UMass Medical School Announces ALS Breakthrough
Grant Welker, Worcester Business Journal
July 8, 2020
Researchers at UMass Medical School and Massachusetts General Hospital have announced a major breakthrough in ALS research: a therapy that suppresses a harmful gene that causes the nervous system disease.
The advancement, announced by researchers Wednesday and set to appear in the New England Journal of Medicine, is the latest made by UMass Medical School researchers, who’ve also identified critical genes that influence the development of ALS.
A gene called SOD1 carries a genetic defect causing ALS, or amyotrophic lateral sclerosis, which gradually harms a victim’s ability to move. New work by UMass Medical School researchers has been able to reverse that damage using something called a vector.
In a lab, researchers have been able to transform a harmless virus, replacing its DNA with a synthetic RNA, or ribonucleic acid, that’s specifically designed to attack the SOD1 gene. In a study, that altered virus was injected into participants’ spinal fluid, delivering the lab-modified DNA. Far less harmful proteins were created by the gene after the injection, researchers found.
The researchers are the first to safely treat two research participants with a synthetic microRNA, the medical school said. The hope is that by targeting the SOD1 gene, the breakthrough can slow down or even reverse the progression of that type of ALS.
The study was led by two UMass Medical School researchers: Dr. Robert Brown Jr., a professor of neurology and director of the school’s neurotherapeutics program, and Dr. Christian Mueller, an associate professor of pediatrics. They worked in consultation with two doctors at Mass. General: Dr. Merit Cudkowicz, the hospital’s chief of neurology, and Dr. James Berry, the chief of the hospital’s division of ALS and motor neuron diseases.
“The biggest takeaway from this study is that we delivered a new class of silencing gene therapy to patients and suppressed levels of the ALS gene SOD1 quite effectively,” Dr. Mueller said in a statement.
Apic Bio, a Cambridge company founded by Brown and Mueller, has licensed the technology from the medical school and is leading the next phase of clinical trials.
Dr. Brown has long been a leader in ALS research. In 1993, a team he led discovered the first gene linked to hereditary ALS, which is now known as SOD1.
Among other ALS research done at UMass Medical School, Dr. John Landers, a neurology professor, has also discovered new genes related to ALS that could help researchers find a cure. Some of the research has been funded by proceeds from the Ice Bucket Challenge, a popular fundraising initiative in 2014.
ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. The disease gradually makes it more difficult for a victim to speak, swallow, walk or even breathe.
More than 5,000 people are diagnosed with it each year, and face a life expectancy of two to five years, according to the ALS Association. About 5% to 10% of ALS cases are inherited. Toxic mutations in the SOD1 gene account for about 20% of inherited ALS cases, according to UMass Medical School.
Atea Pharmaceuticals Announces IND Clearance of AT-527 for COVID-19 and $215 Million Financing
May 20, 2020
BOSTON, May 20, 2020 (GLOBE NEWSWIRE) — Atea Pharmaceuticals, Inc., a biopharmaceutical company engaged in the discovery and development of next-generation therapeutics for severe human viral infections, today announced a $215 million Series D financing. The financing was led by Bain Capital Life Sciences and also included new investors RA Capital Management, Perceptive Advisors, Rock Springs Capital, Adage Capital Management, funds and accounts managed by T. Rowe Price Associates, Inc., Redmile Group, and Omega Funds. Existing Atea investors, including Morningside Ventures, Cormorant Asset Management, Ally Bridge Group, and Sectoral Asset Management, as well as other investors also participated in this financing.
Atea also announced today that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug application (IND) for AT-527, a novel, oral, purine nucleotide prodrug, for the treatment of adult patients hospitalized with moderate COVID-19 disease, with one or more risk factors for poor outcomes. A Phase 2 clinical trial, scheduled to begin shortly, will evaluate the safety and efficacy of AT-527 in this patient population.
AT-527 is a highly selective, orally administered direct acting antiviral, (DAA) designed to inhibit the RNA polymerase enzyme, a key element in the replication machinery of RNA viruses. Antiviral activity of AT-527 has been observed in vitro and in vivo against replication of multiple RNA viruses including, but not limited to, human coronaviruses and flaviviruses.
In addition to supporting its work to find a treatment for COVID-19, Atea expects to also apply proceeds from this financing towards advancing its diverse pipeline of highly selective DAAs that target other severe RNA viral infections. Atea’s pipeline currently includes investigative treatments for hepatitis C virus, dengue virus, and respiratory syncytial virus, in addition to its COVID-19 program.
“We are delighted to have the strong support of this group of blue-chip healthcare investors,” said Jean-Pierre Sommadossi, PhD, Atea’s Founder, Chairman, and Chief Executive Officer. “Atea’s portfolio is focused on developing novel, best-in-class, potent DAA’s and we have shifted all of our immediate resources and our team’s deep expertise in virology and pharmacology to help address the unmet needs in the fight against the COVID-19 pandemic. An oral treatment for COVID-19 patients should prevent progression of the disease and may help lessen the burden on critical inpatient resources. Atea is moving rapidly, in concert with regulatory authorities, to determine if our oral DAA is a safe and effective therapeutic against COVID-19.”
“Atea’s team has an outstanding track record in developing novel, potent DAAs, which we believe can contribute to the urgent fight against the COVID-19 pandemic and other RNA viruses,” said Andrew Hack, M.D., Ph.D., Managing Director of Bain Capital Life Sciences. “We are pleased to partner with Atea’s leadership team and an outstanding group of leading healthcare investors as Atea advances its diverse pipeline of transformative antiviral medicines”
AT-527 is an investigational, oral, purine nucleotide prodrug, which has demonstrated in vitro and in vivo antiviral activity against several enveloped single-stranded RNA viruses, including human flaviviruses and coronaviruses. This highly selective purine nucleotide prodrug was designed to uniquely inhibit viral RNA dependent RNA polymerase, an enzyme that is essential for the replication of RNA viruses. Antiviral activity and safety of AT-527 has been demonstrated in Phase 2 clinical studies of hepatitis C patients. AT-527 is not yet licensed or approved for any indication in the U.S. or any other country.
About Atea Pharmaceuticals
Atea Pharmaceuticals is a clinical stage biopharmaceutical company engaged in discovering and developing best-in-class therapies to address the unmet medical needs of patients with severe viral diseases. Our lead programs are focused on the development of orally-administered direct acting antivirals for the treatment of patients with mild to moderate COVID-19 in the hospital and community settings, the treatment of patients with chronic hepatitis C infection, the treatment of patients with dengue, and the treatment of high-risk patients with severe respiratory syncytial virus infection. Our medicinal chemistry, virology, and pharmacology expertise, bolstered by our collective experience in drug development, enables us to pioneer new advancements in antiviral science. Leveraging the power of our purine nucleotide prodrug platform, our goal is to rapidly advance novel drug candidates with optimal therapeutic profiles for RNA virus targets. Founded by its Chairman and Chief Executive Officer, Jean-Pierre Sommadossi, PhD, Atea began operations in 2014 and is headquartered in Boston, MA. For more information about Atea and our pipeline of products please visit our company website at https://www.ateapharma.com/.
Inotrem Secures Strategic Licensing Agreement for a Companion Diagnostics Test in Septic Shock
PARIS, January 21, 2020 (BUSINESS WIRE) — Inotrem S.A., a biotechnology company specialized in the development of immunotherapies targeting the TREM-1 pathway with potential applications for acute and chronic inflammatory syndromes, announced today that it has entered a worldwide licensing agreement with Roche Diagnostics for the commercialization of a mechanism-based companion diagnostic test using a soluble plasma protein (sTREM-1).
This agreement consolidates existing ties between Roche Diagnostics and Inotrem who are jointly developing since 2017 an in vitro assay for measurement of sTREM-1 in plasma samples of septic shock patients. Measurement of sTREM-1 in blood provides a valuable indicator for the severity and outcome prediction of septic shock patients.
One of the main issues with septic shock is the heterogeneity of the patient population. The companion diagnostic test will allow a stratification of patients to identify those who are more likely to respond to Inotrem’s treatment. Septic shock is the ultimate complication of sepsis. The incidence of septic shock continuously raises, and mortality remains elevated (35%) in developed countries. There is currently no specific mechanism-based therapy approved for this indication besides antibiotics and symptomatic treatment. Inotrem’s therapeutic solution has the potential to become the first targeted treatment for septic shock.
“We are delighted to extend our partnership with Inotrem to make the sTREM-1 test, which we are co-developing at Roche, available for patients globally. Through the development and commercialization of this novel companion diagnostic test we are committed to delivering a solution that enables much needed, better decisions for sepsis patients”, said Ann Costello, Global Head Centralised and Point of Care Solutions, Roche Diagnostics.
“Roche Diagnostics’ licensing agreement is an important milestone for us: it is a strong endorsement of Inotrem’s innovative approach targeting the TREM-1 pathway, and it allows us to further focus on our core mission: the development of a nangibotide-based septic shock treatment” added Dr. Jean-Jacques Garaud, CEO of Inotrem.
Inotrem has recently initiated its Phase IIb study in septic shock patients (ASTONISH trial) and enrolled its first patient. The study aims at demonstrating efficacy of its lead compound, nangibotide, and bring a clinically relevant proof of clinical activity in septic shock patients. This study also intends to confirm the value of soluble TREM-1 as a potential companion diagnostic test to identify patients more likely to benefit from nangibotide treatment.
PhoreMost Enters Multi-Project Drug Discovery Collaboration With Otsuka Pharmaceutical
CAMBRIDGE, England, January 21, 2020 (BUSINESS WIRE) –PhoreMost Limited, the UK-based biopharmaceutical company dedicated to drugging ‘undruggable’ disease targets, today announced it has entered into a multi-project collaboration with Otsuka Pharmaceutical Co., the world-leading pharmaceutical company dedicated to the research and development of highly-innovative drugs and diagnostics. Financial details of the agreement are not disclosed.
PhoreMost will deploy its in-house expertise and next-generation phenotypic screening platform, SITESEEKER®, towards disease relevant pathways nominated by Otsuka. Novel targets identified will be further validated and characterised by Otsuka as part of its internal development pipeline, with an initial focus on gene therapy applications of identified targets.
PhoreMost’s SITESEEKER platform exploits protein shape diversity to find functionally active peptides, significantly enhancing the power of phenotypic screening and translation into therapeutic modalities. Based on proprietary protein interference, or ‘PROTEINi®’, technology PhoreMost uses SITESEEKER to probe the entire proteome in a live cell environment for novel druggable targets linked to any chosen disease. This enables the systematic discovery of functionally active peptides which are directly linked to useful therapeutic applications.
Dr Chris Torrance, CEO of PhoreMost, said: “This collaboration with Otsuka is further recognition of the power of the SITESEEKER approach to drive the identification of novel, druggable targets. We are particularly excited to be exploring not only small molecule therapeutics but also gene therapy applications of our platform.”
Alyce Named Leader in the G2 Winter 2020 Account-Based Execution Software Grid Report
BOSTON, MA, January 15, 2020 (BUSINESS WIRE) – Alyce, the AI-powered gifting platform that’s redefining sales and marketing outreach, today announced its position in the Leader quadrant in G2’s Winter 2020 Grid Report for Account-Based Execution. Leaders are determined by their high levels of customer satisfaction and likeliness to recommend ratings from real users on G2, the world’s leading business solutions review website. As part of this Winter 2020 report, Alyce also scored highest in G2′s Relationship Index for the Account-Based Execution category (with a score of 9.59 out of 10), which is measured based on ease of doing business, likely to recommend, and quality of customer support.
Alyce also scored highest in the same Account-Based Execution category (9.21 out of 10) in G2′s Usability Index, ranking companies based on ease of administration, ease of use, and meeting other key requirements.
“Enterprise companies are turning to Alyce to transform their approach to growing their business, looking to create personal experiences and authentic connections that resonate with buyers and customers,” said Sean MacPherson, Head of Customer Success. “Alyce’s commitment to creating lasting and sustained relationships with our customers and helping them see success is resonating with the market and we are thrilled by G2’s recognition as a category leader. This is truly a testament to the dedication, spirit, and strength of our customer success team.”
“Rankings on G2 reports are based on data provided to us by real users of the products,” said Michael Fauscette, Chief Research Officer at G2. “Technology buyers use the ranking on G2 to find the best software products based on the real-world voice of the user, accelerating their digital transformation projects and gaining competitive advantage.”
Alyce has received more than 125 G2 Crowd reviews. Review highlights include:
- “Alyce is the best door opener – companies that I’ve been working for months, not responding whatsoever, finally answering me because it’s a unique AND personal way to reach someone.” ~Allison F.
- “Creative and personalized way to engage prospects. Alyce’s is a great way to engage with prospects on a personal level. The AI will recommend gifts to send to prospects based on a variety of factors – all of which increase your chance of getting a response from them. Giving the option to exchange gifts or donate the value of the gift to charity is also an awesome aspect of Alyce – I am really a big fan of the charity donation.” ~William R.
Since its inception, Alyce has been focused on creating real personal experiences that help businesses grow with the only platform that gives sales, marketing, and customer teams the ability to create individual personal gifts at scale. Fully focusing on the recipient and customers’ interests, rather than sending everyone the same generic item, Alyce bridges the physical and digital marketing worlds to help companies create breakthrough connections engaging people on their own terms. Providing the power of choice, recipients can select the personal gift recommended, pick something else from the Alyce gift marketplace, or even choose to donate the value of a gift to a charity of their interest.
“We’ve had a huge year at Alyce with 1,200% growth, closing out the year with some of the largest enterprise brands, including 6Sense, Bandwidth, Citrix, Dialpad and ServiceNow selecting our platform, and more importantly, investing in our unique personal experience business approach to help them scale and grow more effectively,” said Greg Segall, founder, and CEO of Alyce. “And now being recognized by G2 is an amazing milestone honor and testament to our focus on making our customers incredibly successful, with them accomplishing tremendous ROI – leading to greater expansion, adoption, and utilization of the Alyce platform throughout their organizations.”
Evonetix and Imec develop MEMS IC to scale up production of gene synthesis system
By David Manners
January 15, 2020 | ElectronicsWeekly.com — Evonetix’s technology controls the synthesis of DNA at many thousands of independently controlled reaction sites or ‘pixels’ on the chip’s surface in a highly parallel fashion.
Following synthesis, strands are assembled on-chip into double-strand DNA in a process that identifies and removes errors, enabling accuracy, scale and speed that is several orders of magnitude better than conventional approaches.
Under the terms of the collaboration, Imec will work with Evonetix to scale up manufacturing of the MEMS technology on 8-inch silicon wafers, enabling Evonetix to supply customers in volume.
Imec is able to leverage its experience in manufacturing silicon for life sciences applications to transfer the novel Evonetix process to their foundries and to manage further expansion in volume.
“Evonetix has developed an innovative approach that integrates physics and biology to enable the production of high-fidelity long DNA in a highly parallel fashion,” says Imec’s Peter Peumans.
Freight Farms goes to school
The shipping containers are used for growing food — and for teaching vital lessons
By Andrea Pyenson
January 15, 2020 | The Boston Globe
Inside the big white shipping container parked behind a classroom building on the campus of the Rivers School in Weston, it smells like a verdant field on a warm spring day, with a degree of humidity that is completely at odds with the cold, dry air outside. A variety of lettuces, herbs, and a smattering of other vegetables grow on vertical towers in adjustable rows. The sixth-grade students who maintain the school’s Freight Farm cycle through in groups of four to reap the bounty of work they started at the beginning of the 2019-2020 academic year. The first harvest day was in late October.
“They all love to come in here,” says Emily Poland, who teaches eighth-grade science and is the farm director at this independent school for grades 6 through 12. The Freight Farm and related projects are built into the sixth-grade curriculum, incorporating humanities, social justice, and science, among other subjects. Students spend time there once a week planting, cleaning, and harvesting. Farming is a club activity for the school’s high school students, who can go in during their free time.
Based in Boston, Freight Farms manufactures technologically advanced hydroponic farming systems. In 320-square-foot, climate-controlled shipping containers, users can grow up to 13,000 plants at a time, vertically, without soil. The company was founded in 2010 by Brad McNamara and Jon Friedman. Several area schools, among them Rivers, Boston Latin School, and Worcester State University, are using the farms to grow food for their own communities, for their neighbors, and as educational tools.
For Poland, managing the farm was a natural extension of her teaching. “I like to create curriculum. I care about food. I like to be outside,” she says. One of the sixth-graders’ annual activities, which combines academics with community service, is cooking a meal for the Natick Open Door at St. Paul’s Episcopal Church. These are hosted every week and attended primarily by seniors. Poland explains that planning the meal incorporates math skills because the students have to scale recipes to feed up to 45 people. And naturally they use their own greens in the salads.
The students run a farmers market in the spring. And this year they are maintaining a (very micro) CSA, which one parent won in an auction. The school’s chef, Michael Clancy, also gets involved, using the students’ produce in the dining room, and helping them cook with what they grow — so far this year they have made pesto and herb vinaigrette. “Their pride is really amazing,” he says.
Boston Latin, a public exam school for grades seven through 12, acquired its farm in 2013 after students in the Youth Climate Action Network won the $75,000 prize in the Global Green Schools Makeover Competition. Farming is a student-run after-school activity here, under the guidance of eighth-grade history and civics teacher Cate Arnold, whose evident affection for her students appears to be reciprocal.
Though roughly 70 students have signed up as student farmers, there is a smaller core group that farms regularly, with an even smaller leadership team that is trained by Freight Farms. At the beginning of each academic year the leaders meet to plan what they will grow, who will work to train new students, and organize schedules. They keep track of chores on a whiteboard in the farm.
Addy Krom, a junior, notes of the farm, “You can come in, it’s a whole different environment. All the stress from school [goes] away.” Adds sophomore Azalea Thompson, “This makes locally grown food more accessible to the city.” The students give the food they grow to faculty members, bring some home, and are working to create a CSA. With Arnold’s help they are also trying to reestablish a more consistent connection to a food pantry in Jamaica Plain, where a former Boston Latin parent, recently deceased, used to deliver their greens.
At Worcester State, Mark Murphy, associate director of dining services, oversees the Freight Farm, which sits outside of Sheehan Hall, the school’s newest dormitory and site of its main cafeteria. Rich Perna, former director of dining, made the decision to purchase the farm five years ago, says Murphy, “to bring hyperlocal produce to the campus.”
Murphy has been responsible for the farm for the last two years. An employee of Chartwells, which has the contract for all of the school’s food services, he grows almost all of the greens for the cafeteria, as well as for alumni catering events, and the salad bar in the food court in the building next door. “I’m learning from trial and error,” he says.
At full capacity, Murphy explains, the farm produces about two acres’ worth of crops. He is constantly looking for different varieties of lettuce that will appeal to the students and is currently “trying to figure out a gourmet mix.” In addition to three varieties of lettuce, he grows kale, rainbow Swiss chard, parsley, and basil. He coordinates with the cafeteria’s cooks, telling them what he is growing so they can plan menus to incorporate the farm’s production.
Though WSU students are not currently working in the farm Murphy says he promotes the fact that most of the greens in the cafeteria are grown right outside the door. And, he says, “We’re trying to get the word out to get students involved.”
Through a partnership with the Worcester Public Schools and its program that helps young adults with differences transition from school to the workforce Murphy has three part-time helper/trainees. Once a week three students, who have completed high school with a certificate, come (often with a job coach from the program) to seed, plant, harvest, and clean. Murphy is in the process of hiring one of the students, who has aged out of the program. She “has a lot of passion for the farm,” he says.
“I never thought we’d be growing food inside a container,” Murphy says. “I think it may become a necessity someday.”
BPS and 2020 On-site Launch ‘Vision for Boston’ Pilot Program
BOSTON, MA, January 13, 2020 — The Boston Public Schools (BPS), 2020 On-site and eyewear brand Warby Parker have partnered to launch “Vision for Boston,” a pilot program offering BPS students free, comprehensive eye examinations and corrective eyewear.
The program brings the eye doctor to students in their schools. 2020 On-site licensed optometrists and specialists will visit 13 BPS schools over 20 days beginning January 6. All students in participating schools are eligible to receive free eye exams, and any form of health insurance will be accepted. Students without health insurance will not be asked to pay. Any student found to need eyeglasses can choose a pair at no cost.
“The Boston Public Schools is committed to providing our students with the resources they need to thrive academically and throughout their lives,” said BPS Superintendent Brenda Cassellius. “A healthy body means a healthy mind, and this all hands on deck community support will allow our young people to become more engaged in their education, further preparing them for success.”
2020 On-site, a leading provider of on-site vision care for businesses and schools, founded in Boston, previously partnered with individual BPS schools beginning in 2015. In recent years, 2020 On-site has visited over 30 schools in the Boston area. Other organizations, such as the New England College of Optometry (NECO), Boston Medical Center, and Prevent Blindness have also partnered with BPS Health Services to assist with state-mandated vision screenings for many years. BPS Health Services met with these organizations to combine and expand efforts, with the goal of creating a district-wide vision initiative.
“It’s fantastic that we’re kicking off 2020 by making sure our students’ vision needs are being met inside their own schools,” said Boston School Committee Chairperson Michael Loconto. “I am grateful to 2020 On-site and all the partner organizations that help us to provide essential vision screenings and optometry services for students, easing the financial burden on our families.”
There are several mandated health screenings for students required by state law. All BPS students in grades K0-5, 7, and 10 must receive a vision screening in school. Students are screened by BPS nurses and health paraprofessionals. In addition, NECO students and instructors also visit schools to assist with screenings. All students who do not pass their vision screenings are flagged as requiring a full eye examination.
Families were asked to complete consent forms to permit students to participate in the pilot program. 2020 On-site will provide students in grades 4-12 a complete, comprehensive eye exam. Any student found to need corrective eyewear will have the opportunity to choose a pair at no cost. Students in grades K0-3 can also choose eyeglasses, for no cost, if the school has a copy of a recent prescription, written within the last year.
Through the pilot program, Warby Parker will provide free prescription eyeglasses to students that need them. The company has designed a line of glasses specifically for students that are part of this program and Warby Parker’s Pupils Project initiative.
“Warby Parker opened its first store in Boston nearly seven years ago. We’ve loved being part of the city’s rich, historic community and look forward to making an impact on its students by providing access to free glasses through this public-private partnership,” says co-founder and co-CEO Neil Blumenthal.
Vision exams will take place both inside schools and onboard 2020 On-site’s mobile vision center, an eye clinic on-the-go. 2020 On-site optometrists and vision specialists will conduct pre-testing, including capturing a high-definition image of the retina and an eye pressure test for glaucoma detection, before students receive a comprehensive eye exam using fully-digital, top-of-the-line equipment.
“So much of learning is visual. While we know that long-term educational performance is closely linked to a child’s ability to see clearly in class, many students lack access to routine eye exams,” said Alexis McLaughlin, CEO of 2020 On-site. “2020 On-site is incredibly proud to once again partner with Boston Public Schools to bring free eye exams and glasses to students who need them. Expanding access to vision care for all is central to 2020 On-site’s mission, and we can’t think of a better place to channel our passion than in our own community.”
Thirteen BPS schools are participating in the pilot. Schools were recommended by Boston Public Schools Health Services Department due to their high need of vision support, while school administration and school nurses advocated for their school communities to participate. The participating schools are: Blackstone Elementary, BTU Pilot K-8, Conley Elementary, Curley K-8, East Boston High, Henderson Upper, Hernandez K-8, Irving Middle, JFK Elementary, King K-8, McCormack Middle, Sumner Elementary and Umana Academy.
Apellis Pharmaceuticals Announces Closing of Public Offering of Common Stock and Full Exercise of Option to Purchase Additional Shares
WALTHAM Mass., Jan. 13, 2020 (GLOBE NEWSWIRE) — Apellis Pharmaceuticals, Inc. (Nasdaq:APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced the closing of its previously announced underwritten public offering of common stock, including the exercise in full by the underwriters of their option to purchase an additional 1,425,000 shares at the public offering price of $37.00 per share. The exercise of the option to purchase additional shares brought the total number of shares of common stock sold by Apellis to 10,925,000 shares and increased the amount of gross proceeds, before deducting underwriting discounts and commissions and expenses payable by Apellis, to approximately $404.2 million.
Citigroup, J.P. Morgan and Evercore ISI acted as joint book-running managers for the offering. Cantor Fitzgerald & Co. and Baird acted as lead managers for the offering.
Meissa Vaccines Receives U.S. FDA Fast Track Designation for Respiratory Syncytial Virus Vaccine, MV-012-968
SOUTH SAN FRANCISCO, Calif., Jan. 10, 2020 (BUSINESS WIRE) — Meissa Vaccines (“Meissa”), a biotechnology company developing vaccines to prevent viral respiratory infections, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to MV-012-968, an investigational, live attenuated vaccine for protection against respiratory syncytial virus (RSV) infection. The FDA’s Fast Track program expedites the development and review of drugs and biologic agents demonstrating potential to fill an unmet medical need in the treatment or prevention of serious conditions.
With Fast Track Designation, Meissa is eligible for early and frequent interactions with FDA reviewers to discuss all aspects of the clinical development plan for MV-012-968, ensuring that the appropriate data are collected to support an application for vaccine licensure. The frequency of communication granted by Fast Track Designation assures that questions and issues are resolved quickly, often leading to earlier approval and access by patients.
Additionally, Fast Track Designation may allow Meissa to submit data for the MV-012-968 Biologics License Application (BLA) before the full application is complete (“Rolling Review”) and receive BLA review under an expedited time frame (“Priority Review”), if relevant criteria are met. A Phase 1 clinical trial evaluating the safety and immunogenicity of MV-012-968 is presently recruiting healthy adult volunteers.
“Fast Track Designation of MV-012-968 accelerates our ability to develop a much-needed RSV vaccine,” said Martin Moore, Ph.D., cofounder and CEO of Meissa. “A safe and effective RSV vaccine is a significant global health priority, and if we are successful, we can save thousands of lives and help millions of patients around the world.”
RSV is the most common cause of acute lower respiratory tract infections in young children and infects nearly all children by two years of age. The virus is responsible for more than 30 million acute lower respiratory infections and nearly 60,000 deaths per year among children under five worldwide.
Elysium Health™ Announces Clinical Trial to Study Correlations Between Epigenetic Aging and Nicotinamide Adenine Dinucleotide (NAD+) Levels in Healthy Adults
NEW YORK, Jan. 9, 2020 (PRNewswire) – Elysium Health, Inc.™, a life sciences company developing clinically validated health products based on advancements in scientific research, today announced the initiation of a clinical trial at Duke Clinical Research Institute to study correlations between biological age and circulating NAD+ levels in healthy adults, in addition to other clinical biomarkers that have been implicated in aging. As opposed to marking calendar years, biological age is the average age an individual’s body is expected to function and is a powerful measure of overall health and wellness. Biological age will be calculated using Elysium Health’s next-generation epigenetic platform, Algorithmic Platform for Epigenetic Examination (APEX), which the company developed to support the 2019 launch of its at-home epigenetic test Index.
Published biological age applications such as DNAm PhenoAge typically look at hundreds of sites on the genome. To ensure greater accuracy at the individual level, Elysium Health head of bioinformatics and Yale School of Medicine professor Dr. Morgan Levine led the development of a proprietary measure that examines global methylation patterns across more than 100,000 sites in collaboration with Elysium Health bioinformatics director Dr. Tina Hu-Seliger. A key consideration for developing the methodology that underlies APEX and Index was the need to refine the technology to address the inherent variability of earlier epigenetic age predictors. Later this year, Elysium Health intends to publish the data that supports its methodology, and which demonstrates that Index is a better predictor of biological age than earlier measures.
As an essential coenzyme involved in hundreds of metabolic processes, NAD+ is critical to cellular metabolism; and its reduced form, NADH, is required for mitochondrial metabolism and the resulting synthesis of ATP for cellular energy creation. NAD+ is also utilized by sirtuins, often referred to as the “guardians of the genome.” NAD+ levels have been shown to decline with age in humans in circulating blood as well as skin and brain tissue. Although the underlying causes for deteriorating NAD+ levels have not yet been fully determined, understanding correlations between epigenetic aging and NAD+ levels may help elucidate the benefits of maintaining NAD+ levels with age.
“Over the past decade, epigenetic tests have evolved to measure biological age with an increasing level of accuracy,” said Elysium Health chief scientist and director of the Glenn Center for Biology of Aging Research at MIT Dr. Leonard Guarente. “Having studied aging for more than 30 years, it’s my belief that the field is at a tipping point. The newfound ability to measure biological age allows us to study whether interventions can have an impact, thus unlocking the potential to realize the benefits of aging research in our lifetime.”
This study is intended to further the understanding of the links between NAD+ levels and other markers of biological age. In the study, NAD+ levels in whole blood, interleukins, inflammatory cytokines, growth factors, omega-3 polyunsaturated fatty acids, and patterns in DNA methylation will be measured and compared in age-matched individuals to determine whether NAD+ levels are associated with biological age.
One hundred and seventy healthy adults ages 25–80 will be enrolled in the study, which is scheduled to complete in May 2020. More information can be found on www.clinicaltrials.gov under the identifier NCT04220658.
Allysta Pharmaceuticals, Inc. Doses First Patient with ALY688 Ophthalmic Solution in Phase 1/2a Dry Eye Study
SAN MATEO, CA, January 8, 2020 (ACCESSWIRE) — Allysta Pharmaceuticals, Inc. (Allysta) today announced dosing of the first patient in its Phase 1/2a trial (ALY688-201) of ALY688 Ophthalmic Solution for the treatment of dry eye disease. Dry eye is a very common condition affecting millions of people in the US and causes eye symptoms (e.g., burning, foreign body sensation, pain) which can limit daily activities such as computer use, reading and driving. In advanced cases, significant inflammation and even scarring of the eye surface can occur.
ALY688 is a novel peptide agonist that binds to and activates adiponectin receptors which are widely distributed on the ocular surface. Following receptor binding, it acts to reduce inflammation and promote healing of injured cells lining the ocular surface. In animal models of dry eye disease, this resulted in significant improvement in corneal damage, tear integrity, and tear volume associated with reductions in inflammatory cells and cytokines in the eye.
“This marks a significant milestone for Allysta, as we now progress from a preclinical into a clinical stage company. We look forward to completing this trial by mid-year and reporting data in the second half of 2020,” said Henry Hsu, M.D., Chief Executive Officer and President of Allysta.