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.
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: firstname.lastname@example.org; 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: email@example.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.
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.”
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.
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.”
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.
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.”
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.”
‘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.
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.”
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.
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.
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.
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.
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.
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.
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.
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.”
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."
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.
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.’
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.
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.
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.
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.”
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.
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.
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.
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.
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.
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.”
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."
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.”
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.”
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.”
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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).
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.”
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.
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.
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.
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.
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.
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.
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.
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.”
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.
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.iii, iv
“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.”
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.
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.”
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."
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.”
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.
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."
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”.
Cognoa raises $11.6M to continue validation, FDA submission for child development assessment app
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.
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.
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.
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.
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.
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.”
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.
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.
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. ”
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.”
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.
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.
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.
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.
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.
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.”
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.”
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."
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.”
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.