collaborators.

25

NuCana Announces FDA Clearance to Commence Phase III Study of Acelarin (NUC-1031) for the First-Line Treatment of Patients with Biliary Tract Cancer

EDINBURGH, United Kingdom, Oct. 25, 2019 (GLOBE NEWSWIRE) -- NuCana plc (NASDAQ: NCNA) announced that the US Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for its Phase III study (NuTide:121) of the investigational drug Acelarin in combination with cisplatin for patients with previously untreated locally advanced or metastatic biliary tract cancer. “We are pleased to have received clearance of our IND from the FDA,” said Hugh S. Griffith, NuCana’s Founder and Chief Executive Officer.  “We are also excited by NuTide:121’s potential, if successful, to support both accelerated and full approval.  Regulatory clearance to begin the study in other countries has also been received and we look forward to enrolling the first patients in the coming weeks.” Mr. Griffith continued: “We believe our investigational drug product Acelarin in combination with cisplatin has the potential to significantly improve the survival outcomes of patients with advanced biliary tract cancer. In the Phase Ib ABC-08 study, we observed an approximate doubling of the response rate with Acelarin plus cisplatin compared to previously reported data for gemcitabine plus cisplatin. Our goal is to establish Acelarin in combination with cisplatin as the global standard of care for the first-line treatment of patients with advanced biliary tract cancer, if Acelarin is approved for marketing.” NuTide:121 will be a global, multi-center, randomized Phase III study that will enroll up to 828 patients in approximately 120 sites across North America, Europe, Asia and Australia. Patients will be randomized 1:1 and treated with either a combination of Acelarin (725 mg/m2) plus cisplatin (25 mg/m2) or the current standard of care regimen, gemcitabine (1,000 mg/m2) plus cisplatin (25 mg/m2). The primary objectives of NuTide:121 are Overall Survival (OS) and Objective Response Rate (ORR). Three interim analyses, including two designed to support accelerated approval, are planned as part of the Phase III study protocol in addition to the final analysis. Based on discussions with the FDA and subject to any further regulatory guidance, the Company believes that a statistically significant improvement in ORR at either of the first two interim analyses, supported by positive trends in other endpoints, could potentially allow for an accelerated approval of a new drug application (NDA) for Acelarin.  Accelerated approval requires a confirmatory clinical study to verify the drug’s clinical benefit. If accelerated approval were to occur, NuTide:121 would continue and the Company anticipates that data from subsequent analyses could provide the confirmatory data to support full (regular) approval. Dr. Jennifer J. Knox, Professor of Medicine at the University of Toronto, Clinician Investigator at the Princess Margaret Cancer Centre and Chief Investigator of NuTide:121 stated: “I am very excited to be leading the NuTide:121 study. I believe Acelarin plus cisplatin may represent an important advance in the treatment of biliary tract cancer, a devastating disease for which there is a significant need for more effective medicines.” Dr. Juan Valle, Professor and Honorary Consultant in Medical Oncology at the University of Manchester and The Christie, Manchester, United Kingdom was Chief Investigator of the ABC-02 study that established gemcitabine plus cisplatin as the current standard of care for the first-line treatment of patients with advanced biliary tract cancer. Professor Valle said: “As Co-Chief Investigator of the ABC-08 Phase Ib Study, I was encouraged by the efficacy signals observed with Acelarin plus cisplatin, so I am enthusiastic about further investigating Acelarin in combination with cisplatin in NuTide:121, the largest study ever conducted in patients with advanced biliary tract cancer.”  

24

Cognoa Launches Pivotal Study of the First Digital Diagnostic for Autism

Palo Alto, Calif., Oct. 24, 2019 (GLOBE NEWSWIRE) -- Cognoa, a company at the forefront of pediatric behavioral health, announced today initiation of its Pivotal Study to demonstrate the effectiveness of its diagnostic aid for autism spectrum disorder (ASD). The company anticipates submitting the results of this multi-site, prospective, blinded, active comparator study to the U.S. Food and Drug Administration (FDA) in 2020. The Cognoa digital diagnostic has the potential to become the first cleared medical device to help diagnose autism. It is intended to enable pediatricians and physicians in primary care settings to make an autism diagnosis in children as young as 18 months, so that children can receive earlier treatment. Currently, children are diagnosed for autism by a specialist at an average age of 4 years 4 months, which is after the primary window of brain development when interventions can have the greatest impact. Cognoa is developing AI-powered digital therapeutics and medicines to advance the standard of care in pediatric behavioral health and improve lifelong outcomes for children. Cognoa has received FDA Breakthrough Device designation for its two lead products, the first digital diagnostic for autism and a digital therapeutic to treat autism. This designation provides priority review for clearance by the FDA. “Cognoa believes digital therapeutics can fundamentally improve the standard of care for pediatric behavioral health. Our breakthrough products will empower both parents and their trusted family physicians so that every child can receive an earlier diagnosis and treatment when there is the greatest ability to impact the child’s development and improve lifelong outcomes,” said Brent Vaughan, CEO of Cognoa. “Building upon Cognoa’s previous successful clinical studies, this pivotal study is an important milestone for our company. In tandem, we have established our commercial model with our partner, EVERSANA, that will enable access to our digital therapeutics by physicians through existing prescription, distribution and reimbursement infrastructures for utmost efficiency.” “The current system does not serve children with autism or families who are concerned about their child’s development. Barriers to treatment will not change as long as access to diagnosis and treatment stays heavily reliant on specialty care, where we face a growing shortfall. We believe digital medicines can help address the limitations of our current system, making both primary and specialty care physicians more efficient and effective,” said Dr. Sharief Taraman, Chief Medical Officer of Cognoa, Division Chief at CHOC Children’s and Vice President of the American Academy of Pediatrics Orange County, California Chapter. Recent published studies reinforce the current crisis in access to timely autism diagnosis and care. In September, a study published in Pediatrics found that the most commonly used screener by pediatricians, the M-CHAT, was less effective in identifying autism than previously reported. Another study found that more than two-thirds of children flagged for autism were not referred to a specialist for treatment. In published studies, prior versions of Cognoa’s algorithm have been clinically validated to screen for autism as early as 18 months of age. Results demonstrate continuous improvements and higher accuracy compared to other behavioral health screening tools including the Revised M-CHAT.  

16

Hepagene Therapeutics, Inc, Announces Dosing of First Healthy Subject in a Phase I Study of HPG1860 for NASH

SHANGHAI, Oct. 16, 2019 (PRNewswire) -- Hepagene Therapeutics, Inc, a clinical stage drug discovery and development company which devotes its efforts towards discovering, developing and delivering innovative medicines that help patients prevail over liver diseases, announced today that it has dosed the first healthy volunteer in a phase I clinical trial of HPG1860 in the United States. HPG1860 is a non-bile acid, potent, selective and full farnesoid X receptor (FXR) agonist. Hepagene's Phase I study of HPG1860, entitled "A Randomized, Double-Blind, Placebo Controlled, Sequential Parallel Group, Single and Multiple Ascending Doses (SAD/MAD) Study following Oral Administration in Healthy Subjects to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of HPG1860", is to assess the safety, tolerability and pharmacokinetics and pharmacodynamics in single- and multiple-ascending dosed healthy volunteers. The study will also evaluate the food effect of HPG1860 in healthy subjects. "HPG1860 is a non-bile acid FXR agonist and exhibits excellent differentiation profiles based on our preclinical research study," stated Dr. Michael X. Xu, Hepagene's CEO. "The primary goal of HPG1860 Phase I study is to understand the drug safety profile. At the same time, the results from PD biomarker study will help to establish a safe and efficacious dose for Phase II study. PBC/PSC indication will also be considered for HPG1860."

10

Alexion and Stealth Announce Agreement for Option to Co-Develop and Commercialize Late-Stage Therapy for Mitochondrial Diseases

BOSTON, MA, October 10, 2019 -- (BUSINESS WIRE)  Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) and Stealth BioTherapeutics Corp (NASDAQ:MITO) today announced an agreement for an option to co-develop and commercialize elamipretide for mitochondrial diseases. Currently being evaluated in a Phase 3 study in people with primary mitochondrial myopathy (PMM) - a genetic mitochondrial disease - elamipretide is a novel, potential first-in-class therapy that targets mitochondrial dysfunction. There are currently no therapies approved to treat PMM, which is characterized by debilitating skeletal muscle weakness, chronic fatigue and exercise intolerance. Under the terms of the agreement, Alexion will have the opportunity to exercise the option following the delivery of results from the Phase 3 study currently underway in PMM.

“Mitochondria play a critical role in normal organ function and, when dysfunctional, can have devastating consequences on multiple organ systems, leading to many serious diseases, such as primary mitochondrial myopathy,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “Given our strong existing relationships with neuromuscular specialists – who play a critical role in treating PMM – we believe this is an exciting potential opportunity to further expand our rare neurology portfolio and look forward to the possibility of working with Stealth to realize the promise of elamipretide for patients.”

“Our mission is to deliver therapies to patients suffering from devastating mitochondrial diseases, and this partnership will enhance our ability to rapidly deliver on that mission,” said Stealth Chief Executive Officer Reenie McCarthy. “Together with Alexion, which is widely recognized for its demonstrated ability to bring important new therapies for rare diseases to children and adults in need, we believe we can achieve synergies of execution that will both expedite and increase patient access following achievement of key upcoming clinical and regulatory milestones for our PMM and Barth syndrome programs.”

Under the terms of the agreement, Alexion will receive an exclusive option to partner with Stealth in the development of subcutaneous elamipretide based on final results from the Phase 3 study currently underway in PMM. If Alexion chooses to exercise the option, the companies will co-develop subcutaneous elamipretide in the U.S. for PMM and Barth syndrome, as well as Leber’s hereditary optic neuropathy (LHON), which is currently in earlier stage clinical development. Upon commercialization, the agreement would provide for a 50-50 co-promote between the two companies in the U.S. and Alexion would receive exclusive rights to develop and commercialize subcutaneous elamipretide outside the U.S. Alexion will make initial payments to Stealth totaling $30 million, including an option fee, an equity investment and development funding. If the option is exercised, the agreement provides for additional payments, including an option exercise fee, an additional equity investment, development funding and potential regulatory and commercial milestone payments. Stealth’s other pipeline assets, including SBT-272, are not included in the option.

09

IM Therapeutics Raises $10 Million Financing Co-Led by JDRF T1D Fund and Morningside Ventures

AURORA, Colo. & BOSTON, October 9, 2019 (BUSINESS WIRE) -- ImmunoMolecular Therapeutics (“IM Therapeutics”), a clinical stage company developing personalized therapies for autoimmune disease, today announced that it has raised $10 million in Series A financing to advance its novel HLA-targeted discovery platform and develop its lead drug candidate in type 1 diabetes (T1D). The funding was co-led by the JDRF T1D Fund and Morningside Ventures, with participation from the Colorado University Healthcare Innovation Fund. IM Therapeutics was founded by world-class physician scientists at the Barbara Davis Center for Diabetes, University of Colorado. Their seminal research showed that blocking action of certain human leukocyte antigen (HLA) gene variants known to be high risk factors blocks the corresponding autoimmune response. As the first such personalized therapy approach in autoimmunity, the Company demonstrated this in a Phase 1b human study in patients recently diagnosed type 1 diabetes who were positive for the HLA DQ8 variant. The study was funded in part by JDRF. The Company is advancing its lead oral drug, IMT-002, through IND development for type 1 diabetes and expanding its core therapeutic platform of HLA blockade to other targets of autoimmune disorders including celiac disease. “Our value has been clear since day one – getting to the root cause of autoimmunity with a targeted therapy approach and making an impact on diseases such as type 1 diabetes,” said Nandan Padukone, Ph.D., CEO of IM Therapeutics. “We believe the expertise brought by the JDRF T1D Fund and Morningside Ventures will help us build a broad portfolio in autoimmune disease.” Dr. Padukone worked with the T1D Fund team as an Entrepreneur-in-Residence and with IM Therapeutics founders, Drs. Peter Gottlieb and Aaron Michels, to launch the Company’s platform and advance its clinical pipeline. “We are excited to be a lead investor in IM Therapeutics and are encouraged by the early results of their lead drug in T1D,” said Katie Ellias, Managing Director, at the JDRF T1D Fund. “We believe the company’s platform could be a game-changer not only for T1D but also for other autoimmune diseases.” IM Therapeutics’ innovative therapeutic approach is currently directed at HLA-DQ8 and DQ2 variants known to predispose individuals to both T1D and Celiac disease. HLA-DQ8 is present in about 60% of T1D patients and in 10% of Celiac disease, while nearly 100% of Celiac patients have DQ2 and/or DQ8 with DQ2 present in about 90% of Celiac disease. The Company has been the first to demonstrate in a clinical study that treating patients based on HLA DQ8 can inhibit the autoimmune response in T1D. With a robust technology platform in place that combines computational chemistry, bioassays, and rational drug design, IM Therapeutics is developing a pipeline against HLA targets in various autoimmune diseases. Sean Doherty, Executive Chairman, JDRF T1D Fund and Dr. Jason Dinges, Investment Advisor, Morningside Ventures, have joined the IM Therapeutics Board of Directors.

September 2019

19

Elysium Health Announces FDA Acceptance of Investigational New Drug Application to Evaluate BASIS for Prevention of Acute Kidney Injury in Surgical Cardiac Patients

NEW YORK, NY, Sept. 19, 2019 (PRNewswire) -- Elysium Health, Inc.,™ a life sciences company developing clinically validated health products based on aging research, today announced the acceptance of an Investigational New Drug ("IND") application by the Food and Drug Administration (FDA) to evaluate the efficacy of BASIS™ for the prevention of acute kidney injury (AKI) in surgical cardiac patients. In 2018, a successful Phase I dose-escalating study to evaluate the safety of Basis in patients with AKI was conducted at Massachusetts General Hospital. The approved IND enables the initiation of a Phase II, randomized, double-blinded, placebo-controlled clinical trial to evaluate the efficacy of Basis for kidney protection against AKI in patients treated by complex aortic aneurysm repair and aortic arch reconstruction, which is scheduled to begin later this year.

The IND marks the first acceptance for the company and its cellular health supplement Basis, which is designed to reverse the age-related decline of the critical coenzyme NAD+ and activate sirtuins. In an earlier published clinical trial, Basis was shown to increase NAD+ levels on average by 40% from baseline in healthy adults. Often referred to as "longevity genes," sirtuins are NAD-dependent, anti-aging enzymes that play a diverse role in cellular energy metabolism and gene regulation. In preclinical studies, animals that were deficient in SIRT1 have been shown to be more susceptible to developing AKI.

"While upwards of 30% of surgical cardiac patients are estimated to experience an incidence of AKI, it is a complication that generally affects 3% of all hospitalized patients," said Elysium Health chief scientist and director of the Glenn Center for Biology of Aging Research at MIT, Dr. Leonard Guarente. "Preclinically, it's been demonstrated that the regulation of NAD+ biosynthesis plays a pivotal role in renal recovery from injury. The sirtuin, SIRT1, is highly protective against kidney damage due to oxidative injury, and raising the activity of this sirtuin with NAD+ precursors is also protective and restorative of kidney function. Our Phase I trial was a collaboration that was made possible by Elysium Health's open-access research model, and it combined work that has come out of numerous labs with ongoing work related to AKI and chronic kidney disease at Massachusetts General Hospital. This IND is an exciting advancement for Elysium Health but also more broadly for the field of aging research, as it allows for the further exploration of a potential treatment that targets a fundamental process of aging."

AKI is a frequent complication in cardiac patients who undergo open surgical or endovascular repair and is associated with significant morbidity and mortality. According to the CDC's 2014 report – Trends in Hospitalizations for Acute Kidney Injury – the total number of hospitalizations with acute kidney injury increased from 953,926 in 2000 to 1,823,054 in 2006 and 3,959,560 in 2014. Clinicians increasingly recognize acute kidney injury as an in-hospital complication of sepsis, heart conditions, and surgery. It is associated with higher likelihood of long-term care, increased incidence of chronic kidney disease, increased hospital mortality, and higher health care costs. A number of U.S. studies have indicated an increasing incidence of dialysis-treated acute kidney injury since the late 1990s.

"Directed by Elysium Health's mission, our work focuses on identifying unmet needs and developing products to support lifelong health with the goal of preventing age-related conditions," said Elysium Health CEO Eric Marcotulli. "While it's incredibly rare for a supplement to receive IND approval, we are most excited about the further study that this acceptance enables and the potential to establish a clinically proven method for the prevention of a condition that has life-changing implications and no available treatments."

18

Phase 2 Study Published in Ophthalmology Highlights Efficacy and Safety Data of Intravitreal APL-2 for Geographic Atrophy Secondary to Age-Related Macular Degeneration

CRESTWOOD, Ky., and WALTHAM Mass., September 18, 2019 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals Inc., (NASDAQ:APLS) a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds to treat disease through the inhibition of the complement system, today announced details about its Phase 2 FILLY study investigating intravitreal (IVT) APL-2 (pegcetacoplan) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) have been published in Ophthalmologythe journal of the American Academy of Ophthalmology. Publication of the full study report follows the release of topline study results earlier this year and shows that treatment with APL-2 resulted in statistically significant reductions in the growth of GA lesion area compared to sham at month 12.

“There is a significant need to develop treatments for people living with GA, a progressive, chronic disease that often results in permanent loss of vision,” said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. “The FILLY study results show preliminary positive benefits of targeting the complement cascade at C3, which is implicated in the destruction of eyesight-protecting retinal pigment epithelium cells, and the potential for APL-2 to be an effective treatment option for people living with GA.”

The FILLY study was a Phase 2, multicenter, randomized, single-masked, sham-controlled clinical trial evaluating APL-2 in 246 patients with GA secondary to AMD conducted at over 40 clinical sites in the United States, Australia and New Zealand. APL-2 was administered as an intravitreal injection monthly or every other month (EOM) for 12 months, followed by six months of monitoring after the end of treatment. The primary efficacy endpoint was the change in GA lesion area from baseline to month 12 compared to sham.

At 12 months, patients treated with APL-2 showed a 29% reduction in the growth of GA lesion area in the monthly treatment group (p=0.008) and a 20% reduction in the EOM treatment group (p=0.067) compared to the pooled sham group. Statistical significance was defined as p<0.1 for this study. Post-hoc analysis showed that the effect was more pronounced in the last six months of treatment, with observed reductions of 45% (p=0.0004) and 33% (p=0.009) for APL-2 monthly and EOM, respectively, compared to sham.

The administration of APL-2 was generally well tolerated. There was an increased incidence of exudation in APL-2 treated eyes (20.9% in the monthly group and 8.9% in the EOM group) compared to sham-treated eyes (1.2%), which was manageable with the administration of standard-of-care treatment.

“There are currently no approved treatments for GA, which means that the approximately five million GA patients globally live knowing that they will lose vision over time,” said David S. Liao, M.D., lead author and retina specialist at the Retina-Vitreous Associates Medical Group. “I’m very encouraged by the FILLY data and look forward to seeing the upcoming results from Apellis’ Phase 3 studies of APL-2 in GA.”

The U.S. Food and Drug Administration (FDA) granted APL-2 Fast Track Designation for the treatment of GA, which facilitates the development and expedites the review of investigational therapies to treat serious conditions and fill an unmet medical need.

Apellis is currently enrolling two global confirmatory Phase 3 studies (DERBY and OAKS) for patients with GA. These identical, prospective, multicenter, randomized, double-masked, sham-injection controlled studies are designed to assess the efficacy and safety of multiple IVT injections of APL-2 in patients with GA secondary to AMD.

More information regarding DERBY and OAKS can be found at https://gastudy.com/.

10

Cognoa and EVERSANA™ announce partnership to advance the commercialization standard for prescription digital medicines

PALO ALTO, Calif. and CHICAGO, Sept. 10, 2019 (PRNewswire)--Cognoa, a company at the forefront of pediatric behavioral health, and EVERSANA™, the leading independent provider of global commercial services to the life science industry, today announced a partnership to advance the industry standard by which digital medicines will be ordered, dispensed, and covered by insurance. By leveraging the existing healthcare infrastructure utilized by pharmaceutical companies and medical device manufacturers, EVERSANA will ease adoption by physicians, payers and patients at commercial launch of Cognoa's prescription digital medicines.

Cognoa is developing digital medicine solutions to change the standard of care in pediatric behavioral health to improve the lifelong outcomes for children. Cognoa has received Breakthrough Device designation from the U.S. Food and Drug Administration (FDA) for the first digital diagnostic and first digital therapeutic device for autism, the company's first area of therapeutic focus. These devices utilize Cognoa's AI-powered digital medicine platform, designed to support earlier identification and treatment of pediatric behavioral health conditions.
Cognoa selected EVERSANA, a fully integrated and independent commercial services platform, to develop and manage a go-to-market strategy that ensures comprehensive market access; deploys highly trained sales and clinical field force to reach and educate providers; and effectively supports the patient journey through prescription and intake, benefits verification, distribution, and adherence through personalized Hub and specialty pharmacy services. "Cognoa is excited to be entering the commercial phase working with EVERSANA, the leader in pharmacy distribution of digital medicines, enabling us to fulfill our mission of changing the standard of care in pediatric behavioral health by ensuring that every child and family has access to earlier diagnosis and treatments," said Brent Vaughan, CEO, Cognoa, Inc. "EVERSANA's national footprint and extensive payer contracts will support the routine ordering and reimbursement of prescription digital medicines. EVERSANA is helping us - as an industry - to unleash the potential of digital medicine. For Cognoa, that means empowering every trusted family physician to improve lifelong outcomes for children." "Cognoa is working to solve the critical unmet need in behavioral healthcare by enabling earlier interventions and more personalized, accessible care. It's our privilege to ensure that Cognoa's prescription digital medicines are available to every physician, so that any child can get access earlier when those innovations have the greatest impact," said Jim Lang, CEO, EVERSANA. "While advancements in digital medicine take us into a new frontier, these devices can and should be available through existing prescription, distribution and reimbursement infrastructures. The commercial model for digital medicine will be efficient, effective and valuable to payers, providers and most importantly, for the patients and caregivers who need them."

10

CellCentric starts dosing patients in blood cancer trial, expanding clinical testing of CCS1477

CCS1477 is the first drug to be used in patients that specifically targets p300/CBP, offering a new route to treat specific drug-resistant cancers

Trial in late stage prostate cancer well underway across multiple clinical centres, under the leadership of Johann de Bono of The Royal Marsden Hospital, The Institute of Cancer Research, London

Dosing in patients with haematological malignancies has now begun, starting at the Christie Hospital, Manchester

CAMBRIDGE, U.K., September 10, 2019, Cambridge--CellCentric has expanded the clinical development programme of its novel anti-cancer drug CCS1477, a highly potent and specific small molecule inhibitor of p300/CBP. Dosing has commended in a new study involving patients with multiple myeloma and will include those with acute myeloid leukaemia and certain lymphomas. Trials are already underway for patients with late stage drug resistant prostate cancer. Dr Nigel Brooks, CellCentric's Director of Translational Science, commented: "Inhibiting p300/CBP is of growing interest to researchers.  We are delighted to now be testing our first-in-class inhibitor in patients with different blood cancers.  CCS1477 has the potential to be a major new drug to treat patients whose disease has relapsed, and have tumours that are resistant to current treatments." Inhibiting the twin gene regulator proteins p300/CBP disrupts the drivers of late stage prostate cancer, as well as the resistance mechanisms to existing anti-cancer drugs, such as abiraterone and enzalutamide. CCS1477 is aimed for use after, or in combination with these drugs, to address a major unmet need for patients with metastatic disease. P300/CBP inhibition also impacts the IRF4-Myc pathway, which has been proven to have a significant effect on certain blood cancers in multiple pre-clinical models. CCS1477 is now being evaluated for its effectiveness against late stage multiple myeloma (MM), acute myeloid leukaemia (AML), and non-Hodgkin lymphoma (NHL).  The lead Chief Investigator for the blood cancer programme is Prof Andy Davies, University Hospital Southampton. CCS1477 binds highly selectively into the conserved bromodomain pocket of both proteins. Other ways of targeting p300/CBP are being investigated, such as binding to the catalytic acetyltransferase site. CellCentric's approach appears to deliver a positive anti-tumour positive mechanism of action whilst minimising the range of other pathways effected. CellCentric originally was formed from the Gurdon Institute, University of Cambridge with one of the pioneers of epigenetics and gene regulation, Azim Surani CBE FRS.  The company evaluated over 50 potential epigenetic-related drug targets, before focusing on p300/CBP and the development of CCS1477. Dr Will West, CellCentric's CEO, commented: "CellCentric was a pioneer in the area of epigenetics.  It is really fulfilling to now see our science translated for patients with different types of cancer, whose tumours that are not responsive to existing treatments. Our strong science base, coupled with a highly experienced discovery and development team, have been key."  

09

MicuRx Pharmaceuticals Reports Positive Top-Line Results from a US Phase 2 ABSSSI Clinical Trial of Novel Antibiotic Contezolid Acefosamil

FOSTER CITY, California, and SHANGHAI, China, September 9, 2019--MicuRx Pharmaceuticals, Inc., today announced positive top-line results for study MRX4-201, a US Phase 2 randomized, double-blind clinical trial comparing contezolid acefosamil (MRX-4) with linezolid for the treatment of acute bacterial skin and skin structure infections (ABSSSI). Contezolid acefosamil met all primary and secondary efficacy endpoints with a potentially improved hematologic safety profile. “We sincerely appreciate the participation of the patients and the investigators and their study staff in this Phase 2 clinical trial,” said Dr. Zhengyu Yuan, President and CEO of MicuRx. “We are very pleased with the excellent results of this study and plan to advance contezolid acefosamil into Phase 3 development next year,” continued Dr. Yuan. The Phase 2 trial enrolled 196 ABSSSI patients in a 2:1 ratio (contezolid acefosamil 131 subjects, linezolid 65 subjects) at 7 centers in the United States to evaluate the safety and efficacy of the intravenous (IV) and oral formulations of contezolid acefosamil for 10-14 days of therapy compared to linezolid. Contezolid acefosamil subjects started with a loading dose of 1500 mg IV followed by twice daily 1000 mg IV doses with the option after at least 3 IV doses to switch to 1300 mg oral doses twice daily; linezolid subjects started with 600 mg IV twice daily for at least 3 doses, after which they could switch to oral 600 mg twice daily. For the primary efficacy endpoint, the percentages of subjects in the intent-to-treat (ITT) population with favorable early clinical responses at the early assessment (EA) visit (48-72 hours after the start of study drug) were 77.9% in the contezolid acefosamil group and 78.5% in the linezolid group. Outcomes were also similar in the ITT population at the post-therapy evaluation (PTE) visit (7-14 days after end of therapy [EOT]), with favorable responses of 76.3% in the contezolid acefosamil group and 73.8% in the linezolid group. Similar results between the study groups were observed in other secondary efficacy outcomes in clinically evaluable and microbiological populations at the EA, EOT, and PTE visits. Methicillin-resistant Staphylococcus aureus (MRSA) was the most commonly identified pathogen, and efficacy outcomes were similar in subjects between the two study arms with MRSA infections. The overall incidence of treatment-emergent adverse events (TEAEs) was similar between the study arms, including TEAEs considered to be related to study drug (contezolid acefosamil 16.3%, linezolid 14.1%). Nausea and vomiting were the most common TEAEs, and most were mild or moderate in severity. There were no drug-related TEAEs considered to be serious or that led to discontinuation of study drug. Overall, safety laboratory changes were similar, though the proportions of subjects with neutrophil and platelet values that were below the lower limit of normal (LLN) or substantially abnormal (SA) were lower in the contezolid acefosamil group than in the linezolid group (neutrophils: contezolid acefosamil below LLN 3.7% and SA 0%, linezolid below LLN 7.4% and SA 3.7%; platelets: contezolid acefosamil below LLN 7.6% and SA 2.5%, linezolid below LLN 12.1% and SA 5.2%). “The IV and oral formulations of contezolid acefosamil appear to be effective in treating ABSSSI, including MRSA infections, as evaluated by endpoints consistent with FDA and EMA bacterial skin infection clinical trial guidance documents, with a dosing regimen that was safe and well-tolerated,” said Edward Fang, MD, Senior Vice President, Clinical Development. “The Phase 2 data support proceeding to pivotal Phase 3 ABSSSI and diabetic foot infection (DFI) studies, and the lower frequencies of hematologic laboratory abnormalities observed with contezolid acefosamil compared with linezolid suggest a potential improvement in safety over other oxazolidinones that may prove even more significant in patients with DFI who often require treatment beyond 14 days,” added Dr. Fang. Linezolid became a “blockbuster” antibiotic for the treatment of multidrug-resistant (MDR) Gram-positive infections, including MRSA and vancomycin-resistant enterococci (VRE) infections, despite well-known hematologic toxicity which often limits clinical utility. MicuRx plans Phase 3 clinical trials in ABSSSI and DFI in order to confirm the safety and efficacy profile of contezolid acefosamil, including with longer duration therapy.

04

Aduro Biotech Announces First Patient Dosed in Phase 2 Study of ADU-S100 (MIW815) in Combination with KEYTRUDA® (pembrolizumab) for First-Line Treatment of Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma (HNSCC)

BERKELEY, Calif., September 4, 2019 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (NASDAQ: ADRO), a clinical-stage biopharmaceutical company focused on developing therapies targeting the Stimulator of Interferon Genes (STING) and A Proliferation Inducing Ligand (APRIL) pathways for the treatment of cancer, autoimmune and inflammatory diseases, today announced that the first patient has been dosed in a Phase 2 clinical trial of ADU-S100 (MIW815), a novel STING pathway activator, in combination with KEYTRUDA® (pembrolizumab), an approved anti-PD-1 antibody, as a first-line treatment for recurrent or metastatic head and neck squamous cell carcinoma (HNSCC).

“The dosing of the first patient in the Phase 2 clinical study of ADU-S100 in combination with pembrolizumab marks an important advancement for Aduro as we shift from heavily pre-treated, heterogenous patient populations to earlier lines of treatment for patients with specific tumor types,” said Dimitry S.A. Nuyten, M.D., Ph.D., chief medical officer of Aduro. “There is increasing evidence that the potential benefit from immunotherapies is greater in patients with fewer prior therapies. With the recent FDA approval of pembrolizumab as first-line treatment for patients with metastatic or unresectable, recurrent HNSCC whose tumors express PD-L1 [Combined Positive Score (CPS) ≥1], we look forward to continuing the progress of our clinical program by investigating the potential synergistic benefit ADU-S100 may provide as a combination treatment option.”

The Phase 2, open-label, multicenter trial, which is part of an ongoing research and development collaboration with Novartis, is designed to evaluate the efficacy and safety of ADU-S100 (MIW815) administered intratumorally in combination with pembrolizumab in the first-line setting. The planned population will consist of 33 adults with PD-L1 positive recurrent or metastatic head and neck cancer (see www.clinicaltrials.gov, identifier NCT03937141).

03

Apellis Dosed First Patient in Phase 3 Study of APL-2 for Treatment-Naïve Patients with Paroxysmal Nocturnal Hemoglobinuria

CRESTWOOD, Ky. and WALTHAM, Mass., September 3, 2019 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals Inc. (NASDAQ: APLS) announced today the dosing of the first patient in the Phase 3 clinical study PRINCE (APL2-308), evaluating the efficacy and safety of APL-2 for treatment-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH). PRINCE is the second Phase 3 study that Apellis has initiated to investigate the potential of APL-2 to treat PNH.

“PNH is a rare, chronic blood disorder that can progress to cause life-threatening complications. We are conducting the PRINCE study to better understand how targeting the complement cascade at C3 can help us improve treatment for all patients with this disease,” said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. “At Apellis, we believe there is a significant opportunity for APL-2 to be a transformative therapy that could benefit people living with PNH.”

The PRINCE study is a Phase 3, randomized, multicenter, open-label, controlled study that aims to enroll 54 treatment-naïve adult patients with PNH. The primary objective of this study is to evaluate the efficacy of APL-2 in patients with PNH who are currently not being treated with complement inhibitors, as assessed by hemoglobin stabilization from baseline in the absence of transfusion through Week 26 and reduction in lactate dehydrogenase (LDH) level from baseline to Week 26.

PNH is a rare, acquired, potentially life-threatening disease characterized by complement-mediated hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes and/or some degree of bone marrow dysfunction. Some of the prominent symptoms of PNH include severe anemia, abdominal pain, headaches, back pain, excessive weakness, fatigue and recurrent infections. If not treated, PNH results in the death of approximately 35 percent of affected individuals within five years of diagnosis and 50 percent of affected individuals within 10 years of diagnosis.

August 2019

29

Vehicle scanning technology at the border is about to ruin the drug trade

By Leandra Bernstein All-day, everyday passenger and commercial vehicles line up for miles at dozens of U.S. ports of entry along the southwest border. For agents working to stop drugs from entering the country, it can feel like finding a needle in a haystack. Customs and Border Protection can only stop what its agents can see. What isn't detected at the border makes its way into the interior where it fuels the multibillion-dollar cross-border drug trade and the worst drug overdose epidemic in U.S. history. According to the Drug Enforcement Administration, the majority of fentanyl, heroin and methamphetamine is trafficked across the southwest border. It's primarily smuggled into the United States through legal ports of entry, meaning it comes within feet of a Customs and Border Protection officer. At the same time, CBP has acknowledged it currently scans only 2% of all private passenger vehicles and 16% of commercial vehicles at land borders. That is about to change as the government and private sector work together to deploy the systems and technologies to give officers at the country's entry points total visibility—through steel, aluminum, and loads of cargo. This technology isn't years away, but months away. By the start of 2020, CBP will begin installing and testing several X-ray imaging systems at ports along the southern border. The systems promise to provide officers and agents with a clearer view of what is crossing the border, so they can more easily detect drugs, weapons, cash and disrupt transnational criminal networks. "We need to start closing those gaps, scanning more," said Robert Watt, CBP's director of the Non-Intrusive Inspection Division. Watt is in charge of acquiring and deploying the equipment and detection technologies for the nation's roughly 320 land, sea, air and rail ports. TECHNOLOGY ARRIVING SOON The biggest challenge at the southern border is that the sheer volume of traffic has made it impossible to inspect every vehicle. For example, at San Ysidro, the busiest land border crossing in the Western Hemisphere, roughly 70,000 vehicle passengers travel northbound from Mexico into the United States every day. A typical officer has less than 20 seconds to scrutinize a vehicle and determine whether to let it through or subject it to additional, secondary screening. "It's a daunting task," Watt told Sinclair. Officers have to balance security against the demands of trade, try to prevent drugs and other contraband from entering the country without slowing cross-border trade and hurting the economy. "The better equipment we have out there, especially with all this new technology that's coming down the pike, we'll be doing a much better job than we're currently doing right now." X-ray imaging technology has been used for years across the Department of Homeland Security to detect everything from drugs to explosives, in cargo, mail shipments, rail freight and at airports. At the southern border, that non-intrusive scanning technology has mostly been used during secondary inspections. Each day, a relatively small number of vehicles deemed suspicious or randomly selected are diverted to a holding area and run through a large portal. Existing systems used in secondary screening employ both backscatter and transmission X-ray technologies. According to Watt, these systems are effective but because of their size, installing them at every port would likely halve the number of vehicle lanes. The effects on the economy would be felt immediately throughout North America. One backscatter image from a recent drug bust, showed a passenger vehicle packed with 134 pounds of methamphetamine, 2.6 pounds of heroin and 4.94 pounds of fentanyl. Another massive haul was discovered by agents using an older transmission detector. The image showed the bulky outline of more than 10,0000 pounds of marijuana and 463 pounds of crystal meth stashed in the rear of a tractor-trailer carrying picture frames. Even with the limited number of vehicle scans, Customs and Border Patrol agents seized, on average, more than 4,650 pounds of drugs each day last year. In 2019, agents have already seized 2,000 pounds of fentanyl, the potent drug responsible for more than half of all U.S. opioid overdose deaths and they are on track to seize more than 800 tons of other narcotics. Those numbers are expected to increase as demand for the drug grows and seizure tactics improve. In the coming months, CBP will be completing several pilot programs to move the non-intrusive scanning technology from secondary inspections to the front line. Similar to the idea of an E-Z pass system, the scanners should keep traffic moving while generating a detailed image of the inside of every vehicle before it reaches the official checkpoint. In most cases, the pre-primary scan will give officers a significant head start on suspicious cargo. According to Watt, by the end of the year, there will be a new scanning system in Anzalduas, Texas at the Hidalgo, Pharr port of entry for every passenger vehicle and another in Brownsville, Texas to scan every cargo vehicle. The agency is also expected to finish work on a new command center at the World Trade Bridge in Laredo. The command center will receive real-time imaging data on all trucks entering and exiting the high-volume inspection station. Finally, by early 2020, CBP will begin testing X-ray scanning systems from several top technology vendors. The systems will be installed at five small land ports along the border that have about four lanes of traffic. CBP will then test and assess which is the best system to handle large-scale, high-volume vehicle scanning. SEIZURES 'MEASURED IN THE TONS OF DRUGS' Viken Detection is one of the companies developing a full vehicle scanning portal for passenger vehicles at one of those border entry points. "The goal for us would be to scan every vehicle that's going across the border," said Viken Detection CEO Jim Ryan. The mission is analogous to Transportation Security Administration scanning every person and piece of luggage that makes it onto an airplane as quickly as possible. "Every vehicle will go through it quickly and you'll be able to see the entire vehicle," he said. "Once we get to that point we'll see the magnitude...the amounts of drugs that are coming across the border." Viken's system employs backscatter technology, which requires little energy to generate an image. It also emits a very low dosage of radiation, making it safe for regular use in a passenger environment. The company has already pioneered a handheld scanning device which is currently being used by several law enforcement agencies across the United States, including in what the DEA has designated High-Intensity Drug Trafficking Areas. Ryan described the 8-pound HBI-120 as a "scaled-down" version of the full-vehicle portal that will soon be put to the test at the border. In a demonstration, Ryan showed how the device can penetrate through up to 3 mm of steel, as well as aluminum panels and vehicle parts. The small device allows law enforcement officers to zero-in on areas of interest where traffickers may be concealing bundles of drugs or bricks of cash. Before X-ray imaging technology, it could take hours to physically dismantle a vehicle looking for compartments hidden deep inside the engine, in the transmission, inside transfer cases, underneath the vehicle or inside gas tanks. Now, a full interrogation of the vehicle can take a few minutes using the handheld device. In the past month, a significant number of Viken's HBI-120s were deployed to the border where they are currently in use by CBP agents. According to Watt, officers have already made a number of seizures with it. Viken was awarded a $28.8 million contract with CBP in October for the devices. About 500 of the instruments have been in wide deployment for about a year. According to Ryan, "Our seizures are measured in the tons of drugs and the millions of dollars in cash." APPLICATIONS FROM THE LAW ENFORCEMENT PERSPECTIVE Mike Tamez is one of the nation's top drug interdiction officers and currently works as part of a highway interdiction team in South Texas, about 60 miles from the southern border. In his position, he regularly interdicts loads of drugs that make it past Customs and Border Protection. "It's not the individual agent's fault. The factors are just against him at those checkpoints and at those entry points," Tamez said. Referring to the massive flow of daily traffic across the border, he added, "The numbers are just astronomical." Tamez has been training officers and using the HBI-120 for about a year. He said the scanning technology "has changed the game tremendously" for him and his officers. In a recent stop, Tamez was given consent to search a passenger vehicle en route from Montamores, Mexico to Houston. In a 10-second scan of the vehicle using the HBI-120 imager, he and another officer discovered 8 kilos of crystal meth hidden in the car. Without the scanner, an officer may have spent hours physically dismantling the vehicle looking for hidden contraband. The backscatter technology has also helped officers defeat a common smuggler tactic of hiding narcotics inside other organic materials, like gasoline. In another recent bust, Tamez was able to detect 100 lbs. of marijuana in a gas tank after a checkpoint stop. The advantages of law enforcement officers having access to advanced imaging technologies are obvious when it comes to disrupting the drug trade. One advantage that is less obvious is officer safety. "All you have to do is Google fentanyl and police officer and you're going to see countless stories and videos of police officers who were exposed to fentanyl....going down and they're having to bring them back," Tamez said. About five years ago, Tamez was working with a partner to inspect the cargo space of an 18-wheeler that he suspected had a hidden compartment. Before the handheld X-ray imager, the only way to see if the compartment was loaded was to drill into it and insert a scope. Tamez got underneath the vehicle and drilled through the floor into the compartment, accidentally penetrating the contraband. "As soon as I bring my drill out, cocaine powder just starts falling out," he said. "Now you apply that to today, right, what are we looking at? That much fentanyl goes airborne, I'm dead...the officers around me are dead." While it's important for citizens to understand the tools available to law enforcement agencies, Tamez emphasized, "I want our officers to know that this device will make them safer." DESIGNING A 'FUTURE-PROOF' BORDER SECURITY SYSTEM In 2019, Congress appropriated $675 million for the Department of Homeland Security to install the non-intrusive inspection technology at pre-primary lanes. The appropriation was $631 million more than the White House's initial request and Congress' initial offer. Given the size of the investment, Watts and vendors like Viken Detection are concerned about demonstrating it is worth the high cost. "We appreciate all the support by Congress," Watt said. "We are getting the new equipment out there, we are testing it out there and I think you're going to see a big change." As the full-vehicle scanners go into place at the border, Watts' division will aim to deliver statistics to Congress on the numbers of seizures as CBP assesses the effectiveness of each system. Ryan acknowledged that upgrading America's land ports with advanced imaging technology will be a significant, long-term investment. He emphasized that it must be "future-proof," in other words, adaptable to new threats and tactics. "These portals will probably remain intact for 15 to 20 years, so they have to be upgradeable. They have to be future-proof; they have to have the latest technology," Ryan said. In some cases, smugglers have already started to change their tactics to defeat X-ray technology. In response, Viken developed "different modalities of technologies" that not only generate images but alert agents to suspicious materials that might be present as part of a concealment tactic. That is the focus of the company's next-generation system. CBP and private sector partners are also looking ahead to the possibilities of integrating artificial intelligence and machine learning into the image-based systems. "There's a lot of really great technology coming out. A lot in the artificial intelligence field that we can put on for our cameras to see if something is going on that shouldn't be going on," Watt said. Through years of experience, well-trained officers become highly attuned to certain anomalies, whether it's an unusual mechanical configuration of a vehicle or an individual's behavior. It is possible to do something similar with artificial intelligence, by synthesizing the images generated by the scanning devices with information from technologies now in use, like biometrics, license plate readers, radio frequency identification (RFID) and other visual systems. The challenge is being able to use that information in real time. "That's where I think AI and machine learning comes into play of how do we process all of this information and create a risk profile so the officer can take action," Ryan said. That may also involve building new sensors into scanning systems and creating a truly multilayered approach. "I think it's only going to get better," Ryan said, emphasizing the breakthroughs in sensor technology, biometrics and artificial intelligence. "Those three things coming together are going to create an incredibly powerful tool for our law enforcement."  

06

Inotrem Announces FDA Clearance of Investigational New Drug (IND) for the Phase IIB ASTONISH Trial in Septic Shock Patients to Demonstrate Nangibotide Efficacy

PARIS, France, August 6, 2019 (BUSINESS WIRE) -- Inotrem S.A., a biotechnology company specialized in immunotherapy for acute inflammatory syndromes, announced today the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for the ASTONISH trial (Phase IIb) where the safety, tolerance and efficacy of nangibotide (LR12), its lead compound for septic shock, will be studied. The IND is now effective allowing Inotrem to begin its planned Phase IIb study in septic shock patients in the US.

The Phase IIb study will aim at demonstrating efficacy of nangibotide and bring a clinically relevant proof of clinical activity in septic shock patients. It will be a global multicentric study conducted in the United States and Europe. In addition, this study intends to validate a personalized medicine approach using soluble TREM-1 as potential companion diagnostic test to identify patients more likely to benefit from nangibotide treatment.

The FDA decision was based on the review of the information provided on the previous preclinical and clinical experience with nangibotide in particular the Phase IIa clinical study which demonstrated the safety and tolerability of nangibotide in patients suffering from septic shock. Preclinical models reviewed by the FDA showed that nangibotide was able to restore appropriate inflammatory response, vascular function, and improved survival in septic shock animal models. The design of the ASTONISH clinical trial has been previously discussed with the FDA during a preIND meeting. The regulatory process for clinical trial authorization in European countries is currently ongoing.

Septic shock is the ultimate complication of sepsis and currently constitutes a high unmet medical need. The incidence of septic shock continuously raises and mortality remains elevated (35%) in developed countries. There is currently no specific therapy approved for this indication besides antibiotics and symptomatic treatment. Inotrem’s solution is based on a novel approach of immunomodulation which targets the TREM-1 pathway: a crucial mediator of the septic shock and has the potential to become the first mechanism-based treatment for septic shock.

Jean-Jacques Garaud, CEO of Inotrem, said: “The FDA’s clearance of IND for nangibotide is an important milestone for Inotrem. It confirms the strong potential of this novel therapeutic approach and gives us the opportunity to validate both the proof of clinical activity of nangibotide and our personalized medicine strategy in septic shock, a severe and often fatal condition for which there is currently no specific targeted therapies”.

Mitchell Levy, Division Chief of Pulmonary, Critical Care and Sleep Medicine at the Warren Alpert School of Medicine at Brown University and Medical Director, Medical Intensive Care Unit at Rhode Island Hospital and Inotrem’s main investigator in the United States, added: “We are thrilled by the FDA’s decision which will allow to begin Inotrem’s planned Phase IIb study in septic shock patients, and which as such recognizes the company’s unique position around the TREM-1 pathway”.

06

Cinder, Powered by Desora, Partners with Michelin Starred Chef John Critchley to Bring High-Quality Recipes to All Cinder Users

CAMBRIDGE, Mass., Aug. 6, 2019 (PRNewswire) -- Cinder, the world's first 1° precision countertop grill, announced today the launch of its Chef Partnership Program, an exclusive program that will develop high-end recipes designed exclusively for Cinder Grill. For a limited time, existing Cinder users will receive a free lifetime subscription to recipes developed by Michelin Starred Chef John Critchley. The recipes are designed to highlight Cinder's unique Sous-Vide and Grilling capabilities. Users can expect to receive new recipes every week from Chef Critchley, with a first batch released today on Cinder's website. Cinder's proprietary Temp-Sense™ algorithm cooks to Sous-Vide perfection without the need for water or plastic bags. The release also coincides with the launch of the all-new Cinder App for Android. The all-new App now guides users through every step of the cooking process, from ingredient selection to food preparation. Once your food is ready to cook, Cinder takes over with its autopilot cooking, which makes overcooking impossible. "Our mission with Cinder is to give every person the ability to make restaurant-quality food from the comfort of their home," said Michel Maalouly, Co-Founder and CEO of Desora, the company behind Cinder. "Our partnership today with Chef John Critchley is a huge step in that direction."

July 2019

18

Apellis Pharmaceuticals Will Commence APL-9 Program to Control the Complement System in Host Responses to AAV Vector Administration for Gene Therapies

CRESTWOOD, Ky. and WALTHAM, Mass., July 18, 2019 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals Inc., (Nasdaq:APLS) a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds to treat disease through the control of the complement system, today announced its plans to develop APL-9 for the prevention of complement immune system activation coincident with adeno-associated virus (AAV) vector administration for gene therapies. APL-9, an investigational drug, is a pegylated synthetic cyclic peptide designed to modulate the complement cascade centrally at C3.

“Gene therapies are among the most exciting medical advancements of our time. However, they also pose a great technical challenge, since the AAV particles designed to deliver genes to tissues are subject to immediate and overwhelming immune attack, specifically by complement C3, which may result in significant safety and efficacy constraints,” said Cedric Francois, CEO and co-founder of Apellis. “We believe that the targeted control of C3 may prevent the C3-mediated attack on AAV particles, yielding three important potential advantages. First, controlling C3 may significantly increase the efficiency of gene therapy delivery by protecting the AAV particles on their journey from the site of administration to the target tissue. Second, control of C3 may minimize the formation of anti-AAV neutralizing antibodies that can complicate subsequent retreatment with the same AAV. Finally, by countering the rapid activation of C3 upon administration of AAV particles, control of C3 may prevent inflammatory reactions that can lead to significant organ damage, for example in the kidneys. Apellis is committed to the development of APL-9 to target host responses to AAV vector administration and thus potentially improve patients’ well-being while undergoing AAV administered gene therapies.”

In Phase I testing, Apellis treated 20 healthy volunteers with single doses of APL-9 ranging from 30 mg to 600 mg, administered as an intravenous infusion. APL-9 demonstrated control of complement through modulation of C3 within 1 hour of administration, that lasted up to 12 hours after the end of the infusion. Multiple doses tested achieved complete suppression of the AH50 hemolytic activity. In this Phase I study, APL-9 was well tolerated with no serious adverse events reported.

18

A Technology Assist For Autistic Children

By Cade Metz July 18, 2019 | The New York Times

SAN FRANCISCO -- When Esaïe Prickett sat down in the living room with his mother, father and four older brothers, he was the only one wearing Google Glass.

As Esaïe, who was 10 at the time and is 12 now, gazed through the computerized glasses, his family made faces -- happy, sad, surprised, angry, bored -- and he tried to identify each emotion. In an instant, the glasses told him whether he was right or wrong, flashing tiny digital icons that only he could see.

Esaïe was 6 when he and his family learned he had autism. The technology he was using while sitting in the living room was meant to help him learn how to recognize emotions and make eye contact with those around him. The glasses would verify his choices only if he looked directly at a face.

He and his family tested the technology for several weeks as part of a clinical trial run by researchers at Stanford University in and around the San Francisco Bay Area. Recently detailed in The Journal of the American Medical Association, Pediatrics, the trial fits into a growing effort to build new technologies for children on the autism spectrum, including interactive robots and computerized eyewear.

The Stanford study's results show that the methods have promise and indicate that they could help children like Esaïe understand emotions and engage in more direct ways with those around them. They could also measure changes in behavior, something that has historically been difficult to do.

Experts believe that other new technologies may help in similar ways. Talking digital assistants like Amazon's Alexa, for example, could help children who misuse their pronouns. But even as these ideas spread, researchers warn that they will require rigorous testing before their effects are completely understood.

Catalin Voss started building software for Google Glass in 2013, not long after Google unveiled the computerized eyewear amid much hullabaloo from the national media. An 18-year-old Stanford freshman at the time, Mr. Voss began building an application that could automatically recognize images. Then he thought of his cousin, who had autism.

Growing up, Mr. Voss's cousin practiced recognizing facial expressions while looking into a bathroom mirror. Google Glass, Mr. Voss thought, might improve on this common exercise. Drawing on the latest advances in computer vision, his software could automatically read facial expressions and keep close track of when someone recognized an emotion and when they did not.

''I was trying to build software that could recognize faces,'' Mr. Voss said. ''And I knew that there were people who struggled with that.''

At the time, the brief moment Google Glass spent in the national spotlight was already coming to an end. Google stopped selling the device to consumers amid concerns that its built-in camera would compromise personal privacy.

But Google Glass lived on as something to be used by researchers and businesses, and Mr. Voss, now a Ph.D. student, spent the next several years developing his application with Dennis Wall, a Stanford professor who specializes in autism research, and others at the university.

Their clinical trial, conducted over two years with 71 children, is one of the first of its kind. It spanned everything from severe forms of autism, including children with speech impairments and tactile sensitivities, to much milder forms. Children who used the software in their homes showed a significant gain on the Vineland Adaptive Behavior Scales, a standard tool for tracking the behavior of those on the autism spectrum, Mr. Voss said.

The gain was in line with improvements by children who received therapy in dedicated clinics through more traditional methods. The hope is that Mr. Voss's application and similar methods can help more children in more places, without regular visits to clinics.

''It is a way for families to, on some level, provide their own therapy,'' Mr. Voss said.

Jeffrey Prickett, Esaïe's father, said he had been drawn to the study because he had known it would appeal to his son, who enjoys using iPad apps and watching DVD movies.

''He does fine interacting with people,'' Mr. Prickett said. ''But he does better interacting with technology.''

Mr. Prickett found it hard to judge whether the Google device helped his son recognize emotions, but he saw a marked improvement in Esaïe's ability to make eye contact.

Heather Crowhurst, who lives near Sacramento, said she had experienced something similar with her 8-year-old son, Thomas, who also participated in the trial. But Thomas was not entirely captivated with the digital therapy. ''It was kind of boring,'' he said.

The concern with such studies is that they rely on the observations of parents who are helping their children use the technology, said Catherine Lord, a clinical psychologist at the University of California, Los Angeles, who specializes in the diagnosis and treatment of autism. The parents are aware of the technological intervention, so their observations may not be reliable.

Still, the Stanford team considers its study a first step toward wider use of this and other technologies in autism. It has licensed the technology to Cognoa, a Silicon Valley start-up founded by Dr. Wall. The company hopes to commercialize the method once it receives approval from the Food and Drug Administration, which oversees the use of medical devices in the United States. That may still be years away.

Other companies are taking a different approach. Brain Power, a start-up in Massachusetts that has built similar software for Google Glass, is selling its technology to local schools. The company considers it a teaching tool, not a medical device.

Patrick Daly, the assistant superintendent of the school district in North Reading, Mass., is testing Brain Power's technology after watching its effect on his 9-year-old son, who is on the spectrum. The district intends to test the technology over the next few years.

Previously, the district tried to teach similar skills through iPad computer tablets. Mr. Daly sees Google Glass as a big improvement.

''It can actually maintain eye contact,'' he said. ''They are not looking down while they try to learn an emotion.''

Robokind, a start-up in Dallas, applies the same philosophy to different hardware. The company spent the past several years designing a robot that attempts to teach many of the same skills as technologies built for digital eyewear. Called Milo, the doll-like, two-foot-tall robot mimics basic emotions and tries to make eye contact with students. It also asks questions and tries to engage students in simple conversations.

Robokind has sold hundreds of the robots to schools for testing. Each one costs $12,000, plus more than $3,500 for additional software.

In some ways, such a device is a poor substitute for real human interaction. But the strength of this and other technologies is that they can repeat tasks time and again, without getting tired or bored or angry. They can also measure behavior in precise ways, said Pam Feliciano, the scientific director of the nonprofit Simons Foundation Powering Autism Research.

For these reasons, Ms. Feliciano also sees promise in Amazon's Alexa. Her 14-year-old son is on the spectrum and struggles with his pronouns. He sometimes calls himself ''you,'' not ''I.''

Her task is to correct him each time he makes a mistake. But she's human and gets tired. She does not always remember. A device like Alexa could help, she said, provided that researchers can show it is reliable and effective.

''The technologies are there,'' she said. ''It is just a matter of the right technologists working with the right clinicians.'

15

AVROBIO Announces 87% Substrate Reduction in First Kidney Biopsy and Additional Positive Data from Clinical Trials of AVR-RD-01 Investigational Gene Therapy in Fabry Disease

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jul. 15, 2019-- AVROBIO, Inc. (NASDAQ: AVRO) (the “Company”) today announced the first kidney biopsy result and additional positive data from two ongoing clinical trials of its AVR‑RD‑01 investigational gene therapy in Fabry disease. To date, eight patients have been dosed in the trials – three patients in the Phase 2 FAB-2011 trial and five patients in the Phase 1 FACTs2 trial.

AVR-RD-01 Summary of Interim Clinical Data

The primary efficacy endpoint for the Phase 2 FAB-201 trial is the change from baseline in the average number of Gb3 inclusions per peritubular capillary (PTC)3 as measured in a kidney biopsy one year post-treatment with AVR-RD-01. Gb3, or globotriaosylceramide, is a substrate (or fat) that accumulates in the cells of Fabry patients and can result in damage to multiple organs including the kidneys and heart.

In addition to safety, the FAB-201 and Phase 1 clinical trials are examining a number of secondary efficacy and other endpoints, including biomarkers, such as measurements in the plasma of Gb3 and lyso-Gb3 (the toxic metabolite of Gb3), AGA enzyme activity levels in leukocytes and plasma, vector copy number (VCN), as well as indicators of kidney and cardiac function.

The following is a summary of key observations from the most recent interim clinical data set:

Substantial Gb3 substrate reduction in kidney biopsy. The kidney biopsy for the first treatment-naïve patient dosed in the FAB-201 trial, as reviewed by two independent examiners, showed a reduction from an average of 3.55 Gb3 inclusions per PTC at baseline to an average of 0.47 inclusions per PTC one year after administration of the Company’s AVR-RD-01 investigational gene therapy, representing an 87% reduction. Sustained plasma lyso-Gb3 reductions. The first Phase 2 patient had an 87% reduction in plasma lyso-Gb3 at one year. The first four Phase 1 patients have seen their plasma lyso-Gb3 levels reduced between 33% and 41% versus their ERT pre-treatment levels. In particular, the 41% reduction level has stabilized at more than two years for the first Phase 1 patient. Durability data for AVR-RD-01 continues to show sustained results across multiple parameters. All six patients across the trials for whom data are reported at six months or longer post-treatment with AVR-RD-01 show sustained AGA enzyme activity in leukocytes and plasma and exhibit consistent VCN trends, with VCN levels for the first Phase 1 patient stable at more than two years post-treatment. Kidney and cardiac functions stable. Kidney and cardiac functions, as measured by GFR4 and cardiac MRI Left Ventricular Mass parameters, were stable and in a normal range in the first Phase 2 patient at one year. Phase 1 patients who have discontinued ERT remain off ERT. The two patients in the Phase 1 trial who discontinued ERT post-AVR-RD-01 treatment remain off ERT to date. These patients have now been off ERT for 10 and 9 months, respectively. A third patient is in the process of discontinuing ERT. No unexpected trends or safety events were identified. Serious adverse events (SAEs) and adverse events (AEs) reported were generally consistent with myeloablative conditioning, underlying disease or pre-existing conditions.

“We are excited by the magnitude of the Gb3 reduction observed in the first patient’s kidney biopsy at 12 months. This is the primary efficacy endpoint in FAB-201 and an efficacy endpoint that has previously been utilized by the FDA in evaluating and approving treatment for Fabry disease,” commented Birgitte Volck, MD, PhD, AVROBIO’s President of Research and Development. “Our prior data readouts have shown AVR-RD-01 is associated with reductions of Gb3 and lyso-Gb3 levels in the plasma, and today’s data further support its potential to reduce Gb3 levels in tissue, including in the kidney. We believe the 87% Gb3 clearance in the kidney biopsy may be considered clinically relevant since Gb3 accumulation in organs of Fabry patients is associated with significant morbidity and early mortality.”

Dr. Mark Thomas of the Department of Nephrology at Royal Perth Hospital and Clinical Professor at the University of Western Australia Medical School, the lead investigator for the FAB-201 trial, noted, “I believe today’s data indicate that AVR-RD-01 is substantially reducing the build-up of Gb3 substrate in kidney tissue to potentially effective clearance levels. This, along with the sustained reduction in Gb3 and lyso-Gb3 in plasma observed to date, could translate into substantially improved patient outcomes over the current standard of care.”

Interim clinical data for all eight patients dosed to date in the FAB-201 and Phase 1 clinical trials appear to indicate that the Company’s AVR-RD-01 investigational gene therapy has been generally well tolerated with no unexpected trends or safety events identified. No serious adverse events (SAEs) related to the AVR-RD-01 drug product were reported as of the safety data cut-off dates of July 10, 2019 for the FAB-201 trial and May 24, 2019 for the Phase 1 trial. As of the respective safety data cut-off dates, four SAEs were reported in the FAB-201 trial and two SAEs were reported in the Phase 1 trial and were consistent with expectations for the myeloablative conditioning regimen, stem cell mobilization, underlying Fabry disease, or pre-existing conditions. Low anti-AGA antibody titers have been detected in two patients, one in each of the trials, and the Company believes neither is considered to be of clinical relevance.

“With eight Fabry patients across two trials now treated with our investigational gene therapy, we are extremely pleased with the emerging data set and its support for AVR-RD-01 to potentially address the genetic basis of Fabry disease. As these clinical trials progress, we aim to position AVR‑RD‑01 as a first-line therapy,” said Geoff MacKay, AVROBIO’s President and CEO. “Looking beyond Fabry disease, we remain on track for other important anticipated milestones, including the dosing of the first patients in the Gaucher and cystinosis Phase 1/2 trials in the second half of 2019 as well as the introduction into our clinical programs of our commercial-scale plato platform.”

Enrollment in the FAB-201 clinical trial is ongoing, and further details are available on clinicaltrials.gov.

08

PhoreMost and inStem Enter Structural Biology Alliance; inStem's Centre for Chemical Biology and Therapeutics to work with PhoreMost to progress selected targets

CAMBRIDGE, England--(BUSINESS WIRE)--PhoreMost, the UK-based biopharmaceutical company dedicated to drugging ‘undruggable’ disease targets, today announced it has entered into a structural biology focussed collaboration with the Centre for Chemical Biology and Therapeutics (CCBT), Bangalore, India. CCBT at the Institute for Stem Cell Science and Regenerative Medicine (inStem) is funded by the Department of Biotechnology, Government of India,. The aim of the collaboration is the structural visualisation of novel druggable sites across multiple targets, to rapidly advance new therapies.

PhoreMost’s next-generation phenotypic screening platform SITESEEKER® probes the entire proteome in a live cell environment for novel druggable targets, enabling the systematic unmasking of cryptic druggable sites. PhoreMost has built a pipeline of novel targets and early drug discovery programmes using this platform, and the new collaboration will draw on CCBT’s structural and chemical biology expertise to progress selected targets within PhoreMost’s discovery portfolio.

CCBT is a state-of-the-art multidisciplinary effort, which was formed to pioneer innovative approaches to create chemical tools that modulate novel classes of targets. The Centre integrates biochemistry, genetics and cell biology with structural biology, computational chemistry and synthetic chemistry.

Dr Chris Torrance CEO of PhoreMost, said: “Since its inception, PhoreMost’s model has been to forge a new approach to drug discovery, working collaboratively with partners in order to progress new medicines. We are delighted to announce this alliance with the CCBT and inStem, which represents an important milestone for PhoreMost and has enormous potential to rapidly advance new therapies.”

 

02

mytaxi Becomes FREE NOW; Rebranding of Europe's biggest taxi app also marks a milestone in the transition to a multiservice mobility provider

 HAMBURG, Germany, July 2, 2019, (PR Newswire) -- mytaxi has rebranded to FREE NOW today. Europe's biggest Taxi app has not only changed its branding, but with the new name, it is also set up as a broader mobility provider, and will add more mobility options to the app in future. As a first step, the company has announced it will integrate hive scooters into the app, in the FREE NOW markets, where hive is already active (Portugal, Poland and Austria) within the next few months. Further mobility options will also be implemented in the future.

"This day is a huge milestone for the whole organisation," Eckart Diepenhorst, CEO Europe for FREE NOW, says. "We started the transition to a multiservice mobility provider a while ago by launching the hive scooter brand last year. The rebrand to FREE NOW marks another step towards offering different services within the app. Whilst Taxi will always play a crucial role in our offering, we strongly believe that we will need to have further mobility options in the future. User demands, regulatory framework and the technical options for mobility are constantly changing. In order to maintain and grow our satisfied customer base, we need to develop our proposition in order to fulfill our mission: We want to make mobility available for everyone - independent of age, income and heritage."

FREE NOW is part of the brand family created through the mobility merger of BMW and Daimler. The other verticals within that family are SHARE NOW (Carsharing) REACH NOW (multi-modal), PARK NOW and CHARGE NOW.

"We believe that being part of a global mobility brand family will be a very strong advantage for us, especially when all verticals have finished their rebranding," Thomas Zimmermann, FREE NOW Chief Marketing Officer (CMO), explains. "For us, FREE NOW means freedom of mind. We want to free our customers - both passengers and drivers - from everyday worries and stress. We want our passengers to lean back, enjoy their ride and let us take care of the details of getting them from A to B. We want our drivers to be confident in us  taking care of getting them enough jobs so they can earn their well deserved living," Zimmermann reveals. In order to raise brand awareness FREE NOW is investing a double digit million euro budget into an international brand campaign, which starts today.

Neither passengers nor drivers will have to install a new app. The change from mytaxi to the new FREE NOW app will happen through a regular update, which is available on Google Play Store and the iOS Appstore from today on. Also all accounts and their settings remain unchanged.

June 2019

12

NuCana Receives Orphan Drug Designation from the U.S. Food and Drug Administration for Acelarin® for the Treatment of Biliary Tract Cancer; NuCana Remains on Track to Open Global Phase III Study in 2019

EDINBURGH, United Kingdom, June 12, 2019 (GLOBE NEWSWIRE) -- NuCana plc (NASDAQ: NCNA) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for the Company's investigational drug, Acelarin® (NUC-1031), for the treatment of biliary tract cancer. Acelarin is a new chemical entity and is NuCana's ProTide transformation of gemcitabine.

"We are pleased to have received orphan drug designation from the FDA for Acelarin in biliary tract cancer," said Hugh Griffith, NuCana's Founder and Chief Executive Officer.  "There is a high unmet need for patients suffering from this cancer type.  Our Phase Ib study of Acelarin combined with cisplatin showed an approximate doubling of the response rate expected with the standard of care, gemcitabine plus cisplatin, with several patients achieving significant reductions in their tumor volume as well as further tumor shrinkage over time.  We believe Acelarin represents a potential significant advance in biliary tract cancer and we remain on track to open our global Phase III study in combination with cisplatin as a front-line treatment for patients with advanced biliary tract cancer in 2019."

Orphan drug designation is granted by the FDA to drugs that are defined as those intended for the treatment, prevention or diagnosis of rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug designation provides certain benefits and incentives that may include tax credits towards the cost of clinical trials and prescription drug user fee waivers.

04

Viken Detection Introduces Broadwing-LAD™, a Large-Area Detector Accessory for Its Market-Leading HBI-120 Handheld Backscatter X-Ray Imager

NEWTON, MA., June 4, 2019, (BUSINESS WIRE) -- Viken Detection, pioneer of handheld x-ray imaging and analytical devices, today announced the introduction of its Broadwing-LADTM accessory for the HBI-120 handheld x-ray imager. The detachable, lightweight, large-area detector enhances vehicle inspection capabilities for drug interdiction and enables new applications of the HBI-120, including building searches, tactical raids and counter-surveillance.

The HBI-120 is currently in use by law enforcement agencies around the world and with Customs and Border Protection (CBP) at the U.S. border. The HBI-120 is responsible for significant seizures of cash, drugs and weapons concealed in vehicles. The addition of the Broadwing-LAD accessory provides not only a deeper scan but also a larger view of items being inspected, including vehicles, and even allows authorities to “sweep” rooms to ensure safe entry for officers.

No other technology on the market can provide this breadth of capabilities.

“Our vision is to take Viken technology anywhere there is a public safety problem that technology can solve,” said Viken Detection CEO, Jim Ryan. “The addition of our Broadwing-LAD accessory enables superior threat detection across multiple public safety scenarios. As technology plays an ever more significant role in law enforcement, Viken is at the leading edge, developing new devices that are futureproof, multi-layered and open-sourced to allow them to work together with other solutions and keep the public safe.”

May 2019

16

At Bevi, it's pretty much all watercooler talk; Company's Charlestown offices reflect its mission to promote sustainability

By Andy Rosen May 16, 2019 | The Boston Globe  

Eliza Becton is the cofounder and head of product at Bevi, a Charlestown startup that's producing "smart" watercoolers.

Houseplants abound in the Bevi offices; engineers Matt Zmetra (left) and Mike Peterson tested some new components.

Bevi staffer Megan Camara got a drink from one of the company's machines.

Eliza Becton could be anywhere in Bevi's Charlestown office: at a hot desk near the sales team, downstairs with her design and research-and-development colleagues, or in the workshop looking over the company's latest prototypes for high-tech seltzer and flavored-water dispensers.

But wherever you find Becton, the company's cofounder and head of product, she's likely to have her trusty reusable water bottle in tow. She's had it since the early days of the company — so long that its pink, rubberized jacket has faded to white.

"I'm kind of emotionally attached to this one," she says. "People use their bottles as kind of a representation — they're proud of their bottles."

Of course, most people don't think about drinking water nearly as much as Becton, a Concord native who grew up sailing and has long been horrified by the amount of plastic waste making its way into the world's oceans. She developed the germ of the idea for Beviwhen she was a graduate student at Rhode Island School of Design looking for creative approaches to the problem.

Bevi makes drink machines that dispense glasses of naturally flavored water and allow users to customize the taste and the amount of carbonation. Six years after the company began, its "smart water machines" are in the offices of Google, Netflix, and some of the trendiest offices in Boston's tech scene. One even appeared in an episode of "Silicon Valley," HBO's comic send-up of the industry. Along the way, Becton says, the company has kept 65 million bottles out of the waste stream.

Bevi's offices contain many elements that align with the company's environmental ethos. Becton recently showed the Globe around the firm's multi-floor arrangement in the former Schrafft's Candy Factory near Sullivan Square.

Plants everywhere

Bevi's bright-white color scheme might suggest a sort of clinical sterility, but the surroundings are actually teeming with life. The main entrance is set apart by cubic shelves bearing dozens of potted plants.

Becton says these are among several touches intended to keep the workplace mindful of Bevi's overarching commitment to the environment. The company also has compost bins on site (Becton sometimes brings in her biodegradables from home), and it has named its conference rooms after various ecological settings (Amazon, taiga).

"We try to make sustainability a part of how we do business, from the product all the way to how we are walking the walk," she says. "We want to have reminders of that in what we're doing day-to-day."

The plants are also a labor of love for Bevi's staff. Office manager Jill Rudnicki this spring repotted the plants by hand, and Rudnicki has developed a decorative motif around the flora. She created a large display of hand-cut paper flowers to hang prominently at Bevi's main entrance.

Just a regular old coffee machine

For a company whose product is an upgrade of the traditional water coolerBevi has a back-to-basics approach to coffee. A Mr. Coffee drip coffee maker was brewing a pot on a recent afternoon, in contrast with the barista-run espresso machines that grace other high-tech offices.

"We're minimalists, I would say. We're not one of those startups that just has a ridiculous amount of perks," Becton says. "We spend money where it matters."

But employees do have options at Bevi. There is cold-brew coffee on tap, and in the main office kitchen Bevi has a fancy "Cafection" machine cranking out top-shelf beverages. Becton says that high-end option is mostly for research — though she does cop to enjoying it.

"I like the machine upstairs, honestly," Becton says. "But I also bring my own."

'Roadkill'

It doesn't sound pretty, but this is what Becton and her colleagues call the experimental version of their water cooler, which "lives" down in Bevi's shop.

Unadorned by any of the usual slick finishes and mounted on a utility cart, the Roadkill gives Bevi's designers and engineers a way to try new configurations and features without worrying about looks.

That allows them to experiment with things like pressure, flavor, and even how it feels to hold a glass as the machine fills it with water.

The name is actually a relatively common term in the field of robotics, referring to the stripped-down nature of the design.

"It's like the guts. You don't care about making it pretty. You just care about functionality and usability," she says.

06

First Clarke Freight Farm Harvest in Massachusetts Exemplifies the Importance of Fresh Food and Superior Food Preservation; Clarke, New England's Official Sub-Zero/Wolf/Cove Showroom and Test Kitchen, innovates again by investing in a 40-foot Freight Farm to grow produce year-round in Massachusetts. Clarke's Culinary Team will use the produce for cooking demonstrations, events and daily tastings to exemplify the importance of fresh, healthy food and the fact that Sub-Zero refrigeration provides superior food preservation. Every showroom visitor will enjoy a gift of fresh lettuce and the entire effort is powered by solar atop the Clarke headquarters complex in Milford, MA.

MILFORD, MA., May 6, 2019 (PRNewswire-PRWeb) -- Clarke,New England's Official Sub-Zero/Wolf/Cove Showroom and Test Kitchen, once again exemplifies kitchen industry innovation with its investment in a 40-foot self-contained hydroponic farm to exemplify the importance of fresh food as part of a healthy lifestyle. "Our farm was delivered outside our Milford, Massachusetts showroom eight weeks ago and our farmer Francesca Mazzilli has been planting and tending to our crops inside the corrugated shipping container ever since," said Sean Clarke, president of the family-owned company. "We are really excited about our first harvest happening on May 7th."

Built by Freight Farms, an innovative Boston-based company launched in 2010, Clarke's exciting initiative will allow every showroom visitor to go home with a gift of fresh butter bibb lettuce. The farm will also supply fresh produce (delicately preserved in Sub-Zero refrigerators) to be used by the Clarke Culinary Team for cooking demonstrations, events and daily tastings in all three of its showroom locations (Boston Seaport, Milford, MA and South Norwalk, CT.) In addition, every Clarke employee will enjoy a weekly share of the produce to take home for their own families.

Farmer Francesca's first harvest will include a lettuce mix, mint, parsley, thyme, swiss chard, mixed radishes, red veined sorrel, sorbet mix viola and arugula. She will continue to add new crops to the mix and harvest fresh vegetables every week throughout the year.

"When I learned about Freight Farms, I could think of no better way to exemplify the Sub-Zero and Wolf mission of superior food preservation and preparation than launching a year-round Freight Farm right here," said Clarke. "As New Englanders, we crave fresh food year-round and many of our own employees can't wait to get back into their gardens each year. Now we will have fresh produce all year round to cook with in our showrooms and share with customers and employees."

The arrival of Clarke's Freight Farm launched their "Live Deliciously" initiative to encourage all members of the design community and the homeowners they serve to remember that kitchens are about gathering to enjoy fresh, delicious meals. As the icing on the cake, Clarke is powering the farm with the solar panels that provide electricity for their entire Milford complex. "We achieved net zero electrical consumption in Milford when we installed 2304 solar panels on our Milford headquarters in 2011," said Clarke. "It makes it all the more satisfying that we are now also able to power a hydroponic farm from this source."

April 2019

29

AVROBIO, Inc. Announces FDA Clearance of Investigational New Drug Application for AVR-RD-01 Gene Therapy for the Treatment of Fabry Disease; The plato(TM) platform represents a significant advance towards a commercial-stage gene therapy solution designed to treat thousands of patients; AVROBIO to incorporate U.S. clinical sites into its ongoing global FAB-201 Phase 2 clinical trial in Fabry disease

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apr. 29, 2019--AVROBIO, Inc. (NASDAQ: AVRO) (the "Company"), a Phase 2 clinical-stage gene therapy company, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company's Investigational New Drug (IND) application for AVR-RD-01, its gene therapy candidate for the treatment of Fabry disease. The Company now expects to move forward on two key initiatives in its Fabry clinical program: the incorporation of AVROBIO's plato(TM) platform into its FAB-201 Phase 2 trial, and the dosing of patients at clinical sites in the U.S. The plato platform consists of a state-of-the-art four-plasmid vector system, automation of a closed cell manufacturing process and a conditioning regimen that utilizes therapeutic drug monitoring (TDM).

"We are very pleased that FAB-201 remains on track to expand into sites in the U.S in the second half of 2019," said Geoff MacKay , President and CEO of AVROBIO. "Importantly, we believe this U.S. FDA clearance represents a major milestone as we transition to plato, our optimized commercial-scale platform for our anticipated future worldwide commercialization activities. We have now achieved initial regulatory clearances for clinical trials in Australia, Canada and the U.S. which incorporate our plato platform."

Clinical data presented at WORLDSymposium in February 2019 continued to support the potential of AVR-RD-01 as a gene therapy for Fabry disease. A total of 7 patients have been dosed with AVR-RD-01 across the investigator-sponsored Phase 1 clinical study and FAB-201, the Phase 2 clinical trial of AVR-RD-01 in Fabry disease, which is currently underway in Australia. The Company plans to provide additional preliminary clinical data from these trials this summer.

26

"Mytaxi" to be renamed "Free Now" and offer more services

26 April 2019 | Hamburg News Rebranding in summer - rental cars with driver and e-scooter to complement taxi business The Hamburg-based mytaxi app is to be renamed Free Now from summer 2019, a press release said Thursday (April 25, 2019). Mytaxi is already part of Free Now, the taxi-hailing joint venture launched by BMW and Daimler in February. Europe’s leading taxi app seeks to position itself as a mobility service provider with a broader offer and will include e-scooters in the app pending national approval. A rental car offer with driver can also be booked on the app after the brand change in Germany. E-scooters to complement mobility mix E-scooters are gaining popularity all over Europe and are likely to be registered in Germany soon, according to the German Ministry of Transport. Alexander Mönch, CEO of mytaxi Germany, said: “I am firmly convinced that e-scooters will very quickly become an important part of urban mobility after their nationwide approval. The company already operates its Hive scooters in five European capitals. We will integrate this offer into Free Now.” In mid April, Hive launched a pilot project on the DESY research centre in Hamburg-Bahrenfeld. Fixed-price entry into rental car and taxi sharing Free Now also plans to launch its own fleet of rental cars in association with taxi entrepreneurs. The entry into the rental car business is a strategic step for mytaxi. Mönch noted: “As a company with European roots, we do not want to leave the market to competitors from other continents. We continue to argue in favour of merging taxi and rental cars in a reformed joint trade with the same rules and fair competitive conditions.” At the same time, the modernisation of the taxi industry is crucial to staying competitive. Mytaxi’s taxi sharing service has been available at a fixed price since April 2019. In future, the taximeter will be replaced by a binding, electronic, price-fixing system. Urban mobility "Made in Hamburg" Founded in June 2009 in Hamburg, mytaxi now has 14 million passengers and more than 100,000 registered drivers making it Europe’s leading taxi app. The company employs over 700 people in 100 cities across nine European countries.

25

Stealth BioTherapeutics Completes Enrollment of Pivotal Study in Primary Mitochondrial Myopathy

BOSTONApril 25, 2019 /PRNewswire/ -- Stealth BioTherapeutics (NASDAQ: MITO), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced completion of enrollment for MMPOWER-3 (SPIMM-301), with top-line data expected by year end. MMPOWER-3 is a Phase 3, randomized, double-blind, parallel-group, placebo-controlled study to evaluate the efficacy and safety of daily subcutaneous injections of elamipretide in patients with primary mitochondrial myopathy (PMM) followed by an open-label treatment extension.

"The debilitating fatigue and muscle weakness due to primary mitochondrial myopathy often has a negative impact on the quality of life of affected patients," said Dr. Michio Hirano, professor of neurology at Columbia University Medical Center in New York, who enrolled the most patients across the 28 sites involved in the trial. "I am encouraged by the data demonstrated with elamipretide in prior trials and was pleased to be able to offer our patients the opportunity to participate in this trial."

Approximately 40,000 individuals in the U.S. have been diagnosed with PMM, a rare primary mitochondrial disease. There are currently no therapies approved by the U.S. Food and Drug Administration (FDA) for the treatment of PMM.

"We are grateful to the scientific, advocacy and patient communities, as well as the FDA, for partnering with us in our efforts to reach this important milestone, which we hope will pave the way toward providing potentially transformative therapy for affected individuals," said Stealth Chief Executive Officer Reenie McCarthy. "We are particularly encouraged by the strong patient interest, which enabled us to exceed our enrollment goal."

The MMPOWER-3 study exceeded its expected enrollment with 218 patients out of a targeted 202 patients. The primary endpoints of the six-month, parallel-design study are change in distance walked (meters) as measured by the six-minute walk test, and change in the Total Fatigue score on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA), a patient-reported outcome measure. Secondary endpoints include safety and tolerability, as well as patient- and clinician-reported outcomes. Patients completing the double-blind portion of the study (Part 1) are eligible to continue into an open-label extension (Part 2); 80 of the 85 patients who have completed Part 1 so far are participating in Part 2.

In December 2015, the FDA granted Fast Track designation for elamipretide for the treatment of PMM. In October 2017, the FDA Office of Orphan Products Development granted Orphan Drug Designation for elamipretide for this indication.

 

16

Stealth BioTherapeutics Announces Positive Results for Elamipretide in Barth Syndrome Open-Label Extension Trial

Design and results from TAZPOWER and the TAZPOWER open-label extension were presented as late-breaking abstracts at the 2019 MDA Clinical & Scientific Conference

BOSTONApril 16, 2019 /PRNewswire/ -- Stealth BioTherapeutics (NASDAQ: MITO), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced positive results from the open-label extension of the Phase 2/3 TAZPOWER study of elamipretide, an investigational drug, in patients with genetically confirmed Barth syndrome. Barth syndrome is an ultra-rare mitochondrial disease in which reduced levels of the mitochondrial phospholipid cardiolipin are associated with skeletal muscle weakness, debilitating fatigue, cardiac abnormalities, recurrent infections, delayed growth and reduced life expectancy. The TAZPOWER open-label results, presented at the 2019 MDA Clinical & Scientific Conference, demonstrated potential for elamipretide to improve the relative levels of normal to abnormal cardiolipin which are diagnostic for the disease, as well as measures of exercise performance and patient-reported outcomes.

"These Phase 2/3 results support previous pre-clinical findings suggesting that elamipretide may help improve cardiolipin levels," said Dr. Hilary Vernon, TAZPOWER trial investigator, Associate Professor of Pediatrics at the McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine and Director, Barth Syndrome Clinic at Kennedy Krieger Institute, Baltimore, MD. "Based on the correlation of the cardiolipin ratio with disease severity, these changes in the cardiolipin ratio data may suggest that elamipretide has the potential to modify the course of disease in Barth syndrome rather than merely addressing the symptoms."

TAZPOWER was a Phase 2/3 crossover study of elamipretide in 12 patients with Barth syndrome followed by an open-label extension in which 10 of the 12 patients participated. Although statistical significance was not achieved in the intent-to-treat population during the blinded, placebo-controlled portion of the study, a pre-specified analysis of subjects with relatively higher levels of normal cardiolipin showed improvement on several endpoints.

Today's presentation of the open-label extension data, demonstrating improvements in various endpoints irrespective of baseline cardiolipin levels, suggests that a longer duration of elamipretide therapy is particularly important for patients with lower baseline levels of normal cardiolipin. Overall, a significant (almost 40%) decrease in the average ratio of abnormal to normal cardiolipin was observed from the start of the study to week 12 of the open-label extension.

Additionally, significantly increased exercise performance in the Six Minute Walk Test (6MWT) was observed both in patients with relatively more normal cardiolipin as well as in those with relatively less normal cardiolipin.  Reductions in fatigue, measured through the BarTH Syndrome Symptom Assessment (BTHS-SA) tool, a novel patient-reported outcome assessment measure, were also observed, with greater improvements observed with longer duration of elamipretide treatment. Improvements were also seen across additional secondary and exploratory endpoints including functional assessments and patient-reported outcomes.

Patients reported improvements in various symptoms, including fatigue/energy levels (90% of patients reported improvement), stamina/endurance (90%), muscle strength (80%) and appetite (70%). In addition, most patients reported improvement in daily life activities (90%), physical activities (90%) and quality of life (80%).

"Our patient community is in dire need of a therapy that addresses the complex, multi-system challenges that occur in Barth syndrome," said Emily Milligan, Barth Syndrome Foundation Executive Director. "The positive findings presented today that elamipretide may improve fatigue and overall quality of life for people living with this devastating disease are encouraging that a potential therapy could finally be on the horizon."

In the placebo-controlled and open-label extension portions of the study, most adverse events were mild to moderate in severity. The most commonly reported adverse events included injection site reactions.

"We are encouraged by these signals that elamipretide may benefit individuals affected by the severe cardiolipin deficiency characteristic of Barth syndrome, which we believe to be one of the most challenging mitochondrial diseases for elamipretide due to reduced opportunity for elamipretide to engage with its target, cardiolipin," said Reenie McCarthy, Chief Executive Officer of Stealth BioTherapeutics. "These findings, which align with the improvements in activities of daily living reported by TAZPOWER subjects in our patient and caregiver perception of change video protocols, will inform our upcoming discussions on a regulatory path forward."

The U.S. Food and Drug Administration (FDA) has granted Fast Track and Orphan Drug designations for elamipretide for the treatment of Barth syndrome. Last summer, the Barth Syndrome Foundation (BSF) hosted a patient-focused drug development meeting with the FDA, from which the organization published a Voice of the Patient Report. The BSF recently shared the report with the FDA during a Professional Affairs and Stakeholder Engagement meeting.

10

First ICA Maxi Store in Sweden, One of 84 Grocery Locations, Launches Sale of Onsite-Grown Produce

Freight Farms customer ICA Maxi Högskolan now offers customers leafy greens grown onsite just steps from in-store shelves BOSTONApril 10, 2019 /PRNewswire/ -- Freight Farms is pleased to announce the launch of ICA Maxi Högskolan's own line of produce grown onsite for shoppers. Located in Halmstad Sweden, Freight Farms customer ICA Maxi Högskolan is one of ICA Gruppen's 1,300 grocery stores in the country. This month, the store began harvesting a range of hydroponically-grown greens for shoppers from Freight Farms' flagship container farm, the Leafy Green Machine.

"We're excited to be the first ICA Maxi store to implement an onsite farm," said Rikard Hillarp, owner ICA Maxi Högskolan. "By growing crops just steps from our shelves, we're able to offer our customers what are truly the freshest greens possible."
Freight Farms' containerized farming technology allows ICA Maxi Högskolan to create and maintain the optimal growing conditions to harvest produce year-round using less than 5 gallons of water per day. Beyond the store's initial offering of butterhead lettuce, spinach, and herbs, the farm's integrated IoT data platform, farmhand, will also allow the store to grow non-native crops otherwise unavailable in the region, regardless of seasonal limitations in Sweden's Nordic climate. "Freight Farms' technology is especially helpful in Sweden, where our short growing seasons can limit crop availability throughout the year and increase our reliance on imported produce," added Hillarp. "We're now able to shorten the distance food travels to get to our customers from 2,000 kilometers to just 30 meters." By removing the miles between the food source and consumers, produce maintains nutrient density and stays fresh for far longer, significantly reducing food waste for both retailers and consumers. On March 29, ICA Maxi Högskolan kicked off its launch with a Harvest Festival for customers, selling produce and offering free samples of the newly-harvested greens and a Q&A with store employees Max Rydberg and Douglas Klang, now the newest onsite farmers. "Our team innovated the technology to empower individuals and businesses all over the world to decentralize the food system in meaningful ways specific to their local community or environment," said Freight Farms CEO Brad McNamara. "We're thrilled to work with industry leaders like Rikard Hillarp and retailers like ICA Maxi, who together have the forward-thinking vision and reach to disrupt the grocery industry internationally."

10

Cognoa Licenses Innovative Digital Therapeutic Technology; Technology will be productized into readily accessible digital applications and incorporated into Cognoa's precision health platform

PALO ALTO, Calif., April 10, 2019 (GLOBE NEWSWIRE) -- Cognoa, a company at the forefront of digital behavioral health for children, today announces it has exclusively licensed technology from the Stanford University School of Medicine to accelerate development and worldwide commercialization of a novel digital therapeutic for children with autism spectrum disorder (ASD). Underscoring the need for earlier access to personalized therapies, Cognoa has already received Breakthrough Device designation from the U.S. Food and Drug Administration (FDA) for its first digital therapeutic device using the technology.

Cognoa's ASD digital therapeutic uses the licensed technology to target the core deficit of social-emotional reciprocity in autism. Social-emotional reciprocity is the ability to recognize, interpret and respond to social cues such as facial expressions and emotional states. Study results from a randomized clinical trial, recently published by peer-reviewed medical journal, JAMA Pediatrics, showed children who used the technology on Google Glass tested better for socialization, play and coping skills than those who only received standard of care behavioral therapy for ASD. Cognoa is actively working to further develop and productize the technology for increased accessibility on more broadly available digital platforms.

"Digital therapeutics can extend a therapist's reach into the home, complementing in-person therapies for improved life-long outcomes," said Brent Vaughan, CEO and co-founder of Cognoa, Inc. "We know that many families are facing obstacles to get their children the help they need. This technology accelerates our goal to empower parents with access to evidence-based solutions so they can more directly impact the progress of their children."

Today, most children in the U.S. who receive an autism diagnosis, and subsequent treatments, do so after the age when interventions have the greatest opportunity to impact the life of the child. Cognoa will integrate the licensed technology into its precision health platform for the earlier diagnosis and treatment of pediatric behavioral health conditions. By uniquely combining artificial intelligence machine learning diagnostics and therapeutics within the same platform, Cognoa is working to create a new standard in behavioral healthcare so that children receive an earlier diagnosis and more effective therapies for the greatest potential impact on their lives.

03

Amylyx Pharmaceuticals Announces First Patients Dosed in PEGASUS Phase 2 Trial of AMX0035 in Alzheimer's Disease and Expansion of the Trial

CAMBRIDGE, Massachusetts, April 3 -- Amylyx Pharmaceuticals, Inc., in collaboration with the Alzheimer's Association, the Alzheimer's Drug Discovery Foundation (ADDF) and the Cure Alzheimer's Fund, today announced the dosing of its first patients in a recently expanded Phase 2 clinical trial (PEGASUS) to assess AMX0035 in individuals with Alzheimer's disease. AMX0035 is Amylyx's proprietary two-drug combination therapy in development to prevent nerve cell death and degeneration.

"The urgent need for a new approach to Alzheimer's is clearer now than ever. As this is one of the first major combination therapy clinical trials for Alzheimer's Disease, we're optimistic about AMX0035's potential to slow disease progression in individuals through its novel mechanisms of action," said Steven E. Arnold, M.D., Translational Neurology Head of the Interdisciplinary Brain Center at Massachusetts General Hospital and Harvard Medical School and the study's principal investigator.

Alzheimer's is a complex disease that likely has multiple, interrelated causes. A growing number of experts believe combination therapy-with multiple therapeutics-will be required to effectively treat it.

"Although targeting the classic pathologies of Alzheimer's disease, beta-amyloid and tau, has proven challenging in clinical trials, this research has awarded us with a wealth of knowledge in our understanding of the disease," said Rudolph Tanzi, Ph.D., of Massachusetts General Hospital, chair of the Cure Alzheimer's Fund Research Leadership Group and chair of the Amylyx SAB. "Cure Alzheimer's Fund is proud to take initiative and provide additional funding to expand Amylyx's trial to support the development of novel approaches that patients need," Dr. Tanzi added.

With additional funding from the Cure's Alzheimer's Fund, the PEGASUS trial has doubled its planned enrollment to 100 participants, with 20 patients enrolled and dosed to date. The trial was first supported in 2017 through the Alzheimer's Combination Therapy Opportunities (ACTO) program, a joint research funding initiative created by the Alzheimer's Association and the ADDF to support clinical trials combining multiple treatment approaches.

"There have been trials targeting amyloid, and there have been trials targeting tau, but this is the first trial to target two novel mechanisms in tandem," said Howard Fillit, M.D., the ADDF's founding executive director and chief science officer. "The ADDF was one of the earliest supporters of innovative targets for Alzheimer's, and we believe combination therapies are a critical next step in finding effective treatments for the disease."

"The rationale for combination therapies has already been demonstrated in diseases like HIV/AIDS, cancer and heart disease. We believe this type of approach will also work for Alzheimer's and other dementias and are encouraged by the launch of this combination therapy trial," said Maria C. Carrillo, Ph.D., Alzheimer's Association's chief science officer. "The Alzheimer's Association is honored to be at the forefront of the mission to keep expanding the research pipeline for therapies that attack this complex disease from a variety of angles."

PEGASUS is a 3:2 randomized, double-blind, multi-center, placebo-controlled study evaluating the safety, tolerability and activity of AMX0035 in patients with late mild cognitive impairment or early dementia due to Alzheimer's disease over 24 weeks. The biomarker-focused trial design will assist in demonstrating the effects of AMX0035 and its potential in treating Alzheimer's disease. Study results are expected in 2020.

"We believe AMX0035 has potential across multiple neurodegenerative diseases. We're moving quickly to determine the synergistic efficacy of these two therapies and deeply value the support from the Alzheimer's Drug Discovery Foundation (ADDF), the Alzheimer's Association and the Cure Alzheimer's Fund to expand this unique trial," said Kent Leslie, Amylyx's chief scientific officer.

01

CollegeVine Secures $24 Million In Series B Funding To Continue Its Disruption Of The College Guidance Industry With Affordable And Effective Solutions

CAMBRIDGE, Mass., April 1, 2019 (PR Newswire)   Today, CollegeVine, the leading provider of affordable, data-driven high school guidance and college admissions advising, announced it has secured $24 million in Series B funding from Maywood Street Investments (Maywood"), Fidelity Investments ("Fidelity"), Morningside Technology Ventures ("Morningside"), and University Ventures ("UV"). Using over a million data points to power and propel their technology, CollegeVine has helped over 10,000 students navigate the path into college, making it the largest marketplace for college advisory services in the country.   "The pricing in the college advising industry can get outrageous. Our mission is to bring data tools to families and drive down costs to expand access," said Jon Carson, the CEO of CollegeVine. "We plan to use the Series B investment to expand our data science and engineering teams and to support our ability to bring affordable services to families making one of the most consequential decisions in their lifetime."   CollegeVine's mission is to address the increasingly significant college guidance gap in U.S. public high schools, where the average student typically receives 38 minutes of guidance over four years even as they confront a high degree of information asymmetry in college selection. Approximately ninety-one percent of its customers are public school students and packages start at less than one-thousand dollars. CollegeVine provides them the tools to not only navigate the high school to college journey and but also recommends the highest ROI schools to minimize debt and improve career outcomes.   "We felt CollegeVine was affordable and offered the best value for the investment," said Christine Sloss, the mother of a CollegeVinstudent. "They helped us negotiate the offer from my son's first choice school, and got us a significant boost in aid. CollegeVine was a gift for our family. The amazing advice and emotional support they gave us was worth every penny and more."   "CollegeVine is leading the way in creating an affordable, data-driven approach to decision making for students hoping to get into college," said Daniel Pianko, University Ventures Managing Director.  

March 2019

22

Amylyx Completes Patient Recruitment for Phase 2 Study Testing AMX0035 for ALS

Amylyx has completed patient recruitment for the Phase 2 clinical trial CENTAUR, which addresses the safety and effectiveness of AMX0035 as an oral treatment for amyotrophic lateral sclerosis (ALS).

In 2017, Amylyx launched the CENTAUR study (NCT03127514) at several centers across the U.S. with plans to enroll 132 individuals diagnosed with sporadic or familial ALS. Now, the required number of patients has been reached, completing the recruitment phase.

Given the urgency for patients with ALS, were encouraged to reach this milestone, Justin Klee, president and co-founder of Amylyx told ALS News Today.

AMX0035 is an oral combination therapy made of two small molecules, tauroursodeoxycholic acid (TUDCA) and sodium phenylbutyrate (PB).

The medication was designed to reduce nerve cell death by blocking key cellular pathways implied in cellular stress and death.

In preliminary studies, AMX0035 reduced those signals at two important cell compartments, the mitochondria and the endoplasmic reticulum, both of which are strongly implicated in the death of nerve cells.

Preclinical testing in animal models of ALS, Alzheimers disease, and mitochondrial diseases showed that AMX0035 can effectively inhibit nerve cell death and neurotoxic inflammation.

Each of the agents that make up AMX0035, PB or TUDCA, have demonstrated strong effectiveness in several preclinical models of ALS.

But more recent studies suggest that combining both can improve effectiveness compared with single therapy.

In addition, TUDCA or PB alone have given signs of being safe and well-tolerated in clinical studies with ALS patients.

In 2017, AMX0035 was granted orphan drug status by the U.S. Food and Drug Administration (FDA) as a potential medicine for ALS.

In the CENTAUR trial, patients are randomized to receive either twice-daily oral doses of AMX0035 or a placebo for 24 weeks.

The trials primary goals are to see if therapy with AMX0035 is safe and well-tolerated and able to delay the loss of capacities during the treatment period.

The later will be measured by the ALSFRS-R scale, a doctor-reported instrument for monitoring disability progression in ALS.

Other efficacy measures will be the treatments impact in muscle strength, respiratory function, and biomarkers of ALS, including markers of neuronal death and neuroinflammation.

At the end of the 24-week trial period, patients who wish may move to an open-label extension study and continue to receive treatment.

The study is a collaboration between Amylyx, ALS Finding a Cure Foundation, ALS Association, Northeast ALS Consortium, Massachusetts General Hospital Neurology Clinical Research Institute and the Leandro P. Rizzuto Foundation.

This collaborative effort to assess the impact of AMX0035 on disease progression involves a committed team of individuals who share a strong personal connection to ALS, Sabrina Paganoni, MD, PhD, from Healey Center for ALS at Massachusetts General Hospital and the studys principal investigator said.

We thank the CENTAUR study participants and their families, site investigators and staff who are devoted to pushing ahead with us on this mission. Its exciting to be fully underway as we continue to study AMX0035, she added.

Thousands of people suffer from this disease, so were working as quickly as possible to evaluate AMX0035 as a treatment for ALS, said Joshua Cohen, Amylyxs CEO and co-founder. 2019 Global Data Point.

15

Engineers Making Waves in Aquaculture

by Tim Sprinkle, ASME.org

The human race is staring down a crisis like nothing it has ever faced before. As the population around the world continues to grow, humanity is faced with increasingly short supplies of food.

The United Nations estimates that 815 million out of the 7.6 billion people in the world, or 10 percent, suffered from chronic undernourishment in 2016. While global food production needs to substantially increase to meet this ever-growing demand, we are also running out of places to produce this food. The Earth has already lost a third of its arable land, and climate change will only continue this trend.

The answer to these challenges is being found not on land, but under water. Aquaculture, or fish farming, has become viewed as an inexpensive, efficient way to feed the world. Fish varieties grow quickly to maturity, are very lean, and offer other health benefits, including those from Omega-3 fatty oils. At the same time, properly managed production facilities can limit the farms’ impact on the environment.

Seafood is, in many ways, an ideal food source. It’s healthy, plentiful, and sustainable. And the market knows it. The industry was worth roughly $176 billion in 2017 and is on track to reach $219 billion by 2022. That’s a five percent compounded annual growth rate.

Given this soaring demand, engineers in many fields are currently working to ensure that aquaculture as an industry is as clean and efficient as possible. This includes remote sensing technology, targeted feed additives, and recirculating water systems that can be built and function on dry land. For example, Prospective Research, a startup out of Massachusetts, is leveraging bacteria to create naturally occurring replacements for antibiotics and other pharmaceuticals used in aquaculture. They’re doing this by modulating individual bacteria using signaling molecules that can trick the bacteria into turning on its natural biotics, or defense mechanisms. “Maybe in 10 years this technology will be used in humans and maybe in five years in cattle and poultry to replace antibiotic growth promoters,” said Dakota Hamill, founder and CEO. “But we focused on aquaculture because it was fast growing and is faced with a very large challenge, which is how do you control disease in a population of animals in which governments have completely banned the use of antibiotics?” The company’s solution has been to create what it calls “stim keys,” which are in effect chemical listeners that mimic what bacteria and fungi use to communicate in the soil to detect competitors or threats. In response to that signal, they’ll turn on their biodefense weapons, which are antibiotic, antifungal, and antiparasitic. As many as 70 percent of the drugs we know today come directly from these bacterial sources, which have been evolving these tools for billions of years. Researchers can access the blueprints to as many as 40 different secondary metabolites just from the bacteria that’s found at the bottom of the ocean or in the soil.

14

An Hour of Light and Sound a Day Might Keep Alzheimer’s at Bay

Playing a flashing white light and a trilling sound reversed signs of Alzheimer’s in mice. Researchers are now trying it in humans

By Angus Chen

Scientific American     March 14, 2019

There is no cure for Alzheimer’s disease. Although a few drugs manage temporarily certain cognitive symptoms of the illness, none can stop or meaningfully slow its progression. “We really don’t have much to offer people,” says Shannon Macauley, a neuroscientist at Wake Forest School of Medicine. Virtually all new treatments have failed in clinical trials. But new research is looking beyond drugs to see what relief might come from a simple LED light and a speaker.

Bathing patients in flashing light and pulsing sounds both tuned to a frequency of 40 hertz might reverse key signs of Alzheimer’s in the brain, according to a paper published in Cell on Thursday. “I think it’s an absolutely fascinating paper to be honest,” says Macauley, who was not involved in this work. “It’s a very provocative idea. It’s noninvasive and easy and low cost, potentially, so if it were to come to fruition in humans—that’s fabulous.”

Still, all this is a big if, Macauley acknowledges. The work was done in mice with genetic alterations that doomed them to develop key symptoms and pathology of Alzheimer’s disease. One batch of mice formed neurofibrillary tangles inside their neurons—dysfunctional knots of a protein called tau that can lead to the cell’s death.  Another batch of the mice developed amyloid beta plaques—sticky heaps of protein that dam the flow of communication between neurons. All the mice also had a third hallmark of the disease—irregular brain activity in the gamma range of brain waves that oscillate between 30 and 100 times a second.

In 2015 neuroscientist Li-Huei Tsai, director at The Picower Institute for Learning and Memory at Massachusetts Institute of Technology, was working on an experiment to manipulate that brain activity by flashing a white light at these mice.  Like light strobes, our brains flicker. Brain waves are generated when large groups of neurons oscillate on and off together. Neurons encode our thoughts and actions and senses in this rhythmic electrical flutter. So when Tsai tuned her light to flash 40 times a second, or 40 hertz, and flickered it at the mice, their brains flickered back— generating gamma waves at a corresponding 40 hertz. Then, something unexpected happened.

When Tsai dissected the mice brains afterward, the amount of amyloid plaques and tau tangles in the mice that saw the light had plummeted. “It was the most remarkable thing,” Tsai says. “The light flicker stimulation triggers a tremendous microglia response. These are the brain’s immune cells that clear cell debris and toxic waste including amyloid. They’re impaired in Alzheimer’s disease, but [the light] seems to restore their abilities.”

 This clearing-out process only happened in the visual cortex where the brain processes light information. To get these effects to penetrate deeper into the brain, she added a clicking sound like a dolphin’s chirrup that also had a 40-hertz frequency. When the mice sat in a room with both the flashing light and the droning sound for an hour day, seven days in a row, amyloid plaques and tau tangles began falling in not just the audio and visual cortices but the prefrontal cortex and the hippocampus as well. “This was one of the big jumps in the new paper,” Macauley says. “These are the learning and memory centers of the brain. And there was about a 40 or 50 percent decrease in amyloid and tau levels. It’s an absolutely impressive feat.”

That showed when Tsai put the mice through a set of cognitive tests. In one, where the mice were given a familiar and an unfamiliar object to explore, mice that didn’t get the treatment acted as though they’d never seen the familiar object. “That shows some memory problems,” Tsai says. Mice that saw the light and heard the sound spent about two thirds of the time that untreated mice did examining the familiar object. “It was unbelievable,” Tsai says. “This is the first time we’ve seen that this noninvasive stimulation can improve cognitive function. It’s not a drug or an antibody or anything, it’s just light and sound.”

One possible explanation for this is brains with Alzheimer’s have irregular, often hyperactive, neurons, says Jorge Palop, a neurologist at the University of California, San Francisco, who did not work on the study. By providing the brains with a steady and regular beat, the repeating light and sound might work as a kind of metronome for brain activity. “This could be like resetting the mice every day and correcting some of this abnormal activity that they have,” he says. “Then downstream of that are all these beneficial effects.”

All of this is still at the level of speculation. Researchers simply do not know why these brain waves, specifically ones rising from light and sound stimulation at 40 hertz and no other frequencies, can lead to a reversal of Alzheimer’s disease symptoms. “That’s a mystery,” says Terrence Town, a neuroscientist, at the University of Southern California who was not involved with the work. It’s also not clear if these beneficial effects would appear or if 40 hertz is the “magic” frequency in humans, he says.

Tsai is already working on answering those questions. In human studies underway at Cognito Therapeutics, a start-up she founded with her colleague Ed Boyden, she says light and sound seem to increase gamma waves in healthy participants without negative side effects. “Nobody gets sick or even complains about it,” Tsai says. “But to see a [therapeutic] effect in humans, you’ll have to wait a long time. If this approach has an impact, the experiment could easily take five years to have some conclusive answer.”

14

Flashing Lights and Sounds Improve Memory and Learning Skills in Mice

Researchers hope the techniques can be applied to help people with Alzheimerʼs.

By Pam Belluck The New York Times     March 14, 2019 Could people’s eyes and ears help fix the damage Alzheimer’s disease does to the brain? Just by looking at flashing light and listening to flickering sound? A new study led by a prominent M.I.T. neuroscientist offers tantalizing promise. It found that when mice engineered to exhibit Alzheimer’s-like qualities were exposed to strobe lights and clicking sounds, important brain functions improved and toxic levels of Alzheimer’s-related proteins diminished. What’s more, the rapid-fire soundtrack appeared to make mice better at cognitive and memory skills, like navigating mazes and recognizing objects. Of course, mice are not people. And many drugs that have helped Alzheimer’s-engineered mice haven’t done much for people with Alzheimer’s, which affects 44 million people worldwide, including 5.5 million Americans. Also, because the technique didn’t have long-lasting effects — results faded about a week after the sensory stimulation was stopped — any therapy developed from the research might have to be repeated regularly. Still, seeing that a noninvasive daily dose of light and sound could have such significant effects in mice give some experts reason for optimism. “It’s exciting, I think,” said Dr. Lennart Mucke, director of the Gladstone Institute of Neurological Disease, who was not involved in the study. “Reading the paper made me quite enthusiastic about seeing this move forward into some well-crafted clinical trials.” The experiments were led by Li-Huei Tsai, director of MIT’s Picower Institute for Learning and Memory. She and her colleagues showed that light and sound delivered to mice at a certain frequency — 40 hertz or 40 flashes or clicks per second — appears to synchronize the rhythm of the brain’s gamma waves, which is disrupted in patients with Alzheimer’s. Gamma waves are among several types of electrical brain waves believed to be involved in concentration, sleep, perception and movement. Somehow — neither Dr. Tsai nor outside experts are quite sure how — 40 hertz produces a gamma-wave oscillation that appears to increase activation of cells called microglia, which perform trash-clearing and immune-regulating functions. The microglia became more efficient at chewing up the amyloid protein that forms toxic plaques in Alzheimer’s. Another Alzheimer’s-related protein, tau, which forms tangles, also decreased. And in the sound experiments, brain blood vessels also worked better, further helping clear harmful proteins.  Especially striking was that these effects occurred in brain areas active in memory formation, planning and decision-making, and that the mice became better at learning and remembering. “The effects on cognitive function are pretty big,” said Dr. Walter Koroshetz, director of the National Institute of Neurological Disorders and Stroke, which funds some of Dr. Tsai’s work. He said the results of the study, published Thursday in the journal Cell, are “definitely something that I don’t think anybody could have predicted.” Enhancing or regulating electrical brain activity through techniques like surgically implanted electrodes is used to treat some other conditions, like Parkinson’s and obsessive-compulsive disorder. And previous research has shown that the activity of gamma waves, the highest frequency waves ranging from 25 to 140 hertz, decreases in the brains of patients with Alzheimer’s. Intrigued, Dr. Tsai began experimenting with light, and in 2016, she and colleagues showed, after trying different frequencies, that light flickering at 40 hertz, beamed at mice an hour daily for a week, decreased amyloid and tau and revved up microglia in the brain’s visual cortex. Aiming to reach other brain areas, she tried sound, settling on clicks because “your brain seems to be able to perceive clicks more than a tone,” she said. Her team found that 40 hertz clicks, broadcast from speakers over mouse enclosures, produced the same brain changes in the auditory cortex and the nearby hippocampus, an area active in forming memories that is damaged early in Alzheimer’s. The mice also performed better at maze navigating and recognizing objects they had seen before. Light and sound combined magnified the brain effects and extended them to the prefrontal cortex, a key area for planning and executing tasks. “It’s stunning that the intervention had beneficial effects on so many different aspects of Alzheimer-like pathology,” said Dr. Mucke, who is also a professor of neurology and neuroscience at University of California San Francisco. “On the other hand, it shouldn’t be surprising that the brain is influenced by outside stimuli because what it was designed for was to adapt to a changing environment.” The results also dovetail with findings by Dr. Mucke and his colleagues, who have genetically altered brain cells called interneurons, which he likened to conductors of the brain’s orchestra.  The altered interneurons enhanced gamma rhythm activity, generating cognitive improvement in mice. “When there isn’t proper brain rhythm, there is disharmony and everyone is sort of playing when they want to, a little like the tuning up of an orchestra,” he said. His colleagues are also developing a drug that would have similar effects. So there might be several ways to enhance gamma rhythms, he said. Because the brain changes subsided somewhat after a week without the light or sound treatment, experts said it’s likely that people would need such stimulation consistently, essentially a sensory version of a daily pill to control a chronic condition. Dr. Tsai’s team has tested light and sound on healthy people, using a four-by-three-foot light panel and high-quality stereo speakers, “so the sound is more tolerable to humans, because it’s not melody, it’s clicks,” she said. Electroencephalogram measurements show the desired gamma-wave effect, she said, and “nobody complains about any discomfort or headache or anything.” They will soon start testing on people with mild Alzheimer’s. Dr. Tsai and a co-author, Edward Boyden, co-director of the M.I.T. Center for Neurobiological Engineering, have also co-founded a company, Cognito Therapeutics, which is testing a goggles-like light-and-sound device on Alzheimer’s patients, she said. Dr. Tsai said the company is not involved in her team’s academic research, which was funded by several foundations and the National Institutes of Health. Experts cautioned that people should wait for clinical trial results and shouldn’t suddenly start illuminating their homes with disco strobes or pipe clicking sounds through their earbuds. Still, said Dr. Koroshetz, sensory treatment is likely to be safe for most people. “Can’t think of any harm that can come out of this one,” he said. And because Alzheimer’s is so devastating, he added: “I think people would participate in studies, even if they require flashing lights for an hour and listening to a very quick drummer.”

12

Desora Launches Cinder Countertop Smart Grill to Help Consumers Cook Food Perfectly and Effortlessly

Officially available for purchase, Desora reintroduces the crowdfunded countertop grill featuring next-gen technology

CAMBRIDGE, Mass.March 12, 2019 /PRNewswire/ -- Desora, the leading food and grilling technology company, announced today the launch of Cinder, the world's first countertop grill with 1° Precision that cooks like magic. Now available for purchase online in the United States, Cinder Grill, powered by Desora, combines waterless sous vide cooking with the searing capabilities of an outdoor grill to create restaurant-worthy meals without ever overcooking food again.

Cinder Grill offers exact temperature control by simply selecting a temperature for the food or by pairing Cinder with its mobile app. The app automatically reads the thickness of food and, through "Temp-Sense," its proprietary algorithm, determines the temperature. It then cooks to that temperature and alerts via mobile when completed, holding food perfectly until users are ready to eat.

"We are so pleased to announce retail availability for Cinder Grill," said Michel Maalouly, Co-Founder and CEO of Desora. "Cinder Grill, along with our other Desora grilling products, provides effortless precision grilling for which home cooks don't need extensive culinary backgrounds - incredible food is now accessible to all."

Acquired in November 2018, Cinder launches as part of Desora's family of cooking and grilling products, including the iKamand, a smart grill controller, and ProJoe, the high-caliber grill powered by Desora.

Cinder Grill is now available for purchase at www.cindergrill.com for $429.00.

All consumers who ordered Cinder Grill through the previous crowdfunding campaign will be contacted and their order will be fulfilled.

11

Liquidia’s LIQ861 Meets Primary Endpoint in Pivotal Phase 3 INSPIRE Study in Patients with Pulmonary Arterial Hypertension

  • LIQ861 was well-tolerated in PAH patients at two months of treatment
  • INSPIRE enrollment complete, including PK sub-study
  • Anticipate submitting NDA for LIQ861 to the FDA in late 2019

RESEARCH TRIANGLE PARK, N.C., March 11, 2019 (GLOBE NEWSWIRE) -- Liquidia Technologies, Inc. (Nasdaq: LQDA) (“Liquidia”), a late-stage clinical biopharmaceutical company focused on the development and commercialization of human therapeutics using its proprietary PRINT® technology, today announced top-line results of its pivotal Phase 3 INSPIRE study in patients with pulmonary arterial hypertension (“PAH”) treated with LIQ861, the first inhaled dry powder formulation of treprostinil. Initial analysis indicates the study has met its primary endpoint of safety and tolerability of LIQ861 at the two-month timepoint.

Nicholas Hill, MD, Chief Pulmonary, Critical Care & Sleep Division and Professor of Medicine at Tufts University School of Medicine and INSPIRE Principal Investigator, stated: “The top-line analysis of LIQ861 from the INSPIRE study is highly encouraging for physicians and patients. LIQ861 was safely titrated to therapeutic levels across a wide range of inhaled doses and was very well tolerated. This means that we are moving closer to having an inhaled therapy available for PAH that is much more convenient than previous ones. Most patients tolerated relatively high doses of treprostinil, raising the possibility that the PRINT technology, by virtue of its ability to make microscopic particles of uniform size, could improve distribution of drug to the lung, enhancing therapeutic effect.”  

LIQ861 was observed to be well-tolerated in 109 patients, with 101 patients (93%) completing at least two months of treatment. During the two-month period, LIQ861 was evaluated at doses up to 150 mcg capsule strength with no study-drug related serious adverse events observed. Reported treatment-emergent adverse events (“TEAEs”) were mostly mild to moderate in nature. The most common TEAEs reported with LIQ861 in ≥4% of PAH patients were cough (33%), headache (18%), throat irritation (14%), dizziness (10%), diarrhea (8%), oropharyngeal pain (6%), nausea (6%) dyspnea (6%), flushing (6%) and chest discomfort (5%). These observations are consistent with the safety data at the two-week timepoint reported on January 7, 2019. Of the TEAEs observed, most were reported during the first two weeks of initial exposure and occurred in patients previously naïve to prostacyclin-based therapy in which LIQ861 was added to oral therapy.

Neal Fowler, Chief Executive Officer of Liquidia, commented: “We are extremely grateful to the patients participating in the clinical trial and for the effort and speed with which our investigators completed enrollment. We believe the commitment to this study signals an increasing need for safe, more convenient inhaled treatment options. We are preparing the new drug application submission, while collecting additional longitudinal data on the benefits from LIQ861.”

In addition to meeting the primary endpoint, the one-directional crossover sub-study to compare bioavailability and pharmacokinetics of treprostinil as the patients transition from Tyvaso to LIQ861 has been fully enrolled. Liquidia expects to report its pharmacokinetics results in the second quarter of 2019 and plans to provide more detailed clinical results through scientific disclosures at upcoming congresses and in peer-reviewed publications.

About LIQ861

LIQ861 is an inhaled dry powder formulation of treprostinil designed using Liquidia’s PRINT technology to enhance deep-lung delivery using a convenient, palm-sized, disposable dry powder inhaler (“DPI”) for the treatment of PAH. Liquidia believes LIQ861 can overcome the limitations of current inhaled therapies and has the potential to maximize the therapeutic benefits of treprostinil in treating PAH by safely delivering higher doses into the lungs.

About INSPIRE Clinical Trial

Liquidia’s pivotal open-label Phase 3 clinical trial, known as INSPIRE, or Investigation of the Safety and Pharmacology of Dry Powder Inhalation of Treprostinil,  is designed to evaluate patients who have either been under stable treatment with nebulizer-delivered treprostinil for at least three months and are transitioned to LIQ861 under the protocol or patients who have been on stable treatment with no more than two non-prostacyclin oral PAH therapies for at least three months and have their treatment regimen supplemented with LIQ861 under the protocol. The primary objective of the INSPIRE study is to evaluate the long-term safety and tolerability of LIQ861. For more information, please visit https://clinicaltrials.gov/ct2/show/NCT03399604.

01

MyTaxi hailed a success; 400 CAB DRIVERS SIGN UP TO APP IN ITS FIRST YEAR IN CITY

by Phoebe Ram, Nottingham Post

A taxi booking app has celebrated its first year of business in Nottingham, and cabbies have managed to rack up some impressive mileage.

MyTaxi was founded in 2009 and launched in Nottingham on March 1, 2018. Since then, the drivers who have signed up to the company have collectively travelled over 430,000 miles - a distance equivalent to driving 17 times around the earth.

Nottingham was the first location for MyTaxi to launch the app outside of London, and is the most successful city in terms of business growth, after the capital.

The company now has almost 400 licensed taxi drivers in Nottingham, with over 41,000 passengers using the service, which is similar to Uber.

The launch last year, in partnership with Nottingham City Council, has helped provide the city with skilled and knowledgeable drivers and a guarantee of safe, reliable, convenient and cost-effective travel for residents.

The business now has a central role in Nottingham's transport system.

David Savage, general manager at MyTaxi, said: "We're really pleased with how well the business is doing in Nottingham.  It's great to see the results after a year in the city, with almost 400 drivers now signed up to the app.  We've really enjoyed working with the local council on this partnership and getting to know some of the drivers in the city who are using the app.  Drivers using mytaxi in the city see, on average, four extra jobs per day, or almost 30 per week, so the app really is benefiting them.  Passengers are also enjoying using the app to get them around safely."

Kaleem Ashraf, a taxi driver from Nottingham, added: "Using the MyTaxi app has really improved my work life and I haven't looked back. It has enabled me to become more flexible with my jobs and has given me extra work when I need it."

Amer Alam, another taxi driver from Nottingham, said: "I recommend MyTaxi to all of my friends who are cabbies.  The app gives you so many more extra jobs a week."

Portfolio holder for community protection, Councillor Toby Neal, said: "We were very pleased to partner with MyTaxi a year ago to help improve the experience for passengers using hackney cabs in Nottingham, the first city outside London to do so.

"Taxis are an important part of the transport options available in Nottingham and MyTaxi is one part of a wider drive to raise the quality of local hackney services, with cleaner, modern vehicles being added all the time, and drivers being held to higher standards. “It’s brilliant, and no great surprise, that people have taken to the app in such large numbers in Nottingham. "With other apps becoming a popular way of booking taxis, it makes complete sense to have something similar for hackneys so it’s as easy and appealing as possible.”

February 2019

26

CollegeVine Closes 2018 as the Largest Marketplace for College Advisory Services in the U.S.

Company Marks Milestone of Helping Over 10,000 Students Navigate the High School to College Journey

CAMBRIDGE, Mass.Feb. 26, 2019 /PRNewswire/ -- Today, CollegeVine, the leading provider of data-driven high school guidance and college admissions advising, announced a major milestone reached in 2018, making it the largest marketplace for college advisory services in the country. The company has now helped over 10,000 students navigate the path into the college and has over a million data points to power and propel their technology forward in 2019.

"CollegeVine is addressing the increasingly significant college guidance gap in U.S. public high schools where the average student typically receives 38 minutes of guidance over four years," said Jon Carson, CollegeVine CEO. "That alarming statistic, coupled with the fact that this is the one of the biggest financial decisions most families make, allows CollegeVine to provide students with the tools to navigate the high school to college journey. In addition, the technology we released in 2018 also helps them determine schools with the best return on investment for each families' unique needs."

This year, counselors in CollegeVine's ecosystem used technology powered by more than a million data points from past students, resulting in:

  • Unparalleled admissions results: 74% of CollegeVine were students accepted to one or more of their top three reach schools. Additionally, CollegeVine students experienced a 3x higher likelihood of getting accepted to a top school relative to their peers.
  • Increased Early Action/Early Decision Acceptance: CollegeVine EA/ED acceptance rate was 3.5 times higher than the market rate for top 15 schools; and 2.5 times higher than the top 30 schools.
  • A record amount of scholarship funding for clients for a second straight year: CollegeVine clients received an average scholarship award of roughly $83,000 during the 2017-2018 admissions season -- an increase of $15,000 year over year.
  • Recognition as leaders in the college admissions space: Data and insights recognized in top-tier media including Business InsiderCBSForbesFox News and NBC.
  • The launch of CollegeVine Applications™ Platform: CollegeVine released its end-to-end college advising platform including data-driven school list generation, deadline management, and predictive career ROI tools.
  • Expansion of HQ staff and management team: Added 35 new employees at its Cambridge headquarters and strengthened the management team by adding Scott Powers as Chief Financial Officer and Rob Merklinger as Senior Vice President of Enrollment.
  • Record customer satisfaction: 91% of families' say they are "extremely satisfied" with CollegeVine's admissions services; 89% are "extremely satisfied" with their re-launched SAT advising platform.

"Getting help from CollegeVine to get us organized and know what we needed to have before we applied was essential," said Bety Gegundez, the mother of a CollegeVine student.

"If we approached it on our own, we probably would have missed two or three schools we never would have thought about," Christopher Arnold, the father of a CollegeVine student.

"In 2019, the growth in our counseling ecosystem will fuel more data insights, higher quality counseling, and improved student outcomes," said Carson. "Simultaneously we will be expanding our offerings to younger students with the launch of some free tools to get families started in the right direction in this process, as early as possible."

About CollegeVine 
CollegeVine is a technology platform that enables high school families to navigate the college admissions process using data. With software powered by millions of past student data points, CollegeVine intelligently analyzes colleges based on students' odds of acceptance and recommends the highest ROI schools to minimize debt and improve career outcomes. Families can also choose to receive personalized guidance from an ecosystem of hundreds of expert admissions consultants recruited from the nation's top universities. Consultants use CollegeVine's guidance technology to triple their clients' odds of acceptance and increase their average scholarship awards by $25,000. With a data-driven model that enables affordable, high quality advising to families, CollegeVine has helped over 10,000 students navigate the path to college. For more information, visit www.collegevine.com.

26

How Freight Farms Plans To Grow Sales (And More Veggies) With Its Next-Gen Farm In A Box

Robin D. Schatz, Contributor February 26, 2019    www.forbes.com

Almost a decade ago, the cofounders of Boston-based Freight Farms pioneered the idea of outfitting used shipping containers with everything a farmer needs to grow greens and herbs in a pesticide-free, climate-controlled  environment  without soil. These hydroponic vertical farms, which grow plants in nutrient-enriched water, are turnkey operations that enable a farmer to get up and running in a matter of days. And the whole thing takes up just 320 square feet.

Cofounders Jon Friedman and Brad McNamara formed their company in 2010, raised their first funds on Kickstarter in 2011 and introduced their Leafy Green Machine a year later. Freight Farms has since raised about $12 million from VC firms. To date, it has sold more than 250 of those shipping-container farms, which use off-the shelf parts and their own proprietary software, in more than 15 countries.

After about four years of working with that model and pushing it as far as we could go, we realized we needed to make a more quantum leap, said Friedman, who is chief operating officer.

Freight Farms says its new LED lighting system produces higher yields and heavier plants

Today Freight Farms will announce its next-gen product, called  the Greenery. Instead of using recycled shipping containers, the units are built from the ground up for Freight Farms with all new components. Growing racks can now be configured to accommodate bigger plants, such as tomatoes. Lighting has improved too. Its energy-efficient LED arrays, with three times the power of the old ones, result in heavier plants and greater yields, according to the company.

Water and energy usage are also more efficient than the old design. The farm's new climate-control system condenses and recycles ambient moisture. The company said it offers 70% more growing space, among other features. Its IoT-connected sensors continuously send the farmer data on temperature,  lighting, moisture content, CO2 levels and other environmental variables to allow them to monitor and adjust conditions remotely from a smart phone.

I first wrote about Freight Farms in December 2015, when revenue was about $3 million and the company had about 50 customers. Granted, the business is still small, but it s growing steadily and adapting, much as plants do, to a changing environment. The company declined to disclose its revenue this time around, citing intense industry competition. Indeed, a quick Google search shows they aren't alone anymore in the shipping-container farm business. Competitors include the   CropBox and Growtainer. Customers might also opt for other types of vertical, hydroponic systems that don't use a shipping container.

In 2015, Leafy Green Machines had a base price of $76,000. The new Greenery costs $104,000.

Some of the most enthusiastic customers of the freight-container farms are big corporations, who see it as an extension of health and wellness programs or social action initiatives. You can find a Leafy Green Machine on the Google campus in Mountain View, Calif., where it's used to grow greens and edible flowers. In Detroit, Ford Motor Company Fund, the automaker's philanthropic arm, also has a freight farm, that it's operating in partnership with a nonprofit to help supply its community kitchen with fresh produce and provide job training. Everlane, the clothing company, uses its shipping-container farms  in Vietnam to provide food for its workers.

Academic institutions find freight farming particularly appealing, said McNamara, who is CEO. We're seeing more and more of the education segment who want onsite local high quality produce and want to connect students and employees to where their food is coming from.

The University of Georgia has just ordered two Greenery farms to help supply their dining halls with local greens more efficiently and sustainably while educating students about where their food comes from, the school told me in an e-mail.

Of course, not every university or corporation wants to actually do the work of farming, the cofounders realized. So, in late 2018, they launched  Grown by Freight Farms,  where customers sign up for a service contract. We provide them with a farm and a farmer, said McNamara, who is CEO.

Small farmers are also an important market for Freight Farms. In Guam, a farmer uses his Leafy Green Machine to supply Wendy's with lettuce. In North Carolina, Heather David and John Peters, who sell pasture-raised meat to chefs from their WhyNot Farms with two locations in North Carolina and a new one in Tennessee, are among the first in line for a Greenery. They expect to receive delivery in May.

We were looking to diversify our business in a way that would be less labor intensive, and we wanted something that would weigh less than cows or pigs, said Davis. We re middle-aged and felt it would be more sustainable for us to be raising vegetables.

Davis, president and chief investment officer at Nuveen Private Markets in Charlotte, N.C., moonlights as her husband's farmhand on weekends and she happens to have a local vertical, hydroponic farming operation in her portfolio. There were plenty of options for Davis to consider for their own hydroponic system, which they hope to power mostly with solar energy.

What appealed to me about Freight Farms is that they are very focused on the sustainability aspect and conservation of water, she said. It s in a compact unit, and they just drop it on your site.

Davis also liked the fact that the company provides intensive farmer support and education.

It s not clear to me yet the extent of what we ll be able to grow in this, she said. I'll grow what anybody will buy as long as it's not a total pain in the neck and I can make a profit.

19

Apnimed Listed a New Clinical Trial of AD036 in Obstructive Sleep Apnea

Contify Life Science News

Feb. 19 -- Apnimed has listed an interventional clinical trial, Phase 2, for placebo-controlled, parallel group dose-finding study to evaluate the efficacy and safety of three dose levels of AD036 in adults with obstructive sleep apnea. The trial's principal investigators are Mark Gotfried, MD, Paul E Wylie, MD, Daniel Norman, MD, Helene Emsellem, MD, James P Maynard, MD and James Andry, MD.

The trial for a sample of 140 participants from USA is currently open for recruiting.

The study is being conducted for the following purpose:

This is a randomized, double blind, placebo-controlled, repeat-dose, parallel arm, outpatient and inpatient phase 2 clinical study to examine the efficacy and safety of three dose levels of AD036 versus placebo in patients with obstructive sleep apnea.

The full document can be viewed at https://clinicaltrials.gov/ct2/show/NCT03845023?recrs=abdfm&type;=Intr&cntry;=US&phase;=0123&sfpd;_s=02%2F19%2F2019&sfpd;_e=02%2F19%2F2019&rank;=10.

Source: ClinicalTrials.gov

14

Stealth BioTherapeutics Announces Pricing of Initial Public Offering

BOSTON – February 14, 2019 – Stealth BioTherapeutics (Stealth), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced the pricing of its initial public offering of 6,500,000 American Depositary Shares (“ADSs”), each representing 12 ordinary shares of Stealth, at an initial public offering price of $12.00 per ADS. The total gross proceeds of the offering, before deducting underwriting discounts and commissions and other offering expenses payable by Stealth, are expected to be $78.0 million.

In addition, Stealth has granted the underwriters a 30-day option to purchase up to an additional 975,000 ADSs on the same terms and conditions. The offering is expected to close on February 20, 2019, subject to satisfaction of customary closing conditions. Stealth’s ADSs have been approved for listing on The Nasdaq Global Market and are expected to begin trading on February 15, 2019 under the ticker symbol “MITO.” Jefferies LLC, Evercore Group L.L.C. and BMO Capital Markets Corp. are acting as joint book-running managers for the offering. Nomura Securities International, Inc. is acting as the lead manager for the offering. A registration statement relating to these securities has been filed with the Securities and Exchange Commission and was declared effective on February 14, 2019. Copies of the registration statement can be accessed by visiting the SEC’s website at www.sec.gov. The offering is being made only by means of a prospectus. When available, copies of the final prospectus may be obtained from Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by telephone at 877-821-7388 or by email at prospectus_department@jefferies.com; Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, New York, NY 10055 or by telephone at (888) 474-0200 or by email at ecm.prospectus@evercore.com; or BMO Capital Markets Corp., Attention: Equity Syndicate Department, 3 Times Square, 25th Floor, New York, NY 10036, by telephone at (800) 414-3627 or by e-mail at bmoprospectus@bmo.com.

12

CellCentric launches clinical programme in blood cancers led by Prof Andy Davies

CellCentric is initiating a clinical study of CCS1477, a first-in-class p300/CBP inhibitor, in haematological malignancies. CCS1477 is already in Phase I studies for late stage prostate cancer. The clinical programme is being extended to include acute myeloid leukaemia (AML), multiple myeloma (MM) and non-Hodgkin lymphoma (NHL) following new pre-clinical data showing profound anti-tumour effects of CCS1477 in models of these haematological malignancies. The Chief Investigator for the new Phase I/II clinical study will be Prof Andy Davies of University Hospital Southampton. The trial is expected to open for recruitment in Q2 2019.

CCS1477 is a novel, highly potent and specific inhibitor of the common bromodomain of two critical cancer regulatory proteins, p300 and CBP. The drug is formulated as a capsule to be taken orally. Inhibiting p300/CBP in blood cancers affects the IRF4-Myc pathway and can have a significant impact on tumour growth in pre-clinical models. CellCentric is now transitioning the product into haematological cancer clinical trials.

Andy Davies is Professor of Haematological Oncology and Consultant in Medical Oncology within The Faculty of Medicine at the University of Southampton. He has specific interests in the clinical course of malignant lymphoma, the investigation of novel therapeutic approaches and biomarker identification. He has extensive experience in early phase trials, including leading several first in man lymphoma studies. He is Chair of the UK National Cancer Research Institute High Grade Lymphoma Study Group and Director of Cancer Research UK/National Institute of Health Southampton Experimental Cancer Medicine Centre.

CCS1477 is currently enrolling patients in a Phase I/II clinical trial for late stage prostate cancer, with Prof Johann de Bono of the Royal Marsden Hospital, as the Chief Investigator. The drug is positioned to be used after or in combination with second generation anti-hormonal drugs such as abiraterone, enzalutamide and apalutamide. The mode of action of CCS1477 addresses the primary resistance mechanisms to these current agents, whether inherent or acquired.

Dr Nigel Brooks, CellCentric’s Director of Translational Research, commented: “At CellCentric and with our network of scientific collaborators, we have generated new and compelling pre-clinical data showing profound anti-tumour effects of CCS1477 in models of AML, MM and NHL. We are excited to now translate these findings into the clinic. We look forward to working with Prof Andy Davies and other clinical experts as we develop this novel approach for the benefit of patients with haematological cancers.”

The clinical need and potential market for a new agent for late blood cancers remains significant. Particularly in late stage relapsed refractory multiple myeloma the market could be in the tens of thousands of patients to treat each year.

CellCentric will be presenting CCS1477 in the ‘New Drugs on the Horizon’ session at AACR in Atlanta, on the 31st March 2019.

07

Cognoa scores FDA breakthrough designations

by Liz Hollis, Staff Writer, BioWorld MedTech

Palo Alto, Calif.-based Cognoa Inc. received breakthrough device designations from the U.S. FDA for its digital diagnostic and digital therapeutic devices for autism. Cognoa's digital precision health platform weds machine learning and predictive analytics to parental inputs and diagnostic data and responses to therapeutics to create more personalized care. The single platform aims to help clinicians to come to accurate diagnostic and therapeutic decisions faster and modify treatments.

About 1 in 59 children has been identified with autism spectrum disorder, according to estimates from CDC's Autism and Developmental Disabilities Monitoring Network. The company noted that the average age of autism spectrum disorder diagnosis has remained unchanged for more than 15 years, standing at more than 4 years of age.

"We believe [artificial intelligence]-based precision health can empower parents and their pediatricians to act on early concerns that are highly predictive of developmental delays, like autism, with potentially life-changing results for children and their families," Brent Vaughan, CEO and co-founder of Cognoa, explained. "We are thankful that the FDA recognizes the critical need for innovative solutions to help address these challenges. We look forward to working closely with them to further our clinical studies and support our development."

The company said its diagnostic device is intended to help health care professionals provide a diagnosis of autism spectrum disorder in children between the ages of 18 months and 72 months who are at risk for developmental delays. That is important because a diagnosis often comes later, after the window of brain plasticity that is crucial to maximizing treatment outcomes.

With its therapeutic device, the company will aim to help boost socialization and responsiveness of children with autism spectrum disorder. The device will be intended for use outside the clinician's office to supplement existing therapies, giving patients timely and convenient access to care, with the potential of reducing wait times.

Earlier diagnosis

"Cognoa's diagnostic device is unique in that we expect it to be the first medical device intended for use by pediatricians and primary care physicians to enable a diagnosis up to two-and-a-half years sooner than the current standard of care," Vaughan told BioWorld MedTech. "The Cognoa device is also unique in that it engages both the parent and their child's trusted doctor to enable earlier diagnosis and treatment."

The company is hoping for a quick turnaround with the FDA. "We are focused on our product development and continue to work with the FDA to gain regulatory clearance. We expect to make substantial progress towards the goal of FDA clearance in 2019," Vaughan said.

In terms of other offerings, he noted that the company provides the Cognoa Child Development app via partnerships with employers, payers and applied behavior analysis therapy centers.

Last fall, Baltimore-based Learn Behavioral, a network of providers serving children with autism and other special needs, reported a partnership with the company that aims to improve the timeliness of care. The company's solution will be deployed in nine regions within the Learn network. Just last month, the company said Cambia Health Solutions Inc., of Portland, Ore., would incorporate its evidence-based platform within its subsidiary health plans.

Feedback for the development app, which, so far, has been used by more than 250,000 families, has been positive. Vaughan said parents like that it can help them obtain answers sooner, allowing for earlier diagnosis and treatment. "Regardless of whether their child's development is on track or not, parents have reported feeling empowered with our evidenced-based activities to support their child's unique developmental progress. We have also received strong support and enthusiasm from clinicians wanting to participate in our clinical studies," he added.

When asked about the potential for going to other markets, Vaughan said the U.S. is the main focus right now, but the company will evaluate expansion. Also, the company is looking to go beyond its first therapeutic area of autism. "We intend to develop further devices based on our precision health platform for ADHD and other behavioral health indications. We are committed to generating and sharing clinical data and trials and submitting further results to peer-reviewed medical journals," Vaughan said.

Another company trying to help identify those with autism spectrum disorder early is Madison, Wis.-based Neuropointdx, a division of Stemina Biomarker Discovery. Last fall, the company, which focuses on metabolomics, unveiled the Npdx AA test. That test identifies metabolic subtypes associated with the condition. (See BioWorld MedTech, Nov. 2, 2018.) The test, provided through Neuropointdx's CLIA-certified laboratory, may be used to screen children as young as 18 months.

01

Heuresis Announces Its Rebrand to Viken Detection

Viken Detection Committed to Leveraging Emerging Technology to Address Urgent Homeland Security and Public Safety Threats

NEWTON, Mass.--(BUSINESS WIRE)--Heuresis, pioneer of handheld x-ray imaging and analytical devices, today announced it is rebranding the company as Viken Detection (effective February 11). The brand name, Viken, and tagline, “One Sense Ahead,” reflect the vision of the company, led by CEO Jim Ryan and CTO Dr. Peter Rothschild, to significantly increase the use of cutting-edge technologies to address urgent homeland security and public safety challenges.

Viken Detection is already the recognized leader in handheld imaging used by law enforcement to uncover contraband such as opioids, cash and explosives hidden in vehicles, cargo containers, boats, buildings and other concealed areas. Viken Detection is also revolutionizing lead paint detection with its Pb200i, a handheld analyzer that can identify the hazard at much lower levels than currently being tested. In addition to the advanced physics, all Viken’s ergonomic handheld devices are Android-based, and include smartphone technologies such as a camera, GPS, WiFi, third-party app integration and other functionality.

“There are many areas of homeland security and public safety that can be addressed with new technology advances,” said Ryan. “Our focus is to quickly get this technology into the hands of professionals who need it to protect the public. Now our brand name reflects that vision and mission.”

“VIKEN” is derived from “sixth sense” through the blending of concepts “VI” the roman numeral for six and “KEN” meaning knowledge or range of sight. Along with its new tagline, “One Sense Ahead, the brand VIKEN is intended to signify the company’s focus on detecting public threats with breakthrough technology.

Viken Detection recently announced a 5-year IDIQ contract award, valued up to $29 million, with U.S. Customs and Border Protection for the HBI-120 (Viken’s handheld x-ray imaging device) to help thwart drug-trafficking across the border. Viken also announced contracts with the U.S. Department of State and the World Customs Organization for wide international deployment of the handheld device.

The new website can be found by visiting vikendetection.com.

January 2019

22

Bevi Lands $35M for Smarter Water Cooler That Gets to Know You

News Bites Finance January 22, 2019  

The water cooler will see you now.

The beverage engineers at Boston-based startup Bevi are hard at work building a drink dispenser that can call up the finest details of your workplace drink order. How much lime flavoring is enough? How effervescent should the carbonation be? What mix of vitamins should be added?

"We see that people are getting more and more specific about what they want for themselves," says Sean Grundy, co-founder and CEO of Bevi, a six-year-old Boston startup that makes a beverage kiosk that dispenses flavored drinks.

The company says today it has landed a $35.5 million Series C funding round led by Bessemer Ventures. The sum pushes the company's total venture capital haul to more than $63 million. Kent Bennett of Bessemer-who's worked with food startups Blue Apron, MealPal, and Toast--is joining Bevi's board as part of the financing, the company says. Earlier Bevi investors Horizons Ventures and Trinity Ventures both joined in the new round.

Bevi was founded in 2013, and by 2017, its Internet-connected beverage kiosks had been deployed in the offices of approximately 370 companies, including Apple, AT&T, Fidelity, GE, and Netflix. Grundy says the company's sales grew "well over 100 percent" this past year. Its machines have a touchscreen that lets thirsty users select still or sparkling water, and add any variety of flavors.

People's taste preferences for drinks are all over the map, Grundy says, making it harder to nail down the stock for a drink machine that will satisfy the masses. He says Bevi customer surveys show scant agreement about how drinks should be made, such as how much raspberry flavoring is enough or what is the "correct" bubble strength for a carbonated drink. Bevi is working to provide users the ability to dial in the details and let the beverage machines recall those preferences when people return for a refill.

Exactly how the machines will identify return customers is still in the works, with phone apps, badges, and radio-frequency-tagged bottles all being explored, Grundy says. In fact, the proprietary bottle was an early focus for Bevi when it aimed to control each step in the drink dispensing process.

"In the future, we would definitely be interested in bringing that back," Grundy said.

Worried that big tech is taking over your afternoon strawberry fizz? Grundy says the identification system will stay an opt-in system and users will still be able to use Bevi to get a drink without it saving their preferences.

PepsiCo's recent $3.2 billion acquisition of SodaStream, the Israeli company that makes a kitchen drink carbonation machine, is an encouraging signpost for Bevi's quest to customize drinks in the workplace.

Bevi's push for personalization comes as the company is also hiring to boost its brand and put its products in more offices. Bevi currently has about 90 employees (with about 40 openings scheduled to be filled in 2019), and the company is in about 30 states and a few cities in Canada.

Over the last year, Bevi has been stocking its executive ranks. The company hired Bob Doucette, who ran global operations and logistics at Amazon Robotics, to be its chief operating officer. It hired Boston Dynamics engineering veteran Thomas Allen to head up hardware. And it brought on LogMeIn sales director Todd Johnston to head Bevi's sales.

"We've been recruiting executives from the best of the best of Boston technology companies in order to become one of the best ourselves," Grundy says, adding that the company is currently hiring a marketing VP.

Bevi's previous funding round in mid 2017 clocked in at $16.5 million and prompted predictions that the company could seek expansion while also starting to make a profit. Now in 2019, Grundy says the goal-which even then he admitted was "easier said than done"-went unrealized. But he hasn't given up on it.

"Now, I'm saying the same thing again," Grundy says, "that one of our goals is to become net income positive with a Series C, and ideally this time we can do it."

(Being on the wrong end of a rosy prediction is far from the worst place Grundy has been. For that, ask him about his time working with a non-governmental organization in China wearing a well-used gibbon suit and meeting now-retired NBA star Yao Ming at an event run by the Ministry of Forestry. It ends with Ming learning down, sniffing his hair, and saying, "I can smell that you were the gibbon.")

But back to profitability: Grundy says it wouldn't surprise him if Bevi is still seeing red ink in two years and chasing a profit after it raises its next funding round.

   

21

Enyo Pharma Listed a New Clinical Trial for Safety, Tolerability, Pharmacokinetics and Efficacy of EYP001a in Patients With Nonalcoholic Steatohepatitis (NASH)

Contify Life Science News

Jan. 21 -- Enyo Pharma has listed an interventional clinical trial, Phase 2, for randomized, double-blind, multicenter, placebo-controlled study to assess the safety, Tolerability, Pharmacokinetics, and Efficacy of EYP001a in patients with Nonalcoholic Steatohepatitis. The trial's principal investigator is Harrison Stephen, MD.

The trial for a sample of 160 participants from USA is currently open for recruiting.

The study is being conducted for the following purpose:

The purpose of this study is to assess the effects of EYP001a with respect to safety, tolerability, pharmacokinetics and on markers of liver inflammation in patients with NASH.

The full document can be viewed at https://clinicaltrials.gov/ct2/show/NCT03812029?recrs=abdfm&type;=Intr&cntry;=US&phase;=0123&sfpd;_s=01%2F21%2F2019&sfpd;_e=01%2F21%2F2019&rank;=8.

07

Liquidia Technologies Reports Positive Interim LIQ861 Safety Data on 109 Patients from Pivotal INSPIRE Trial

RESEARCH TRIANGLE PARK, N.C., Jan. 07, 2019 (GLOBE NEWSWIRE) – Liquidia Technologies, Inc. (Nasdaq:LQDA) (“Liquidia”), a late-stage clinical biopharmaceutical company focused on the development and commercialization of human therapeutics using its proprietary PRINT® technology to transform the lives of patients, today reported positive interim safety data from its open-label, multicenter Phase 3 clinical trial (INSPIRE) evaluating LIQ861, an inhaled dry powder formulation of treprostinil, for the treatment of pulmonary arterial hypertension (“PAH”). The safety data at the two-week timepoint addresses the U.S. Food and Drug Administration’s (“FDA”) data request for inclusion in a New Drug Application (“NDA”) submission. Liquidia anticipates submitting the full NDA for LIQ861 to the FDA in late 2019. LIQ861 was observed to be well-tolerated at the two-week timepoint in PAH patients. During this time period, LIQ861 was evaluated at doses up to approximately 125 mcg with no study-drug related serious adverse events or dose-limiting toxicities. Reported treatment-emergent adverse events (“TEAEs”) were mostly mild in nature and consistent with inhaled prostacyclin therapy. The most common TEAEs reported with LIQ861 in ≥4% of PAH patients (n=109) were cough (25%), headache (13%), throat irritation (12%), diarrhea (7%), dizziness (6%), oropharyngeal pain (5%) and chest discomfort (5%). Patients have continued to receive treatment beyond two-weeks with the first patient dosed in March 2018. To date, a maximum tolerated dose of LIQ861 has not yet been reached, with patients having been administered doses up to approximately 150 mcg. “LIQ861 has the potential to maximize the therapeutic benefit of inhaled treprostinil in treating PAH by safely delivering higher doses into the lungs,” stated Nicholas Hill, MD, Chief Pulmonary, Critical Care & Sleep Division and Professor of Medicine at Tufts University School of Medicine and INSPIRE Principal Investigator. “Enabled by Liquidia’s proprietary PRINT technology, LIQ861 is designed to provide the benefits of delivering prostacyclin analogs locally to the lungs via inhalation, potentially offering a targeted and effective approach with an acceptable systemic side effect profile.” The INSPIRE clinical trial is designed to evaluate patients who have either been under stable treatment with nebulizer-delivered treprostinil for at least three months and are transitioned to LIQ861 under the protocol or patients who have been on stable treatment with no more than two non-prostacyclin oral PAH therapies for at least three months and have their treatment regimen supplemented with LIQ861 under the protocol. Patients adding LIQ861 to current non-prostacyclin oral therapies started at a dose of approximately 25 mcg and those transitioned from nebulizer-delivered treprostinil at a stable dose were initiated at a dose of LIQ861 lower than their current stable treprostinil dose. In both cases, LIQ861 was uptitrated in 25 mcg incremental doses to symptom relief or the limit of tolerance. “Patient demographics and baseline characteristics in the trial suggest that LIQ861 may be attractive across disease severity and may have utility as a first-line prostacyclin,” added Robert Roscigno, PhD, Liquidia’s Senior Vice President of Product Development and LIQ861 Program Lead. “Interestingly, enrollment of the safety portion of the trial was driven primarily by stronger than anticipated interest from New York Heart Association Functional Class II add-on patients, which may imply that dry-powder delivery could be an alternative to oral delivery in prostacyclin naïve patients. We are pleased with these findings and believe they support the therapeutic potential and versatility of LIQ861 among patients cross different functional classes.” Liquidia continues to enroll patients in the INSPIRE clinical trial in support of the one-directional crossover pharmacokinetic (“PK”) sub-study. The sub-study is designed to compare bioavailability and PK of treprostinil as patients are transitioned from nebulizer-delivered treprostinil to LIQ861. PK results are expected to be reported in the second quarter of 2019. To further support Liquidia’s future marketing and commercial activities with additional medical information, Liquidia expects to continue to treat patients and collect data until the launch of LIQ861 in the United States, if approved.

July 2018

30

Liquidia Technologies Announces Closing of Initial Public Offering

RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--Jul. 30, 2018-- Liquidia Technologies, Inc. (the “Company”), a late-stage clinical biopharmaceutical company focused on the development and commercialization of human therapeutics using its proprietary PRINT® technology to transform the lives of patients, today announced the closing of its initial public offering of 4,545,455 shares of common stock at a public offering price of $11.00 per share for gross proceeds of $50.0 million before underwriting discounts and commissions. In addition, the Company has granted the underwriters a 30-day option to purchase up to 681,818 additional shares of common stock at the initial public offering price, less the underwriting discount. The Company’s common stock began trading on the Nasdaq Capital Market under the ticker symbol “LQDA” on July 26, 2018. All of the shares sold in the offering were offered by the Company.

Jefferies and Cowen acted as joint book-running managers for the offering. Needham & Company and Wedbush PacGrow acted as co-managers.

A registration statement relating to these securities was declared effective by the U.S. Securities and Exchange Commission on July 25, 2018. The offering was made only by means of a prospectus. Copies of the final prospectus may be obtained from Jefferies LLC, Attn: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by telephone at 877-547-6340 or by email at Prospectus_Department@Jefferies.com; or from Cowen and Company, LLC, c/o Broadridge Financial Services, 1155 Long Island Avenue, Edgewood, NY 11717, Attn: Prospectus Department, by telephone at (631) 274-2806 or by fax at (631) 254-7140.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

May 2018

18

Nuance buys Voicebox Technologies, scooping up speech-recognition and natural-language pioneer

Nuance Communications has quietly acquired Voicebox Technologies, a Bellevue, Wash.-based company that was an early leader in speech recognition and natural language technologies, GeekWire has learned. The acquisition wasn’t publicly announced, and Nuance didn’t respond to requests for comment, but an email autoreply from Voicebox CEO and co-founder Mike Kennewick confirms that the company “has recently been acquired by Nuance Communications.” It’s the latest indication of the booming market for conversational speech technology and voice assistants, driven by new types of devices and the rise of artificial intelligence. Founded in 2001, Voicebox had amassed a large patent portfolio during its 17-year history. Nuance’s latest quarterly filing with the Securities and Exchange Commission references an $82 million cash acquisition of an unnamed company in its automotive segment in April. Voicebox has been focusing largely on connected car voice technology since its work on Samsung’s S Voice technology took a hit following the Galaxy Note 7 discontinuation last year. Voicebox supplied behind-the-scenes voice technology used in a variety of applications and devices, but it was competing in a larger market defined and controlled in recent years by tech giants including Amazon, Google and Microsoft. The surprise deal is the latest in a string of Seattle-area acquisitions by Nuance. The speech and imaging technology giant, headquartered in Burlington, Mass., has a large presence in the region as a result of previous deals. It bought text-input pioneer Swype for $102.5 million back in October 2011; it bought in-car speech software startup Tweddle in 2013; and acquired automated message developer Varolii later that year. In 2009, it acquired Jott, another Seattle startup. The acquisition of Voicebox “shows that the market for conversational assistants is really heating up — it’s not about mere speech recognition any more,” said Dan Weld, a Madrona Venture Group venture partner and University of Washington professor who was an adviser to the company. This shows that the market for conversational assistants is really heating up — it’s not about mere speech recognition any more. “The market wants full dialog capabilities with semantic parsing of what the user is saying, the ability to take actions on the user’s behalf, and multi-turn, contextual response,” Weld said. “Voicebox has been a leader in this technology for many years with an impressive white-label conversational assistant shipping in Toyota cars, Samsung phones and other products.” The voice and speech recognition market is expected to be worth $6.7 billion by 2025, from $1.1 billion in 2017, according to a recent report by ResearchandMarkets.com. The market is fueled as companies add new voice technologies, specifically conversational assistants, to a wide variety of devices and services. Voicebox focused on conversational voice technology for cars, homes, mobile devices, and more. Its customers include Samsung, Toyota, AT&T, TomTom, and Fiat Chrysler. The company was co-founded by brothers Mike Kennewick and Rich Kennewick As of earlier this month, the company was #65 on the GeekWire 200, our index of the Pacific Northwest’s top privately held technology companies. With the acquisition, the company is no longer on the index. As shown in this 2006 news report, Voicebox was attempting to build the equivalent of Alexa long before Amazon was. Publicly-traded Nuance, known for products including Dragon NaturallySpeaking, is a leader in speech and imaging applications. It posted revenue of $518 million in the most recent quarter and has a market capitalization of nearly $4 billion. Voicebox had more than 180 employees, according to LinkedIn, with additional offices in Europe and Asia. The company had raised more than $25 million, according to CrunchbaseVoicebox made major job cuts in January 2017 due to a “combination of unforeseen circumstances,” related in part to the Galaxy Note 7 discontinuation. Two months prior, the company announced that it had leased a new office in Bellevue, totaling nearly 67,400 square feet, to accommodate what it described at the time as its growing staff. The company’s Twitter and LinkedIn pages have been quiet since the end of March. The last post on its news page was March 29. There are no job postings on its careers page.

April 2018

09

Stealth BioTherapeutics Granted Orphan Drug Designation for Elamipretide for Treatment of Barth Syndrome

BOSTON, April 9, 2018 /PRNewswire/ -- Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing therapeutics to treat mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug Designation to Stealth's investigational drug candidate, elamipretide, for the treatment of patients with Barth syndrome. "There are currently no FDA-approved therapies for patients with Barth syndrome, a debilitating condition characterized by heart and skeletal muscle weakness," said Reenie McCarthy, Stealth's chief executive officer. "We believe elamipretide represents a promising, novel approach with the potential to offer hope for patients with Barth syndrome, and we are very pleased to have received Orphan Drug Designation for this indication." The Orphan Drug Act, enacted in 1983, encourages development of therapies for rare diseases. A disease is considered rare if it affects fewer than 200,000 persons in the United States. Once granted, Orphan Drug Designation provides various development benefits for an investigational drug, including seven-year exclusivity after marketing approval is received. In March 2018, Stealth announced the completion of enrollment in the TAZPOWER clinical trial, a phase 2/3 crossover study evaluating the effects of daily treatment with elamipretide in 12 patients with genetically confirmed Barth syndrome followed by an open-label treatment extension. The primary endpoints include change in distance walked during the six-minute walk test and change in total fatigue as measured by a patient-reported outcome measure, the Barth Syndrome Symptom Assessment. Secondary endpoints include additional functional assessments, patient-reported outcomes and safety. Data from the TAZPOWER trial are expected later this year. In November 2017, Stealth announced that the FDA granted Fast Track designation for elamipretide for the treatment of Barth syndrome. For additional information on the TAZPOWER study or elamipretide, please refer to Stealth's website.  

04

Allysta Pharmaceuticals, Inc. Doses First Patient in Phase 2A Study in Glaucoma

BELMONT, CA (ACCESSWIRE) Allysta Pharmaceuticals, Inc. (Allysta) today announced that that the first patient was dosed in its Phase 2A trial of H-1337 for the treatment of patients with glaucoma. H-1337 was developed by D. Western Therapeutics Institute, Inc., (DWTI) in Nagoya, Japan and, in its arrangement with Allysta, Allysta is providing management and oversight of the pre-clinical, regulatory, and clinical development through Phase 2A for H-1337. Allysta has an option to license the compound outside of Japan under certain conditions after completion of a phase 2 clinical trial. This first-in-human study will evaluate the safety, tolerability, and preliminary efficacy of three concentrations of H-1337 and vehicle administered as an eye drop in a parallel group, double-masked design for 28 days of dosing in patients with elevated intraocular pressure (IOP) and will enroll approximately 80 subjects across 5 ophthalmic centers in the US. “This marks a significant milestone for Allysta as we successfully advanced this promising novel drug from lead through IND and into the clinic, transitioning Allysta from pre-clinical status to a clinical stage company. We look forward to completing enrollment with the intent to finish the study in Q4 of this year,” said Henry Hsu, M.D., Chief Executive Officer and President of Allysta.

February 2018

21

Cognoa’s AI platform for autism diagnosis gets first FDA stamp

Cognoa has gained regulatory recognition for its machine learning software as a class II diagnostic medical device for autism — meaning the digital health startup is now positioned to submit an application for full FDA clearance. It’s a first but important regulatory step for a business that was founded back in 2014, and plays in a still nascent digital health space where untested ‘wellness’ apps are far more plentiful than medical technologies with robust data to prove out the efficacy of their interventions. Discussions with the FDA started in early 2017, says Cognoa CEO Brent Vaughan, adding that it’s hoping to gain full FDA clearance this year. He says the ultimate goal for the US startup is to become a standard part of domestic health insurance-covered medical provision — and for that FDA clearance is essential to opening the doors. We first covered the Cognoa at launch in 2014 and the following year when it was still being careful to describe its technology as a screening rather than a diagnostic system. It’s since gathered enough data to be confident in using the ‘D’ word — having run a pilot with 250,000 parents, offering free screening for their children so it could gather more data to refine its machine learning models. “We were lucky that we had investors,” says Vaughan. “There’s not a huge business model in providing free screening services to kids, right, because we were certainly never going to sell ads. That wasn’t the goal. To view the full document, please visit https://techcrunch.com/2018/02/21/cognoas-ai-platform-for-autism-diagnosis-gets-first-fda-stamp

21

FDA recognizes Cognoa's child development app as autism diagnostic

Palo Alto, California-based Cognoa’s machine learning app for pediatric behavioral health has received categorization from the FDA as a Class 2 diagnostic medical device for autism, according to an announcement from the company. By analyzing parent-provided information and videos of a child’s natural behavior, the company’s app uses machine learning to provide an assessment of whether that child is developing at the right pace, as well as to evaluate their behavioral health. The clinically-validated app can identify autism among children as young as 18 months, according to Cognoa, and so far has been used by hundreds of thousands of families. “Today, there is a profound, unmet need for earlier and more accurate diagnoses of behavioral health conditions which we know can create life-changing results for children and their families,” Cognoa CEO Brent Vaughan said in a statement. “We are very encouraged by this FDA determination of Cognoa’s AI-based software as a medical device for diagnosis. Cognoa is committed to conducting additional clinical studies and working with both the FDA and clinicians to further validate both our diagnostic software as well as integrated digital therapeutics that will dramatically improve the standard of care for physicians, children, and their families." Founded in 2013 on the back of AI technology developed at Harvard and Stanford’s respective medical schools, Cognoa looks to reassure parents that their child’s development is on track, or alert them if additional attention may be necessary. In addition to serving as a diagnostic tool, the app also gives personalized recommendations for activities or interventions that parents can do at home to support development in their child. Outside of the benefits for families, Cognoa positions its product as a way for healthcare payers or similar companies to reduce long-term behavioral health costs. The company also notes that its app can reduce the burden on clinicians and support their diagnoses. “For years, primary care physicians have been referring children with suspected behavioral and developmental conditions for more extensive evaluations for autism spectrum disorders. This results in a logjam of children, with varying levels of severity, waiting for these assessments,” Dr. Daniel Coury, professor of pediatrics and psychiatry at Nationwide Children's Hospital and The Ohio State University, and medical director of the Autism Speaks Autism Treatment Network, said in a statement from Cognoa. “Cognoa has completed several well-designed clinical trials and has engaged early with the FDA. What’s exciting is how Cognoa will be able to empower primary care physicians to make a primary diagnosis and refer children directly for treatment, greatly reducing the time from diagnosis to meaningful interventions and leading to better outcomes.” Last March, Cognoa raised $11.6 million in a funding round led by Chinese private investment group Morningside, bringing the company’s total funding to more than $20 million. At the time, Cognoa said that the money would be used for continued validation studies for regulatory submission. Speaking to TechCrunch, Vaughn said that his company will likely raise another round of investments by the end of the year.

November 2017

15

NuCana Announces First Patients Enrolled in Phase 2 Study of Acelarin in a Platinum-Resistant Ovarian Cancer

EDINBURGH, United Kingdom, Nov. 15, 2017 (GLOBE NEWSWIRE) -- NuCana plc (NASDAQ:NCNA) announced the enrollment of the first patients in both the United States and the United Kingdom in its PRO-105 study evaluating single-agent Acelarin (NUC-1031) in patients with platinum-resistant ovarian cancer. Hugh Griffith, NuCana’s Chief Executive Officer, stated: “The initiation of this Phase 2 study of Acelarin in both the US and the UK is a major step in the expansion of the NuCana product pipeline and advances our strategy of rapidly developing our ProTides to benefit cancer patients globally. We are grateful to the patients and clinicians who are making this study possible.” The PRO-105 study will enroll up to 64 patients with platinum-resistant ovarian cancer who have relapsed following three or more prior lines of chemotherapy. Patients will receive Acelarin on day 1, 8 and 15 of a 28-day cycle and will be treated to progression. The primary endpoint of the study will be Objective Response Rate, and secondary endpoints include Duration of Response, Progression-Free Survival, Overall Survival and safety parameters. Part one of the study will enroll up to 20 patients in each of two dose cohorts: 500mg/m2 and 750mg/m2. In part two of the study, NuCana will select one of these doses and enroll at least an additional 24 patients at the selected dose. NuCana expects to announce interim data from this study in 2018. More information about this study may be found at https://clinicaltrials.gov/ct2/show/NCT03146663. Professor Bradley J. Monk of Arizona Oncology and co-Chief Investigator of PRO-105 stated: “Platinum-resistant ovarian cancer remains an area of significant unmet medical need and we are excited to participate in this study and advance Acelarin as a potential treatment for women with ovarian cancer. Acelarin’s ability to overcome key cancer cell resistance mechanisms resulting in significantly greater levels of the active anti-cancer metabolite differentiates it from other treatment approaches.” Professor Charlie Gourley, of the University of Edinburgh and co-Chief Investigator of PRO-105, added: “Acelarin has shown meaningful clinical activity in advanced recurrent ovarian cancer and has been well-tolerated in clinical studies to date. We are pleased to be a part of this important clinical study.” Acelarin is a potential first-in-class ProTide that has been evaluated in over 140 patients. In the first-in-human Phase 1 dose-ranging PRO-001 study in 49 evaluable patients with advanced metastatic solid tumors, Acelarin was well tolerated and achieved a 78% disease control rate. Acelarin achieved a disease control rate of 93% in a subset of 14 evaluable patients with advanced gynecological cancers in the PRO-001 study. This was followed by the Phase 1b dose-ranging PRO-002 study where Acelarin in combination with carboplatin achieved a 96% disease control rate and 39% response rate in 23 evaluable patients with recurrent ovarian cancer.

13

Apellis Pharmaceuticals Announces Closing of its Initial Public Offering

CRESTWOOD, Ky., Nov. 13, 2017 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds to treat disease through the inhibition of the complement system, today announced the closing of its initial public offering of 10,714,000 shares of common stock at a public offering price of $14.00 per share. The total gross proceeds to Apellis were approximately $150.0 million, before deducting underwriting discounts and commissions and expenses payable by Apellis. All of the shares were sold by Apellis. In addition, Apellis granted the underwriters a 30-day option to purchase up to 1,607,100 additional shares of common stock at the public offering price, less underwriting discounts and commissions, to cover over-allotments, if any. The shares commenced trading on the NASDAQ Global Select Market under the ticker symbol “APLS” on Thursday, November 9, 2017. Citigroup, J.P. Morgan and Evercore ISI acted as joint book-running managers for the offering. A registration statement relating to the securities sold in this offering was declared effective by the Securities and Exchange Commission on November 8, 2017. The offering was made only by means of a prospectus. When available, copies of the final prospectus relating to the offering may be obtained by contacting: Citigroup Global Markets Inc., c/o Broadridge Financial Services, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: 1-800-831-9146, or email: prospectus@citi.com; J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: (866) 803-9204; or Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 36th Floor, New York, NY 10055, telephone: (888) 474-0200, or email: ecm.prospectus@evercore.com. This press release shall not constitute an offer to sell, or the solicitation of an offer to buy, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.  

10

Aduro Biotech Announces Promising Preclinical Data that Validate Anti-CTLA-4 Antibody ADU-1604

BERKELEY, Calif., Nov. 10, 2017 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, today announced data from preclinical studies with ADU-1604, the company’s humanized anti-CTLA-4 monoclonal antibody. Data from these in vitro and in vivo studies demonstrate the potency of ADU-1604 and its ability to inhibit tumor growth and enhance T cell-dependent antibody responses. These data, which will be highlighted later today in a poster presentation (Poster #335) at the 32ND Annual Meeting of the Society for Immunotherapy of Cancer (SITC), underscore the potential application of ADU-1604 for the treatment of multiple cancer types, either as monotherapy or in combination with other therapies. “These data from preclinical studies of ADU-1604, a novel anti-CTLA-4 product candidate derived from our proprietary B-select antibody platform, are encouraging and provide support to file an Investigational New Drug Application to advance ADU-1604 into clinical studies,” stated Andrea van Elsas, Ph.D., chief scientific officer of Aduro Biotech. “As a company with multiple programs and proprietary technology platforms, we are well positioned to leverage our product candidates, as monotherapies and in rational combinations, to develop new treatment options for patients in need.”

08

Synlogic Reports Positive Top-Line Phase 1 Data Demonstrating Safety and Tolerability and Proof of Mechanism in Healthy Volunteers for SYNB1020, a Synthetic BioticTM Medicine for the Treatment of Hyperammonemia

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Nov. 8, 2017-- Synlogic, Inc.,(Nasdaq:SYBX) a clinical-stage drug discovery and development company applying synthetic biology to probiotics to develop novel Synthetic Biotic medicines, today announced positive top-line clinical data from its Phase 1 placebo-controlled single (SAD) and multiple ascending dose (MAD) clinical trial of SYNB1020 in healthy volunteers. The trial successfully met the primary objectives demonstrating safety and tolerability in healthy volunteers and identifying the maximum tolerated dose. Furthermore, proof of mechanism was demonstrated by a clear signal in a plasma nitrogen endpoint. SYNB1020, is a novel, first-in-class, Synthetic Biotic medicine that is orally delivered and designed to treat elevated blood ammonia levels, or hyperammonemia, in genetic urea cycle disorders (UCD) or in chronic liver disease "The positive data from our Phase 1 study in healthy volunteers, demonstrates that SYNB1020 was well-tolerated and had a statistically significant dose-dependent effect on the level of a nitrogen endpoint, providing evidence to support its mechanism of action," said Aoife Brennan, M.B., B.Ch., Synlogic’s chief medical officer. "These data support the hypothesis that SYNB1020 treatment may provide clinical benefit in patients with UCDs or liver disease, and will inform dose selection in our planned Phase 1b/2a study of SYNB1020 in patients, which we expect to begin in the first half of 2018." “This first-in-human study represents a significant milestone for our new class of Synthetic Biotic medicines and demonstrates that they can operate from the gastrointestinal tract to metabolize systemic toxins,” said JC Gutiérrez-Ramos, Ph.D., Synlogic’s president and chief executive officer. “We look forward to evaluating SYNB1020 in patients, and to moving our second program, SYNB1618 for the treatment of phenylketonuria into clinical trials in 2018. SYNB1020 was safe and well tolerated in subjects in multiple ascending dose cohorts who received total daily doses of up to 1.5x1012 CFU for 14 days. There have been no serious adverse events (SAEs), and no cases of infection with the bacteria in this study. While the study is ongoing, there is no evidence of colonization by SYNB1020 as all subjects who have completed follow-up have cleared the bacteria from their systems within the expected timeframe. In the MAD component of the Phase 1 study, daily dosing of SYNB1020 over 14 days in healthy volunteers enabled identification of a dose-response relationship between SYNB1020 oral administration and changes in a nitrogen endpoint in plasma which was found to be statistically significant in the highest dose cohort compared to placebo. In addition, viability and evidence of mechanistic activity of the Synthetic Biotic was demonstrated in feces of subjects who received SYNB1020, but not in control subjects. As expected, SYNB1020 did not lower blood ammonia levels in these healthy individuals who had normal blood ammonia levels at baseline. Collectively, the data support the hypothesis that SYNB1020 treatment may enable metabolism of potentially neurotoxic blood levels of ammonia in patients with hyperammonemia stemming from UCDs or liver damage.

October 2017

26

This 'Uber' Of Staffing App Could Revolutionize The Hospitality Industry

Jitjatjo is a new app specifically designed to solve temporary hospitality staffing problems. Catering companies, event planners and restaurateurs can use Jitjatjo (which sounds like tic tac toe) to ease their “supply and demand” challenges of staffing. In one week managers might need to hire staff to accommodate an intimate black-tie dinner party for 30 diners, a 3,000-person corporate event and a promotional cocktail hour for 300 people. Finding talented workers during busy periods, retaining them during slow periods, is both stressful and time consuming. “How can I get part-timers that come in and give me the available supply to satisfy the demand?” asks Ron McCulloch rhetorically in his strong Scottish accent. Jitjatjo’s co-founder and Executive Chairman, 64-year-old McCulloch sits in his Manhattan office that overlooks Soho, explaining that his 25+ years in the hospitality industry, including the colossal Home Nightclub in Sydney where he lived for 17 years, taught him the elastic nature of hospitality staffing. “So rather than just taking someone off the street and say, ‘Come and work for me for one night,’” says McCulloch, demonstrating the level of desperation some managers feel when looking for hospitality staff, especially last-minute; instead he suggests, managers turn to the more efficient and targeted Jitjatjo app, which launched 11 months ago. McCulloch, along with co-founder and CEO Tim Chatfield, play down the comparison, Jitjatjo is arguably the Uber of temporary hospitality staffing. If a catering company suddenly needs five extra servers and a bartender for an event in two hours, they input the specifics of the job through the Jitjatjo app and its algorithm books the most qualified workers available within minutes. For better or worse, servers and clients don’t choose each other, Jitjatjo’s algorithm makes the match. “People in hospitality have so many things to do,” says McCulloch, “they want simple solutions.”

26

ENYO Pharma opens a new subsidiary in Australia as a new managing center for clinical trials

Lyon, October 26, 2017. ENYO Pharma, a biopharmaceutical company developing new molecules with an innovative approach inspired by viruses, made a strategic decision and has recently decided to open its first wholly owned subsidiary in Melbourne, Australia, because of its excellent clinical trials development capabilities and attractive conditions offered by the government. The Australian Government is very supportive of biotech startups and the Victorian Government Business Office provides good business development networking. This office will be the new ENYO Pharma center to manage EYP001 (an FXR agonist) clinical trials in the Asia-Pacific region for chronic Hepatitis B. A Phase 1b study of EYP001 is currently submitted in 4 countries and two new Australian centers in Sydney and Perth have joined the study and started recruitment. ENYO Pharma bases its innovation on mimicking viral strategies to discover new cellular targets and is currently developing drug candidates in therapeutic areas such as infectious and metabolic diseases. One of the company’s leading programs is a new drug EYP001, against chronic Hepatitis B which is a major public health concern in the Asia-Pacific region. More than a half of the global population infected worldwide by Hepatitis B are located in this region, representing 129 million patients. “ENYO Pharma is currently looking forward to working in this region as the well-established clinical expertise in this part of the world adds important value to ENYO Pharma R&D efforts and specifically our expeditious development of EYP001” commented Pietro Scalfaro, M.D., Chief Medical Officer of ENYO Pharma SA. “Melbourne’s strong biotechnology sector is the leading research and clinical trial hub in the Asia Pacific region. ENYO Pharma is a leading biotechnology innovator in infectious diseases and the Government of Victoria is proud to welcome their new research subsidiary into Melbourne, Australia.” said the Minister for Innovation and Trade, Minister Philip Dalidakis.

24

Innovative Alzheimer’s Disease Combination Therapy Trial Supported By New Joint Funding Initiative

NEW YORK AND CHICAGO, Oct. 24, 2017 /PRNewswire-USNewswire/ — The Alzheimer’s Association and Alzheimer’s Drug Discovery Foundation (ADDF) are collaborating to jointly fund a new combination therapy clinical trial for Alzheimer’s disease to be conducted by Amylyx Pharmaceuticals. The $1.85 million grant is the first award under an initiative created by the Alzheimer’s Association and the ADDF to fund combination therapies. The grant will support a phase 2 clinical trial of AMX0035, a combination of sodium phenylbutyrate (PB) and tauroursodeoxycholic-acid (TUDCA). The trial, expected to begin in the first half of 2018, will include approximately 50 people with mild cognitive impairment or mild-to-moderate Alzheimer’s disease and test the drug’s effectiveness at slowing or stopping brain cell death. “Combination therapies hold great potential to slow the progression of Alzheimer’s,” said Howard Fillit, MD, the ADDF’s founding executive director and chief science officer. “The innovation of Amylyx’s combination therapy is that it targets multiple causes of brain cell loss, and the two drugs given in tandem create additional protective effects. The ADDF was an early supporter of innovative targets for Alzheimer’s, and we believe combination therapies are a critical next step in finding effective treatments for the disease.” Alzheimer’s is a complex disease with multiple, interrelated causes. A growing number of experts believe combination therapy—with two or more drugs, or drugs and lifestyle interventions—will be required to effectively treat it. “We have witnessed the success of combination therapy in HIV/AIDS, cancer, and heart disease. There is strong reason to believe that to successfully address Alzheimer’s, and its extraordinary complexity, we need to attack the disease on multiple fronts,” said Maria Carrillo, PhD, Alzheimer’s Association chief science officer. “Meetings convened in 2015 by the Alzheimer’s Association (proceedings published in 2016) and the ADDF led the two organizations to recognize the potentially important role of combination therapies in Alzheimer’s and paved the way for this exciting partnership and initial clinical trial funding.” New approaches to Alzheimer’s are urgently needed, as deaths from the disease continue to rise precipitously as more and more Baby Boomers reach the age of greatest risk. The grant was made through the Alzheimer’s Combination Therapy Opportunities (ACTO) program, a joint research funding initiative created by the Alzheimer’s Association and the ADDF to support clinical trials combining multiple treatment approaches. The ACTO program specifically called for study proposals using repurposed drugs that have been determined safe for use in treating other conditions. Repurposing may speed the drug development process because researchers can often begin with phase 2 trials including outcome measures of effectiveness, rather than phase 1 safety tests. The drug to be tested, AMX0035, is a proprietary oral formulation of two existing therapeutics. PB is an FDA-approved therapy—currently prescribed for urea-cycle disorders—that activates genes responsible for protecting brain cells from toxic unfolded proteins. TUDCA is an acid produced in small amounts by the body that targets cellular energy loss. In preclinical studies conducted by the company and with academic collaborators, combination dosing of PB and TUDCA protected brain cells from inflammation and oxidation. Amylyx received FDA clearance for AMX0035’s Investigational New Drug application in April 2017 and has an ongoing multicenter clinical study of the compound in people with amyotrophic lateral sclerosis (ALS). “We are very grateful for the support from the Alzheimer’s Association and the Alzheimer’s Drug Discovery Foundation, and we’re honored to partner with them in this new combination therapy initiative,” said Kent Leslie, Amylyx chief scientific officer. “Through a combination approach targeting two different and independent pathways, AMX0035 is designed to benefit both neurodegeneration and neuroinflammation, key drivers of Alzheimer’s and ALS. The biomarker-focused trial design will assist in translating the promising preclinical effects observed in models of Alzheimer’s to an improved understanding of the potential of AMX0035 to help individuals living with this disease.”

24

Synlogic Receives Orphan Drug Designation for SYNB1618, a Synthetic BioticTM Medicine for the Treatment of Phenylketonuria

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Oct. 24, 2017-- Synlogic (Nasdaq: SYBX), a clinical stage company applying synthetic biology to probiotics to develop novel living medicines, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to SYNB1618, Synlogic’s preclinical-stage drug candidate for the treatment of phenylketonuria (PKU) , an inborn error of metabolism (IEM) caused by a mutation in the gene that breaks down the amino acid phenylalanine (Phe). Phe accumulation in the blood and brain can lead to neurocognitive abnormalities and treatment currently requires severe dietary protein restriction. SYNB1618, an orally administered medicine, is designed to complement the missing function in patients with PKU by providing alternative metabolic pathways to consume Phe. Synlogic plans to file an investigational new drug application (IND) with the FDA for SYNB1618 for the potential treatment of PKU in early 2018. “We believe our Synthetic Biotic medicines could transform the treatment of PKU,” said Aoife Brennan, M.B., B.Ch., Synlogic’s chief medical officer. “Consequently, we were pleased to receive the FDA’s orphan drug designation which validates our approach and represents an important step toward achieving our goal of bringing novel treatments to the patients and families affected by this challenging disease.”

16

‘Farm in a Box’ Could Protect Food Supplies After Extreme Weather Events

Boston-based Freight Farms has developed what it calls the Leafy Green Machine, a method for vertical, hydroponic farming inside a shipping container. BY MOLLY FOSCO OCTOBER 16, 2017 3:53 PM EDT - Vertical and hydroponic farming has gained popularity in recent years. And with the frequency and intensity of some extreme weather events on the rise, innovative techniques offer enticing solutions to some agricultural problems. Boston-based company Freight Farms is offering one approach: It’s helping people grow food inside shipping containers 365 days a year, no matter the weather conditions. Brad McNamara and Jon Friedman started Freight Farms in 2010 with the goal of making it easier for anyone to grow food on a commercial scale. “We really focused on putting farming into the hands of regular people so that you can have a manageable footprint but still produce at a commercial level,” McNamara, the company’s CEO, told Seeker. “We were really inspired by what’s becoming possible with new technology and the fact that no one had changed their thinking yet from big agriculture and large-scale farms.” McNamara and Friedman wanted to create something small that could still produce large crop yields. The result is the Leafy Green Machine, a produce farm that operates inside a shipping container that is forty feet long, eight feet wide, and nine and a half feet high. The LGM uses a vertical, hydroponic farming method that allows leafy greens like kale, lettuce, and cabbage, as well as herbs like basil, mint, and oregano, to be harvested once a week, year round. It grows 2-4 tons of produce annually using less than 10 gallons of water per day. Its water, air, and nutrient inputs can be monitored and controlled remotely using a software called "farmhand." The LGM costs about $85,000 and around $13,000 to operate annually. Freight Farms offers financing options, as well as video tutorials and two-day, in-person trainings at their Boston headquarters on how to operate an LGM.

04

Forensic Logic Announces Acquisition of COPLINK Platform from IBM

The acquisition will create the nation’s largest network of law enforcement users, information, and technology WALNUT CREEK, CA and TUCSON, AZ – October 4, 2017 – Forensic Logic, the premier provider of network search technology and cloud-based information services to law enforcement, today announced it has acquired the COPLINK suite of products from IBM. COPLINK is a leading public safety information sharing platform and provider of analytical, tactical and operational software for law enforcement. The acquisition will deliver an unprecedented level of information access and technological interoperability to the U.S. law enforcement community, while greatly streamlining data integration, product acquisition, and customer service among their customer base. Created in 1998, COPLINK provides public safety software and services to over 5100 law enforcement jurisdictions throughout the United States. In addition to its core analysis platform, the COPLINK system offers an integrated array of software solutions for investigations, operations, and compliance. Founded in 2003, Forensic Logic has become one of the fastest-growing public safety technology providers in the country built on the success of its search engine optimized for public safety. The acquisition will further Forensic Logic’s strategy to deploy a fully integrated suite of world-class information access technology across the widest possible network of public safety agencies. “The COPLINK acquisition reinforces our commitment to bring a compelling combination of technological capabilities to this market,” said Bob Batty, CEO of Forensic Logic. “Our customers will no longer have to choose between the best search capability, the finest analytical tools, and the richest network of data – once the integration is done they will all be on one platform.”

02

Stealth BioTherapeutics Granted Orphan Drug Designation of Elamipretide for Treatment of Patients with Primary Mitochondrial Myopathy

BOSTON – October 2, 2017 – Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing therapeutics to treat mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) has granted Orphan Drug Designation to Stealth’s investigational drug candidate, elamipretide, for the treatment of patients with primary mitochondrial myopathy (PMM). “We are thrilled to achieve this key regulatory milestone for the treatment of PMM, a debilitating condition characterized by muscle weakness and fatigue with no FDA-approved treatments,” said Reenie McCarthy, Stealth’s chief executive officer. “We will continue to work closely with the FDA as we advance into our Phase 3 trial of elamipretide in patients with PMM.” The Orphan Drug Act was enacted in 1983 to encourage development of drugs for rare diseases, which are diseases that affect fewer than 200,000 persons in the United States. Once granted, Orphan Drug Designation provides various development benefits for an investigational drug, including seven-year exclusivity after marketing approval is received. In June 2017, Stealth announced results from MMPOWER-2, a Phase 2 continuation trial evaluating safety, tolerability and efficacy of treatment with elamipretide for PMM, which showed benefit of elamipretide across multiple endpoints assessed and supports a Phase 3 trial in this patient population. Stealth is currently recruiting for the RePOWER trial, an observational study of patients with PMM. Patients enrolled in RePOWER may have the opportunity to participate in the Phase 3 trial to further evaluate the potential efficacy, safety and tolerability of elamipretide for patients with PMM.

02

NuCana Plc Announces Closing Of Initial Public Offering Of ADSs And Exercise Of Underwriters' Option To Purchase Additional ADSs

EDINBURGH, United Kingdom, Oct. 02, 2017 (GLOBE NEWSWIRE) -- NuCana plc (Nasdaq:NCNA), a clinical-stage biopharmaceutical company focused on significantly improving treatment outcomes for cancer patients by applying its ProTide™ technology to transform some of the most widely prescribed chemotherapy agents into more effective and safer medicines, announced today the closing of its initial public offering of 7,596,505 American Depositary Shares ("ADSs") at a price to the public of $15.00 per ADS, which includes 929,505 shares sold upon partial exercise of the underwriters' option to purchase additional ADSs, for total gross proceeds of approximately $114 million, before deducting underwriting discounts and commissions. Each ADS represents one ordinary share of NuCana. The ADSs began trading on the NASDAQ Global Select Market on September 28, 2017 under the ticker symbol "NCNA." All ADSs were offered by NuCana. Citigroup, Jefferies and Cowen acted as joint book-running managers for the offering, and William Blair acted as co-manager for the offering. A registration statement on Form F-1 relating to these securities has been filed with the U.S. Securities and Exchange Commission (the "SEC") and was declared effective on September 27, 2017. Copies of the registration statement can be accessed by visiting the SEC's website at www.sec.gov. The offering was made only by means of a prospectus. Copies of the final prospectus relating to the offering may be obtained by contacting Citigroup Global Markets Inc., c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by phone at (800) 831-9146, Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, or by telephone at (877) 547-6340 or by e-mail at Prospectus_Department@Jefferies.com, or Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attention Prospectus Department or by phone at (631) 274-2806. This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state or jurisdiction.

September 2017

27

Aduro Biotech Announces First Patient Dosed in Phase 1 Clinical Trial of Personalized Immunotherapy pLADD Based on Proprietary Neoantigen Technology

Trial has begun in Patients with Metastatic Colorectal Cancer Who Have Few Treatment Options BERKELEY, Calif., Sept. 27, 2017 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, announced today that the first patient has been dosed in the Phase 1 clinical trial (see www.clinicaltrials.gov, identifier NCT03189030) designed to evaluate the safety and tolerability of a personalized live, attenuated double-deleted Listeria monocytogenes (pLADD) immunotherapy for adults with metastatic colorectal cancer that is microsatellite stable (MSS). The personalized immunotherapy has been engineered with patient-specific neoantigens that were identified and selected using state-of-the-art neoantigen identification technology developed by Hanlee Ji, M.D., associate professor of medicine at the Stanford University School of Medicine. “Our pLADD program leverages our extensive capabilities relating to the use of Listeria as a delivery mechanism for cancer antigens and Dr. Ji’s innovative neoantigen technology used to identify immunogenic antigens specific for an individual patient,” said Natalie Sacks, M.D., chief medical officer of Aduro Biotech. “Together, we believe these two cutting edge technologies represent a new approach to treating patients who have relapsed following prior chemotherapy. We look forward to evaluating initial proof-of-concept in this Phase 1 clinical trial.” Clinical Design of Phase 1 PLADD Trial in Adults with Metastatic Corlorectal Cancer The Phase 1 clincial single-arm trial is designed to evaluate the safety and tolerability of a personalized immunotherapy made using patient-specific antigens and Aduro’s proprietary live, attenuated, double-deleted Listeria monocytogenes platform technology. The trial is seeking to enroll approximately 10 patients with metastatic colorectal cancer that is MSS. Patients will receive their patient-specific immunotherapy once every three weeks.

27

NuCana plc Announces Pricing of Initial Public Offering

EDINBURGH, United Kingdom, Sept. 27, 2017 (GLOBE NEWSWIRE) -- NuCana plc (Nasdaq:NCNA), a clinical-stage biopharmaceutical company focused on significantly improving treatment outcomes for cancer patients by applying its ProTide ™ technology to transform some of the most widely prescribed chemotherapy agents into more effective and safer medicines, announced today the pricing of its initial public offering of 6,667,000 American Depositary Shares ("ADSs") at a price to the public of $15.00 per ADS, for total gross proceeds of approximately $100.0 million, before deducting underwriting discounts and commissions. Each ADS represents one ordinary share of NuCana. In addition, NuCana has granted the underwriters a 30-day option to purchase up to 1,000,050 additional ADSs at the initial offering price to cover over-allotments, if any. All of the ADSs are being offered by NuCana. NuCana's ADSs have been approved for listing on the NASDAQ Global Select Market and are expected to begin trading under the symbol "NCNA" on September 28, 2017. The offering is expected to close on October 2, 2017, subject to customary closing conditions. Citigroup, Jefferies and Cowen are acting as joint book-running managers for the offering, and William Blair is acting as co-manager for the offering. A registration statement on Form F-1 relating to these securities has been filed with the U.S. Securities and Exchange Commission (the "SEC") and was declared effective on September 27, 2017. Copies of the registration statement can be accessed by visiting the SEC's website at www.sec.gov. This offering is being made only by means of a prospectus. A copy of the final prospectus relating to the offering will be filed with the SEC and may be obtained, when available, by contacting Citigroup Global Markets Inc., c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by phone at (800) 831-9146, Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, or by telephone at (877) 547-6340 or by e-mail at Prospectus_Department@Jefferies.com, or Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attention Prospectus Department or by phone at (631) 274-2806. This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state or jurisdiction.

26

Aduro Biotech Announces Advancement of ADU-S100 into Global Combination Trial With PDR001 for the Treatment of Solid Tumors and Lymphomas

First Patient Dosed in Phase 1b Clinical Trial BERKELEY, Calif., Sept. 26, 2017 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, announced today that the first patient has been dosed in the Phase 1b dose escalation and dose expansion clinical trial (see www.clinicaltrials.gov, identifier NCT03172936) designed to evaluate the safety and efficacy of ADU-S100 (also known as MIW815), a novel STING pathway activator, in combination with PDR001, Novartis’ investigational PD-1 checkpoint inhibitor, for the treatment of advanced/metastatic solid tumors or lymphomas. The trial, which is being conducted in collaboration with Aduro’s partner, Novartis, is expected to enroll approximately 175 patients at sites located in the United States, Europe, Canada, Australia and Japan. “We are pleased with our early progress in the ongoing Phase 1 dose escalation trial of ADU-S100 as a single agent and are eager to expand our investigation into a separate clinical trial to evaluate the effect of ADU-S100 used in combination with the PDR001 checkpoint inhibitor,” said Natalie Sacks, M.D., chief medical officer of Aduro Biotech. “As a leader in STING activation, we look forward to gaining more insight into the potential therapeutic application of this novel combination therapy.” Clinical Design of Phase 1b ADU-S100 (MIW815)/PDR100 The Phase 1b multi-center, open-label study is designed to evaluate the safety and efficacy of ADU-S100 (MIW815) in combination with PDR001 in patients with accessible solid tumors or lymphomas. The trial will evaluate two treatment schedules of ADU-S100 in dose escalation. One group will receive a fixed dose of intravenous PDR001 on day 1 and an intratumoral injection of ADU-S100 three times in a 28-day cycle. Another group will receive a fixed dose of intravenous PDR001 on day 1 and intratumoral injection of MIW815 (ADU-S100) on day 1 of every 28-day cycle. Once the maximum tolerated dose and/or recommended dose for expansion is determined, the expansion part of the study will open.

19

Amylyx Pharmaceuticals Receives FDA Orphan Drug Designation for AMX0035 for the Treatment of Amyotrophic Lateral Sclerosis

September 19, 2017 09:00 AM Eastern Daylight Time CAMBRIDGE, Mass.–(BUSINESS WIRE)–Amylyx Pharmaceuticals, Inc., announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AMX0035, an oral therapeutic in clinical development for the treatment of amyotrophic lateral sclerosis (ALS). The Company recently began a Phase 2 clinical study, the CENTAUR trial, to evaluate the safety and efficacy of AMX0035 in ALS patients. The U.S. Orphan Drug Act provides incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the U.S. Orphan drug designation by the FDA conveys up to seven years of marketing exclusivity if the compound receives regulatory approval, and offers various development incentives, including tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design. Background on AMX0035 AMX0035 is a combination of two small molecules, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). Each compound has demonstrated strong efficacy in several cellular and animal models of ALS. When individually tested in ALS clinical trials, PB and TUDCA have both shown safety, tolerability, and preliminary signs of efficacy. In preclinical trials, Amylyx has demonstrated a synergistic effect between the two compounds, suggesting that the combination may have improved efficacy compared to the individual agents. About the CENTAUR Trial CENTAUR is a 24-week, randomized, double-blind, placebo-controlled Phase II clinical trial in 132 participants with ALS. The trial’s primary objectives are to evaluate the safety and tolerability of AMX0035 and assess the drug’s impact on disease progression as measured by the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) over the 24-week study period. The trial will also evaluate the impact of AMX0035 on isometric strength as measured by ATLIS, respiratory function, and exploratory biomarkers. There will also be an open label extension to the trial so that all enrolled patients will have the option of continuing treatment once the 24-week treatment period concludes.

18

Green Biologics’ Renewable Chemicals Granted Kosher and Halal Certification

Company’s n-butanol and acetone can now serve as ingredients for the growing markets for kosher and halal consumer goods Ashland, Virginia and Abingdon, Oxfordshire U.K. (September 18, 2017) – Green Biologics, Inc., the U.S. subsidiary of Green Biologics Ltd., a U.K. industrial biotechnology and renewable chemicals company, announced today that it has achieved kosher and halal certification for the 100 percent biobased n-butanol and acetone, produced at the company’s commercial facility in Little Falls, Minnesota. These certifications enable Green Biologics’ chemicals to serve as ingredients for personal, homecare and food products. “The kosher and halal certifications that have been granted to our Little Falls, MN manufacturing facility will further enable us to build partnerships within the major food, cleaning and cosmetic brands, to name a few,” said David Anderson, Vice President of Marketing at Green Biologics. “These certifications are becoming popular, not only for those with religious motivations, but also to the wider market as they are being used as an effective method for identifying high quality goods.” The recent certifications are an important milestone for Green Biologics’ BioPure™ n-butanol and acetone, which are entirely produced using renewable feedstocks. Consumers and retailers are increasingly demanding products produced from natural or sustainable ingredients. Both n-butanol and acetone can also serve as additives and solvents in personal care, cosmetic, and both household and industrial cleaning products. For cosmetics, while acetone is most often used as a nail polish remover, BioPure™ n-butanol can be combined with acids derived from natural plant based oilsto produce a wide array of 100 percent renewable butyl esters. These esters can be utilized in natural lipsticks, moisturizers, and nail polishes. Butyl esters are also utilized as aroma chemicals in fragrance products and flavor additives within the food industry. In addition to the kosher and halal certifications, provided by the respective OK Kosher and Islamic Services of America certifying bodies, Green Biologics is also a member of the American Chemistry Council (ACC), and its commercial facility, Central MN Renewables LLC, participates in the ACC’s Responsible Care® program. The company’s n-butanol and acetone have received 100 percent biobased, USDA BioPreferred® certification.

13

NuCana Announces Interim Data from First-In-Human Study of Transformative Anti-Cancer Agent, NUC-3373

Received Best Poster Presentation Award at ESMO 2017 Edinburgh, United Kingdom, September 13, 2017 (GLOBE NEWSWIRE) – NuCana plc announced the presentation of interim first-in-human data from a Phase 1 clinical study of NUC-3373, its ProTide transformation of the active anti-cancer metabolite of 5-fluorouracil (5-FU), at the European Society for Medical Oncology (ESMO) 2017 Congress held earlier this week in Madrid, Spain. Interim pharmacokinetic (PK) and pharmacodynamic (PD) data were presented at ESMO 2017, and the presentation was awarded “Best Poster” status by the organizing committee. Key findings from the study included PK and PD data from 21 patients with 10 different advanced solid tumor cancer types and indicated that NUC-3373 has the potential to have greater activity and an improved safety profile as compared to 5-FU. The results indicated a linear and reproducible dose-response across all dose ranges as well as a well-tolerated safety profile. After a short administration, plasma half-life of NUC-3733 was 9.7 hours, as compared to the 8 to 14 minute half-life associated with 5-FU. Evidence of rapid and efficient intracellular activity were noted, with high levels of FUDR-MP, the active anti-cancer metabolite of 5-FU detectable in cells 5 minutes after infusion and still detectable after 48 hours. NUC-3373 bound to and inhibited the target enzyme, thymidylate synthase, and depleted the pool of dTMP, a nucleotide necessary for DNA replication, 2 to 4 hours after administration. Additionally, NUC-3373 did not generate the key toxic metabolites of 5-FU (F-BAL or dhFU) intracellularly or in the patient’s plasma. “NUC-3373 appears to have considerable advantages over 5-FU, with much higher levels of the active, anti-cancer metabolite, that may overcome key cancer resistance pathways and allow for a more favourable safety profile and a more convenient dosing regimen,” said Dr. Sarah Blagden, Associate Professor of Experimental Cancer Medicine at the University of Oxford and Chief Investigator of the study. “5-FU remains one of the most important and widely used anti-cancer drugs in the world. This interim analysis of NUC-3373’s Phase 1 data strongly supports its continued development.” Hugh Griffith, NuCana’s Chief Executive Officer, said: “NUC-3373 is our second product candidate that uses our proprietary ProTide technology with the goal of improving the efficacy and safety of important anti-cancer agents. We are excited to see these interim data and especially gratified that it was chosen for a Best Poster award at ESMO 2017. These data support that NUC-3373 may have a key role to play in the treatment of cancer and we look forward to continuing its rapid development, along with the development of other product candidates in our pipeline.”

12

NuCana Announces Positive Results of NUC-1031 (Acelarin®) in Advanced Ovarian Cancer at ESMO 2017

Acelarin combination with carboplatin achieved 96% disease control rate and 39% response rate in patients with recurrent ovarian cancer Edinburgh, United Kingdom, September 12, 2017 (GLOBE NEWSWIRE) – NuCana plc, a clinical-stage biopharmaceutical company developing a portfolio of novel anti-cancer medicines called ProTides™, announced the presentation of data from its recently completed trial of its lead product candidate, NUC-1031 (Acelarin®), at the European Society for Medical Oncology (ESMO) 2017 Congress held September 8th-12th, 2017 in Madrid, Spain. Results from NuCana’s Phase 1b trial showed that Acelarin, when combined with carboplatin, was well tolerated and demonstrated clinical activity in women with recurrent platinum-resistant and platinum-sensitive ovarian cancer. An overall response rate of 39% was observed amongst the 23 evaluable patients, including 1 (4%) who achieved a complete response, 8 (35%) with partial responses, and 13 (57%) with stable disease that lasted at least 12 weeks. This yielded an overall disease control rate of 96% (22 patients). The responses were durable, with an average progression free survival of 7.4 months. The most common adverse events across all dose levels were neutropaenia, leukopaenia and thrombocytopaenia. No unexpected adverse events were observed with the combination to date. All patients in the study were previously treated with an average of three prior chemotherapy regimens. Seventeen of the evaluable patients were either refractory or resistant to their last platinum-containing regimen. “The fact that the Acelarin combination with carboplatin achieved these results in heavily pre-treated and platinum-resistant patients clearly demonstrates Acelarin is a very active agent,” said Dr. Sarah Blagden, Associate Professor of Experimental Cancer Medicine at the University of Oxford and Chief Investigator of the Phase 1b study. Professor Blagden added, “importantly, the favorable toxicity profile of Acelarin enabled us to combine it with carboplatin at AUC 5, whereas with gemcitabine, carboplatin has to be given at AUC 4. Thus, we are able to deliver both Acelarin and carboplatin at their optimal dose.” Hugh Griffith, NuCana’s Chief Executive Officer, said: “The high disease control rate and durable responses achieved with the combination of Acelarin and carboplatin are exciting. We remain focused on advancing Acelarin’s development for the treatment of patients with ovarian cancer as well as exploring its use for the treatment of other solid tumours."

07

Project ALS and Amylyx Enter Collaboration to Test AMX0035

Pre-clinical studies at Columbia University’s Motor Neuron Center Complement Amylyx Phase 2 Clinical Studies of Lead Therapeutic Compound New York, NY and Cambridge, MA (September 7, 2017) – Project ALS and Amylyx Pharmaceuticals today announced a collaboration to undertake pre-clinical studies with Amylyx’s oral compound AMX0035 to advance the understanding of the compound’s neurobiological effects. The studies to be conducted at the Project ALS Pre-Clinical Core at Columbia University’s Motor Neuron Center will complement the company’s recently initiated Phase 2 clinical program of AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). The Project ALS Pre-Clinical Core at the Columbia University’s Motor Neuron Center has established a unique integrated and standardized platform for the testing and validation of new therapeutic strategies in recognized experimental models of ALS and for biomarker discovery. The Core, developed in collaboration with Project ALS, will accelerate the translation of new promising therapies to patients by facilitating speedy testing of new therapeutic leads discovered by laboratories studying motor neuron biology, genetics and genomics. “The collaboration will bring together a promising therapeutic candidate for a devastating disease with leading edge, fundamental neuroscientific research at Columbia,” said Valerie Estess, director of research for Project ALS. “The studies in this collaboration will provide greater insight into the neurobiological effects of AMX0035, and hopefully optimize its beneficial effects.” The collaboration is an outgrowth of previous studies by The Project ALS Pre-Clinical Core at Columbia University of tauroursodeoxycholic acid (TUDCA), one of the components of AMX0035. “We look forward to evaluating AMX0035 in models of ALS. It is exciting that TUDCA has independently shown promise in both our labs and in studies conducted by the company,” said Drs. Hynek Wichterle and Serge Przedborski, co-directors of The Project ALS Pre-Clinical Core and tenure faculty in the Departments of Pathology and Neurology at Columbia University. AMX0035 is a combination of two drugs, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). Previous studies of PB and TUDCA as individual agents demonstrated efficacy in cellular and animal models of ALS. AMX0035 has been shown to synergistically prevent nerve cell death and neurotoxic inflammation, hallmarks of ALS, in preclinical models. In addition, PB and TUDCA have been individually tested in ALS clinical trials and each demonstrated safety, tolerability, and preliminary signs of efficacy. AMX0035 recently entered Phase 2 clinical development to evaluate its safety and efficacy in ALS. The trial, called CENTAUR, is a 24-week, randomized, double-blind, placebo-controlled study in 132 participants with ALS. More information on the CENTAUR trial can be found at www.clinicaltrials.gov, NCT03127514 and at www.Amylyx.com/Trials. “Amylyx is very excited to partner with Project ALS and the researchers at Columbia’s Motor Neuron Center to advance these experiments. We hope these studies will provide valuable insights into both AMX0035 and ALS biology that will ultimately improve the lives of patients with ALS,” said Kent Leslie, Chief Scientific Officer of Amylyx.

05

CellCentric exploring CCS1477 and p300/CBP inhibition for haematological cancers

September 5th 2017 CellCentric has signed an agreement with Laura Pasqualucci, Professor of Pathology and Cell Biology at Columbia University Medical School, New York USA, to explore the relevance of the company’s drug Candidate, CCS1477, in treating certain haematological cancers. This is in addition to existing collaborations with Professor Tim Somervaille, Consultant Haematologist and Senior Group Leader at the Cancer Research UK Manchester Institute and Professor Brian Huntly, Consultant Haematologist and Professor of Leukaemia Stem Cell Biology at the Cambridge Stem Cell Institute. CCS1477 is a novel, potent and highly selective p300/CBP inhibitor candidate drug. The compound is progressing to the clinic with a lead indication for the aggressive form of prostate cancer (CRPC). Additionally, it has been known for some time that inhibiting p300/CBP has relevance to treating other tumour types, including blood, bladder and lung cancers. However, to date, there has been a lack of clinical-quality compounds with which to explore such new applications. CellCentric has been exploring a range of potential clinical indications for CCS1477 through its own research, as well as through multiple collaborations with leading academic groups. The latest agreement will see Laura Pasqualucci test CCS1477 in a series of in-house haematological cancer models. She has a long-standing interest in epigenetic-related targets and their role in cancer. Alongside Riccardo Dalla-Favera, she originally identified p300/CBP as common target mutations in both diffuse large B cell lymphoma (DLBCL) and follicular lymphoma (FL). Tim Somervaille separately, is exploring the potential for CCS1477 in acute myeloid leukaemia (AML). Tim has a long-standing interest in the mechanisms and therapies in myeloid malignancies. Brian Huntly is an internationally recognised expert in the role of p300/CBP in haematopoietic stem cell function and will be researching the effects of CCS1477 in his unique collection of haematological cancer models. CellCentric is also collaborating with academic groups for CCS1477’s primary indication, castrate resistant prostate cancer. Inhibiting p300/CBP drives down levels of the androgen receptor, its adaptive splice variants, as well as key biomarkers including PSA. Further mechanistic aspects are being explored by CellCentric with Karen Knudsen (Director, Kimmel Cancer Center, Thomas Jefferson University); Johann de Bono (Drug Development Unit Director, ICR, Royal Marsden, London); and Luke Gaughan (NICR, University of Newcastle). CCS1477 is in the final stages of toxicological evaluation prior to submission of a Clinical Trial Application (European equivalent of an IND) to initiate human testing. First patient dosing is anticipated early in 2018, under the leadership of Johann de Bono. Commenting, Nigel Brooks, CellCentric’s Director of Translational Science, said ‘Recent data has strongly supported the potential of CCS1477 and p300/CBP inhibition for the treatment of tumours beyond castrate resistant prostate cancer. We are delighted to be investigating these further with multiple world-leading researchers. We look forward to further validating our strategy for testing CCS1477 for multiple tumour types, as CellCentric moves to the clinic.’

August 2017

28

Synlogic Completes Merger with Mirna Therapeutics

- Synlogic Commences Trading on NASDAQ Capital Market under Ticker Symbol “SYBX” - Combined Company has Approximately $100 Million in Cash and Cash Equivalents Following Transaction Close - Company Strengthens Board of Directors with addition of Michael Powell, Ph.D., and Richard Shea CAMBRIDGE, Mass. & AUSTIN, Texas--(BUSINESS WIRE)--Aug. 28, 2017-- Synlogic, Inc. and Mirna Therapeutics, Inc. today announced that the proposed merger of the two companies has closed following the approval of Mirna’s stockholders received on August 24, 2017. The merged company will operate as Synlogic, Inc. and will focus on advancing Synlogic’s platform for development of Synthetic Biotic™ medicines, which are designed using synthetic biology to genetically reprogram probiotic bacteria to treat metabolic and inflammatory diseases and cancer. Synlogic will commence trading on the NASDAQ Capital Market today, August 28, 2017, under the ticker symbol “SYBX”. “The close of this merger, in combination with our recent financing, provides Synlogic with significant resources to move forward as a public company and realize our goal of developing a new class of living medicines that have the unique potential to compensate for dysfunctional pathways in serious diseases,” said Jose Carlos Gutiérrez-Ramos, Ph.D., Synlogic’s president and chief executive officer. “Earlier this year we initiated the first human clinical trial of our lead Synthetic Biotic investigational medicine for hyperammonemia, and in the first half of 2018 we expect to initiate clinical trials of a second Synthetic Biotic medicine candidate for the treatment of phenylketonuria (PKU). Our solid financial position enables us to continue to execute on advancing our novel development programs through the clinic to demonstrate the therapeutic potential of our Synthetic Biotic platform.” The combined company’s cash and cash equivalents, as of immediately following the closing of the merger, is approximately $100 million. This includes proceeds from a Series C financing that closed immediately prior to the signing of the merger agreement in which Synlogic raised approximately $42 million from leading biotechnology investors, including Aju IB Investment, Ally Bridge Group, Arctic Aurora LifeScience, CLI Ventures, Perceptive Advisors, Rock Springs Capital, and other undisclosed new investors. Existing investors, Atlas Venture, Deerfield, New Enterprise Associates (NEA), and OrbiMed also participated in the financing. As a result of the closing of the merger, Synlogic stockholders and option holders own, or have rights to acquire, approximately 82 percent of the combined company, and former Mirna stockholders own approximately 18 percent of the combined company.

24

Apellis Pharmaceuticals Announces that APL-2 Met its Primary Endpoint in a Phase 2 Study in Patients with Geographic Atrophy, an Advanced Form of Age-Related Macular Degeneration

Statistically Significant Slowing of Disease Progression Seen at 12 Months LOUISVILLE, KY., August 24, 2017 - Apellis Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing a platform of novel therapeutic compounds for the treatment of autoimmune diseases, today announced that its complement C3 inhibitor, APL-2, met its primary endpoint in a Phase 2 clinical trial (FILLY) in patients with geographic atrophy (GA) associated with age-related macular degeneration (AMD). At 12 months, APL-2, administered monthly via intravitreal injection, showed a 29% (p=0.008) reduction in the rate of GA lesion growth compared to sham. With every other month administration, a 20% (p=0.067) reduction was observed. Additionally, in a post hoc analysis, a greater effect was observed during the second six months of the study: a reduction in growth rate of 47% (p<0.001) with monthly administration, and a reduction of 33% (p=0.01) with every other month administration. The most frequently reported adverse events in the study eye were associated with the injection procedure. A higher incidence of exudative AMD was observed in the treatment groups, predominantly in subjects with a history of exudative AMD in the fellow eye, and was managed with the administration of standard-of-care therapies. “We are very excited about the results of this study,” said Cedric Francois, MD, PhD, founder and chief executive officer of Apellis. “In addition to demonstrating a statistically significant slowing of disease over 12 months, APL-2’s effect appears to increase in the second six months of the study, slowing down the rate of degeneration by almost half. We plan to move forward with Phase 3 studies as soon as possible.” David Boyer, MD, of Retina-Vitreous Associates Medical Group, said, “These results are very exciting for all people afflicted with dry AMD with geographic atrophy. It is currently an untreatable condition, and the reduction of the progression of atrophy in this trial offers new hope for vision maintenance for our patients.” Results, including an analysis of genetic markers, will be presented at an upcoming major medical meeting.

11

Aiming at Soliris, Apellis loads for PNH phase III with $60M series E

By Marie Powers, News Editor, BioWorld Apellis Pharmaceuticals Inc. extended its string of financings with a $60 million series E preferred stock round led by Sectoral Asset Management that included new investors Sofinnova, Vivo Capital, F-Prime Capital Partners, investment funds advised by Clough Capital Partners LP and Venbio Select. Existing investors Morningside Ventures, Cormorant Asset Management, Venbio Global Strategic Fund and Epidarex Capital also participated in the financing. In conjunction with the financing, Maha Katabi, private equity partner at Sectoral Asset Management, is set to join the Apellis board, chaired by Morningside co-founder Gerald Chan. The raise represents the largest to date for the Louisville, Ky.-based company, topping last year’s $47 million series D. Late in 2015, Apellis considered a run at the public markets to fund development of its lead program, APL-2, but the timing was “unfortunate,” according to Cedric Francois, co-founder, president and CEO, with the company’s S-1 dropping at about the same time the markets were doing the same. Still, Apellis, founded in 2009, has had little trouble securing the funds to keep moving forward, raising $92 million prior to the series E. “The real purpose of this round was to broaden our existing syndicate by bringing in investors that can help us take this company to the next level,” Francois told BioWorld. The round, which came together in less than three months, positions Apellis to move its complement C3 inhibitor into a phase III program in paroxysmal nocturnal hemoglobinuria (PNH) and to advance studies in other indications. A synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b, APL-2 blocks all three pathways of complement activation (classical, lectin and alternative) with a particularly high potency against the alternative pathway. In December 2016, Apellis disclosed interim results from two phase Ib open-label, dose-escalation trials of the self-injected APL-2, reporting that the drug reduced the breakdown of red blood cells in patients when given daily as a monotherapy or as an add-on to standard-of-care therapy, the intravenous Soliris (eculizumab) from Alexion Pharmaceuticals Inc., of Cheshire, Conn. At the time of the report, 15 patients dosed across the two trials had completed a month of dosing, and five had completed more than three months of treatment. With APL-2 (270 mg) as a monotherapy, three of three PNH patients achieved a reduction in levels of the biomarker lactate dehydrogenase (LDH) to below the standard for control in PNH (500 U/L). With APL-2 (270 mg) as a Soliris add-on, six of six previously transfusiondependent PNH patients did not require transfusions during the study, and five of six achieved hemoglobin levels within the normal range for healthy people. “We might be on to something,” Francois said at the time. (See BioWorld Today, Dec. 5, 2016.) The phase III program will help to advance that thesis by confirming whether inhibition of C3 offers advantages over drugs like Soliris, which targets complement C5. Apellis plans to begin enrolling the first phase III in PNH in the fourth quarter, “where, ultimately, the goal will be to find out if APL-2 can be superior to Soliris in its mechanism of action,” Francois said.

10

Apellis Pharmaceuticals Announces $60 Million Series E Financing

Funding Will Advance Trials of APL-2 in Paroxysmal Nocturnal Hemoglobinuria LOUISVILLE, KY., August 10, 2017 - Apellis Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing a platform of novel therapeutic compounds for the treatment of autoimmune diseases, today announced the closing of a $60 million Series E preferred stock financing led by Sectoral Asset Management. New investors include Sofinnova, Vivo Capital, F-Prime Capital Partners, certain investment funds advised by Clough Capital Partners L.P., and venBio Select. Existing investors Morningside Ventures, Cormorant Asset Management, venBio Global Strategic Fund, and Epidarex Capital also participated in the financing. The proceeds of the financing will be used to initiate Phase 3 trials with APL-2 in paroxysmal nocturnal hemoglobinuria (PNH), a rare, chronic, life-threatening blood disorder, and advance development in other indications. Positive Phase 1b results in PNH were recently reported, and, later in 2017, results are expected from a Phase 1 study in autoimmune hemolytic anemia and a Phase 2 study in geographic atrophy, an advanced form of age-related macular degeneration. “Data generated to date with APL-2 across a range of clinical indications support our belief that C3 inhibition has great potential to deliver novel and commercially successful treatments,” commented Cedric Francois, MD, PhD, founder and Chief Executive Officer of Apellis. "This group of top tier investors supports our vision of developing APL-2 to its full potential, and of providing a range of differentiated treatments to patients with serious unmet medical needs,” he added. “The remainder of this year will see significant milestones for Apellis.” As part of this financing, Maha Katabi, PhD, CFA, Partner, Private Equity at Sectoral Asset Management will join Apellis’ Board of Directors. “APL-2 is a molecule with the potential to disrupt current treatment paradigms in a range of complement-mediated conditions,” noted Dr. Katabi. "We are pleased to support Apellis, and help the Company reach its next growth objectives.”

09

Aduro Biotech Bolsters Intellectual Property Position in STING Field with Two New Patents

BERKELEY, Calif., Aug. 09, 2017 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, today announced that the United States Patent and Trademark Office has issued two patents to Aduro related to the activation of the STING (Stimulator of Interferon Genes) Pathway. The first, U.S. Patent 9,724,408, jointly owned with the University of California and licensed to Aduro Biotech, covers ADU-S100 (MIW815), an investigational STING Pathway Activator being developed as a cancer therapy, and certain methods for its use. The second patent, U.S. Patent 9,695,212, claims methods of using certain additional cyclic dinucleotides (CDNs) to induce STING-dependent immune responses. “As leaders in cutting edge research and development of STING technologies, it is imperative that we protect our discoveries and expertise related to the modulation of the STING pathway,” said Stephen T. Isaacs, chairman, president and CEO of Aduro Biotech. “These issued patents strengthen and broaden the intellectual property coverage for fundamental aspects of our clinical CDN, ADU-S100, in addition to protecting our innovative research recognizing the importance of STING as a central mediator in triggering the development of tumor-specific immunity. We have demonstrated preclinically that activating STING promotes an inflamed tumor phenotype, which led to a profound anti-cancer immune response. ADU-S100 is the first STING Pathway Activator to enter into the clinic and is in an ongoing Phase 1 dose escalation study in patients with cutaneously accessible tumors.” The issued patents bolster our patent portfolio for STING, where Aduro owns and licenses families of patents and patent applications for compounds that target the STING receptor, which would expire, or if issued would expire, between 2025 and 2038. These include both U.S. and international patent applications.

08

Amylyx Pharmaceuticals Doses First Patient in Phase II Clinical Trial of AMX0035 for the Treatment of Amyotrophic Lateral Sclerosis

Cambridge, MA, August 8, 2017 – Amylyx Pharmaceuticals, Inc., in collaboration with The ALS Association, ALS Finding a Cure®, and the Massachusetts General Hospital, today announced that the first patient was dosed in the CENTAUR study, a Phase II clinical trial assessing the efficacy and safety of AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). The Neurological Clinical Research Institute (NCRI) at the Massachusetts General Hospital (MGH) and the Northeast ALS Consortium (NEALS) will oversee the trial, and 25 NEALS member medical centers across the United States will participate. CENTAUR (“Combination of Phenylbutyrate and Tauroursodeoxycholic Acid”), which will enroll 132 participants nationwide, was designed in collaboration with patients and caregivers along with clinical leaders in ALS. The design is intended to facilitate trial access, ease of trial site visits, patient engagement, and to measure both functional and biochemical changes over time. Amylyx plans to provide an open label extension to people who participate in CENTAUR. “The CENTAUR trial builds on promising results with AMX0035 in preclinical studies and clinical experience with the drug’s two components,” said Sabrina Paganoni, M.D., Ph.D., Principal Investigator for the study and physician at the Massachusetts General Hospital and Spaulding Rehabilitation Hospital. “The team at NCRI worked closely with Amylyx to design an innovative trial that will evaluate AMX0035’s safety and efficacy, advance our understanding of several biomarkers, and provide insights into ALS disease biology.” Merit Cudkowicz, M.D. MSc, Chief of Neurology at MGH, added, “The design and launch of the CENTAUR trial reflects a close collaboration between academia, industry, ALS organizations, and patients. Collaborative efforts among key stakeholders is essential to developing treatments for this complex and serious disease.” The study will be the first multi-center, interventional trial to utilize ATLISTM (“Accurate Test of Limb Isometric Strength”), a novel technology developed by Pat Andres, DPT, to quantitatively assess changes in patients’ muscle strength. PET imaging of neuroinflammation and blood-based markers of neurodegeneration will also be utilized to assess changes in disease pathology. Support for CENTAUR comes in part from a $2.96 million grant from The ALS Association and ALS Finding A Cure®. Amylyx CEO and co-founder, Joshua Cohen commented, “We are tremendously grateful for the support we have received from the ALS community.” Justin Klee, President and co-founder added, “The study reflects the outstanding progress the Amylyx team and our collaborators made to build a strong clinical program aimed at ultimately improving the lives of patients with this devastating disease.” The Amylyx therapeutic candidate, AMX0035, is an oral combination of sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA) that works synergistically to reduce neuronal death and inflammation. Both PB and TUDCA have been individually tested in previous ALS clinical trials; both compounds demonstrated safety, tolerability, and preliminary signs of efficacy.

02

CollegeVine Completes First Six Months of 2017 With Accelerating Momentum

CAMBRIDGE, Mass., Aug. 2, 2017 /PRNewswire/ -- CollegeVine, a national online provider of student mentoring and college admissions guidance for high schoolers and their families, today reported company growth and capital raises through the first half of 2017. CollegeVine doubled its Series A to $6.7m in March with $3.6m additional funds from Morningside Technology Ventures and University Ventures. "Our momentum is a direct result of addressing the changes needed in the college admissions process," said Jon Carson, CollegeVine CEO and 30 year edtech entrepreneur veteran. "Our platform and process of having near-peer mentors (college students) work with high schoolers and their families helps lessen the stress of the college admissions process. This year is extremely pivotal in our progress because we are disrupting the industry with the use of data to help students match with schools that are best for them. " These milestones help validate CollegeVine's value proposition and proliferation in the edtech industry, but what particularly highlights its success is the amount of students CollegeVine has helped find and be admitted to the best schools for them, exceeding the market acceptance rate close to four times. "One of the most important things CollegeVine was able to provide for my family and my teenager was a sense of regaining control over a stressful process," said Denise Hoffner, parent of a CollegeVine student from the 2017 admissions class. "Navigating the college admissions process is tough when you have little to no guidance from the high school, but CollegeVine helped my son stay organized and created a path to help him get admitted to some of his top choice schools. Having stellar, incisive assistance took pressure off of us individually and collectively." Continuing to demonstrate its leadership, innovation, and commitment to high school students and their families, CollegeVine has made major strides in achievements including: Doubled capital raise: CollegeVine closed an additional $3.6 million Series A funding round in April to bring its total series A financing to 6.7M. Release of Mentorship 2.0: To better serve the growing needs of its customer base, CollegeVine extended its mentorship program by increasing the number of meetings between mentors and mentees, providing students with access to more interest-specific resources such as exclusive office hours with experts in a student's particular field of interest. Total offering of scholarship dollars: CollegeVine's robust client base has received a total offering of $14,500,000 in scholarship dollars for the 2016-17 college acceptance season. Progressive pro bono program: The company's ongoing devotion to its pro bono program saw a 90 percent acceptance rate to a top 50 school, as well as a 71 percent acceptance rate to Ivy League or equivalent schools. Company recognition: CollegeVine has been recognized as one of top 50 startups to watch by Built in Boston, slated to make a huge impact in tech over the next 12 months. The company's momentum has been featured in key media outlets including The Wall Street Journal, Business Insider and Boston Business Journal. Incremental inbound inquiries and website traffic: CollegeVine has seen a steady uptick in website traffic by 3.5 times and a six-time growth in inbound inquiries over the previous year. Expansion of leadership: The company has grown its leadership team with the addition of Aaron Rissler, who previously ran sales at 2020 Onsite. Upon joining CollegeVine at the beginning of the year, Aaron has led the team to beating the sales plan every month so far in 2017. CollegeVine also recently added Stephen Smith, co-founder of Naviance, to its Advisory Board. Smith joins Doug Williams, CTO of iSpecimen on the Advisory Board, and will provide guidance for continued growth of CollegeVine as a strategic advisor.

01

Voicebox Launches Linguistic Code-Switching Capability

The technology allows speech systems to draw from one language to learn another. By Phillip Britt - Posted Aug 1, 2017 Semantic parsing and similar techniques can revolutionize natural language processing, according to Phil Cohen, chief scientist for artificial intelligence at Voicebox, a provider of voice technology for the automotive, mobile, home, and internet-connected devices markets. However, acquiring training data for each domain in each supported language remains cumbersome and expensive. This limitation has become increasingly significant as companies demand intelligent, conversational voice applications to support their global product strategies. Late last week, Voicebox's Advanced Technologies Team announced a significant innovation that reduces the burden of data collection. More important, the innovation helps overcome the challenge of parsing multilanguage utterances, known in linguistic circles as code-switching. Voicebox's approach applies the learning of one language to another. The team evaluated utterances in German, English, and a mix of both in a single utterance. They developed a neural network model, trained on both English- and German-only sentences. As part of a single semantic parsing process, this model transfers information from one language to the other, thus leveraging English data to reduce the amount of data needed for German. As a result, performance on each language improved. The team also evaluated utterances that could contain a mix of both languages and weren't in any of the training data. Results were similarly impressive. "We're excited about the ground-breaking benefits this innovation brings to users," Cohen says. To date, voice navigation systems, for example, have struggled with multilingual use cases. This has been a serious usability issue in multilanguage regions, such as Europe or Asia, where code-switching is common. For example, a French person driving through Germany might ask a question in French. A person who is multi-lingual might ask a question or phrase a sentence in a mix of languages, using the language that best describes what he or she is discussing. "My grandmother did this constantly," Cohen says. "She said, 'It sounds better this way.'" Being able to offer multiple language speakers a multilanguage speech recognition solution in connected cars has been a problem for automotive companies ever since the first inception of connected cars at the end of the last decade, according to Cohen. But the Voicebox development is only the first step in providing a true multilinguistic speech recognition system, he adds. A large company specializing in voice recognition would need to develop a comprehensive voice recognition system that then the auto manufacturers would need to add to their systems. The new voice recognition systems come out every two years, according to Cohen. The new 2017 versions are out now. So the next versions will be out in 2019, with the next generation after that out in 2021. Cohen says it is possible for a multilanguage voice recognition system to be ready for the 2019 models, and hinted that a company like Voicebox could develop it. While he wouldn't go as far to say that customers were demanding such a system, he says that once such a system is available, customers will come to expect they have a car equipped with that capability.

July 2017

30

Talking Cars like Toyota and Can Now Talk in Multiple Languages with help from VoiceBox

July 30, 2017 by Lynn Walford Voicebox Advanced Technologies Team announced an innovation that reduces the burden of data collection while producing impressive, industry-leading results. More importantly, the innovation helps overcome the challenge of parsing multi-language utterances, known in linguistic circles as “code-switching.” Voicebox software is shipping in all new 2018 Toyota cars. Voicebox’s approach applies the ‘learning’ of one language to another. The team evaluated utterances in German, English and a mix of both in a single utterance. They developed a neural network model, trained on both English- and German-only sentences. As part of a single semantic parsing process, this model transfers information from one language to the other, thus leveraging English data to reduce the amount of data needed for German. As a result, performance on each language improved, yielding state-of-the-art accuracy comparable to the likes of Google. The team also evaluated utterances that could contain a mix of both languages and weren’t in any of the training data. Results were similarly impressive.

27

Voicebox Announces Pioneering Work in AI to Streamline Development Process and Improve User Experience

Innovative transfer learning method using multiple languages to be presented at the prestigious Conference on Natural Language Learning (Association for Computational Linguistics) July 27, 2017 09:10 PM Eastern Daylight Time BELLEVUE, Wash.--(BUSINESS WIRE)--AI techniques such as semantic parsing hold the promise of revolutionizing natural language processing. However acquiring training data for each domain in each supported language remains cumbersome and expensive. This limitation has become increasingly significant as companies demand intelligent, conversational voice applications to support their global product strategies. Today, the Voicebox Advanced Technologies Team announced a significant innovation that reduces the burden of data collection while producing impressive, industry-leading results. More importantly, the innovation helps overcome the challenge of parsing multi-language utterances, known in linguistic circles as “code-switching.” Voicebox’s approach applies the ‘learning’ of one language to another. The team evaluated utterances in German, English and a mix of both in a single utterance. They developed a neural network model, trained on both English- and German-only sentences. As part of a single semantic parsing process, this model transfers information from one language to the other, thus leveraging English data to reduce the amount of data needed for German. As a result, performance on each language improved, yielding state-of-the-art accuracy comparable to the likes of Google. The team also evaluated utterances that could contain a mix of both languages and weren’t in any of the training data. Results were similarly impressive. “We are excited to present this innovation at CoNLL,” said Dr. Phil Cohen, Chief Scientist, AI at Voicebox. “And we’re more excited about the ground-breaking benefits this innovation brings to users.” To date, voice navigation systems, for example, have struggled with multi-lingual use cases. This has been a serious usability issue in multi-language regions, such as Europe or Asia where code-switching is common. For example, a French person driving in Germany may say, “Wie viele Universitäten ont Paris?” (How many universities does Paris have?) Similarly, it is very common for a person in China to ask, “播放歌曲 Let It Be.” “At the end of the day,” Cohen added, “the goal of Voicebox Advanced Technologies research is to bring practical value and a better experience to users.”

25

Kezar Life Sciences Announces Successful Completion of Phase 1a Study and Secures $50 Million in Series B Financing

Funding to support continued development of KZR-616, a first-in-class immunoproteasome inhibitor, and advance discovery program targeting the protein secretion pathway SOUTH SAN FRANCISCO, Calif., July 25, 2017 /PRNewswire/ -- Kezar Life Sciences, a private, clinical-stage biopharmaceutical company developing novel small molecule therapeutics targeting the immunoproteasome and the protein secretion pathway, announced today that it has closed an oversubscribed Series B investment round of $50 million led by Cormorant Asset Management and Morningside Venture. New investors participating in the financing include Cowen Healthcare Investments, Pappas Capital, Chiesi Venture Fund, Qiming Venture Partners and Bay City Capital, joined by additional existing investors EcoR1 Capital, Omega Funds, and Aju IB Investment. Kezar has now raised a total of $73 million since its inception in 2015. Kezar also announced the successful completion of the Company's Phase 1a healthy volunteer study with their lead drug candidate, KZR-616, a first-in-class selective immunoproteasome inhibitor. The placebocontrolled study enrolled a total of eighty-two subjects, sixty-one of which received single or multiple doses at varying dose levels. The trial identified multiple doses that resulted in desired levels of inhibition of the immunoproteasome and that were well tolerated with repeat dose administration. Additional results from the study are anticipated to be presented at the American College of Rheumatology's Annual Meeting in San Diego in November. "We are pleased with the results of our healthy volunteer study, and grateful for the support of such an excellent group of investors to finance our upcoming clinical trials," said John Fowler, CEO of Kezar Life Sciences. "The strong demand for this financing reflects growing excitement for the potential of immunoproteasome inhibition in treating autoimmune disorders and recognizes the clear leadership position enjoyed by Kezar." Christopher Kirk PhD, President and CSO, added, "These initial clinical trial results demonstrate that KZR-616 is achieving the desired levels of immunoproteasome inhibition that correlate with anti-inflammatory activity seen in laboratory models. By selectively targeting the immunoproteasome, we believe we can avoid the toxicities associated with dual proteasome inhibitors like VELCADE™ and KYPROLIS™, as exhibited by the early safety findings from this study."

19

Stealth BioTherapeutics Initiates Phase 2/3 Study of Elamipretide in Patients with Barth Syndrome

Study will evaluate elamipretide in rare genetic mitochondrial disease characterized by muscle weakness, cardiac abnormalities, recurrent infections and delayed growth BOSTON – July 19, 2017 – Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing therapeutics to treat mitochondrial dysfunction, today announced the initiation of TAZPOWER, a Phase 2/3 study evaluating elamipretide in patients with Barth syndrome. Barth syndrome is a rare genetic mitochondrial disease, caused by mutations in the TAZ gene, and characterized by cardiac abnormalities, skeletal muscle weakness, recurrent infections and delayed growth. “The severe problems experienced by patients with Barth syndrome are caused by misshapen and dysfunctional mitochondria, which reduce the energy production in the affected tissues. The resulting muscle weakness can lead to severe fatigue, heart failure and death,” said Stealth Chief Medical Officer Doug Weaver. “In this study, we hope to show that elamipretide may have clinical benefit by improving function in these affected mitochondria.” TAZPOWER is a randomized, double-blind, placebo-controlled crossover study that will evaluate the effects of daily elamipretide treatment in a minimum of 12 patients with genetically confirmed Barth syndrome. Patients will be randomized to one of two sequence groups: 12 weeks of single daily subcutaneous injections of elamipretide in Treatment Period 1, followed by 12 weeks of treatment with placebo in Treatment Period 2, with a four-week wash-out period between periods, or vice versa. The primary endpoint is change in distance walked during the six-minute walk test. Secondary endpoints include functional assessments, patient-reported outcomes and safety. “Our understanding of Barth syndrome and how it manifests has evolved significantly, but current treatment efforts are still limited to the management of symptoms,” said Hilary Vernon, M.D., Ph.D., assistant professor of Pediatrics at McKusick-Nathans Institute of Genetic Medicine at Johns Hopkins University and the primary investigator for the study. “The initiation of TAZPOWER represents an important milestone in the potential development of a disease-specific treatment option.” TAZPOWER builds upon Stealth BioTherapeutics’s existing rare disease and cardiorenal programs, including three ongoing Phase 2 studies in adults with heart failure (IDDEA-HF, PROGRESS-HF, RESTORE-HF). “This study underscores our commitment to develop elamipretide for the treatment of rare genetic mitochondrial diseases,” said Stealth Chief Executive Officer Reenie McCarthy. “The cardiovascular and skeletal muscle symptoms affecting this population share a common thread with symptoms experienced in diseases commonly associated with aging, such as heart failure, in which mitochondrial dysfunction contributes to the clinical pathology.”

19

STEALTH BIOTHERAPEUTICS SHARES PROMISING DATA FROM MITOCHONDRIAL MYOPATHY TRIAL

July 19, 2017 - Recently, at the 2017 UMDF Mitochondrial Medicine Symposium, Stealth BioTherapeutics shared very encouraging results from its second clinical trial for primary mitochondrial myopathy, the MMPOWER-2 study. Stealth is a Boston-based biopharmaceutical company developing therapeutics to treat mitochondrial dysfunction; its lead drug under investigation is elamipretide (previously known as Bendavia). Primary mitochondrial myopathy (PMM) is a genetically-acquired mitochondrial disease characterized by signs and symptoms of myopathy (debilitating muscle weakness, easy fatigability, exercise intolerance and pain). The Phase 2 MMPOWER-2 trial was conducted to evaluate safety, tolerability and efficacy of treatment using elamipretide in 30 adult (ages 16-65) patients with PMM. An overall assessment of the top-line MMPOWER-2 results showed benefit across multiple endpoints and is supportive of continuation toward a Phase 3 study in this patient population. Patients enrolled in MMPOWER-2 previously participated in Stealth’s MMPOWER trial, designed to assess dosing, which demonstrated a dose-dependent improvement in distance walked in the 6-Minute walk test (6MWT) after 5 days of once daily intravenous administration of elamipretide or placebo. In MMPOWER-2, patients were randomized to once daily subcutaneous administration of elamipretide or placebo for a longer period of time (four weeks), and then, after a washout period, received the opposite treatment during a second four-week dosing period. Patients, their doctors and investigators at Stealth were unaware of which group patients belonged to during the trial and the subsequent assessments. This type of study design, known as a randomized, double-blind, placebo-controlled crossover study, is considered a “gold standard” in clinical trials evaluating investigational drugs. Randomized controlled trials can be challenging in rare and orphan diseases because there simply are not as many patients available to participate, and there tend to be as many differences as there are similarities between patients who may have the same diagnosis (also known as heterogeneity). Multiple endpoints, or outcomes, were considered and evaluated during the MMPOWER-2 trial in addition to the 6MWT, including the Neuro-QoL (Quality of Life in Neurological Disorders) measurement system, and a new patient-reported outcome tool, the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA), developed by Stealth specifically for this patient population. Other tools and measures for safety, tolerability and functional assessment were also applied. Chuck Mohan, Executive Director of the United Mitochondrial Disease Foundation, comments “Clinical trials for rare diseases are challenging and we are very supportive of Stealth for their efforts to evaluate potential therapeutics in a meaningful, rigorous and scientific manner. The progress of the MMPOWER-2 trial provides hope to our global mitochondrial disease community. ” The primary objective of the study was to evaluate the effect of a single daily subcutaneous dose of elamipretide for four weeks on a PMM patient’s walking distance, as measured by the 6MWT. Patients receiving elamipretide walked an average of 20 meters more than those receiving placebo at the end of the 4-week dosing period. In addition, those patients who were the most impaired at baseline demonstrated the greatest improvement. Data gathered and analyzed for several other secondary endpoints is also informative and promising. Treatment with elamipretide resulted in statistically significant and clinically meaningful improvements in the Neuro-QOL Fatigue Short Form score and in the PMMSA Total Fatigue score. In other words, patients showed overall improvement in fatigue and in symptoms which impacted their daily quality of life and functional abilities. Patients also reported a statistically significant improvement in the PMMSA identified symptom most bothersome to them individually, such as tiredness, muscle weakness, muscle pain, abdominal discomfort, vision problems, or balance problems. Data also continued to demonstrate safety and tolerability of elamipretide, with the most common side effect being redness or itching at the injection site. Kira Mann, CEO of MitoAction, is enthusiastic about these findings. “These results are very promising for patients struggling with fatigue, pain, and weakness due to mitochondrial myopathy. On behalf of patients and families with mitochondrial disease, we are excited about this data and continue to be supportive of future elamipretide studies.” Stealth continues to be committed to developing mitochondrial therapeutics and engaging with the mitochondrial disease clinician and patient/family community. In March of 2017, Stealth initiated RePOWER, a prospective, observational study of patients with mitochondrial myopathy. RePOWER will assess approximately 300 PMM patients, ages 16-65, and will gather information about current symptoms, quality of life, functional abilities and medical history. Findings from MMPOWER, MMPOWER-2 and RePOWER will together help establish and inform a Phase 3 trial to continue to evaluate the potential efficacy, safety and tolerability of elamipretide. Stealth plans to launch its Phase 3 trial around the end of this year. The executive director of the Foundation for Mitochondrial Medicine, Laura Stanley, has a child with mitochondrial disease. She states, “Results such as these demonstrate why it is so important for every patient and family with primary mitochondrial disease to be involved in these very important and groundbreaking studies. Together we are pioneering this field.”

13

UK’S SILICON PHOTONICS CONSORTIUM WELCOMES £4.8 MILLION BOOST TO R&D INNOVATION FUNDING

Rockley Photonics matches government funding from the Engineering and Physical Research Council (EPSRC) in a ‘Prosperity Partnership’ with the University of Southampton Oxford, UK, 13 July 2017 – Rockley Photonics Limited, the UK’s leading integrated technology and systems innovator for next-generation networks, will, over the next five years, match government funding from the EPSRC, and form a ‘Prosperity Partnership’ with the University of Southampton’s Optoelectronics Research Centre (ORC). An official announcement about this partnership, and additional projects involving 10 universities and businesses operating in key areas of innovation, will be made today at 18:00hrs (Thursday 13th July) by Jo Johnson, Minister of State for Universities, Science, Research and Innovation at a special event at BT’s HQ, 81 Newgate Street, London. The money, totalling around £4.8 million, will be used to support research into how silicon photonics technology can be used to improve data centre communication networks and support a new integrated photonics platform for broader mass market applications. Dr Andrew Rickman, founder, CEO and chairman of Rockley Photonics said: “We are honoured to have our technology and business endeavors supported and recognised by the EPSRC in this extraordinary funding initiative.” He continued: “Rockley Photonics and The University of Southampton team has a long-standing history of working together. Our partnership, built up over many years, demonstrates the value of relationships between academia and commercial enterprises such as ours. It gives us the ability to combine resources and academic excellence and focus on ground-breaking, early-stage technologies, such as silicon photonics. “Research in to this area is progressing quickly, and in the very near future, this game-changing, disruptive technology will soon have a huge impact on the future architecture design of large data centres; improve the power and compute capacity of new consumer devices and provide robust sensing solutions in a variety of industry sectors, such autonomous vehicles and biomedical. All this at dramatically lower cost and with considerably less power requirements.” Graham Reed, Professor of Silicon Photonics at Southampton, commented: “Andrew Rickman, Chief Executive Officer of Rockley Photonics, is the world’s leading entrepreneur in this field. We have a long history of working together and this collaboration is almost the perfect fit for the remit of the Prosperity Partnerships – a truly mutual relationship between university and industry. “At Southampton, our expertise and facilities offer a unique environment for silicon photonics research and innovation. One of the world’s most pressing problems is how to handle our relentless desire for more data and we are striving to make significant improvements.”Professor Nigel Titchener-Hooker, Professor of Biochemical Engineering at UCL, who chaired the panel that approved the Prosperity Partnerships projects, said: “The quality of the applications we reviewed was outstanding. The breadth of applications too speaks to the diversity of UK industry and to the alignment between the UK’s very best academic teams and our industrial base. “The grants promise to create a series of exciting avenues of research leading to industrial implementation. It's a wonderful new example of how, in partnership, we can harness our collective capabilities to strengthen our economy and once again underscores the importance of ongoing investment in the higher education research base.”

13

Aduro Announces Milestone Achieved Relating to Collaboration with Merck for Development of Anti-CD27 Antibody for the Treatment of Cancer

Investigational Immunotherapy on Track to Enter Clinical Development in 2018 BERKELEY, Calif., July 13, 2017 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, announced today that the company has earned a $2.0 million milestone payment under its worldwide licensing agreement with Merck (known as MSD outside the United States and Canada) for work supporting the preparation of an Investigational New Drug Application (IND) for its anti-CD27 antibody. “We are pleased with the progress being made on the pre-clinical development of the anti-CD27 antibody, which was created with our proprietary B-select monoclonal antibody technology and selected by Merck for continued development,” stated Hans van Eenennaam, Ph.D., chief operational officer, Aduro Biotech Europe. “Aduro’s anti-CD27 antibody targets the CD27 co-stimulatory pathway, an important component in stimulating an anti-cancer immune response. We look forward to working closely with Merck in their effort to advance this promising and novel approach in the field of immunotherapy into clinical development.” About CD27 and Aduro’s Anti-CD27 Antibody CD27 is a co-stimulatory receptor expressed on different immune cells, such as T-lymphocytes and NK (natural killer) cells. It has been recognized as having an important role in priming, enhancing and sustaining a productive anti-cancer (CD8 T-cell) adaptive immune response. In preclinical studies, anti-CD27 activation in combination with immune checkpoint inhibition has demonstrated the ability to achieve complete tumor eradication. In 2014, Merck, through a subsidiary, entered into a worldwide license agreement for the development and commercialization of CD27 antibody agonists. Aduro’s anti-CD27 antibody, which was identified with its proprietary B-select monocolonal antibody technology, targets a functional epitope on CD27 demonstrating potent activation of the CD27 co-stimulatory pathway in pre-clinical studies. As a part of the worldwide license agreement, and in addition to payments received, including the $15 million up-front payment, Aduro is eligible to receive future development, commercial and net sales milestone payments. In addition, Aduro is eligible to receive royalties in the mid-single digits to low teens based on any net sales of the product, if it is approved for marketing.

11

Why These Harvard Dropouts Hired a 60-Year-Old Yale-Educated CEO

A trio of college entrepreneurs were hauling in $100,000 a month from their Ed Tech startup. They dropped out and gave up the CEO role to a 60 year old serial entrepreneur. Here's why it's working. By Peter Cohan, Founder, Peter S. Cohan & Associates@petercohan Just because you drop out of Harvard it doesn't mean you're the next Bill Gates or Mark Zuckerberg. And a group of mostly Harvard undergraduates who dropped out to run their startup realized that they'd be better off picking as their CEO a 60-year-old serial entrepreneur who had sold three of his companies for about $200 million. In so doing, their startup is riding the crest of a breaking wave that could propel them all to great success. This comes to mind in considering my July 10 conversation with Jon Carson who since January 2016 has been CEO of CollegeVine, a service that connects undergraduate so-called Near-Peer Mentors (NPMs) with high school students seeking college admissions and their parents. As Carson explained, "CollegeVine is a virtual high school guidance platform that enables families of public school students to supplement their high school guidance to get the same quality of guidance as students at private schools.The company has been growing revenue at 3.5 times per year and between 2015 and 2017, the number of NPMs it employs has [skyrocketed] from 80 to 600," Carson said. CollegeVine's "secret sauce is connecting high schoolers and their families to a network of highly talented college students at a range of top schools. These consultants have expertise in navigating the high school journey, proven academic success, and successfully completed the college admissions process. Due to age proximity they relate easily to teenagers. These consultants go through intensive training and are leveraged by proprietary, data-driven decision making tools," he explained. Carson was a startup mentor at the Harvard Innovation Lab (iLab) where he met the Harvard undergraduates who were running CollegeVine. "We had a speed dating session in which 10 mentors met with 10 startup teams. We chose each other. CollegeVine was doing $100,000 in revenue a month from their dorm room. I thought they had something special and helped them to think through the trade-offs of whether they should drop out of college to pursue the idea," said Carson. The CollegeVine founders were three friends from New Jersey who met in seventh grade. Two of them were at Harvard and one was at U. Chicago. They did not get much help from their high school college counselors but with help from slightly older students they figured out what they needed to do to get into these elite schools. Others asked for their help and their reputation spread. In the fall of 2015, they had to leave the iLab and they decided to take a leave from Harvard to work on CollegeVine -- in January 2016 they made Carson its CEO. Why drop out of Harvard? "As our company grew, we found ourselves continuously making tradeoffs between the business and school. There simply wasn't enough time in the day to really get the most out of Harvard and also achieve our goals for the company. My co-founders and I decided we wanted to focus on one thing, and at the time we were all in agreement that we were onto something pretty interesting with the company because of the strong revenue traction and clear market signals we had caught a wave. We all felt like we had stumbled upon a rare opportunity we just couldn't pass up." Perkins and his cofounders trust Carson and he trusts them. As Perkins said, "The relationship only works because there is strong mutual trust; we respect Jon's extensive experience and intelligence; Jon sees us as equals and gives us full autonomy in everything we do, with a healthy dose of his own mentorship." "The juxtaposition is incredibly powerful. We met Jon as we were approaching a pretty critical juncture for the company. The business was completely taking off and our aspirations for what it could be were scaling well beyond what any of us had possibly imagined. Jon started off as a volunteer mentor with us at the iLab," Perkins continued. Perkins believes the company is better off with Carson as CEO. "After working with Jon for a few months, we all came to appreciate a really strong team dynamic; Jon's part was to help synthesize our vision and define the trajectory for the incredible momentum we were building in all aspects of the business. From our perspective, we were onto something so powerful that we wanted someone at the table that had seen the movie a few times to add a new perspective," concluded Perkins. Carson has learned a valuable lesson from his decades of entrepreneurial experience. "It is much better to work for a startup that's catching a wave. It's easy to tell -- customers are seeking out its product and revenues are growing." That is clearly the case with CollegeVine.

07

OMNI FACILITATES THE SHARING OF STORED ITEMS

San Francisco – On-demand storage start-up Omni enables users to rent out their stored items to the local community. Omni creates an inventory of all the items stored by subscribers, photographing, identifying and categorising each one. ‘Omni gives you the ability to make any of your items available to your friends or the local community,’ Ryan Delk, vice-president of product and growth at Omni, tells TechCrunch. The items are categorised as ‘personal’ by default, but subscribers can re-label their belongings to enable them to be shared with ‘friends’ added to the platform’s network, or members of the wider Omni community. Users looking to borrow an item send a request to the item owner and state the desired dates. Users pay a monthly fee of £0.38 ($0.50, €0.44) per item to store small items and £2.3 ($3, €2.6) per item for larger pieces. There is currently no system in place at Omni that enables users to make money off their unused goods, but the company is considering whether to monetise the service, with Omni taking a proportion of the rentals. According to Omni, 29% of items in its inventory are classified as home goods and tools, 25% as apparel and 13% as sports and recreation.

June 2017

30

Far from the big hubs, Apellis is steering its rival to Alexion’s Soliris into a PhIII program

A Kentucky biotech says they’re laying the foundation for a pivotal program for their C3 inhibition therapy, which execs believe can replace Soliris in treating PNH. Louisville-based Apellis has been making progress on its lead drug far away from the spotlight that concentrates attention on the big biotech hubs. But it’s been well funded, with a $47 million D round that dropped early last year after they gave up on an IPO in chilly market waters. And the company says they’ve been nailing down hard human evidence that by moving upstream from C5 inhibition, where Soliris hits, they can do a better job in controlling anemia and transfusion dependence among patients with this extremely rare condition. This week, Apellis is reporting on two tiny studies of 3 and 6 patients. In 3 patients never treated with Soliris, investigators reported that all of them experienced a quick correction on a key biomarker for lactate dehydrogenase, or LDH. In 6 patients not responding well to Soliris, the average hemoglobin level was brought up an average of 36%, LDH was corrected and transfusions dropped from 3.4/month on eculizumab monotherapy to 0.3/month when APL-2 was added to eculizumab. And the biotech raised no unusual red flags on the safety side. As one of the world’s most expensive therapies, Soliris has inspired a range of rivals all looking to replace it with their own drug. Companies like Ra Pharmaceuticals and Akari have been on the trail, while Soliris’ manufacturer, Alexion, has been making advances with a second-gen product for their key moneymaker. ALXN1210 — an anti-C5 antibody that inhibits terminal complement for patients with paroxysmal nocturnal hemoglobinuria (PNH) — was about the only experimental product that earned much respect from new CEO Ludwig Hantson when he took over earlier this year. Apellis has attracted considerable financial support for its work. At the time it filed its S-1, the biotech reported that Morningside Venture Investments owned 32.6% of the company, making the VC their biggest investor. And they say that they’re just getting started with a lead focus on PNH, with a range of other diseases that they believe can be treated through the same pathway. CEO Cedric Francois said he found the data encouraging as he steers the company to a Phase III study in a few months. We believe that C3-inhibitor APL-2 can be the next generation PNH treatment offering patients a powerful solution to meaningfully improve their quality of life.

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Stealth casts a wide net with experimental treatment for mitochondrial diseases

Jun 30, 2017 at 1:55 PM - At first glance, primary mitochondrial diseases are an incredibly hard target for biopharma companies to pursue. There are literally hundreds of different subtypes, caused by mutations in either nucleic DNA (nDNA) or mitochondrial DNA (mDNA). Even within that, patients that carry the same mutations (genotypes) often have variable symptoms and disease characteristics (phenotypes). Perhaps that’s why there are no targeted therapies currently approved for primary forms of the disease and thus, a huge unmet need. But it doesn’t stop there. Secondary mitochondrial dysfunction is implicated in some of the greatest public health burdens facing us to do, from Parkinson’s and Huntington’s disease to heart failure. In this light, there’s a dire need to better understand and develop therapies for the cellular battery packs we call mitochondria. Boston, Massachusetts-based Stealth Biotherapeutics has a good shot at making in-roads. Its lead program seeks to address a common denominator across all primary subtypes — skeletal muscle weakness (myopathy). “Based on epidemiology, our best guess is that approximately 40,000 patients in the U.S. have what we would consider primary mitochondrial myopathy,” said Jim Carr, Stealth’s chief clinical development officer, by phone. According to Carr, myopathy, “seems to be a primary feature and you could say, chief complaint of patients with primary mitochondrial diseases.” Skeletal muscle requires huge amounts of energy when the individual exerts him or herself, which is why patients with these conditions fatigue very easily. In Stealth’s first trial, dubbed MMPOWER, participants were given the experimental drug elamipretide intravenously every day, for five days. A six-minute walk test was then conducted at the end of treatment, which registered clinically meaningful gains in the patient endurance. The major aim, however, was to determine the optimal dose. Stealth was also able to gather additional safety data, which Carr said gave the team confidence to progress. In a subsequent trial, MMPOWER-2, the same participants were invited back to build on those results. Thirty out of 36 patients signed up for a second round. “The primary objective of MMPower-2 was to find identify additional endpoints to take into Phase 3,” Carr explained. Stealth went to great lengths to understand what symptoms really impact the patients and what metrics would correlate with actual improvements in quality of life — an approach FDA encourages with rare conditions. The results from MMPOWER-2 were presented this week at the United Mitochondrial Disease Foundation (UMDF) Symposium in Washington D.C. “We were very gratified with what we saw,” Carr said. “We found additional endpoints that seemed to be very sensitive to the changes that occurred in response to the molecule.” Stealth’s drug elamipretide is a four-amino acid peptide with an affinity for cardiolipin, found in the membrane of the many mitochondria functioning within our cells. Elamipretide’s mechanism of action has not been fully elucidated, but Carr believes it has to do with the stabilization of the mitochondrial membrane. “When there’s disease, the membrane tends to lose some of it integrity and those complexes drift apart,” he explained, which comprises the mitochondria’s electronic transfer chain. “So what we think happens is that the molecule associates with cardiolipin and helps to pull the complexes together and restore normal electronic flow.” Carr said the company has also demonstrated with repeat dosing in different animal models that the drug can improve the morphology of the mitochondria, “making very sick-looking mitochondria look normal again.” With its affinity for cardiolipin, elamipretide is readily absorbed by cells. However, it doesn’t appear to impact healthy mitochondria. “It associates with mitochondria whether there is disease or not, but the drug really only has activity if the mitochondria are dysfunctional. So it’s a built-in safety mechanism,” Carr noted. The toxicity profile has been favorable thus far. In the second study, which moved from IV dosing to subcutaneous injections, a majority (80%) of patients suffered localized effects (itching, redness) at the site of administration. According to Carr, there were no serious events. “We don’t view that as a safety concern. If anything, it may affect compliance and adherence going on, so we’re very conscious of that.” It’s manageable, but one major question remains: How much of an impact will the therapy really have? It’s a very relevant question in this day and age, following the approval of some rare disease drugs that barely move the needle. In some cases, insurers are reluctant to cover them. “Of course we have to prove this definitively, but we think it’s going to make a fairly significant difference, a fairly meaningful difference to patients,” Carr said. “In fact, that’s one of the reasons we wanted to develop the patient reported outcomes; because the patients need to tell us that. To me, that’s the most important deliverable for patients with this molecule, to help them feel better and live a more normal life.” In the MMPOWER-2 study, patients improved on the six-minute walk test — albeit less emphatically than in the first trial. But they also reported less overall fatigue, less fatigue associated with activity, less muscle pain, and less muscle weakness, with “persuasive P-valuations” as Carr put it. The average age of patients in the study was 40. Imagine living for 40 years with extreme fatigue and severe physical limitations. There’s no promise of a miracle gene therapy-like effect, but a five or 10 percent improvement in energy could make a profound difference to their lives. Those patients are now rolling over into an open-label extension, Carr said, to gauge the effects of longer-term treatment. “Our hope is, certainly when we give the drug for even longer, the effects will be intensified.” The continuation is a testament to the relationship Stealth has built with U.S. and international mitochondrial patient advocacy groups. They’re all on the front line of a pivotal battle in the wider war against disease, given how important our tiny cellular energy packs are. To that end, planning for a Phase 3 trial of elamipretide is well underway.

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Elamipretide Shows Therapeutic Potential for Primary Mitochondrial Myopathy in Latest Trial Results

JUNE 30, 2017 - The investigational drug elamipretide has therapeutic potential to improve the physical capacity of patients with primary mitochondrial myopathy (PMM), according to the latest results of the Phase 2 MMPOWER-2 study. The positive results support the further clinical development of the drug to tackle this medical condition that lacks therapeutic options. These and other findings of the clinical trial were presented at the Mitochondrial Medicine Symposium 2017 held by the United Mitochondrial Disease Foundation (UMDF) in Washington DC from June 28 to July 1. “The lives of patients with primary mitochondrial myopathy, for which there are no FDA-approved treatment options, can be significantly impaired by the debilitating muscle weakness and fatigue they experience daily,” Dr. Amel Karaa, trial investigator, internist, and clinical geneticist at Massachusetts General Hospital said in a press release. “We have seen improvements associated with elamipretide in MMPOWER-2 which merit study in a Phase 3 trial.” The injectable therapy, also known as Bendavia or MTP-131, was developed by Stealth BioTherapeutics and designed to target dysfunctional mitochondria. Elamipretide restores the ability of mitochondria to serve as the cell’s power source and reduces the levels of damaging oxidative stress produced by its previous dysfunctional activity. The efficacy, safety, and tolerability of elamipretide are being evaluated in the randomized, placebo-controlled MMPOWER-2 (NCT02805790) clinical trial. All 30 participants with diagnosed mitochondrial disease included in this study had previously completed Stealth BioTherapeutics ‘s first clinical trial MMPOWER (NCT02367014). In the first trial, the distance patients were able to walk in six minutes (6MWT) improved after five days of treatments with elamipretide. This beneficial effect was found to be dose-dependent. In the ongoing MMPOWER-2, increasing the time of treatment to four weeks was associated with patients improving their 6MWT distance by an average of 20 meters compared to those receiving a placebo, although this endpoint did not reach statistical significance. However, a detailed analysis additionally showed that the treatment seemed to most benefit those who presented great physical impairment at baseline (able to walk less than 450 meters). These patients were able to walk an average additional 24 meters compared to an additional 8 meters observed in the group of patients with better physical capacity at the beginning of the trial. This observation was consistent with previous results of MMPOWER. During the trial, elamipretide treatment significantly improved Neuro-Quality of Life Fatigue Short Form score, and the Primary Mitochondrial Myopathy Symptom Assessment Total Fatigue Score — a Stealth BioTherapeutics proprietary patient-reported outcome tool — compared to the placebo group. No serious treatment-associated adverse events were reported during the trial. The most common side effect reports were mild redness or itching at the injection site. “We are highly encouraged by the MMPOWER-2 trial’s identification of endpoints to measure changes in both skeletal muscle function and quality of life issues, which are so crucial in this patient population,” said Reenie McCarthy, chief executive officer at Stealth. “These findings confirm the potential of elamipretide for these patients and help us establish and validate critical details for our planned Phase 3 study. We look forward to working closely with U.S. and European regulatory officials to finalize the design of our Phase 3 trial, which will enroll patients with primary mitochondrial myopathy caused by a variety of genetic mutations,” McCarthy added.

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Storage company Omni now lets users share their belongings with friends

Posted Jun 29, 2017 by Ryan Lawler (@ryanlawler) On-demand storage startup Omni wants to make it easier for you to have access to your favorite items without them taking up space in your closet. But now the company is taking a huge step toward making those items available to your friends, and to other people in your local community. Omni is hardly alone in the market for on-demand storage, with companies like Clutter, MakeSpace and Trove bringing those services online. But where Omni seeks to differentiate from other storage startups is in providing item-level categorization and access to its users’ stuff. When you store your stuff with Omni, it doesn’t just sit in a box or crate collecting dust in a warehouse somewhere. The company goes through the process of photographing, identifying, categorizing and adding each item to an inventory that can be managed in a mobile app. Users can choose to take items out of storage at any time, so long as they give the company at least two hours notice. That allows Omni users who like to surf or bike or golf on the weekends to keep their sporting gear in storage when they’re not using it and take it out only when they need it. But now that the company has accrued a kind of critical mass of items, it wants to allow users to make them available to friends and other people in their local community. “What we’re launching is the ability for you as the item owner to make any of your items available to your friends or to the local community,” Omni VP of product and growth Ryan Delk says. For Omni, which has itemized more than 100,000 goods in the 18 months since launch, this was always part of its master plan. “We positioned ourselves as a storage company knowing that was a Trojan horse,” Delk told me. According to him, Omni was able to accomplish this because “everything happens on the item level.” In retrospect, the plan probably should have been obvious. After all, why go through the trouble of building infrastructure required to pick up items for storage, individually tag and categorize them, and add them to a cloud database of goods unless you would then allow users to actually do something with them? Omni allows users to store small goods for $0.50 per item per month and large items for $3 a month. It also charges pick-up and delivery fees based on how soon a user wants to access something in their inventory. While it’s free to have goods picked up — unless it’s a real rush (3 hours or less) — Omni charges a $3 delivery fee for items that will be dropped off next day and $20 for items needed within 2 hours. Due to the economics of its business, the stuff you store with Omni would probably not be the same type of thing you’d throw into a box and forget about at your local self-storage warehouse. Based on its own categorization, Omni says that 29 percent of items fall in the “home goods and tools” bucket, with apparel making up another 25 percent and sports and recreation accounting for 13 percent of all goods. From those three categories alone, you could imagine an Omni user making a set of power tools available to a neighbor, letting a friend borrow a dress for an event or sharing camping or other outdoor equipment. Omni has already been testing this concept with a limited number of beta users in the Bay Area, and is now opening it up to others. All items a user has stored will by default remain private, but if they would like to share with friends or make their stored goods available to the community at large they can now easily do so.

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Stealth BioTherapeutics Presents Phase 2 Data From MMPOWER-2 Continuation Trial Supporting Phase 3 Development of Elamipretide in Primary Mitochondrial Myopathy

BOSTON ­- JUNE 29, 2017- Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing therapeutics to treat mitochondrial dysfunction, today announced results from MMPOWER-2, a Phase 2 continuation trial evaluating safety, tolerability and efficacy of treatment with elamipretide for primary mitochondrial myopathy (PMM). Detailed results from the trial were presented today at Mitochondrial Medicine Symposium 2017, the United Mitochondrial Disease Foundation (UMDF) symposium. “The lives of patients with primary mitochondrial myopathy, for which there are no FDA-approved treatment options, can be significantly impaired by the debilitating muscle weakness and fatigue they experience daily,” said Dr. Amel Karaa, trial investigator, internist and clinical geneticist at Massachusetts General Hospital. “We have seen improvements associated with elamipretide in MMPOWER-2 which merit study in a Phase 3 trial.” The overall assessment of the top-line MMPOWER-2 results shows benefit across multiple endpoints assessed and is supportive of a Phase 3 trial in this patient population. The 30 patients enrolled in MMPOWER-2 previously completed MMPOWER, Stealth’s first clinical trial in this patient population, which demonstrated a dose-dependent improvement in distance walked in the six-minute walk test (6MWT) after five days’ treatment with elamipretide. In MMPOWER-2, a longer, four-week treatment period with elamipretide was associated with an average 20 additional meters walked versus placebo during the 6MWT (p=0.08), the primary endpoint. Although the 6MWT efficacy endpoint did not reach significance, a pre-specified analysis showed that patients who were more impaired at baseline (pre-treatment 6MWT less than 450 meters) experienced a greater improvement with elamipretide (24 meters on average) than patients who were less impaired at baseline (pre-treatment 6MWT more than 450 meters; eight meters on average). This finding is consistent with observations from MMPOWER. MMPOWER-2 was instrumental in identifying additional endpoints for a Phase 3 trial. At four weeks, treatment with elamipretide resulted in statistically significant improvements in Neuro-QoL Fatigue Short Form score (4 units versus placebo; p=0.01), a validated patient-reported scale for fatigue in neurologic disorders, and in the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA, formerly the Mitochondrial Disease Symptom Assessment) Total Fatigue Score (1.7 units; p=0.0006), a proprietary patient-reported outcome tool developed by Stealth specifically for this patient population. Several other PMMSA assessments also showed significant improvements for elamipretide-treated patients, including improvement in the most bothersome symptom reported by each patient (p=0.01). The triple Timed-Up-and-Go test (3TUG; p=0.8), measuring the time it takes to stand from seating, walk six meters, sit and repeat three times, was completed in less than one minute, which may be insufficient to measure endurance-related skeletal muscle weakness and fatigue. The 3TUG test will not be included in Phase 3. Treatment with elamipretide appeared to be well tolerated, with no serious adverse events. The most common side effect was injection-site reactions (80 percent with elamipretide versus 17 percent with placebo); most were mild redness or itching. “We are highly encouraged by the MMPOWER-2 trial’s identification of endpoints to measure changes in both skeletal muscle function and quality of life issues, which are so crucial in this patient population,” said Stealth Chief Executive Officer Reenie McCarthy. “These findings confirm the potential of elamipretide for these patients and help us establish and validate critical details for our planned Phase 3 study. We look forward to working closely with U.S. and European regulatory officials to finalize the design of our Phase 3 trial, which will enroll patients with primary mitochondrial myopathy caused by a variety of genetic mutations.” In March of this year, Stealth initiated RePOWER, a multi-national pre-trial registry of patients with PMM. RePOWER will assess approximately 300 patients, ages 16-65, at a single enrollment visit, where they will complete questionnaires about their current symptoms and quality of life, perform functional assessments and share data from clinical records. Findings from MMPOWER, MMPOWER-2 and RePOWER will together help inform a Phase 3 trial to further evaluate the potential efficacy, safety and tolerability of elamipretide.

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Green Biologics Achieves REACH Certification, Sets Sights on Europe

Compliance with stringent REACH regulations positions company to further expand market share internationally Ashland, Virginia and Abingdon, Oxfordshire U.K. (June 29, 2017) – Green Biologics, Inc., the U.S. subsidiary of Green Biologics Ltd., a U.K. industrial biotechnology and renewable chemicals company, announced today that it has received REACH certification, a regulation of the European Union that promotes protection of human and environmental health from risks posed by chemicals. By registering with REACH, which stands for Registration, Evaluation, Authorisation of Chemicals, Green Biologics is now able to supply Europe with larger bulk quantities of its bio-based n-butanol. The company has also obtained pre-registration for its bio-based acetone and various derivatives, allowing it to ship up to 100 tonnes of these chemicals to Europe through June 1, 2018. This achievement underlines Green Biologics’ commitment to embracing a global view on customers and business. It also enables the company to solidify its long-term position as a supplier to customers and markets in Europe. “Although Green Biologics’ first manufacturing site is based in North America, as the only producer of bio-based n-butanol and acetone in the world there are a wide range of opportunities for our products to be adopted across Europe as replacements to their traditional petroleum-based counterparts,” said Sean Sutcliffe, Chief Executive at Green Biologics. “Europe will be a key growth market for our company and the REACH registration of our n-butanol will help us navigate the many complex regulatory requirements associated with importing chemicals into the region.” The result of more than 100 years of research and development, Green Biologics’ fermentation platform utilizes a robust library of Clostridium microbial strains as biocatalysts to produce its 100 percent biobased n-butanol and acetone. The company has plans to offer a full suite of BioPure™, 100 percent biobased, high purity, products through its patented Advanced Fermentation Process™ and third-party partnerships. Currently, all Green Biologics chemical products are being produced at the company’s commercial facility, Central Minnesota Renewables, located in Little Falls, MN, which officially began operations in December of last year. Green Biologics is a member of the American Chemistry Council (ACC) and its commercial facility, Central MN Renewables LLC, has been built to meet Responsible Care® standards. The company’s n-butanol and acetone have received 100 percent bio-based, USDA BioPreferred® certification.

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Positive Data from APL-2 Studies Show Rapid and Durable Improvements in LDH and Hemoglobin Levels in PNH

LOUISVILLE, Ky., June 29, 2017 – Apellis Pharmaceuticals, Inc. today provided an update on clinical outcomes in its two ongoing Phase 1b clinical trials with APL-2, a complement C3 inhibitor, in the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare, acquired, potentially life-threatening disease characterized by complement-mediated hemolytic anemia. Apellis is developing APL-2 as a next generation monotherapy, with the goal of resolving anemia and transfusion dependency in PNH patients on standard of care. APL-2 is being developed for newly diagnosed PNH patients as well as for patients who suffer from anemia while receiving standard of care intravenous infusions of eculizumab. Eculizumab is a complement C5 inhibitor that improves anemia in patients with PNH, but leaves up to 75% of PNH patients anemic and 35-40% transfusion-dependent. APL-2 is being evaluated in two Phase 1b clinical trials. PADDOCK is an open label safety and efficacy study of 270mg of APL-2 administered daily by subcutaneous injection to PNH patients (n=3) who have never received eculizumab. In PADDOCK, in the first month, all three patients treated with APL-2 monotherapy experienced rapid corrections in lactate dehydrogenase (LDH), a key marker of hemolytic activity in PNH, from an average of 1,615 U/L to an average of 275 U/L (1.1x upper limit of normal), a decline of 83%. PHAROAH is an open label safety and efficacy study of 270mg of APL-2 administered daily by subcutaneous injection as a complementary therapy to suboptimal responders to eculizumab (n=6), defined as hemoglobin levels (Hb) of less than 10 g/dL at screening or a history of at least one transfusion in the previous year. In the first month, average Hb levels in the six patients increased from 8.8 g/dL to 11.9 g/dL, an increase of 36%. During this period, patients also experienced rapid corrections in LDH from an average of 280 U/L (1.3x upper limit of normal) to an average of 163 U/L (0.8x upper limit of normal), a decline of 42%. After six months, the average Hb level was 11.4 g/dL, and the average LDH level continued to be normal at 184 U/L (0.9x upper limit of normal). During that same period, the average transfusion rate dropped from 3.4/month on eculizumab monotherapy to 0.3/month when APL- 2 was added to eculizumab. Notably, five of six patients on baseline were receiving higher than normal dosing with eculizumab in the form of 1,200 mg every two weeks or 900 mg every week, as opposed to the normal 900 mg every two weeks. There have been no significant drug-related safety concerns and overall APL-2 was well tolerated in both PADDOCK and PHAROAH during the six months of dosing. None of the patients experienced episodes of breakthrough hemolysis, which can occur in patients treated with C5 inhibitors. Both open label studies are ongoing and are designed to support cross-over from eculizumab to APL-2 after a brief period of add-on dosing. Later in 2017, the Company plans to switch from daily to twice per week dosing.

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Aduro Biotech Announces Initiation of Phase 2 Clinical Trial of CRS-207 in Combination with KEYTRUDA® (pembrolizumab) for the Treatment of Previously-Treated Gastroesophageal Adenocarcinoma

BERKELEY, Calif., June 29, 2017 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, today announced  the initiation of the Phase 2 clinical study designed to evaluate the tolerability, safety and efficacy of CRS-207, Aduro’s lead listeria-based immunotherapy construct (LADD), in combination with KEYTRUDA® (pembrolizumab), an anti-PD-1 therapy marketed by Merck (known as MSD outside the United States and Canada), for the treatment of patients with gastroesophageal adenocarcinoma who have failed two prior chemotherapy treatments. Clinical trial sites have been activated and the study is open for enrollment.

“Gastroesophageal adenocarcinoma is an aggressive, difficult to treat cancer for which there is currently no FDA-approved therapy for those in need of a third-line treatment option,” stated Natalie Sacks, M.D., chief medical officer of Aduro Biotech. “We are pleased to be evaluating the CRS-207/KEYTRUDA combination in this Phase 2 clinical trial for late-stage gastroesophageal cancer patients and hope to see similar synergistic anti-cancer activity as observed in preclinical studies with this investigational treatment regimen.”

The multicenter, single-arm, open-label Phase 2 study is designed to evaluate the safety and efficacy of CRS-207 with KEYTRUDA in adults with gastric, gastroesophageal junction, or esophageal adenocarcinoma who have received two prior systemic chemotherapy treatment regimens for advanced disease.  The trial will be conducted at up to 15 sites and will enroll approximately 70 patients. The primary efficacy endpoint is objective response rate, defined as the proportion of patients with either complete or partial responses.  For additional information about the study, please visit www.clinicaltrials.gov (search identifier NCT03122548).

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Newton biotech says drug targeting cell's energy center shows promise

Jun 29, 2017, 8:30am EDT - A Newton biotech says it has moved one step closer to developing the first treatment for rare genetic diseases of the mitochondria — the powerhouses of the cell that create 90 percent of the human body’s energy. Privately held Stealth BioTherapeutics on Thursday unveiled what it characterized as positive data from a Phase 2 study of its potential treatment for "mitochondrial myopathy," muscle weakness caused by a broad class of diseases. The 50-employee company, which has raised around $200 million since being founded in 2007, said that patients who received the drug fared better in a six-minute walking test than those taking a placebo. While improvement on that metric was not statistically significant, Stealth said the trial helped it to identify other potential endpoints, or objectives, for a Phase 3 study later this year. Those endpoints include the level of fatigue felt by patients, which was markedly greater for those taking the placebo. There were no serious side effects, Stealth said. In an interview, Stealth CEO Reenie McCarthy said that mitochondria have long been an intriguing target for drugmakers because of the important role they play in the body. But targeting mitochondria, which have multiple membranes, can be difficult, and many earlier approaches have produced toxic side effects. Most patients with mitochondrial myopathy are prescribed cocktails of vitamins and put on a diet high in antioxidants (think blueberries or red wine). Stealth’s drug is designed to permeate mitochondria and bind to a part of the inner membrane that plays a pivotal role in energy metabolism, McCarthy said. In June 2016, Stealth had unveiled positive data from a Phase 2 trial of the drug, called "elamipretide." The trial data reported on Thursday examined the same patients for a longer period. The company now plans to work with regulators in the U.S. and Europe to finalize the design of the Phase 3 trial, which could begin by the end of the year, McCarthy said. “With this data, we really do feel like we’ve substantially de-risked and prepared for our Phase 3 program,” she said. “We’re very encouraged by the data in a population for which there are no approved treatments.”

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Aduro Biotech Announces First Patient Dosed in Phase 2 Clinical Trial of CRS-207 in Combination with KEYTRUDA® (pembrolizumab) for the Treatment of Patients with Previously Treated Malignant Pleural Mesothelioma

BERKELEY, Calif., June 28, 2017 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, today announced that the first patient has been dosed in the company’s Phase 2 clinical trial in malignant pleural mesothelioma (MPM). The trial, which will involve approximately 35 patients, will evaluate the tolerability, safety and efficacy of CRS-207, Aduro’s LADD (live, attenuated double-deleted) based immunotherapy, in combination with KEYTRUDA® (pembrolizumab), an anti-PD-1 therapy marketed by Merck (known as MSD outside the United States and Canada), for the treatment of patients with MPM whose disease progressed following prior treatment.

“We are excited to initiate this Phase 2 trial to evaluate the combination of CRS-207 and pembrolizumab, an anti-PD-1 therapy, which we believe has the potential to be a synergistic combination therapy for patients with malignant pleural mesothelioma,” said Natalie Sacks, M.D., chief medical officer at Aduro. “Mesothelioma is an aggressive cancer with a poor prognosis and limited treatment options; currently, there are no FDA-approved therapies indicated for second- or third-line treatment. We have received Orphan Drug Designation in the U.S. and E.U. for CRS-207 for this indication, and we are committed to doing all that we can to bring new treatment options to patients facing this difficult disease.”

The multicenter, single-arm, open-label Phase 2 study is designed to evaluate the safety and efficacy of CRS-207 with pembrolizumab in adults with previously treated MPM. The trial will be conducted at up to 10 sites and will enroll approximately 35 patients who have failed one to two prior treatments. The primary efficacy endpoint is objective response rate, defined as the proportion of patients with either complete or partial responses. For additional information about the study, please visit www.clinicaltrials.gov (NCT03175172).

Earlier this year, Aduro announced a clinical collaboration with Merck, through a subsidiary, relating to the investigation of CRS-207 used in combination with pembrolizumab for the treatment of MPM. This is the second clinical collaboration formed this year between the two companies, with the first announced in January 2017 relating to the investigation of CRS-207 used in combination with pembrolizumab for the treatment of gastric cancer.

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Human Cell-Expressed Interferon beta for Stem Cell Research and Regenerative Medicine Applications

Chicago, IL. — June 27, 2017 HumanZyme Inc., a leading supplier of novel recombinant human proteins and growth factors expressed in human cells, today announced the launch of HumanKine® Interferon beta (IFN beta) expressed from HEK293 cells. IFN beta is a member of the type I family of interferons whose function is inhibiting viral infection, along with the regulation and activation in immune responses against bacteria, parasites and tumor cells. IFN beta is deficient in multiple sclerosis, and is currently used in injectable form as a treatment for this disease. According to Scott Coleridge, CEO at HumanZyme, “We are proud to be the only commercial supplier of high-quality, tag-free recombinant human Interferon beta expressed in human cells for research purposes. Our proprietary HEK293 expression system allows us to express difficult proteins in a human cell line to provide the most authentic recombinant products possible. The new IFN beta further expands our animal component-free HumanKine product line, is priced competitively, is and is also available in bulk.” Glycosylation of the Interferon beta protein by a single asparagine-linked sugar chain has been shown to be essential to its activity and stability, both in vitro and in vivo. HumanZyme’s IFN beta expressed in human cells assures native processing, glycosylation and folding of the purified protein compared to other expression systems such as bacterial, mammalian cell lines, or insect cells, preserving its biologic function and activity. HumanZyme’s HumanKine proteins are also animal-derived product free, xeno-free and carrier-free.

15

Stealth BioTherapeutics to Present Top-Line Data From MMPOWER-2 Phase 2 Continuation Trial at Mitochondrial Medicine Symposium 2017

MMPOWER-2 assessed once daily dosing, tolerability and efficacy of elamipretide in patients who completed the MMPOWER clinical trial Company to feature presentation from primary mitochondrial myopathy program BOSTON – June 15, 2017 – Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing therapeutics to treat mitochondrial dysfunction, today announced the presentation from the Company’s primary mitochondrial myopathy (PMM) program will be featured at Mitochondrial Medicine Symposium 2017, the United Mitochondrial Disease Foundation (UMDF) symposium, from June 28 – July 1, 2017 in Washington D.C. The presentation will include top-line results from MMPOWER-2, a Phase 2 continuation trial evaluating the safety, tolerability and efficacy of treatment with elamipretide for PMM in patients with genetically confirmed mitochondrial disease previously treated in the MMPOWER study. Data from the MMPOWER-2 trial will be presented at the UMDF symposium on Thursday, June 29 at 8:00 a.m. EDT. “People with PMM, for which there are no FDA-approved therapies, experience debilitating muscle weakness and severe fatigue that can make even simple daily tasks challenging. We are focused on developing products designed to address this unmet medical need with our mitochondrial medicine platform,” said Stealth Chief Executive Officer Reenie McCarthy. “We look forward to discussing the progress of our clinical programs and the MMPOWER-2 data at the UMDF symposium. The findings, together with our learnings from the MMPOWER study, will be critical in informing our plans for a Phase 3 program in this patient population.” For additional information on Stealth’s program in PMM or elamipretide, please refer to Stealth’s website. About MMPOWER-2 MMPOWER-2 is a Phase 2, randomized, double-blind, placebo-controlled crossover study to evaluate the safety, tolerability and efficacy of four weeks’ treatment with once-daily subcutaneous (SC) injections of elamipretide or placebo in thirty patients with PMM previously treated in the MMPOWER study. Subjects treated in the MMPOWER-2 study were randomized (1:1) to receive either four weeks of treatment with 40 mg elamipretide administered once daily SC in the first treatment period followed by four weeks of treatment with placebo administered once daily SC in the second treatment period, or vice versa. The two treatment periods were separated by a four-week washout period. The primary efficacy endpoint is change in distance walked in six minutes at the end of each four-week treatment period. Secondary endpoints include safety and tolerability assessments, patient-reported outcomes and global impression scales.

14

ENYO Pharma SA announces successful completion of EYP001 first in man Phase 1 study

June 14, 2017 - ENYO Pharma SA, a privately held biopharmaceutical company currently focused on developing treatments for viral infections, today announced that the Phase 1a single and multiple ascending dose trial evaluating EYP001 in healthy subjects has been completed. The results show that EYP001 was safe and well-tolerated at all doses studied in 80 subjects. The safety, pharmacokinetics (PK) and pharmacodynamic (PD) analysis are in line with established FXR biology and reported results from other early stage compounds that are in development for NASH. In particular, the levels and pattern of plasma concentrations changes of C4 (7αhydroxy4cholesten3one) and fibroblast growth factor 19 (FGF19) are consistent with FXR agonism over the dose range of 60mg up to 500mg of multiple single doses administered over 15 days. These clinical results were presented at the international liver conference in Amsterdam earlier this year. In addition, in vitro data were presented confirming that EYP001 inhibits HBV particle release similarly to Tenofovir (TFV) or Entecavir (ETV), with an additive effect when combined. Moreover, EYP001 alone inhibited viral protein (HBsAg and HBeAg) production, reduced cccDNA and pgRNA, while TFV or ETV mono-treatment had negligible effect on these HBV markers in vitro. EYP001 is a synthetic farnesoid X receptor (FXR) agonist with a favorable profile for oral therapy. The first Phase 1 study was designed to determine the safety, tolerability and pharmacokinetics of EYP001 in healthy subjects. Another ongoing Phase 1 study evaluates the safety, food effect and PK of EYP001 in subjects with chronic HBV infection. Jacky Vonderscher, Ph.D., Chief Executive Officer of ENYO Pharma SA commented: “We are pleased with the profile emerging in our Phase 1 development with EYP001. We look forward to bringing the compound into further clinical development and believe EYP001 has the potential to be explored in additional indications such as NASH.” “Throughout the clinical program completed thus far, EYP001 has demonstrated an excellent tolerability and safety profile. By using the novel approach of enriched digitized ECG analysis we also showed that EYP001 does not impact QT. This supports our focus to progress swiftly with efficacy trials, while advancing the required regulatory clinical package.” added Pietro Scalfaro, M.D., Chief Medical Officer of ENYO Pharma SA.

08

Voicebox’s Advanced Technologies Team Tackles Natural Language Understanding With New Semantic Parsing Methods

Although Voicebox Technologies offers best-of-breed Automated Speech Recognition (ASR) products, speech recognition alone does not make for a voice interface. Natural Language Understanding (NLU), the process by voice-driven systems understand and respond to a user’s commands, are what make each Voicebox product a success. For Voicebox’s Advanced Technologies team, semantic parsing is essential to building an accurate next-generation NLU system. Semantic parsing is a method of mapping a user’s language to its meaning, even in cases with complex phrasing. In other words, semantic parsing is how a voice-enabled device knows the difference between such similar commands as “what is close” and “what is the closest.” Mark Johnson, Chief Scientist of Voicebox Australia, described semantic parsing as an important advance beyond rule-based NLUs of the past. “Semantic parsing is a crucial component for Conversational Intelligence, as it permits users to say what they want the device to do—for example, ‘Send this photo to all the people in it’—rather than [forcing them] conform to a fixed set of templates or patterns.” The Advanced Technologies team at Voicebox aims to solve some of the greatest obstacles facing current NLU technology, from mixed-language speech, to rare and complex utterances. Some of these obstacles were addressed in the team’s recent research paper on multilingual semantic parsing, which Voicebox is proud to say has been accepted for presentation at the CoNLL 2017 conference. The team’s approach to multilingual parsing handles utterances known in research circles as “code-switching,” which contain a mixture of languages. For Voicebox, which has offered monolingual solutions for years, this research is essential to developing multilingual NLU technology that better serves an increasingly multi-cultural world. The research team conducted a series of code-switching experiments in semantic parsing systems, where commands were given in German, English, or a mixture of both languages in the same utterance. The researchers developed a model which transfers information from a source language to a target language in a single semantic parsing process that speeds up the overall training. They combined data from both languages, including low-frequency words, to train the model to overcome lexical complexities and understand utterances in two languages. They also tested deep-learning neural networks on code-switching data and saw similar results. Their approach achieved state-of-the art accuracy comparable to that of Google’s NLU system. The system reached 85.7% and 83% accuracy respectively, on English and German utterances. More surprisingly, the code-switching model achieved 78% exact-match accuracy on multi-lingual utterances—more than 60% better than a corresponding monolingual model—even though it was never trained on code-switching data. “[We asked] can we use the information we learned from one language to make it easier to build semantic parsers in other languages?” Mark Johnson said. “Our goal was to see if having a semantic parser in one language could reduce the amount of data required for a second or even a third language. We were able to show that we could, and significantly so.” Voicebox is tackling the fundamental research necessary to provide reliable NLU products in a variety of languages, dialects, and multi-lingual settings. Since many users are multilingual and mix foreign words into their everyday speech, the ability of NLU technology to accommodate complex speech around the world is key to building more capable and accessible conversational systems.

08

Voicebox Technologies Releases Talisman Embedded Speech Recognition Software

Voicebox Technologies, an innovator of natural language voice applications, recently released Talisman, the latest iteration of its Embedded Automatic Speech Recognition (ASR) product for automotive and embedded IoT applications. The Talisman ASR is designed for small and mid-range embedded platforms like those found in cars, smartphones, and smart appliances. It supports large recognition grammars that can handle thousands of phrases. Historically, Voicebox has worked with third-party ASR solutions. With a high-quality, robust, in-house ASR solution, Voicebox can now double its language catalogue for the Embedded ASR platform over what it introduced in 2016. New languages will include Korean, Dutch, and Portuguese, and others. The expanded catalogue will also offer improved recognition for British, Australian, and Indian English dialects. When asked about the significance of the Talisman ASR Dan Carter, Vice President of Speech for Voicebox, said “the Talisman ASR enables us to deliver the entire end-to-end voice experience, from audio to understanding what the user said, to executing their request without the engineering overhead of integrating components from multiple suppliers.” Voicebox’s Embedded ASR utilizes deep neural networks (DNN), which uses vast quantities of collected data to generate probabilistic text outputs. Voicebox trains its speech models using a GPU compute cluster to include a wide range of speakers, ensuring their voice commands won’t be compromised due to differences in pronunciation or accent. What is remarkable with Talisman ASR is that it uses the latest techniques previously found on high end server based ASR solutions on a low memory embedded platform. Combining Talisman’s excellent on-board performance in embedded systems with Voicebox’s Hybrid SDK provides the additional power of cloud-computing. When network connectivity is available we can leverage Voicebox’s cloud ASR and capture usage and performance data from Talisman. This data can be used to train better models, improving accuracy.

07

InCarda Therapeutics Announces Positive Clinical Data Supporting Development of Inhaled Flecainide for the Treatment of Symptomatic Acute Events of Paroxysmal Atrial Fibrillation (PAF)

Flecainide administered via oral inhalation is well tolerated and elicits ECG changes suggestive of rapid drug delivery to heart

Brisbane, California, June 7, 2017 – InCarda Therapeutics, Inc. (InCarda), a privately held biopharmaceutical company developing therapies for acute cardiovascular conditions via the inhalation route, today announced positive top-line clinical data from a Phase 1 study for its lead investigational product, InRhythmTM (inhaled flecainide), demonstrating rapid drug delivery to the systemic circulation to treat symptomatic acute episodes of PAF.

“Patients with PAF experience acute episodes of heart palpitations, lightheadedness, fatigue and shortness of breath caused by fast heart rate and irregular rhythm, and are at increased risk of strokes.  Many require hospital procedures such as electrical cardioversion (shock), to restore normal heart rhythm,” explained Luiz Belardinelli, MD, chief medical officer of InCarda. “The clinical study results are consistent with our preclinical findings, which suggest that InRhythmTMcould restore normal heart rate and rhythm in patients with PAF.”

InRhythmTM delivers flecainide via inhalation to achieve more rapid delivery of the drug to the heart via the lungs.  Delivery of flecainide via inhalation offers the potential for faster conversion to normal sinus rhythm and more rapid relief of PAF symptoms than either intravenous (IV) or oral flecainide.  Flecainide is approved as an oral anti-arrhythmic drug for first-line therapy of patients with PAF.  An IV formulation of flecainide is approved as first-line therapy in the EU and in selected countries for acute cardioversion of recent onset PAF.

The Phase 1 clinical study was designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of flecainide delivered via inhalation and was conducted in two parts. The first part was a double-blind placebo controlled single sequential ascending dose study evaluating three doses of inhaled flecainide or placebo in 34 healthy volunteers. The second part was a two-period crossover study comparing delivery of flecainide via inhalation with delivery via IV infusion in six healthy volunteers.

The study met its endpoints of safety and tolerability; all doses of inhaled flecainide administered (estimated lung doses of 20 to 60 mg) were found to be safe and well tolerated. Inhalation of flecainide rapidly delivered the drug into systemic circulation (within one to three minutes), yielding venous plasma levels sufficient to elicit its expected electrophysiological effects, in keeping with the therapeutic activity of flecainide.

“We are excited about these results and the potential for inhaled flecainide to safely restore normal heart rate and rhythm as well as relieve symptoms from episodes of PAF within minutes after inhalation without the need to go to a hospital or emergency room,” stated Dr. Belardinelli.

“In the US alone, atrial fibrillation affects over five million patients and results in an annual expenditure of over $26B.  Providing patients a way to treat their episodes of PAF soon after the onset of symptoms, whether at home, at work or anywhere else, should markedly improve their quality of life, make the overall management of PAF more efficient, and reduce healthcare utilization and costs,” stated Grace E. Colon, Ph.D., chief executive officer and president of InCarda. “With these exciting data in hand, we are actively preparing for a Phase 2 trial.”

01

Aduro Biotech Announces FDA Clearance of Investigational New Drug Application to Evaluate the Combination of ADU-S100 with PDR001 for the Treatment of Solid Tumors and Lymphomas

Early Phase 1 Dose Escalation Signals Support Advancement into Phase 1b in Second Half of 2017 BERKELEY, Calif., June 01, 2017 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, announced today that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug application (IND) for ADU-S100 (also known as MIW815), a novel STING pathway activator, to be evaluated in combination with PDR001, Novartis’ investigational anti-PD-1 checkpoint inhibitor, for the treatment of advanced/metastatic solid tumors or lymphomas.  The Phase 1b study is expected to be initiated in the second half of 2017. “The initial insights we have gained from the ongoing trial of ADU-S100, coupled with preclinical data that suggests an anti-cancer synergy between an anti-PD-1 checkpoint inhibitor and ADU-S100, underscores the importance of evaluating these two novel approaches to treating cancer in combination with one another,” stated Natalie Sacks, M.D., chief medical officer of Aduro.  “With the IND now cleared, working with Novartis, our STING program collaborator, we look forward to initiating a Phase 1b trial in the second half of the year to gain clinical insights into the STING/anti-PD-1 inhibitor combination for the treatment of multiple tumor types. At the same time, we will continue to evaluate the potential of ADU-S100 as a monotherapy in cutaneously accessible tumors as well as viscerally accessible tumors.” The open label, global, multicenter Phase 1b study is designed to evaluate the safety and efficacy of ADU-S100 administered by intratumoral injection with PDR001 to patients with advanced/metastatic solid tumors or lymphomas.  

May 2017

24

Liquidia Technologies Announces Positive Phase 1 Data for LIQ865, Sustained-Delivery PRINT® Formulation of Bupivacaine for Post-Surgical Pain Relief

RESEARCH TRIANGLE PARK, NC – May 24, 2017

Liquidia Technologies, Inc., today announced initial data from its LIQ865 internal clinical development program, which is a PRINT® formulation for the sustained-delivery of free base bupivacaine for post-surgical pain relief. The phase 1 trial, marking the first evaluation of LIQ865 in humans, was a randomized, controlled, double-blind study evaluating the safety, pharmacokinetic profile and pharmacodynamic response of a single-ascending dose in healthy adult males. Topline data indicate that LIQ865 doses were well tolerated and the pharmacodynamic response was consistent with a local anesthetic effect lasting for three or more days.

“According to the National Institute on Drug Abuse, a component of the National Institutes of Health, 2.1 million people in the United States suffer from substance use disorders related to prescription opioid pain relievers, many of whom began taking opioids as post-surgical patients,” said Mike Royal, M.D., LIQ865 Program Leader and Senior Vice President, Clinical Development at Liquidia. “Our intent with LIQ865 is to increase the options for long-lasting, safe, effective post-operative pain relief that can reduce the need for opioids in the early days following surgery.”

Liquidia is developing LIQ865 with the goal of providing at least three days of post-surgical pain relief with a single administration, potentially minimizing or avoiding the need for opioid analgesics. There are over 80 million inpatient and outpatient surgeries performed every year, with the majority of surgeries requiring opioids to treat moderate to severe post-operative pain.i,ii  A minority of these individuals will become long-term users and have the potential for opioid misuse and addiction.iiiiv 

“The phase 1 clinical trial results for LIQ865 further validate the remarkably broad applicability of the PRINT technology across virtually any therapeutic area,” said Neal Fowler, Chief Executive Officer at Liquidia.  “We look forward to providing additional updates on our PRINT technology-enabled clinical programs throughout 2017.”

 

17

Aduro Biotech Announces Clinical Collaboration with Merck to Evaluate the Combination of Aduro’s CRS-207 with Merck’s KEYTRUDA® (Pembrolizumab) for the Treatment of Mesothelioma

Second Phase 2 Clinical Collaboration between the Two Companies to Evaluate CRS-207/Pembrolizumab Combination BERKELEY, Calif., May 17, 2017 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, announced today the expansion of its clinical collaboration with Merck (known as MSD outside the United States and Canada) to include an additional Phase 2 clinical trial. The companies will investigate the combination of CRS-207, Aduro’s LADD (live, attenuated double-deleted) based immunotherapy, with KEYTRUDA® (pembrolizumab), Merck’s anti-PD-1 therapy, for the treatment of patients with malignant pleural mesothelioma (MPM) whose disease progressed following prior treatment. Earlier this year, Aduro announced a Phase 2 clinical collaboration with Merck, through a subsidiary, to evaluate the combination of CRS-207 with pembrolizumab for the treatment of gastric cancer. “Data from our ongoing Phase 1 clinical trial of CRS-207 with standard chemotherapy as frontline treatment for malignant pleural mesothelioma have been very encouraging, including disease control in 94 percent of patients treated with the CRS-207/chemotherapy combination,” said Natalie Sacks, M.D., chief medical officer at Aduro.  “Based on these clinical data, as well as data from preclinical studies that demonstrate synergistic activity of CRS-207 and anti-PD-1 therapy, we look forward to initiating a Phase 2 trial to evaluate the CRS-207/pembrolizumab combination in patients with malignant pleural mesothelioma who have failed prior treatment.” The multicenter, single-arm, open-label Phase 2 study is designed to evaluate the safety and efficacy of CRS-207 with pembrolizumab in adults with previously treated MPM.  The trial is expected to involve approximately 35 patients who have failed one to two prior treatments.

11

DNAtrix Oncolytic Myxoma Virus Eliminates Treatment-Resistant Cancer

Houston, TX – May 11, 2017 – DNAtrix, a clinical stage biotechnology company developing oncolytic viruses for cancer, today announced a podium presentation on the use of DNAtrix’s oncolytic myxoma virus for treatment-resistant cancer at the upcoming 2017 Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Washington, DC.

Grant McFadden, PhD, Director of the Biodesign Center for Immunotherapy, Vaccines and Virotherapy at Arizona State University, will present his pre-clinical results showing that ex vivo treatment of stem cells with myxoma virus prior to transplantation efficiently eliminates residual chemotherapy-resistant myeloma cells that remain in the transplant recipient.

Myxoma virus is an oncolytic poxvirus with the ability to selectively kill cancer cells without infecting or perturbing normal cells. More importantly, it can exploit T-cells and other white blood cells as virus “carriers,” which can be systemically delivered to target and destroy tumors.

“This report by Dr. McFadden represents years of research to uncover the remarkable cancer-targeting properties of this virus,” said Frank Tufaro, PhD, CEO of DNAtrix.  “It appears that myxoma virus could be especially effective in combination with T-cell-based therapies, such as CAR-T therapy and stem cell transplantation.”

09

Apellis Receives EMA Orphan Drug Designation for APL-2 in PNH

C3 inhibitor is in development for the treatment of PNH, both in patients not previously treated with eculizumab, and in patients who continue to experience hemolysis and require red blood cell transfusions despite receiving treatment with eculizumab LOUISVILLE, Ky., May 9, 2017 /PRNewswire/ -- Apellis Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company focused on inhibition of the complement system, announced today that the European Medicines Agency (EMA) has granted orphan medicinal product ("Orphan Drug") designation to APL-2, a complement C3 inhibitor, in the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare, acquired, potentially life-threatening disease characterized by complement-mediated hemolytic anemia. To qualify for EMA orphan designation, a sponsor must establish that the product is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting not more than 5 in 10,000 persons in the European Union (EU), and that there exists no satisfactory method of treatment of the condition that has been authorized in the EU or, if such method exists, that the product will be of significant benefit to those affected by the condition. An orphan designation by EMA will allow Apellis to benefit from several incentives offered by the EU to companies developing medicines for rare diseases, including protocol assistance, access to the centralized authorization procedure, and market exclusivity once the medicine is on the market. Cedric Francois, M.D., Ph.D., chief executive officer of Apellis, said: "This is another important milestone for the APL-2 program. We believe that APL-2 has the potential to offer an important, and hopefully improved, new treatment option for patients suffering with PNH.  The granting of orphan designation by the EMA and the resulting incentives will benefit us, both in the near term, as we continue to advance our clinical programs, and in the longer term, as we prepare to bring APL-2 to market."

08

Green Biologics Honored with Biorenewable Deployment Consortium’s Commercialization Achievement Award

Ashland, Virginia and Abingdon, Oxfordshire U.K. (May 8, 2017) – The Biorenewable Deployment Consortium (BDC) honored Green Biologics, Inc., the U.S. subsidiary of Green Biologics Ltd., a U.K. industrial biotechnology and renewable specialty chemicals company, with the organization’s Spring 2017 Commercialization Achievement Award at its spring meeting in Des Moines on May 2, 2017. Green Biologics is recognized for its leadership in the biorenewable industry and successful commercialization of renewable alternatives to n-butanol, acetone and its associated derivatives. These chemicals serve as vital components to downstream products across various industries, including CASE (coatings, adhesives, sealants and elastomers), HI&I (household, industrial & institutional cleaners), PCI (personal care intermediates), food ingredients and energy chemicals. “The Biorenewable Deployment Consortium is proud to honor Green Biologics with its Spring 2017 Commercialization Achievement Award,” said BDC Chairman and Co-Founder Ben Thorp. “Green Biologics is a great example of a company that is effectively using its technology platform to produce a wide range of sustainable feedstocks into high performance green chemicals and industrial products. Its commercial plant in Little Falls, MN is the reason that BDC has recognized Green Biologics and awarded its global team for its continued success.” Green Biologics officially began customer shipments at its first commercial facility in Little Falls, MN, in December 2016, marking the culmination of a years’-long effort to create a robust, global network of customers and partners. These relationships have enabled the company to bring its 100 percent bio-based products to numerous downstream markets and specialty applications. As a result, Green Biologics has further strengthened its position as a global renewable specialty chemicals company, and reinforced its ability to meet the needs of today’s producers and consumers, who are demanding environmentallyconscious alternatives to longstanding products. “We’re pleased to be recognized by the Biorenewable Deployment Consortium for these achievements,” said Tim Staub, Green Biologics’ Global VP Business Development, who received the award on behalf of the company. “Our global team’s tireless efforts and dedication to a greener, cleaner tomorrow made all that we have accomplished possible, and we thank all involved for their hard work. It makes us very proud to follow in the footsteps of companies like DuPont Industrial Biosciences, which received this honor last year, and we’ll continue to work hard to bring new products and innovative solutions to market.”

02

Stealth BioTherapeutics Initiates Phase 1/2 Trial of SBT-20 in Patients With Early Stage Huntington’s Disease

SBT-20 is the second investigational compound from Stealth’s mitochondrial platform

Trial results to inform development plan for targeting mitochondrial dysfunction in neurodegenerative diseases

BOSTON – May 2, 2017 – Stealth BioTherapeutics Inc. (Stealth), a clinical-stage biopharmaceutical company developing therapeutics to treat mitochondrial dysfunction, today announced the initiation of CHALLENGE-HD, a Phase 1/2 trial evaluating SBT-20 in patients with early stage Huntington’s disease. SBT-20 is an investigational tetrapeptide aimed at improving mitochondrial function by restoring the physical and biochemical properties of dysfunctional mitochondria.

“Huntington’s disease is a fatal genetic disorder in which nerve cells in the brain deteriorate over time, causing patients to have progressively limited physical and mental abilities. Research shows that mitochondrial dysfunction may play a central role in this deterioration, making the mitochondria a prime target for study,” said Stealth’s Chief Clinical Development Officer Jim Carr. “This trial examines the safety and tolerability of SBT-20 at various doses and begins to explore the possible benefit of the compound in addressing mitochondrial dysfunction in Huntington’s disease.”

CHALLENGE-HD is a two-part, randomized, double-blind, placebo-controlled trial being conducted at a single clinical site, the Centre for Human Drug Research (CHDR) in the Netherlands.  The trial is evaluating the safety, tolerability and efficacy of daily subcutaneous injections of SBT-20 in adult patients with early stage Huntington’s disease (genetically confirmed disease with Unified Huntington’s Disease Rating Scale [UHDRS] Total Motor Score of five or more and Total Functional Capacity Score of seven or more). During part one, patients are administered SBT-20 subcutaneously for seven days at one of three ascending doses (5, 15 and 25 mg). The findings will be used to select a dose for the second part of the trial, in which SBT-20 will be administered subcutaneously for 28 days. The trial aims to enroll 24 patients, all of whom will participate in both part one and part two of the trial. The trial’s primary endpoints are safety and tolerability, and secondary endpoints will measure the effect of SBT-20 on mitochondrial and motor function as well as its pharmacokinetic profile.

“The initiation of CHALLENGE-HD is a significant milestone for Stealth as our second drug candidate enters human trials in a new therapeutic area. As a leader in mitochondrial medicine, we want to pursue the full potential of mitochondria-targeted therapies, in rare primary mitochondrial diseases, common diseases of aging and now in neurodegenerative disorders. We plan to use the results from this trial to better inform our SBT-20 pipeline development plan and the broader potential of our platform in neurodegenerative disorders,” said Stealth Chief Executive Officer Reenie McCarthy.

02

Aduro Biotech Reports First Quarter 2017 Financial Results

Ten Product Candidates Advancing with $356 Million in Total Cash

BERKELEY, Calif., May 02, 2017 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (NASDAQ:ADRO) today reported financial results for the first quarter ended March 31, 2017. Net loss for the first quarter of 2017 was $21.8 million, or $0.32 per share, compared to a net loss of $28.8 million, or $0.45 per share for the same period in 2016.

Cash, cash equivalents and marketable securities totaled $356.0 million at March 31, 2017, compared to $361.9 million at December 31, 2016.

“This will be an important year for Aduro, as we generate data in our ongoing ADU-S100/STING monotherapy trial and our planned Phase 2 trial in mesothelioma, as well as look for data from Janssen’s Phase 1 trials in lung and prostate cancers evaluating LADD therapeutic candidates,” said Stephen T. Isaacs, chairman, president and chief executive officer of Aduro. “We also plan to advance our STING program into additional clinical studies in collaboration with Novartis, and the first antibody from our B-select platform, the novel anti-APRIL antibody, is expected to be cleared for clinical testing this year. With ten product candidates in our diversified portfolio and a healthy balance sheet, we are in a strong position to continue to advance our pipeline and build a leading immunotherapy company."

April 2017

24

Precision Intratumoral Delivery of DNX-2401 with the Alcyone MEMS Cannula

Houston, TX & Lowell, MA – April 24, 2017 – DNAtrix, a clinical stage biotechnology company developing virus-based immunotherapies for cancer, and Alcyone Lifesciences, a leader in neural intervention systems and advanced drug delivery, will reveal clinical data demonstrating the precise administration of DNX-2401, an oncolytic adenovirus, into brain tumors using Alcyone’s MEMS Cannula (AMCTM) at the Annual Meeting of the American Association of Neurological Surgeons (AANS) in Los Angeles, CA.

Neurosurgeon Frederick Lang, MD, FACS, FAANS, will present results from a Phase 1b study confirming reproducible delivery of DNX-2401 by the AMC into recurrent glioblastoma tumors. Infusion accuracy was determined using the “aura method,” the displacement of DNX-2401 by gadolinium.

DNX-2401 is a potent oncolytic adenovirus that replicates in tumors and activates an anti-tumor immune response. The AMC is a dual-channel, MRI-safe cannula with the smallest-in-class micro-tip, ensuring optimal and consistent drug distribution while eliminating backflow.

“The AMC cannula standardizes the efficient delivery of DNX-2401 into the tumor of patients with recurrent glioblastoma.” said Frank Tufaro, PhD, CEO of DNAtrix. “Based on these results, neurosurgeons in the US and Canada are using the AMC in our Phase 2 clinical trial of DNX-2401 with pembrolizumab (KEYTRUDA®).”

“The lessons learned from more than a decade of attempts at exploiting convection enhanced delivery (CED) for brain cancer treatment weighed critically on the design, development, and implementation of the AMC delivery platform. The drawbacks centered around sub-optimally designed devices that have poor bio-distribution characteristics as well as surgeons' inability to monitor the distribution of these drugs within the tumor,” said PJ Anand, CEO of Alcyone Lifesciences. “It is our vision that the AMC intraparenchymal delivery platform will make this modality of treatment a permanent addition to clinical management of brain malignancies.”

March 2017

31

LUQA PHARMACEUTICALS PARTNERS WITH POLICHEM SA (An Almirall Company) TO DISTRIBUTE MACMIROR (Nifuratel) IN THE PEOPLES REPUBLIC OF CHINA

March 31st 2017 Hong Kong, PRC – Luqa Pharmaceuticals (“Luqa” or the “Company”), the China focused specialty pharmaceutical company, announces that it has entered into an exclusive long-term agreement for the distribution rights of MACMIROR (Nifuratel). MACMIROR (Nifuratel) is a product used for the polyvalent therapy of vulvovaginal infections due to pathogenic micro-organisms: Candida, Trichomonas and Bacteria. The product is used in the treatment of a wide range of infections of the genito-urinary tract. The product is already registered in the Peoples Republic of China and Luqa will be responsible for the sales, marketing and distribution, with initial commercialization expected during the second half of 2017. Luqa is already firmly established in the women’s health market with both its prescription and aesthetic dermatology portfolio, which is actively promoted and distributed by the Company’s own sales force across China. Robert Braithwaite, Luqa’s CEO commented, “Coming after our recent acquisition of Arista- which expanded our commercial footprint in China, we are very pleased to announce this transaction, which extends Luqa’s growing women’s health portfolio with the commercial rights for a registered prescription drug, used in various public and private hospital settings”.

27

Cognoa raises $11.6M to continue validation, FDA submission for child development assessment app

By Heather Mack | March 27, 2017

Palo Alto, California-based Cognoa, which makes an app to assess child development, has raised $11.6 million in a round led by existing investor Morningside. This brings the company’s total funding to over $20 million.

By analyzing parent-provided information and videos of a child’s natural behavior, the Cognoa app uses machine learning to provide an assessment of whether that child is developing at the right pace, as well as to evaluate their behavioral health. The app is intended for use in children aged 18 months to 7 years old, and while it does not provide a diagnosis, parents can take the evaluation to the pediatrician. The company has completed several clinical validation studies and has been used by 300,000 families.

“The trend of using patient-reported outcomes (PROs) and machine learning to provide diagnoses has grown exponentially in the healthcare field, and Cognoa is a leader in the area of assessments for developmental and behavioral conditions” Dr. Isaac Cheng, an investor at Morningside, said in a statement. “We believe Cognoa can significantly improve the standard of care for child behavioral development and are committed to supporting the clinical validation and FDA approval of the first machine learning-based diagnostic for early diagnosis of developmental delays.”

The latest funding will be used to for additional validation studies on the path towards FDA submission as well as to expand the app's use with pediatricians, employers and insurers.

22

Launch of the HBI-120 handheld backscatter imager

Mar 22, 2017 Heuresis Corp., a privately held U.S., company specializing in advanced x-ray instrumentation, is pleased to announce the launch of the HBI-120 handheld backscatter imager. Heuresis’ HBI-120 is the world’s first one-piece handheld backscatter x-ray imager; it supports mission-critical requirements for customs and border protection, narcotics interdiction, bomb squads, VIP security, and other “soft target” protection missions. With optimum scan speeds of 15 cm (6”) per second, the HBI-120 provides one-sided x-ray images of bulk narcotics, hidden currency, explosives, ammunition and other contraband through up to 2.5 mm of steel; typical steel motor vehicle body panels are no more than 1 mm-thick. In prison environments, improvised weapons hidden in in bedding materials can be found quickly. HBI‐120 images objects with a miniaturized, shielded, 5 Watt, 120 keV, 42 microAmp, x‐ray generator and proprietary optics that makes a raster‐scanning pencil beam of x-rays to scan objects of interest. As the HBI-120 is moved over an object, a two‐dimensional backscatter x‐ray image of the object is displayed in real‐time on the imager’s high‐resolution transflective LCD touchscreen and saved in the instrument’s memory. Complete with an Android™ operating system for a broad feature set, the HBI-120 includes built-in Wi-Fi, Bluetooth™, GPS, laser scan guides, and a flashlight, all designed to help you gather and document scan images and share results quickly. Designed for the harshest work environments, the battery-operated HBI-120 is rated for use from –40 °C (–40 °F) to 60 °C (140 °F); the unit it is splash and dustproof, and rated to IP54. “The HBI-120 makes it possible for the first time to quickly find threats and contraband when much larger and more expensive truck and portal-based x-ray imaging systems are not practical or affordable. With its 120 keV x-ray generator, built-in display and ergonomic design, the HBI-120 gives law enforcement officers, customs agents and others a new tool to help them identify anomalies in vehicles, cargo and baggage.” Mr. Grodzins continued: “We have generated a lot of excitement wherever the HBI-120 has been used, particularly by narcotics interdiction teams and customs and border protection agents. From narcotics and currency to hidden weapons and bulk explosives, the HBI-120 is designed to support the global law enforcement community in their ongoing efforts to fight terrorism and combat contraband trafficking.

15

Aduro Biotech to Host Research and Development Day in New York

Company Also Invited to Ring the Opening Bell at the NASDAQ Stock Market

BERKELEY, Calif., March 15, 2017 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (NASDAQ:ADRO) today announced that the Aduro management team will host a Research and Development Day in New York City, New York, on Monday, March 27, 2017, at 8:30 a.m. Eastern Time, to review its three distinct immunotherapy platform technologies and related clinical programs.  Leading immunotherapy expert Thomas Gajewski M.D., Ph.D., leader of the Immunology and Cancer program at the University of Chicago Comprehensive Cancer Center, Director of Melanoma Oncology, and a Professor in the Ben May Department for Cancer Research, will present on the STING (Stimulator of Interferon Genes) pathway as an important new therapeutic target and speak on its potential for groundbreaking innovation in immunotherapy.

The following day, on Tuesday, March 28, Stephen T. Isaacs, chairman, president and chief executive officer of Aduro, and members of management, will ring the opening bell at the Nasdaq Stock Market.

14

InCarda Therapeutics Announces Abstract Acceptances for the Heart Rhythm 2017 Conference

San Francisco, California, March 14, 2017 – InCarda Therapeutics, Inc. (InCarda), a privately held biopharmaceutical company focused on the development and commercialization of therapies for acute cardiovascular conditions via the inhalation route, today announced the acceptance of two abstracts for poster presentations at the Heart Rhythm Society’s 38th Annual Scientific Sessions (HRS) to be held in Chicago, IL from May 10 – 13, 2017. The abstracts are: Rapid Cardioversion of Acute-Onset Atrial Fibrillation with Pulmonary Delivery of Flecainide in Anesthetized Dogs; Authored by Luiz Belardinelli, M.D., and colleagues of InCarda Therapeutics. Accelerated Conversion of Atrial Fibrillation to Sinus Rhythm by Intratracheal Delivery of Flecainide Acetate in a Porcine Model; Authored by Richard L. Verrier, Ph.D., and colleagues of Beth Israel Deaconess Medical Center, Harvard Medical School. Dr. Verrier is a well-known expert in preclinical models of cardiac arrhythmia.  He is also collaborating on InCarda’s clinical research program. “As we continue to advance the clinical development of InRhythm™ (inhaled flecainide) for acute cardioversion of recent onset atrial fibrillation (AF) in patients with paroxysmal atrial fibrillation, we are increasingly optimistic about the future of this exciting product candidate,” stated Luiz Belardinelli, M.D., chief medical officer of InCarda. “We believe that by delivery of flecainide via inhalation, we can develop a safe and effective product. We are looking forward to presenting our results at the HRS as well as at future medical conferences.” InCarda’s Lead Product for PAF Paroxysmal atrial fibrillation (PAF), the most common type of cardiac arrhythmia (abnormal heart rhythm) is characterized by rapid and irregular heartbeats that often lead to palpitations and other disabling symptoms (e.g., fatigue). Orally administered flecainide is a commonly prescribed antiarrhythmic drug. InCarda is evaluating an inhaled formulation of this drug to treat symptomatic recent onset AF.  Inhaled delivery through the lung and pulmonary vein directly to the heart offers the potential for faster conversion of PAF to sinus rhythm with rapid relief of symptoms associated with PAF, both in and out-of-hospital, along with enhanced safety.

13

Stellar Biotechnologies and Matrivax Sign Agreement to Transfer Vaccine Technology

LOS ANGELES and BOSTON, March 13, 2017 /PRNewswire/ -- Stellar Biotechnologies, Inc. (Nasdaq: SBOT), a leading manufacturer of a key protein utilized in immunotherapy development pipelines, and Matrivax Inc., a vaccine biotechnology company, today announced that the companies have entered into a technology transfer and purchase agreement related to Stellar's proprietary Clostridium difficile technology. Under the terms of the agreement, Stellar will transfer its proprietary rights and know-how of immunogens and vaccine technology for a life-threatening pathogenic bacteria known as Clostridium difficile (C. diff). Stellar advanced this technology through exploratory preclinical studies completed under an exclusive license of the patented immunotherapy technology from the University of Guelph, Canada. Stellar President and CEO Frank Oakes said that Matrivax's acquisition of Stellar's interest in C. diff technology underscores the importance of developing immunotherapy treatments for C. diff infections (CDI). "We are pleased with this endorsement of our vision for therapeutic vaccines to fight C. diff. This arrangement provides Stellar the opportunity to advance promising technology and share in successful milestones, without further capital investments," said Mr. Oakes. Matrivax CSO Kevin P. Killeen, PhD, said that, "Matrivax is excited to advance the C. diff prophylactic and therapeutic vaccine technology to the clinic. A key company goal is to develop novel therapeutic and preventative interventions targeting CDI that direct the immune system against both the pathogen and toxins, unlike many alternative approaches that only impact toxin-mediated disease symptoms. By acquiring rights to this enabling protective antigen technology, we can now advance research designed to disrupt the fundamental pathways of C. diff pathogenesis and transmission," said Dr. Killeen. For termination of its exclusive license to the patent rights, and transfer of know how related to its development work, Stellar will receive an upfront fee from Matrivax as well as a percentage of certain fees, milestone payments, sublicensing income and royalties that are paid by Matrivax to the University of Guelph in consideration of the license granted to Matrivax under the patent rights. As part of the arrangement, Stellar and the University of Guelph terminated their existing license agreement, effective March 6, 2017. The succeeding license agreement directly between the  University of Guelph and Matrivax became effective the same date. C.difficile has been categorized as an urgent threat by the Centers for Disease Control. According to published research reports, treatment costs in the United States and Europe are estimated at $7 billion annually.

10

Aduro Biotech Awarded East Bay Innovation Award for its Contributions in Life Sciences and the San Francisco East Bay Community

BERKELEY, Calif., March 10, 2017 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, announced today the receipt of the East Bay Economic Development Alliance’s (East Bay EDA) East Bay Innovation Award for its innovation within the life science industry and unique contributions to the East Bay’s prosperity and culture of innovation.

“We are honored to have been recognized by the East Bay EDA for our innovative approach to the treatment of cancer and for our efforts to support and nurture the continued prosperity and rich culture that makes the East Bay what it is today,” stated Stephen Isaacs, chairman, president and chief executive officer of Aduro. “It is the diversity of our community—the people, the academic institutions, and businesses—that support us in our mission to bring innovative new therapies to individuals battling cancer.  We are grateful to be in the position to make a positive difference in the lives of others, within the East Bay and beyond.”

07

Green Biologics Named No. 12 in Biofuels Digest’s 50 Hottest Companies in the Advanced Bioeconomy

Renewable chemicals company rises 10 spots in annual industry rankings Ashland, Virginia and Abingdon, Oxfordshire U.K. (March 7, 2017) – Green Biologics, Inc., the U.S. subsidiary of Green Biologics Ltd., a U.K. industrial biotechnology and renewable chemicals company, announced today that it was named the No. 12 hottest company in this year’s 50 Hottest Companies in the Advanced Bioeconomy (Hot 50). The rankings, which are compiled by Biofuels Digest, a leading daily news publication on the pulse of the biofuels and biochemical industries, were announced last week in Washington D.C. at ABLC 2017 and mark a significant advancement for the company as compared to last year’s competition, in which Green Biologics held the No. 22 spot. “We’re very excited about this notable rise in the rankings,” said Sean Sutcliffe, CEO of Green Biologics. “This recognition is a true testament to the progress we have made toward establishing ourselves as the speciality chemicals provider of choice for numerous global markets. In just the past year alone, we have strengthened and expanded our network of industry partners, started operations and shipments at our first commercial plant, and launched our first sustainable, consumer-facing product. This year’s ranking is proof that the industry has taken notice of our achievements and has confidence in what’s to come.” Considered an industry staple, this longstanding annual listing recognizes companies leading the charge in innovation and achievement throughout the fuels and biobased chemicals and materials industries. The rankings are developed based on a system that weights inputs from an invited panel of distinguished industry selectors, composed of CEOs, scientists and media among others, with votes from registered subscribers to The Digest and those cast through social media. This recognition is the latest in a string of prestigious industry accolades the company has received in recent months, which include being named in the 2017 Global Cleantech 100 and recognized by the 2016 Global Clean Energy Awards as the Best Renewable Speciality Chemical Company in the UK.

06

ROCKLEY PHOTONICS RECEIVES GLOBAL TECHNOLOGY INNOVATION AWARD FOR BREAKTHROUGH DATA CENTER TECHNOLOGY

Frost & Sullivan recognises Rockley’s ground breaking R&D which is poised to transform the future design of ‘hyper-scale’ data centers

Pasadena, CA and Oxford, UK, 6 March 2017 – Rockley Photonics Limited, an integrated technology and systems innovator for next-generation networks, has been recognised by leading analyst firm, Frost & Sullivan, for its ground breaking R&D in networking infrastructure technology.

Dr Andrew Rickman, Rockley’s founder and CEO, will receive the prestigious Frost & Sullivan 2017 Global Technology Innovation Award for Data Center Networking Infrastructure at a ceremony in London on 14th March.

The proliferation of Internet of Things (IoT), connected living and artificial intelligence is set to dramatically increase the demand for cloud services and faster computing capabilities within data centers. Rockley’s integrated networking technology is designed to enable large data centers to upscale their networking infrastructure so that the projected growth in compute power can be met.

Experts have suggested that data traffic in cloud data centers may require 1000 times more bandwidth than that deployed today in order to keep up with demand for internet services over the next 10 years.

According to Frost & Sullivan, Rockley is poised to play a transformational role in the future design of ‘hyper-scale’ data centers.

03

Luqa Acquires Arista, strengthening its position in China medical aesthetics market

March 3rd, 2017 Hong Kong, China - Luqa Pharmaceuticals (“Luqa”) today announced that it has entered into a definitive agreement under which Luqa will acquire all of the outstanding common stock of Arista, a company focused on aesthetic and surgical solutions. This transaction advances Luqa’s strategy of becoming a China leader in the fast growing, multi-billion dollar aesthetic market and a provider of innovative medical solutions. The new Luqa will be distinguished in the marketplace by its ability to offer a broad suite of solutions to enable healthcare professionals to achieve excellent patient results and satisfaction. The acquisition will bring the flagship product Aethoxysklerol® for the treatment of varicose veins. Luqa will offer healthcare providers a broader range of high quality treatment options, further enhancing Luqa’s customer relationships, its competitive position and enable Luqa to capitalize on opportunities to effectively launch new products from its product pipeline. “Today’s announcement is a significant next step in our journey to become the leader in China’s aesthetics market. These additions to Luqa’s strong platform strengthen our operating foundation and build on Luqa’s mission of providing innovative and effective products with customer centric solutions for the medical community and the patients we serve,” Robert Braithwaite, CEO of Luqa, said. “We are extremely pleased with this transaction, which has been strongly supported by Luqa shareholders. It ensures even greater potential for future growth in the fast growing aesthetics and medical business we focus on. With this acquisition we expand our product offering, increase our direct commercial presence in China and expand our reach to the whole of the Greater China region.” Zona Yim, Managing Director of Arista, “We are pleased to join Luqa. I believe this combination offers a platform for future growth as well as expanded opportunities for our company as a whole. I am confident that with Luqa’s energy, resources, product portfolio and robust pipeline, we will be better positioned to develop and market the solutions our customers need. ”

02

Stealth BioTherapeutics Initiates Observational Study of Patients With Mitochondrial Myopathy

BOSTON – March 2, 2017 – Stealth BioTherapeutics Inc. (Stealth), a clinical-stage biopharmaceutical company developing therapeutics to treat mitochondrial dysfunction, today announced the initiation of RePOWER, a prospective, observational study and pre-trial registry of patients with mitochondrial myopathy (MM). This study will be conducted in North America, Europe and Australia.

“We expect to use this study to help us design an interventional Phase 3 trial and further our understanding of elamipretide’s potential in this patient population,” said Jim Carr, Stealth’s Chief Clinical Development Officer. “Since mitochondrial disease has a heterogeneous clinical presentation, the results of this study will help us better characterize the disease burden impacting patients who may participate in our upcoming Phase 3 trial, as well as add to the scientific understanding of the disease. We hope that our scientific collaboration with the investigators will add to the work of others, and further the knowledge needed to find effective treatments for this disease.”

“As our mission is to be the leader in mitochondrial medicine, we’re committed to help fill the significant treatment gaps in primary mitochondrial disease, as well as in more common diseases associated with aging in which mitochondrial dysfunction is a contributory or causal factor,” said Stealth’s Chief Executive Officer Reenie McCarthy. “Severe fatigue and muscle weakness, or MM, is among the most common clinical presentations of primary mitochondrial disease, making it difficult for patients to complete simple daily tasks. Our goal with our MM program, as with all our programs, is to design clinical trials as thoughtfully as possible taking into account the clinical burden of disease on the patient population. We believe this study will help guide us as we work to develop therapies for MM and other primary mitochondrial diseases.”

February 2017

21

Frequency Therapeutics Announces a Revolutionary Small-molecule Approach to Restore Hearing Published in Cell Reports

Frequency Therapeutics, a company spearheading the movement to restore hearing by harnessing the regenerative potential of progenitor cells in the body, today announced that a team led by Frequency’s scientific co-founders published research highlighting a breakthrough small-molecule approach to regenerate inner ear sensory hair cells. Frequency is advancing the approach to develop a potentially restorative treatment for chronic noise-induced hearing loss. The paper titled, “Clonal Expansion of Lgr5-Positive Cells from Mammalian Cochlea and High-Purity Generation of Sensory Hair Cells,” is a cover feature in the journal Cell Reports, and can be accessed in the current online edition.

16

CellCentric’s novel p300/CBP inhibitor, CCS1477 advancing to the clinic

CellCentric has developed a potent, selective, orally bioavailable small molecule inhibitor of p300/CBP. These targets are key regulators of cancer cells, and specifically play a critical role in the progression of the aggressive form of prostate cancer.  Key pre-clinical efficacy data on CellCentric’s compound CCS1477 is presented today at GU-ASCO, Orlando. GU-ASCO poster 2017: http://www.cellcentric.com/gu-asco-poster-2017 Castrate resistant prostate cancer (CRPC) remains a significant unmet need, despite recent advances including the adoption of new agents such as Enzalutamide and Abiraterone.  CRPC is driven by functional androgen receptor (AR) proteins, and modified AR-splice variants that emerge. Data shared today shows that CellCentric’s candidate drug CCS1477 lowers AR and AR-splice variants, as well as the key cancer regulator c-Myc, causing complete inhibition of prostate tumour growth in vivo (22Rv1 xenograft).  PSA levels are completely suppressed, a well-known biomarker of prostate cancer, as well as AR-regulated genes such as TMPRSS2.  CellCentric is now advancing CCS1477 in to the clinic. p300/CBP are twin (paralogue) proteins that act as transcriptional co-activators, which help govern which genes are used or not used within cells. They are also active acetyl transferases that play a role in turnover of proteins within cells, including AR.  CellCentric’s drug compound CCS1477 binds to a common conserved bromodomain of p300 and CBP. Separate from prostate cancer, certain tumours develop loss of function mutations in either p300 or CBP.  It has been shown that when this occurs, the cancer cell becomes dependent on the non-mutated paralogue3.  Inhibition of the non-mutated protein can drive cancer cell death – known as synthetic lethality. Specific p300 and CBP mutations can be detected to identify patients that could benefit from a targeted drug such as CCS1477.  This opportunity represents up to 20% of lung cancer sufferers (both small cell and non-small cell) and 25% of patients with bladder cancer, as well as up to 30% of haematological cancers. These are significant areas of unmet clinical need.

14

New membrane, Giga-module to be piloted

Clean Membranes, a US-based membrane company founded to commercialize technology developed at MIT, told WDR that it will pilot its Neophil UF membrane at the town of Amherst, Massachusetts. The test will be conducted in cooperation with the department of civil and environmental engineering at the University of Massachusetts, Amherst.   According to Michael Grossman, Clean Membrane’s sales engineering manager, the Neophil membrane is a foulingresistant, PVDF hollow fiber membrane that operates in an outside-in, dead-end filtration mode. Developed jointly with Polymem and Arkema, the fibers are arranged in bundles (elements) and potted for mounting within a Gigamem module. A 24-inch (61cm) diameter Gigamem module contains 52 individual elements, while a 14-inch (36cm) diameter element contains 18 elements.   With recent water restrictions due to severe drought conditions across the state, Amherst is interested in investigating UF as a means for recovering municipal wastewater for beneficial reuse purposes, with a particular focus on the irrigation of town’s recreation fields. The pilot system will filter secondary effluent from the Amherst Wastewater Treatment Plant to demonstrate its ability to generate Class A reuse quality water.

13

ASLAN Pharmaceuticals announces first patient enrolled in phase 1 study of varlitinib in Japan

Singapore, 13 February 2017 – ASLAN Pharmaceuticals (ASLAN), a biotech company focused on the development of immunotherapies and targeted agents for Asia prevalent tumour types, today announced that they are initiating enrolment of the first patient in the phase 1 clinical trial for varlitinib (ASLAN001) in biliary tract cancer in Japan. Varlitinib is a potent reversible small molecule inhibitor of the HER-family of receptor tyrosine kinases (RTKs). The study is expected to enrol up to 36 Japanese patients. Designed in collaboration with Japanese medical experts, the open-label study is open to all patients with solid tumours and biliary tract cancer. The primary study objective is to characterise the safety and tolerability of varlitinib as monotherapy and in combination with capecitabine in Japanese patients with biliary tract cancer. This will make possible for Japanese patients join the global study in varlitinib in biliary tract cancer. Enrolment is planned at two clinical sites in Japan.

06

CollegeVine Reports Accelerating Growth, Achieves Key Milestones In 2016

CAMBRIDGE, Mass., Feb. 6, 2017 /PRNewswire/ -- CollegeVine, a national provider of student mentoring and college admissions guidance, today reported steady growth in 2016, wrapping up its fourth admissions cycle in business with the achievement of several key milestones. CollegeVine revenue grew by 326 percent from 2015—a strong indicator of the growing demand for the company's specialized services and appeal of its unique, near-peer mentorship program. Beyond the significant growth of its revenue, CollegeVine achieved numerous milestones in 2016, including: Paid Client Results – The number of CollegeVine clients rose by 300 percent over 2015, with students applying Early Action and Early Decision achieving a 46 percent acceptance rate to the Ivy League and equivalent schools – more than twice the national average; Pro Bono Program – The company continued its pro bono program, identifying and selecting 21 deserving students, who were accepted into schools such as Princeton, Caltech, and University of Michigan, Ann Arbor; Leadership Expansion – Edtech veteran Jon Carson joined CollegeVine as CEO in 2016, and the company tapped former Zipcar CTO Doug Williams and Harvard Business School professor Deepak Malhotra to join its Board of Advisors and Board of Directors, respectively; Capital Raise – CollegeVine completed a $3.1 million Series A funding round led by Morningside Technology Ventures with participation by New York-based University Ventures, after which Morningside's founder Gerald Chan also joined the CollegeVine board; Staff Growth – The company expanded its network of near-peer consultants from 80 to more than 300 college students at the nation's top universities. The company expanded its recruiting reach from a dozen campuses to over 20; Mentorship Program Launch – CollegeVine launched a robust near-peer mentorship program for high school students, with customized services for freshmen, sophomores and juniors to develop their interests and get the most out of their high school experience; New SAT Tutoring Program – To better prepare students for the SAT while addressing changes implemented by the College Board in March 2016, CollegeVine updated its SAT tutoring program, making it one of the most comprehensive in the nation; New ACT Tutoring Program – To better prepare students for the ACT, CollegeVine updated its ACT tutoring program, making it one of the most comprehensive in the nation as well; Thought Leadership – In 2016, CollegeVine management was invited to speak at multiple sessions including a keynote at GSV/ASU, the premier edtech thought leadership conference. In addition, CollegeVine founders were widely quoted as industry experts in such media outlets as U.S. News & World Report, TeenVOGUE, The Boston Globe and many others; and Successful Blogs – CollegeVine launched its Zen blog in 2016 to address the growing stress families experience throughout the admissions process, and continued its industry-leading CollegeVine blog as well, with up to 100 high-density posts per month.

03

ENVISIA THERAPEUTICS RELEASES INTERIM ENV515 (travoprost XR) PHASE 2 DATA DEMONSTRATING 11-MONTH DURATION-OF-ACTION AFTER A SINGLE DOSE IN PATIENTS WITH GLAUCOMA

RESEARCH TRIANGLE PARK, NC – FEBRUARY 3, 2017 – Envisia Therapeutics, a clinical-stage biotechnology company focused on the development of novel extended-release therapies in ophthalmology, today released an interim analysis of the second cohort of its ENV515 (travoprost XR) phase 2 trial in patients with glaucoma showing a clinically meaningful reduction in intraocular pressure (IOP) for the entire 11-month evaluation period following a single administration. ENV515 also demonstrated an IOP lowering effect comparable to prestudy topical prostaglandin analogs (XALATAN® and LUMIGAN®) and in-study topical timolol maleate 0.5% ophthalmic solution (daily eye drops). Glaucoma is the leading cause of preventable vision loss and blindness due largely in part to poor patient compliance with once-daily eye drops. “A clinically meaningful reduction in IOP over the initial 11 months indicates that ENV515 has the potential to become a once a year therapy for glaucoma patients,” said Benjamin Yerxa, President of Envisia. “We continue to enroll patients into the next cohort of the study where we are studying ENV515 dose levels that have the potential to demonstrate a duration-of-action longer than the current 11 months.”

January 2017

25

Green Biologics Recognized as a Clean Technology Leader by Two Prestigious Industry Awards

Ashland, Virginia and Abingdon, Oxfordshire U.K. (January 25, 2017) – Green Biologics, Inc., the U.S. subsidiary of Green Biologics Ltd., a U.K. industrial biotechnology and renewable chemicals company, today announced that it has been named to the prestigious 2017 Global Cleantech 100 and recognized in the 2016 Global Clean Energy Awards as the Best Renewable Specialty Chemical Company – UK. Both highly-selective awards programs honor companies worldwide for efforts aimed at tackling today’s energy challenges and contributions to a more sustainable future. Green Biologics was recognized for its Clostridium fermentation platform, which uses sustainable feedstocks to produce high-performance chemicals, such as n-butanol and acetone. These high-value chemicals provide a renewable alternative to conventional petrochemical-based commodities for a growing number of consumer and industrial applications. “We are extremely proud to receive these two distinguished clean technology recognitions that acknowledge our tireless efforts in bringing renewable chemical alternatives to market,” said Sean Sutcliffe, Chief Executive of Green Biologics. “Both accolades reinforce the industry’s confidence in our technology and are a testament to the significant progress we’ve made over the past year in strengthening our position as a global renewable speciality chemicals company.”

19

HD Biosciences Merges with WuXi AppTec

Shanghai, China – January 19th, 2017 HD Biosciences Co., Ltd. (HDB), a leading biology-focused preclinical drug discovery contract research organization (CRO), today announces its merge with WuXi AppTec, a leading global pharmaceutical, biotechnology and medical device open-access capability and technology platform. After completion of acquisition, HD Biosciences will become a wholly-owned subsidiary of WuXi, and will continue to focus on growing its core competences and providing greater services. The acquisition will further strengthen WuXi's R&D capability from target validation to lead discovery and optimization, improving and expanding WuXi's open-access enabling service platform. Founded in 2002, HD Biosciences is headquartered in Shanghai with operating facilities in Beijing and San Diego, USA. As a leading biology and preclinical service provider, its plate-based pharmacology & screening capability and AGMTM based target validation are industry leading platforms with great reputation. The company also provides hit identification, lead discovery, in vivo pharmacology and other related services. HD Biosciences has long established close and strategic partnerships with major multinational pharmaceutical companies, biotechs and research institutions worldwide. Over the years, HD Biosciences has been providing award winning services to its worldwide clients, and has won a leading position among the biology CROs in China. "We are excited about this acquisition and the great opportunities it brings along. HB Biosciences has a long-standing mission to grow into a major global player with distinctive core competences that could create special value for our clients. Merging with WuXi AppTec, a clear industry leader in drug R&D enabling services with enormous global exposure, resources and vision, will greatly speed up the process. We are confident that the integration will elevated our services to a new height and will enable us to better meet our growing customer needs," said Dr. Xuehai Tan, Chairman and CEO of HD Biosciences. "We are very pleased to welcome HD Biosciences to WuXi," said Dr. Ge Li, Chairman and Chief Executive Officer of WuXi AppTec. "During the past 14 years, HD Biosciences has developed into a global company with wide recognition, and this business combination is an important step in strengthening WuXi's biology and preclinical service capability. At WuXi, our commitment is to build the most comprehensive capability and technology platform in industry to enable anyone, and any company to discover and develop better medicines and healthcare products for patients, and to realize our vision that every drug can be made and every disease can be treated."

19

Aduro Biotech Enters into Exclusive License Agreement for Proprietary Neoantigen Identification Technology

BERKELEY, Calif., Jan. 19, 2017 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, today announced an exclusive license agreement with Stanford University for state-of-the-art neoantigen identification technology developed by Dr. Hanlee Ji, associate professor of medicine at Stanford.  Aduro will leverage its proprietary live, attenuated double-deleted Listeria (LADD) immunotherapy platform to engineer personalized LADD-based cancer therapies (pLADD) encoding multiple neoantigens identified through this technology.  The company plans to initially evaluate pLADD for the treatment of cancers of the gastrointestinal tract, including colorectal cancer, with a Phase 1 clinical trial expected to be initiated in 2017.

Pursuant to the terms of the agreement, Aduro received an exclusive license to the proprietary bioinformatics algorithms and computational workflows for neoantigen identification and selection. The accurate identification of neoantigens, tumor markers that are unique to an individual’s tumor, is believed to be critical in the development of a patient-specific cancer treatment.  Aduro’s LADD technology, which has been shown in clinical studies to remodel the tumor microenvironment, will be used to create a patient-specific immunotherapy that is engineered to enable the presentation of multiple selected neoantigens in dendritic cells, with the aim of inducing a targeted, robust anti-cancer immune response.

“We are excited to leverage the strength of Aduro’s LADD program with this new expertise in identifying a patient’s unique repertoire of cancer antigens to make personalized immunotherapies a reality for patients in need,” said Thomas Dubensky Jr., Ph.D., chief scientific officer of Aduro. “We look forward to applying the discoveries made possible with this highly sophisticated computational approach to neoantigen identification, with the aim to initiate a Phase 1 clinical trial later this year.”

09

Aduro Biotech Announces Clinical Collaboration with Merck to Evaluate the Combination of Aduro’s CRS-207 with Merck’s KEYTRUDA® (pembrolizumab) for the Treatment of Gastric Cancer

BERKELEY, Calif., Jan. 09, 2017 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, announced today a clinical collaboration with Merck (known as MSD outside the United States and Canada). The companies will investigate the combination of CRS-207, Aduro’s LADD (live, attenuated double-deleted) based immunotherapy, with Merck’s anti-PD-1 therapy KEYTRUDA® (pembrolizumab) for the treatment of gastric cancer.

“CRS-207 has demonstrated the ability to induce an anti-tumor immune response in clinical trials in other tumor types that over express the tumor antigen, mesothelin,” said Dirk G. Brockstedt, Ph.D., executive vice president of Research and Development at Aduro.  “Gastric cancer is an immune-sensitive mesothelin-expressing tumor where PD-1 checkpoint inhibitors have shown some activity. The combination of inducing an immune response through CRS-207, while simultaneously suppressing the cancer’s ability to evade the immune system through a PD-1 checkpoint inhibitor, has resulted in synergistic anti-tumor activity in pre-clinical studies. We aspire to reproduce this activity in the clinic in patients with gastric cancer.”

The multicenter Phase 1 study, planned to begin in the first half of the year, will enroll patients with metastatic gastric cancer who have failed at least two prior therapies to receive the combination of CRS-207 and pembrolizumab.

About LADD and CRS-207 LADD is Aduro's proprietary platform of live, attenuated double-deleted Listeria monocytogenes strains that have been engineered to generate a potent innate immune response and to express tumor-associated antigens to induce tumor-specific T cell-mediated immunity.

CRS-207 is one of a family of product candidates based on Aduro's LADD immunotherapy platform that has been engineered to express the tumor-associated antigen mesothelin, which is over-expressed in many cancers including mesothelioma and pancreatic, non-small cell lung, ovarian, endometrial and gastric cancers.

December 2016

20

Paroxysmal Nocturnal Hemoglobinuria Treatment Receives Fast Track Designation

RDR Staff; Published Online: Tuesday, Dec 20, 2016 Apellis Pharmaceuticals announced that their orphan drug APL-2, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) received Fast Track Designation by the FDA. APL-2 is designed for patients who experience hemolysis and require RBC transfusions even while taking eculizumab. APL-2 currently in two Phase Ib clinical trials. One trial assessing doses APL-2 administered by daily subcutaneous injection (SC) in patients with PNH who have not received the standard of care in the past, and the other is assessing doses of APL-2 as an add on with standard care. Results APL-2 doses of 180mg and 270mg significantly reduced hemolytic activity as early as eight days after the start of dosing, and this inhibition was maintained through the dosing period. About APL-2 APL-2 is a synthetic cyclic peptide conjugated to a polyethylene glycol (PEG) polymer that binds specifically to C3 and C3b, effectively blocking all three pathways of complement activation (classical, lectin, and alternative) with a particularly high potency against the alternative pathway.  About PNH Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder in which red blood cells break apart prematurely. It is an acquired hematopoietic stem cell disorder. Some hematopoietic stem cells in individuals with PNH are defective and consequently produce defective blood cells. These defective red blood cells of PNH are extremely susceptible to premature destruction by a particular part of a person’s own immune system called the complement system.  About Fast Track Designation  The FDA’s Fast Track program is designed to facilitate and expedite development and review of new drugs. Through the Fast Track program, a product may be eligible for priority review at the time of BLA and may be eligible to submit sections of the BLA on a rolling basis as data become available. APL-2 doses of 180mg and 270mg significantly reduced hemolytic activity as early as eight days after the start of dosing, and this inhibition was maintained through the dosing period.

16

ENYO Pharma SA announces successful initiation of the Phase 1 clinical programme with EYP001, its lead candidate for the treatment of Chronic Hepatitis B Virus infection

Lyon, December 16, 2016 - ENYO Pharma SA, a privately held biopharmaceutical company currently focused on developing treatments for viral infections, today announced that the Phase 1 single and multiple ascending dose trial evaluating EYP001 in healthy subjects has been initiated, and that single dose escalation has been completed. The results have shown that EYP001 is safe and well-tolerated at all doses studied in 46 healthy subjects. The safety and pharmacokinetics (PK) analysis of these first Phase 1 data will be complete by Q2 2017. EYP001 is a synthetic farnesoid X receptor (FXR) agonist with a favorable profile for oral therapy. The first Phase 1 study was designed to determine the safety, tolerability and pharmacokinetics of EYP001 in healthy subjects. ENYO Pharma also announced that next Phase 1 clinical studies of EYP001 are already planned during 2017 to test the safety, PK and initial antiviral activity of EYP001 in subjects with chronic HBV infection.

13

Green Biologics Begins Customer Shipments at First Commercial Plant

Little Falls, MN, facility produces 100 percent bio-based n-butanol and acetone Ashland, Virginia USA and Abingdon, Oxfordshire U.K. (December 13, 2016) – Green Biologics, Ltd., a UK industrial biotechnology and renewable specialty chemicals company, announced today the start of commercial shipments of bio-based n-butanol and acetone from its manufacturing facility in Little Falls, Minnesota. Over the past year, Green Biologics has built a robust pipeline of domestic and export customers combined with multiple partnerships to bring its products to downstream markets. These include distribution agreements with Acme Hardesty, Nexeo Solutions, and Caldic as well as a strategic partnership with HOC Industries, a custom blender, packager and distributor of consumer and government products. The company is collaborating with other industry leaders in a range of specialty markets and applications where performance and sustainability drive value. “The start of our first commercial facility is a critical milestone in building our position within the industry as a global renewable speciality chemicals company,” said Sean Sutcliffe,Chief Executive of Green Biologics. “We’re very proud to announce the start of shipments to key customers in highvalue markets and look forward to working with existing and new collaborators to bring a wide range of sustainable, environmentally-friendly products to shelves.” Offered as a high-performance, high purity, fully-sustainable alternative to conventional petrochemical-based commodities, Green Biologics’ speciality chemicalsaim to drive value in customer applications and downstream markets ranging from specialty coatings, pharmaceuticals, cosmetics, personal care and consumer products. Both butanol and acetone products carry the brand name BioPure™ and have receivedUSDA BioPreferred® status. As a member of the American Chemistry Council (ACC), Green Biologics’ commercial facility is actively working towards meeting Responsible Care® standards.

07

Cognito Therapeutics Launched with Exclusive License to Promising Alzheimer’s Research from The Massachusetts Institute of Technology

Boston, Mass. and San Francisco, Calif. — December 7, 2016 – Cognito Therapeutics announced today that the company has secured an exclusive worldwide license to the intellectual property developed from scientific discoveries by Li-Huei Tsai, Ph.D., and Ed Boyden, Ph.D., professors in the Department of Brain and Cognitive Sciences, Massachusetts Institute of Technology (MIT). The company was launched earlier this year with Series A financing from Morningside Venture. The scientific discoveries are featured in the December 8th issue of Nature. Working with mouse models of Alzheimer's disease, the team of scientists showed that by using a unique and totally non-invasive method of stimulation, they were able to restore gamma oscillation in the brains of the mice, which in turn activated the microglia cells to remove beta amyloid plaques in the brains. These plaques are characteristic of the brains of Alzheimer's disease patients, which are also deficient in gamma oscillation. “These results have opened up new doors to our understanding of Alzheimer’s,” said Prof. Tsai, Picower Professor of Neuroscience at MIT and co-founder of Cognito Therapeutics. “By demonstrating the underlying importance of these brain wave signatures, we have potentially uncovered a key to solving this disease in humans.” Tsai and Boyden co-founded Cognito Therapeutics to translate their findings into a treatment for Alzheimer’s patients. The company has filed an extensive portfolio of patents covering the applications of this novel approach to treating a variety of neurological disorders. "This is truly breakthrough science," said Gerald Chan, ScD, founder of Morningside and board member of Cognito Therapeutics. "It has the potential of being a game changer in our struggle to find an effective treatment for Alzheimer's disease. The social impact of such a treatment, if successful, would be enormous, as dementia is becoming the leading medical problem of an aging population." Cognito Therapeutics is based in Cambridge, Massachusetts, and at the San Francisco incubator TheraNova, a medical device developer with expertise in accelerating time-to-market for innovative medical technologies. “A device-based approach to Alzheimer’s is novel,” said Daniel Burnett, M.D., chief technology officer of Cognito Therapeutics and founder of TheraNova. “We are excited to be working with Morningside, which has shown an unwavering commitment to bringing this technology all the way into the clinic.”

06

Aduro Biotech Europe’s Hans van Eenennaam, Ph.D. and John Dulos, Ph.D. Honored with the Intellectual Property Owners Education Foundation’s 43rd Inventor of the Year Award for Contributions in the Discovery of KEYTRUDA® (pembrolizumab)

December 06, 2016 5:00 a.m. ET BERKELEY, Calif. and OSS, The Netherlands, Dec. 06, 2016 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, announced today that Hans van Eenennaam, Ph.D., chief operational officer and executive director of Aduro Biotech Europe, and John Dulos, Ph.D., principal scientist, will be honored tonight by the Intellectual Property Owners Education Foundation (IPOEF) as winners of the 43rd Inventor of the Year Award for their work relating to the discovery of KEYTRUDA® (pembrolizumab). KEYTRUDA is marketed by Merck & Co., Inc., Kenilworth, NJ, USA (known as MSD outside the U.S. and Canada) and is the first FDA-approved PD-1 inhibitor for the treatment of select cancers. “It is an honor to have been part of the team whose work led to the discovery of KEYTRUDA and positively impacted the lives of many cancer patients,” said Dr. van Eenennaam. “Now as part of Aduro Biotech Europe, where we are focused on developing novel B-select antibodies to modulate the immune system, we are pleased to be collaborating once again with the MSD team on what we believe to be the best-in-class anti-CD27 therapeutic antibody.” Dr. Dulos, principal scientist and project team lead for Aduro’s anti-APRIL BION-1301 program, commented, “As a scientist, it is incredibly satisfying to have been part of a team whose discoveries today are having a meaningful impact on the lives of many patients with several cancer types.  We thank the Intellectual Property Owners Education Foundation for this honor and will continue to leverage our knowledge and discoveries in the field of immuno-oncology with the aim of helping cancer patients in need.”

05

ENYO Pharma receives a €2.5 million grant from EU under the Horizon2020/SME Instrument Phase 2 programme for its project MIMESIS

• MIMESIS will accelerate and enable large scale development of ENYO Pharma’s novel discovery approach aimed at identifying new preclinical assets against infectious diseases and cancer from innovative drug discovery starting points inspired by viruses. Lyon, December 5, 2016 - ENYO Pharma, a biopharmaceutical company currently focused on developing treatments for viral infections, today announced that it has received a grant of €2.5 million in response to its application to the highly competitive SME Instrument Phase 2 funding programme (6% success rate). The SME Instrument is part of Horizon 2020, the EU framework programme dedicated to innovation and research managed by the European Commission. It belongs to the pillar “Industrial Leadership” of H2020 and aims to support high growth and highly innovative SMEs with global ambitions. ENYO Pharma’s MIMESIS project applied for the June 2016 cut-off under the topic “Dedicated support to biotechnology SMEs closing the gap from lab to market”. MIMESIS has been selected by an independent jury of four experts recognised for their scientific and business expertise. The company will receive 2.5 M€ corresponding to the financing of 70% of the total project budget (€3.6 million). After a feasibility Phase that generated ENYO’s internal drug development programme on an autophagy target, MIMESIS will be expanded to an industrialised scale to accelerate the discovery of new therapeutic targets and innovative new chemical entities against infectious diseases and cancer. Dr Eric Meldrum, ENYO Pharma's Chief Scientific Officer commented, "We are very honoured to receive the recognition from the Horizon 2020 jury in what was a highly competitive process. MIMESIS represents a real paradigm shift in the development of new innovative drugs for pathologies where the medical need is immense. This financing will enable us to screen our library of original peptides and small molecules on hundreds of intracellular human targets previously untapped by the pharmaceutical industry.” For this project ENYO Pharma has designed a proprietary library of 10’000 small molecules to modulate host cell biology and also capable of disrupting protein:protein interactions (PPIs) between pathogens and the host. This library of developable chemical templates will be screened in phenotypic assays for inhibitors of the replication cycle of several viruses (Influenza, RSV, HRV, Zika) and a mycobacterium (TB). As this library is designed to modulate host cell biology, it will also be screened for inducers of Immunogenic Cell Death (ICD) in tumors. With €3.6 million over 24 months, most promising chemistries will be the starting point for numerous hit to lead optimisation programmes funded within the EU grant. Those optimisation efforts focused on novel intracellular targets will generate valuable Intellectual Property. Upon completion of MIMESIS, ENYO Pharma will further optimise its best chemical series either internally or in collaboration with other pharmaceutical companies up to clinical proof of concept. This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement n° 739086- MIMESIS

05

Aduro Biotech Announces Anti-CD27 Agonist, an Investigational Anti-Cancer Immunotherapy, Advancing in Collaboration with MSD

December 05, 2016 4:00 a.m. ET

Expected Filing of Investigational New Drug (IND) Application in 2017

Company to Host Conference Call Today to Provide Program Update, Including STELLAR, at 5:30 a.m. PT/ 8:30 a.m. ET

BERKELEY, Calif. and OSS, The Netherlands, Dec. 05, 2016 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, announced today that an anti-CD27 antibody developed by Aduro Biotech Europe and derived from its proprietary B-select technology has been selected to be advanced into clinical development by Merck & Co., Inc., Kenilworth, NJ, USA (known as MSD outside the U.S. and Canada), through a subsidiary.  CD27 has been recognized as having a critical role in activating a productive anti-cancer immune response and has demonstrated the potential to be combined with checkpoint inhibitors in pre-clinical studies.

“Pre-clinical studies have shown that an anti-CD27 agonist can induce a T cell-mediated anti-cancer immune response, and in combination with PD-1 immune checkpoint inhibitors complete tumor eradication can be achieved,” said Hans van Eenennaam, Ph.D., chief operational officer, Aduro Biotech Europe.

The CD27 antibody was licensed by MSD to advance as a part of their successful immunotherapy development program and was identified in close collaboration with Prof. Jannie Borst, Ph.D., professor at the University of Amsterdam and division head at the Netherlands Cancer Institute, through Aduro’s B-select monoclonal antibody technology. This technology includes a proprietary ultra-selective functional screening process to identify antibodies with unique binding properties against a broad repertoire of targets that can modulate the innate and adaptive arms of the immune system.

 "In 2014, prior to the acquisition by Aduro Biotech, BioNovion entered into a worldwide license agreement with MSD that covers the product candidate’s development and advancement through commercialization," said Andrea van Elsas, Ph.D., chief scientific officer of Aduro Biotech Europe. "Both companies recognized the significant potential of targeting CD27 as a new and distinct mechanism in cancer immunotherapy, especially in the context of PD-1 checkpoint inhibitors, and were strongly committed to accelerate a quality candidate into the clinic."

03

Aduro Biotech Presents Preclinical Data Supporting Clinical Development of its Anti-APRIL Antibody, BION-1301, for the Treatment of Multiple Myeloma

December 03, 2016 12:00 p.m. ET

Poster #2112 to be Presented at the 58th American Society of Hematology Annual Meeting and Exposition

BERKELEY, Calif. and OSS, The Netherlands, Dec. 03, 2016 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, today announced the presentation of data from preclinical studies supporting the clinical development of the company’s proprietary monoclonal antibody (mAb) BION-1301, a humanized anti-APRIL (A PRoliferation-Inducing Ligand) antibody for the treatment of multiple myeloma.  Data from these in vivo and in vitro preclinical studies demonstrated that BION-1301 effectively neutralized APRIL, preventing its binding to BCMA (B cell maturation antigen), an essential receptor expressed on multiple myeloma cells.  Based on the mechanism of action and anti-tumor activity observed in earlier preclinical studies with the parental anti-APRIL antibody, hAPRIL.01A, BION-1301 has the potential to inhibit multiple myeloma tumor growth, survival and chemoresistance.  These data, which will be highlighted in a poster presentation (Poster #2112) at the 58th American Society of Hematology Annual Meeting and Exposition, further underscore the potential application of BION-1301 for use as a single agent, or in combination with current standard of care therapies, for the treatment of multiple myeloma.

"In patients with multiple myeloma, there is an overabundance of APRIL, a ligand which plays a critical role in the proliferation of multiple myeloma cells," stated Andrea van Elsas, Ph.D., chief scientific officer of Aduro Biotech Europe. "With BION-1301, which was derived from Aduro’s proprietary B-select antibody platform, we are blocking APRIL from binding to its target receptor, thereby inhibiting the growth and survival of multiple myeloma cells."

Dr. van Elsas continued, "Based on the data to be presented later today, we believe BION-1301 represents a novel antibody with a novel mechanism of action that has potential in the treatment of multiple myeloma, alone or in combination regimens.  We look forward to advancing BION-1301 into clinical development in the coming year in our effort to bring much needed new treatment options to patients with multiple myeloma."

02

Apellis Reports Positive Interim Results from Phase Ib Clinical Trials of APL-2 in PNH

C3 inhibitor reduces hemolysis in patients with PNH, is generally well tolerated LOUISVILLE, Ky., December 2, 2016 – Apellis Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company focused on inhibition of the complement system, today will present positive interim results from two ongoing Phase Ib open-label, dose-escalation clinical trials of APL-2, a complement C3 inhibitor, in paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare, acquired, potentially lifethreatening disease characterized by complement-mediated hemolytic anemia. Interim results suggest that APL-2 reduces hemolysis in patients with PNH who receive daily subcutaneous injections of APL-2 as monotherapy or as an add-on to the standard of care, eculizumab. With APL-2 (270 mg) as monotherapy, three out of three (3/3) PNH patients achieved a reduction in lactate dehydrogenase (LDH) levels to below the standard for control in PNH (500 U/L). With APL-2 (270 mg) as add-on to eculizumab, six out of six (6/6) previously transfusion-dependent PNH patients did not require transfusions during the study and five out of six (5/6) PNH patients achieved hemoglobin levels within the normal range for healthy individuals. All 15 PNH patients who have been dosed across the two trials to date have completed at least one month of dosing, with five having received more than three months of dosing. Based on preliminary assessment, APL-2 is generally well tolerated. “Establishing that systemic inhibition of C3 is feasible with daily subcutaneous injections of APL-2 in patients with PNH is a significant achievement that validates C3 as a target in the complement pathway,” said Cedric Francois, M.D., Ph.D., chief executive officer of Apellis. “APL-2’s unique ability to target C3 and block all three pathways of the complement system is indicative of its potential to be an effective treatment for multiple complement-driven diseases. We are encouraged by the preliminary results APL-2 has demonstrated, as well as the favorable safety data observed thus far in the Phase Ib trials and our previous studies in healthy volunteers. All patients in the Phase Ib trials will have completed three months of dosing in January 2017.” Apellis will present the Phase Ib interim results today at the International PNH Interest Group (IPIG) Annual Scientific Assembly and will present the poster “APL-2, a Complement C3 Inhibitor for the Potential Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH): Phase I Data from Two Completed Studies in Healthy Volunteers” tomorrow at the American Society of Hematology (ASH) Annual Meeting.

02

Why High School Students Should Seek Out Peer Mentors for College Applications

Find out how CollegeVine's near peer mentors can help high school students successfully apply for college. DEC 2, 2016 2:59PM EST -- When Ling Ritter applied early decision one of her top college choices, she had doubts about her ability to get in, but her guidance counselor told her she had a strong enough profile that the school would either accept or defer her, but not deny. So, when she received a rejection from that school, she was flummoxed. Ling, now 19, went back to that counselor who, this time, told her the rejection was an “indicator of [her] competitiveness,” and that she should remove any other “reach” schools she was planning to apply to. She didn’t listen. Instead, she added a few "safe" schools, and applied to her "reach" options as well — and was accepted to 13 out of 17, including Princeton University, where she’s currently a sophomore politics major. Ling’s college application process was rocky, perhaps in part because her main source of guidance was an adult counselor who was also responsible for helping out all of her classmates. After all, according to CollegeVine, the average public school student receives only 38 minutes with her school’s guidance department over the course of four years. But, Ling is determined to make it better for other high schoolers. She now works as a "Near-Peer Mentor" with CollegeVine, which pairs high schoolers with college students who not only still have the application process fresh in their minds, but also can lend insight on actual, current college life. As part of her one-on-one relationships with her mentees, Ling (who went through training with CollegeVine for the job) helps with their school selections, applications, and essays, managing deadlines and timelines, and more. And she’s not alone. CollegeVine’s college mentors attend schools around the country, and represent more than 20 different majors, extracurriculars, and individual experiences. We caught up with Ling to find out even more about the program — and why it’s awesome for both the mentors and the mentees. Teen Vogue: How do you think having a near-peer mentor would have changed your college application experience? Ling Ritter: Near-peer mentors help make college applications less of a guessing game. There is no silver bullet, no magic formula. However, after joining the CollegeVine team and going through their consultant training program, I now understand that there are ways mentors can help students authentically maximize their strengths in order to stand out in the applicant pool. TV: What do you wish a college student would have or could have told you during that process? LR: One piece of advice high school seniors frequently hear during the application process is, "Just be yourself." I want to double down on that. You don’t need to be anyone else to succeed — you need to know that you are enough. But I also want to expand on this idea: Be yourself and understand who you are. Colleges are looking for individuals who demonstrate that they have been thinking about the intersections of their different identities, extracurricular interests, and academic pursuits, and that they are exploring how they can apply their unique perspectives to the world around them. An application is a very condensed representation of you, so seek out ways to add complexity and color. TV: As a mentor, what kind of advice do you give? What aspects of your college experience do you share? LR: Although [we follow a] curriculum, I will share anything about my college experience that my student wishes to know. CollegeVine’s pairing process is very specific, so I have thus far always been paired with a student who either is really interested in Princeton or has many shared passions in common with me (or both). This often leads to all sorts of great conversations [and questions, like], "Should I submit an arts supplement?" [or] "How do I begin to navigate the myriad career paths that stem from my interests?" TV: Do you have any moments from your mentoring experiences with high school students that especially stand out to you? LR: My favorite thing to hear from a student is, "Oh! I didn’t think I could do that." Many students come in believing that college applications have to be weighty, and serious, and formal. While it’s important to demonstrate diligence, it’s equally important to convey personality. The times in which I was able to help students discover new and creative ways of expressing themselves, or showcasing quirky hobb[ies] that [don’t] fit within the confines of a traditional resume are definitely special moments for me. TV: What's the best part about being a near-peer mentor? LR: Getting to know my students has been incredible. They are all such kind hearted, multifaceted, and spirited individuals. I am rooting for them in their college searches, and I know that, when spring comes around, the tables will turn, and they will be the ones choosing college[s] that fill them with pride and excitement for the future. TV: How does mentoring high school students affect your current college life and path? LR: Mentoring gives my college life a sense of purpose. Being raised by immigrant grandparents instilled in me the importance of not only being thankful for the resources available to me, but also using those resources to help future generations obtain the same opportunities. CollegeVine gives me a platform to do so on a very personal level. TV: What about your confidence and outlook as a student and a person? LR: Working with CollegeVine has given me insight into some of the potential reasons...behind my own admittance to Princeton. This, coupled with hard work paying off in the classroom, has really helped me prove to myself that I belong here. TV: If you could give one piece of advice to students beginning the college application process, what would it be? LR: The weight of a hundred rejections is still less than that of refusing to try and always wondering if things could be different. Rub some dirt on it and get back to being awesome.

November 2016

30

MicuRx Initiates Phase 1 Clinical Trial in U.S. for Novel Antibiotic Agent MRX-4

HAYWARD, Calif. and SHANGHAI, China – November 30, 2016 – MicuRx Pharmaceuticals, Inc. today announced the start of a Phase 1 clinical trial of a new antimicrobial agent, MRX-4, for the treatment of infections caused by Gram-positive bacteria including methicillin-resistant S. aureus (MRSA) and vancomycin-resistant Enterococci (VRE). MRX-4 is a prodrug form of the oral antibiotic MRX-1, presently in Phase 3 clinical trials in China for the treatment of complicated skin infections.

This comprehensive Phase 1 trial, now enrolling patients at a single center in the United States, will evaluate safety, tolerability, pharmacokinetics and bioavailability of the oral formulation of MRX-4. The study will enroll 122 healthy subjects in nine single- and four multiple-ascending dose cohorts. Concurrently, the safety, tolerability, and pharmacokinetics of the intravenous formulation of MRX-4 will be evaluated in 60 healthy subjects of five single- and four multiple-ascending dose cohorts, along with an oral to IV crossover study.

“We anticipate that success in this Phase 1 trial would enable a rapid transition into advanced clinical studies in the U.S. for this new means of efficient delivery of our antibiotic systemically,” stated Zhengyu Yuan, Ph.D., president and CEO of MicuRx.

MRX-1 is a next-generation oxazolidinone agent that has shown notably improved hematopoietic safety compared to first-generation antibiotics, such as linezolid, while maintaining excellent efficacy characteristics for this class. The prodrug form of MRX-1, named MRX-4, serves to expand its utility into both intravenous and enhanced oral applications.

29

Green Biologics Receives USDA Certification

Renewable Specialty Chemicals Company Receives Certification Under USDA BioPreferred® Program Ashland, Virginia and Abingdon, Oxfordshire U.K. (November 29, 2016) – Green Biologics, Inc., the U.S. subsidiary of Green Biologics Ltd., a U.K. industrial biotechnology and renewable chemicals company, announced today that its high purity bio-based n-butanol and acetone have received official certification under the USDA BioPreferred® program. The products are now certified as 100 percent bio-based and are marketed under the BioPure™ brand. “Having our n-butanol and acetone named as USDA Certified BioBased Products is yet another milestone that we can point to when demonstrating the value and differentiation of our products from conventional petrochemical-based commodities,” said David Anderson, Global Vice President of Marketing for Green Biologics.

21

Partial Clinical Hold Lifted and Enrollment Resumes for Aduro Biotech LADD Clinical Trials

November 21, 2016 8:00 a.m. ET Company’s Ongoing Clinical Trials with LADD-based Therapies Include Mesothelioma and Ovarian Cancers as well as Personalized LADD (pLADD) BERKELEY, Calif., Nov. 21, 2016 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, today announced that the U.S. Food and Drug Administration (FDA) has lifted the partial clinical hold placed on its clinical trials evaluating the LADD (live, attenuated double-deleted) immunotherapy platform, enabling patient enrollment to resume in all Aduro-sponsored clinical studies. "We are pleased to come to a rapid agreement with the FDA to resume new patient enrollment in our LADD clinical studies," said Stephen T. Isaacs, chairman, president and CEO of Aduro Biotech. "With slight protocol modifications implemented, we remain on track to initiate a Phase 2 clinical study using our LADD-based therapy CRS-207 in combination with an anti-PD-1 compound for patients with mesothelioma in the first half of 2017. Additionally, we continue to make significant progress with our STING Pathway Activator and B-select Antibody programs and with our three diverse immunotherapy platforms, we believe we are uniquely positioned to bring innovative therapies to patients with late-stage cancers." In agreement with the FDA, Aduro’s LADD-based clinical trial protocols have been modified to include extended patient surveillance, to exclude patients with certain prosthetic devices that are not easily removed, and to add guidance to administer prophylactic antibiotics following LADD-based treatment for patients who may receive immune-suppressive treatments.

18

Stealth BioTherapeutics Announces Positive Phase 2 Clinical Trial Findings for Patients Undergoing Renal Angioplasty

Data support company’s cardiorenal program, including three ongoing heart failure trials

Single elamipretide infusion improves kidney microvasculature and function after renal artery angioplasty

BOSTON – November 18, 2016 – Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing investigational drugs to treat mitochondrial dysfunction, today presented results from the EVOLVE trial, which demonstrated that a single dose of elamipretide prior to renal artery angioplasty and stenting procedures improved kidney function and blood flow for three months post-procedure. The data were presented as a late-breaking poster at the American Society of Nephrology (ASN) Kidney Week 2016 in Chicago.

“Patients undergoing renal angioplasty and stenting, intended to open up kidney arteries blocked by atherosclerosis, often fail to regain normal kidney function due to tissue damage during the procedure, possibly due to the sudden replenishment of oxygen to starved tissues,” said Dr. Stephen Textor, the principal investigator for the trial. “The study results validate our preclinical findings and our underlying hypothesis that elamipretide may help prevent acute kidney injury by preserving mitochondrial function in cells and ultimately improve measures of kidney function in these patients.”

15

SKSpruce Technologies Finalist WBA Awards

San Jose, CA, November 15, 2016—SKSpruce Technologies, an emerging Silicon Valley leader in carrier-class Wi-Fi, is a finalist for the Wireless Broadband Alliance (WBA) “Best Next Gen Wi-Fi Network Infrastructure” award, recognizing and celebrating excellence in wireless innovation. The WBA recently announced the shortlist for its annual WBA Industry Awards 2016, taking place at the Wireless Global Congress, November 16-17 in San Jose, California. The finalist entry recognizes SKSpruce’s Wi-Fi solution used in the largest integrated Wi-Fi/cellular system in the world, deployed in Japan by SoftBank, a leading global carrier with more than 100 million subscribers. The SoftBank Mobile Wi-Fi system has 460,000 access points throughout the country seamlessly integrated into their 4G LTE network providing transparent uninterrupted handover between 4G LTE and Wi-Fi, delivering a superior user experience. SoftBank Mobile’s entire Wi-Fi network is controlled by the SKSpruce SKG10000 gateway, and its integration to the core mobile network is provided by SKSpruce.

14

Stealth BioTherapeutics Initiates Phase 1 Study of Elamipretide in Dry Age-Related Macular Degeneration

Top-line data from study evaluating elamipretide in patients with dry age-related macular degeneration expected mid-year 2017

BOSTON – November 14, 2016 – Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing investigational drugs to treat mitochondrial dysfunction, today announced the initiation of ReCLAIM, a Phase 1 study evaluating elamipretide in intermediate age-related macular degeneration (AMD). Top-line data are expected mid-year 2017.

“There are currently no FDA-approved treatment options for dry AMD, so we are eager to better understand the effect that elamipretide may have in treating these roughly 13 million patients,” said Dr. Scott Cousins, the trial investigator, and Professor of Ophthalmology and Director of the Duke University Center for Macular Diseases. “Mitochondrial dysfunction that stems from various environmental toxins may be an important causative factor in dry AMD, and in laboratory models, elamipretide appears to prevent mitochondrial dysfunction in the retinal pigment epithelium.”

ReCLAIM is an open-label study to evaluate the safety and tolerability of 12 weeks’ treatment with daily subcutaneous injections of elamipretide in patients, age 55 and above who have at least one eye with intermediate AMD, and have either: a) high-risk protein deposits (drusen) on the retina without any geographic atrophy (GA), a characteristic of advanced AMD which can result in the loss of photoreceptor cells or b) GA with an unaffected central fovea (noncentral GA). The study’s primary endpoints are safety and tolerability, and the secondary endpoints are changes from baseline in physical/ophthalmic examinations and feasibility of subcutaneous injections in this patient population.

“ReCLAIM will build upon our ongoing ophthalmic program to help us better understand the potential of elamipretide to treat back of the eye diseases as well as the effects of the treatment to slow the aging process in eye tissue,” said Stealth Chief Executive Officer Reenie McCarthy. “We look forward to evaluating initial results of this study mid-year 2017.”

12

Aduro Biotech Highlights Positive Clinical Results from Second Cohort of Phase 1b Mesothelioma Clinical Trial

November 12, 2016 8:02 a.m. ET

Data Presented at the Society for Immunotherapy of Cancer (SITC) Annual Meeting Continues to Show Clinical Activity of CRS-207

Two Additional Poster Presentations Detailing Promising Preclinical Results of STING and pLADD Programs

BERKELEY, Calif., Nov. 12, 2016 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO) today announced highlights from a poster presentation at the Annual Meeting of the Society for Immunotherapy of Cancer (SITC) being held in National Harbor, Maryland on the preliminary safety and efficacy of its novel immunotherapy, CRS-207, being evaluated in unresectable malignant pleural mesothelioma. These data were from the second cohort of patients in an ongoing Phase 1b clinical trial of CRS-207 in combination with standard of care chemotherapy and immune-modulating doses of cyclophosphamide (Cy) as a first-line treatment.

Preliminary results as of October 2016 from the 22 patients in the second cohort in the Phase 1b clinical study (Abstract #261) showed that 82 percent (18/22) of patients had disease control, with 55 percent (12/22) of patients achieving a partial response (PR) and 27 percent (6/22) with stable disease. Tumor shrinkage was observed in 77 percent (17/22) of patients. Of these patients, 36 percent (8/22) experienced tumor shrinkage after two doses of immunomodulatory doses of cyclophosphamide combined with CRS-207 (Cy/CRS-207), but prior to initiation of standard of care chemotherapy. Fourteen percent (3/22) of these patients achieved a partial response. No treatment-related serious adverse events or unexpected toxicities were observed. Median duration on study was 9.7 months at the time of data cutoff. Treatment continues, with immune response evaluations and survival follow up ongoing.

Of note, analysis of paired tumor biopsies obtained from two patients showed a marked infiltration of immune effector cells into the tumor microenvironment (TME) following two doses of Cy/CRS-207, as compared to baseline. Post-therapeutic changes included an increase in CD8+ cytotoxic T cells as well as an increase in other immune cell types that are thought to be essential for effective immunotherapy, including dendritic cells and natural killer cells. Together, these data suggest that the Cy/CRS-207 remodeling of the TME may be an important component of the clinical responses observed in this cohort of patients.

“The data from the second cohort, which is a patient population with more advanced disease compared to the first cohort, demonstrate that the addition of immunomodulatory doses of cyclophosphamide, which has been shown to inhibit negative regulatory T cell populations, to the combination of CRS-207 and chemotherapy results in encouraging disease control and tolerability for patients with mesothelioma,” said Dirk G. Brockstedt, Ph.D., executive vice president of research and development of Aduro. “Importantly, we believe these data, together with the results from the first cohort, support further investigation of CRS-207 in mesothelioma, and we intend to initiate a Phase 2 study of CRS-207 used in combination with an anti-PD-1 therapy as an immune-modulator in patients with mesothelioma who have failed at least one prior therapy.”

 

09

Aduro Biotech’s Personalized LADD Therapy Featured in an Oral Presentation at SITC’s New Cancer Immunotherapy Agents in Development Program

November 09, 2016 4:01 p.m. ET -- Personalized LADD, or pLADD, is being developed utilizing patient-specific neoantigens -- BERKELEY, Calif., Nov. 09, 2016 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, announced that Tom Dubensky Jr., Ph.D., chief scientific officer of Aduro, presented today about the company’s personalized LADD technology at the Society for Immunotherapy of Cancer (SITC) distinct session titled New Cancer Immunotherapy Agents in Development being held in conjunction with the SITC annual meeting. LADD is Aduro's proprietary platform of live, attenuated double-deleted Listeria monocytogenes strains that have been engineered to express tumor-associated antigens to induce an innate immune response and tumor-specific T cell-mediated immunity.  Personalized LADD, or pLADD, is a second generation LADD technology that is being designed for individualized, patient-specific immunotherapy.  The pLADD approach leverages the immune activating activity of the Listeria bacterial vector in combination with neoantigens, or the tumor markers specific to an individual’s cancer, which are derived from the patient’s own unique tumor cells. Once administered, pLADD therapies are expected to mobilize the immune system through first an immediate recognition of the presence of Listeria as being foreign and then second a specific and customized immune attack on cells containing the tumor neoantigens presented by pLADD.  An Investigational New Drug (IND) application has been accepted, and a Phase 1 trial evaluating the safety and immunogenicity of pLADD in patients with advanced gastro-intestinal cancers is planned. "There is tremendous excitement in the oncology field to develop personalized therapies as the next new wave in immunotherapy to specifically customize treatment for each patient based on neoantigens that are unique to a patient’s tumor," said Tom Dubensky Jr., Ph.D., chief scientific officer of Aduro. "We are excited about the potential of our pLADD program, which has been shown to induce anti-tumor immune responses specific to tumor neoantigens and correlate with longer survival in preclinical models. Additionally, in these models, we observed a synergistic anti-tumor effect when pLADD was combined with an anti-PD-1 antibody, resulting in a significant reduction in tumor volume and increased survival."

08

DNAtrix Licenses Myxoma Virus for New Immunotherapy Platform

Houston, TX – November 8, 2016 – DNAtrix, a clinical stage biotechnology company developing virus-driven immunotherapies for cancer, announced it has entered into an exclusive license agreement with the University of Florida, Gainesville to develop a novel oncolytic virus platform.  The platform is based on myxoma virus, a poxvirus that has been shown to have beneficial features for treating cancers.

A major advantage of the myxoma virus is its ability to attach to T lymphocytes and other white blood cells, which are then delivered to the patient to trigger tumor cell killing and antitumor immunity.  Myxoma virus can be armed with multiple immune stimulatory genes, a feature shared by other large DNA virus such as herpes simplex and adenovirus.

“The myxoma virus has unique properties for attacking cancer,” said CEO Frank Tufaro, Ph.D. “We think this technology platform provides a new modality for delivery of a potent oncolytic virus to tumors by co-administering it along with T cells.  We look forward to testing this “Trojan horse” strategy in the clinic.”

“The myxoma virus is a novel oncolytic candidate that does not infect normal human cells but has a unique ability to identify the damaged signaling pathways found in the majority of human cancers; thus, resulting in productive infections in the patient’s cancer cells,” stated Grant McFadden, Ph.D., Professor in the Department of Molecular Genetics & Microbiology at the University of Florida, College of Medicine.

08

CellCentric wins Innovate UK award to expand use of p300/CBP inhibitors in cancer

November 8th 2016 CellCentric is developing first in class drug compounds against a key regulator of cancer. P300/CBP are twin (paralogue) histone acetyltransferase proteins, that act as transcriptional co-factors. When inhibited they cause the down regulation of the androgen receptor (AR) and its variants. It also decreases c-Myc, another key cancer driver. CellCentric has been focusing the use of its new potent, specific, orally deliverable compounds for the aggressive form of prostate cancer, CRPC, where AR and AR-splice variants play a key role. Recent scientific publications have shown that cancer cells can gain mutations in p300 or CBP. When this happens, the cell becomes dependent, or addicted, to the corresponding paralogue protein. Inhibition of p300/CBP with drug compounds can then drive synthetic lethality – cancer cell death. This potential new way to treat certain types of cancer occurs is application to up to 20% of lung cancers (both small cell and non-small cell, NSCLC) as well as bladder cancer (up to 25%) and leukaemia (up to 18%). CellCentric has won an award from the UK’s innovation agency, Innovate UK, to expand its work, to investigate the use of p300/CBP inhibitors beyond prostate cancer. The funds will be used to confirm the synthetic lethality mechanism in lung cancer. It will go on to validate if p300 and CBP mutations can be readily detected in circulating tumour cells, thus allowing clinicians to easily identify patients that will benefit from the new inhibitor drugs that are being developed. Lung cancer accounts for 25% of all cancer deaths. Despite other new targeted approaches (e.g. EGFR and ALK inhibitors for NSCLC), over 50% of lung cancers are not molecularly defined. Thus p300/CBP inhibitors offer a significant new opportunity, either as single agents or used in combination with existing or other developmental drugs. This is also a significant commercial opportunity. The lung cancer drug market is set to reach $14.3bn by 2024. Even a small segment such as that representing ALK positive tumours (4-8% of total) are predicted to have sales of $1.2bn.

07

Stealth BioTherapeutics Announces Acceptance of Late-Breaking Clinical Trial Poster at American Society of Nephrology Kidney Week 2016

Late-breaking poster presents data from elamipretide clinical trial in chronic kidney disease patients at risk of acute kidney injury

BOSTON – November 7, 2016 – Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing investigational drugs to treat mitochondrial dysfunction, today announced that the abstract, “Phase 2a Clinical Trial of Mitochondrial Protection (Elamipretide) during Stent Revascularization in Patients with Atherosclerotic Renal Artery Stenosis” has been accepted as a late-breaking clinical trial poster at the American Society of Nephrology (ASN) Kidney Week 2016 taking place in Chicago, November 15-20, 2016.

“The EVOLVE trial underscores our commitment to our cardiorenal program,” said Stealth’s Chief Executive Officer Reenie McCarthy. “We are pleased to present the details of these results both in the context of our three ongoing Phase 2 heart failure studies, as well as to inform our approach to other diseases of aging in which mitochondrial dysfunction is a contributing factor.”

EVOLVE was a double-blind, placebo-controlled study that evaluated 14 patients ages 40 to 80 with chronic kidney disease (CKD) at risk of acute kidney injury (AKI). The primary endpoint was change in kidney function, and the secondary endpoint was a change in renal blood flow and cortical perfusion.

07

Aduro Biotech Presents Preclinical Data Demonstrating Acute and Systemic Immune Activation through STING Pathway Stimulation with ADU-S100

Combination of ADU-S100 and PD-1 Blockade Resulted in Complete Eradication of Local and Distal Tumors BERKELEY, Calif., Nov. 07, 2016 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO) today highlighted an oral presentation given by the company’s chief scientific officer, Thomas Dubensky Jr., Ph.D., at the 4th European Society of Medical Oncology (ESMO) Symposium on Immuno-Oncology held last week in Lausanne, Switzerland.  The data, generated from multiple preclinical models, demonstrated important changes in the tumor microenvironment and the activation of acute and systemic tumor-specific immune cell responses following intratumoral administration of ADU-S100 (also known as MIW815), an investigational STING (Stimulator of Interferon Genes) Pathway Activator immunotherapy.  Importantly, these preclinical data underscore the ability for ADU-S100 to induce tumor-specific memory mediated by immune cells (e.g. T-cells and NK-cells) whereby the immune system is able to eliminate specific cancerous cells upon their reintroduction without further therapy. Additionally, the anti-tumor efficacy achieved with ADU-S100 was enhanced by combination with an anti-PD-1 immune checkpoint inhibitor, and resulted in the complete eradication of local and distal tumors. "We are pleased to have further validated, through multiple preclinical models, our previous discoveries regarding the potential mechanism of action of the STING Pathway and the role it serves in stimulating a robust and systemic T-cell immune response," stated Dr. Dubensky. "We look forward to working in partnership with Novartis on translating our preclinical findings to a clinical experience as we continue to make progress with our ongoing Phase 1 study of ADU-S100."

02

CollegeVine Secures $3.1 Million In Series A Funding

CAMBRIDGE, Mass., Nov. 2, 2016 /PRNewswire/ -- CollegeVine, the fast-growing EdTech startup specializing in high school mentoring for college applications, has closed a $3.1 million Series A funding round led by Morningside Technology Ventures ("Morningside") with participation by New York-based University Ventures. Morningside is a private equity and venture capital firm. Gerald Chan, its co-founder, has joined CollegeVine's Board of Directors. University Ventures is a specialty venture capital fund that exclusively focuses on promising ideas in the higher education space. The announcement was made today by Jon Carson, the CEO of CollegeVine. CollegeVine was started by two Harvard University students and one University of Chicago student working out of the Harvard iLab. The company uses a near peer model of matching talented college students with high schoolers to help them with the college application process. CollegeVine's services quickly earned a reputation for its outstanding college admissions results. Two years ago, the company employed 20 college students as consultants; today it employs almost 300. "There is a huge unmet need for high school students to get proper guidance and mentoring in their planning for college and their application for admission. The fast growth of CollegeVine's business validates that its services are exactly what high school students are looking for. Optimizing the college application process is beneficial both to the applicants and to the universities," said Chan. "With the backing of Morningside and support from University Ventures, we are now positioned to grow the company to meet consumer demand. We plan to use the Series A investment to build out our technology platform and product suite, grow the team, and accelerate our growth," said Carson. "We are pleased to have Gerald join our Board as he has been a valued partner and mentor over several ventures. I am honored, to be working with him on another new and exciting EdTech venture." Morningside was the primary backer of FamilyEducation Network where Carson was both the founder and CEO. FamilyEducation Network became the largest K-12 online network when it was acquired by Pearson Plc for $175 million in 2000. CollegeVine has recently strengthened its management team with the addition of former Zipcar executive Doug Williams as chief technical advisor and Harvard Business School professor Deepak Malhotra as a member of the board of directors.

01

DNAtrix Announces First Patients Treated in Phase 2 Trial with DNX-2401 and KEYTRUDA

Houston, TX – November 1, 2016 – DNAtrix, a clinical stage biotechnology company developing virus-driven immunotherapies for cancer, announced that the first patients have been treated in a multicenter Phase 2 trial investigating its oncolytic adenovirus, DNX-2401, in combination with KEYTRUDA® (pembrolizumab), Merck’s anti-PD-1 therapy, in patients with recurrent glioblastoma.

The CAPTIVE trial is evaluating the potential effect of DNX-2401 and KEYTRUDA in patients with recurrent glioblastoma, a disease for which there is neither a cure nor adequate treatment. Leading medical centers in the United States and Canada are participating.

DNX-2401 is a potent conditionally replicative oncolytic adenovirus that targets and kills cancer cells, while leaving normal cells intact. Multiple clinical studies have shown that DNX-2401 has a favorable safety profile, strong tumor-killing potential and can trigger an antitumor immune response.

KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 (programmed death receptor-1) and its ligands, PD-L1 and PD-L2.  This activity enhances the T cell response and leads to effective tumor destruction. KEYTRUDA is currently approved in the United States for advanced melanoma, metastatic non-small cell lung cancer (NSCLC), and advanced head and neck squamous cell cancer (HNSCC).

“Glioblastoma is a difficult disease to treat with conventional therapies,” said Frank Tufaro, Ph.D., Chief Executive Officer of DNAtrix. “Based on remarkable preclinical data, we anticipate that the addition of KEYTRUDA to DNX-2401 therapy will provide even more benefit to patients with recurrent disease.”

October 2016

31

Myanmar HTI Project Formally Launched

October, 2016 - SKSpruce Technologies formally launched the Myanmar Carrier-grade Wi-Fi project. A dedicated project team led by Henry Wu (VP of Product, SE & Product Marketing) was set up to ensure the smooth implementation of this project. The project marks the inaugural collaboration between SKSpruce and Myanmar HTI. Aimed at providing excellent data communication services and comprehensively improving user experience, the project will deploy the world-class carrier-grade Wi-Fi solution of SKSpruce in major areas of Yangon. During this project, over ten thousand APs will be deployed in 22 towns (10 towns in phase I) of Yangon to provide Wi-Fi services to 6 million Yangon citizens, in order to improve the data communication capacity of Yangon public areas, and to bring great economic profits and social benefits to Myanmar. Myanmar HTI project is SKSpruce’s largest project in Southeast Asia this year. Meanwhile, it indicates that SKSpruce is making a significant move to expand Southeast Asian market. SKSpruce is attracting an increasing number of international clients with its continuous product innovation, solution optimization, and technology development.

25

Stealth BioTherapeutics Initiates Phase 2 Study of Elamipretide in Patients Hospitalized with Congestive Heart Failure

Top-line results anticipated in second half of 2017

BOSTON – October 25, 2016 – Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing investigational drugs to treat mitochondrial dysfunction associated with common diseases of aging and genetic mitochondrial diseases, today announced the initiation of IDDEA-HF, a Phase 2 study evaluating elamipretide in patients hospitalized due to congestive heart failure. Heart failure causes more than two million hospitalizations in the U.S. and Europe each year.

“In heart failure, mitochondrial dysfunction may not only be a causative factor, but may also contribute to the progression of the disease and the associated fluid build-up that causes congestion, due to muscle weakness from a lack of energy production,” said Stealth Vice President of Clinical Development Jim Carr. “In line with our findings in elderly patients enrolled in the   trial, we hope to demonstrate the ability of elamipretide to increase energy production to help the heart muscle work better, subsequently relieving congestion in the body.”

IDDEA-HF is a randomized, double-blind, placebo-controlled study to evaluate the cardiac and renal effects of daily treatment with elamipretide in patients who have been hospitalized with congestive heart failure. Up to 300 patients will be randomized within 72 hours of presentation to receive 20 mg daily elamipretide or placebo intravenously for up to seven days. The primary endpoint is change in NT-proBNP, a cardiac biomarker reflecting the level of congestion. Secondary endpoints include change in clinical status and safety and tolerability.

“IDDEA-HF is a key step in our development of therapies for common diseases of aging. These patients have failed current therapies, experiencing an episode of acute decompensation, which highlights the intense need for new options within the heart failure treatment paradigm where we believe elamipretide can have a significant impact,” said Stealth Chief Executive Officer, Reenie McCarthy. “The data from this study, together with our other ongoing trials in heart failure, will help inform our projected Phase 3 heart failure program as well as our approach to other common diseases of aging for which elamipretide may have therapeutic potential.”

24

Aduro Biotech Reports Partial Clinical Hold to Pause Enrollment in LADD Trials

BERKELEY, Calif., Oct. 24, 2016 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, announced today that it has received notice from the U.S. Food and Drug Administration (FDA) that trials with investigational agents based on its LADD (Listeria-based immunotherapy construct) platform have been placed on partial clinical hold to pause new patient enrollment. Patients currently receiving a LADD-based agent (except one currently identified patient, due to the presence of a pacemaker) are continuing to receive treatment, with several of these patients having been on study drug for six months or longer. The partial hold was initiated following notification to the FDA that a blood culture sample from an indwelling port of a metastatic pancreatic cancer patient who presented with gastrointestinal symptoms tested positive for Listeria, which is suspected to be CRS-207. The patient was administered intravenous antibiotics, subsequent blood cultures tested negative for Listeria, and the patient was reported to be doing well.

Aduro is working with the FDA to lift the partial hold so as to resume new patient enrollment in its LADD clinical trials. The company is revising study protocols in accordance with feedback from the agency, including the modification of antibiotic administration following treatment, extended patient surveillance, and, as a pre-emptive measure, exclusion of patients who are on or will receive certain immune-suppressive treatments or who have certain prosthetic devices. Aduro will be providing proposed revisions to the protocols, patient consent forms, and investigator brochures to the agency later this week.

This partial hold does not impact any ongoing development of Aduro’s two other distinct platform technologies, STING pathway activators and B-select monoclonal antibodies.

 

17

ENVISIA THERAPEUTICS RELEASES ENV515 (travoprost XR) PHASE 2 DATA SHOWING NINE-MONTH DURATION OF ACTION AFTER A SINGLE DOSE IN PATIENTS WITH GLAUCOMA

Results Provide Encouraging Outlook for Extended Treatment of Glaucoma Patients RESEARCH TRIANGLE PARK, NC – OCTOBER 17, 2016 – Envisia Therapeutics, a clinical-stage biotechnology company focused on the development of novel extended-release therapies in ophthalmology, today released an interim analysis of its ENV515 (travoprost XR) phase 2 trial in glaucoma patients showing clinically meaningful reduction in intraocular pressure (IOP) for the entire nine-month evaluation period following a single administration. ENV515 also demonstrated an IOP lowering effect comparable to prestudy topical prostaglandin analogs (XALATAN® and LUMIGAN®) and in-study topical timolol maleate 0.5% ophthalmic solution (daily eye drops). Glaucoma is the leading cause of preventable vision loss and blindness due largely in part to poor patient compliance with once-daily eye drops. “ENV515 continues to show great potential with a favorable safety profile and a sustained, clinically meaningful reduction in IOP over the initial nine months,” said Benjamin Yerxa, President of Envisia. “We plan to initiate enrollment in a new cohort of this phase 2 trial by year-end, which will enable us to evaluate the high dosage form of ENV515 that has been formulated with the goal of achieving efficacy comparable to TRAVATAN Z® with a duration greater than 9 months.”

12

New app helps workers track child development, identify autism early

By Amanda Eisenberg, Published October 12 2016, 1:41am EDT -- As employers struggle with retention rates across the board, healthcare technology company Cognoa is trying to keep a specific population employed: parents of children with developmental disorders. The Palo Alto-based company recently launched Cognoa for Employers, a mobile app that screens children as young as 18 months for developmental disorders, to assist with early detection and cut down on future healthcare costs and missed workdays. The app would be included in a benefits package for parents to assess how their young children are reaching developmental benchmarks and screens for autism, speech delays and ADHD. With one in six children in the United States diagnosed with a developmental disorder, according to the Centers for Disease Control and Prevention, the impact affects parents who work full-time. "There is a greater pressure on one of the parents to leave the workforce and stay home with the child," says Cognoa CEO Brent Vaughan. "The key benefit is it allows valuable employees to be happy and focused at work." The company built the parent-facing app to encourage early detection and intervention; the average age of an autism diagnosis is age 4, according to nonprofit Autism Speaks. An earlier diagnosis, says Vaughan, could be "the difference between attending special or normal schools” and could create a “lifelong change for a family." "We hear from parents constantly who are unable to keep a job," says Wendy Fournier, president of the National Autism Association. "Many get frequent calls to pick up their kids from school, or have lots of medical appointments and need to take time off. It’s difficult for a lot of us to be dependable employees because caring for a child with autism can be volatile: Some days are fine, some are disastrous." Cognoa’s machine learning-based platform, which has been running for two years and has data from more than 140,000 children screened by the app, attempts to minimize the number of doctor’s visits that could leave an employee to take time off, says Vaughan. Parents are asked to fill out a questionnaire that details their child’s play habits, eye contact, stomach and sleep problems, sensitivity to noise, and body and verbal language. They are also able to record videos of their children for a home-based, parent-selected evaluation, which is then compared to other videos and information in the system. The data is kept password protected and has rigorous data encryption that meet or exceed HIPAA compliance, Vaughan says. "In the detailed assessments, parents receive descriptions of specific behavioral areas where their child is meeting developmental milestones, as well as areas that correspond with any elevated risk for delay," he says. "The assessment reports also include the links to the videos the parent provided of the child’s actual behavior that corresponds with the assessment. We have found that both parents and clinicians find it valuable to see the videos of the child’s natural behavior at home." Vaughan says the app’s main competitor is standard care. However, not all pediatricians screen for developmental disorders, which is where the app bridges the gap in care for parents who might not be able to pay the co-pay or bring their child to the doctor’s office.

September 2016

28

ACD Launches BaseScope™ Suite of Tissue Analysis Assays Enabling Breakthrough Advances in Molecular Pathology

The BaseScope assay is unique in enabling spatial localization of splice variants, point mutations and highly homologous sequences within tissue samples NEWARK, Calif., Sept. 28, 2016 /PRNewswire/ -- Advanced Cell Diagnostics (ACD), a brand of Bio-Techne Corporation, announces the release of a breakthrough RNA in situ hybridization (ISH) assay delivering the unprecedented capability of detecting RNA transcripts at single base resolution. The BaseScope™ assay is available commercially as a kit for use in laboratories worldwide and via ACD's Pharma Assay Services. As one of the offerings of the Pharma Assay Services portfolio, BaseScope provides highly sensitive and specific detection of exon junction/splice variants, SNPs, small insertions/deletions, and short targets, including complex and highly homologous gene families such as MAGE and ILT in just four weeks. Dr. Yuling Luo, Founder and President of ACD, explained the significance of the launch: "BaseScope is a truly revolutionary assay providing groundbreaking insights into biological and disease mechanisms not possible with other technologies – again demonstrating our pioneering position in molecular pathology. Before the launch of the BaseScope assay, it was simply not possible to detect a specific exon-exon junction or a point mutation in situ with morphological context."

27

Aduro Biotech Presents Encouraging Preclinical Data Showing Combination Synergy of its Immunotherapy and Checkpoint Inhibitors to Increase Antitumor Efficacy

BERKELEY, Calif., Sept. 27, 2016 (GLOBE NEWSWIRE) -- Aduro Biotech, Inc. (Nasdaq:ADRO) today highlighted two posters presented at the Second CRI-CIMT-EATI-AACR International Cancer Immunotherapy Conference (CRI-AACR) in New York. The preclinical data demonstrate positive changes in the tumor microenvironment and induction of a tumor-specific immune response by Aduro’s LADD (listeria-based immunotherapy construct) and STING (Stimulator of Interferon Genes) Pathway Activator immunotherapy platform technologies. Importantly, adding a PD-1 blockade to either immunotherapy regimen significantly bolstered antitumor efficacy. “These preclinical data demonstrate the underlying mechanisms by which our LADD and STING immunotherapy platforms activate the immune system and induce robust innate immunity, facilitating a change in the tumor microenvironment which results in effective destruction of cancer cells in several preclinical models,” said Thomas Dubensky, Jr., Ph.D., chief scientific officer of Aduro. “Importantly, the combination data are even more impressive, showing increased efficacy when our LADD and STING platforms are combined with an anti-PD1 checkpoint inhibitor to combat the tumor’s ability to hide from the immune system. These data support our strategy to combine our immunotherapy regimens with checkpoint inhibitors for greater anti-tumor activity, looking toward the ultimate goal of better, more effective patient care.”

26

MicuRx Closes $55 Million Series C Financing to Support Development of Next-Generation Antibiotic MRX-I

Funds to Support Phase 3 Clinical Trials in U.S. and China HAYWARD, Calif. and SHANGHAI, Sept. 26, 2016 /PRNewswire/ -- MicuRx Pharmaceuticals, Inc. today announced the close of its $55 million Series C financing. Led by GP Healthcare Capital, the round included new investors GP TMT Capital, 3E Bioventures Capital, and Delian Capital. Mr. Yu Miao of GP Healthcare Capital will join the board of directors. "Our investors have recognized the unique benefits that our next-generation antibiotic, MRX-I, offers to patients who need better tolerated, more convenient oral and intravenous antibiotics to treat MRSA," commented Zhengyu Yuan, Ph.D., president and CEO of MicuRx. "We are very pleased to have the solid support of such a strong syndicate which will fund MicuRx through our first New Drug Application (NDA) filing in China. The fast growing pharmaceuticals market in China, presents an excellent opportunity for MRX-I. In parallel, we are pursuing development in the U.S. and other global markets." Funding from Series C financing will be used to continue development of MRX-I in both the United States and China. With the funds, the company expects to complete its Phase 3 trial using oral MRX-1 to treat complicated skin and skin structure infections (cSSSI) in China, complete one pivotal US Phase 3 trial with oral and intravenous formulations of the drug for the indication of acute bacterial skin and skin structure infections (ABSSSI) as well as file a Chinese NDA for cSSSI. The BFC group provided financial advice to MicuRx.

19

Stealth BioTherapeutics Reports Elamipretide Improved Skeletal Muscle Bioenergetics in the Elderly

Trial evaluating treatment with investigational drug, elamipretide, demonstrated improved mitochondrial function by increasing energy production after single treatment BOSTON – September 19, 2016 – Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing investigational drugs to treat mitochondrial dysfunction, today announced results from the MOTION Trial. This clinical trial evaluated the systemic delivery of elamipretide on skeletal muscle function in elderly subjects with reduced mitochondrial function, and demonstrated improved mitochondrial energy production after treatment. This increase in mitochondrial bioenergetics was comparable to the improvement resulting from six months of exercise training, shown in a similar patient population in other trials. The data from this trial were presented at the Late Breaker Poster Session of the Heart Failure Society of America Annual Scientific Meeting in Orlando, Florida on Saturday, September 17. MOTION was a Phase 2 randomized, double-blind, placebo-controlled study that evaluated 40 patients ages 60-85 with demonstrated mitochondrial dysfunction to determine the effect of elamipretide on skeletal muscle energetics and performance. The primary endpoint showed improved mitochondrial energy production (ATPmax) which increased by 30% over baseline as compared to a 10% change in the placebo group (p=0.055). The improved bioenergetics were associated with greater skeletal muscle function (p=0.004). Furthermore elamipretide was well tolerated with no treatment-emergent adverse events (TEAEs) identified.

13

Kezar Life Sciences Announces Initiation of Phase 1 Clinical Program for Lead Candidate KZR-616, a First-in-Class Immunoproteasome Inhibitor

SOUTH SAN FRANCISCO, Calif., Sept. 13, 2016 /PRNewswire/ -- Kezar Life Sciences, a company focused on the discovery and development of drugs targeting protein homeostasis, announced today the initiation of its Phase 1 randomized, double-blind clinical program for KZR-616, a selective inhibitor of the immunoproteasome. Since enrollment of the first cohort on August 9, Kezar has completed dosing of 24 subjects across three cohorts in the single ascending dose (SAD) portion of the Company's Phase 1a study, which includes both SAD and multiple ascending dose (MAD) portions. The study is expected to enroll a total of 64-80 subjects, with key endpoints including pharmacokinetics and biomarkers of target engagement, as well as safety and tolerability. In the Phase 1 program, KZR-616 is administered once-weekly as a subcutaneous injection. Pending progress of the study, the Company anticipates that it will report topline pharmacokinetics, safety, and target engagement data by the end of 2016. Additionally, in the first half of 2017 the Company anticipates commencing a Phase 1b study of KZR-616, which is expected to enroll up to 40 patients with autoimmune disorders and include a cohort with a placebo control. "We are proud to be the first company to move a selective inhibitor of the immunoproteasome into the clinic," said John Fowler, Kezar's Chief Executive Officer and Co-Founder. "The Kezar scientific team is unparalleled in its understanding of the unique biology and therapeutic potential of the immunoproteasome, and has done a tremendous job moving our lead program rapidly into the clinic.  We look forward to demonstrating the safety profile and target engagement of KZR-616 in the coming months, and launching Phase 1b and 2 trials in patients in 2017."

12

Stealth BioTherapeutics Initiates Phase 2 Study of Elamipretide in Primary Mitochondrial Disease

Study will evaluate the long-term safety, tolerability and efficacy of elamipretide in patients who completed the Phase 2 MMPOWER study BOSTON – September 12, 2016 – Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing investigational drugs to treat mitochondrial dysfunction, today announced the initiation of a longitudinal extension trial for evaluating elamipretide in primary mitochondrial disease. The study, MMPOWER-2, will be limited to patients who completed the initial MMPOWER Phase 2 study. Positive data from MMPOWER were announced in June 2016 showing statistically significant improvement in distance walked in six minutes. “Patients with rare primary mitochondrial diseases have no FDA-approved treatment options to address their needs,” said Stealth Chief Executive Officer Reenie McCarthy. “We’re committed to helping fill these significant gaps in care through our study of elamipretide. Following the promising results from our Phase 2 MMPOWER study, we’re happy to announce the initiation of MMPOWER-2, which will help us better understand the effects of longer treatment with elamipretide for these patients, who often face severe challenges completing even simple daily activities.” MMPOWER-2 is a randomized, double-blind, placebo-controlled crossover study to evaluate the safety, tolerability and efficacy of four weeks’ treatment with once-daily subcutaneous injections of elamipretide in patients with genetically confirmed mitochondrial disease. All patients in this study have previously completed the MMPOWER study.

01

ASLAN PHARMACEUTICALS AND A*STAR ENTER RON ANTIBODY LICENSING AND RESEARCH COLLABORATION AGREEMENT

Singapore, 1 September 2016 – ASLAN Pharmaceuticals (ASLAN), a biotech company focused on the development of immunotherapies and targeted agents for Asia prevalent tumour types, today announced the licensing of a novel immuno-oncology antibody, targeting RON (Recepteur d’Origine Nantais), from Singapore’s Agency for Science, Technology and Research (A*STAR) which ASLAN will develop and commercialise worldwide. RON is a receptor tyrosine kinase, and an overexpression of RON leads to increased tumour metastasis. Consequently, RON activation in tumour cells promotes aggressive disease. The RON antibody licensed by ASLAN was developed by A*STAR’s p53 Laboratory and is currently in preclinical development. The antibody has demonstrated preclinical efficacy in a range of in vivo models of human cancer. Under the terms of the agreement, ASLAN will gain global rights to develop the RON antibody and intends to commence clinical studies in 2018. Commercial terms were not disclosed. To further advance the project towards clinical trials, ASLAN and A*STAR have also entered a three-year research collaboration. ASLAN will be responsible for the design of innovative clinical development programmes, in close collaboration with A*STAR’s p53 Laboratory which will continue to be responsible for the preclinical development of the antibody assets.

August 2016

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InCarda Therapeutics Initiates Enrollment of Phase 1 Clinical Trial of Inhaled Flecainide for the Treatment of Cardiac Arrhythmias

San Francisco, California, August 31, 2016 – InCarda Therapeutics, Inc. (InCarda), a privately-held biopharmaceutical company pioneering a novel approach of treating cardiovascular conditions by delivering medications via the inhalation route, today announced that it has initiated a Phase 1 cl