Amyotrophic Lateral Sclerosis 1

SOD1- ALS is a devastating, uniformly fatal, and ultra-rare genetic form of ALS that represents approximately two percent of all ALS cases1 Health Canada regulatory decision on tofersen New Drug Submission expected in early 2025 TORONTO, March 19, 2024 /CNW/ - Biogen Canada Inc. announced today that Health Canada has accepted for review a New Drug Submission(NDS) for tofersen for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. If approved, tofersen will be the first treatment indicated in Canada to target a genetic cause of ALS. "The tofersen New Drug Submission represents a critical milestone for the Canadian ALS community and those suffering from this ultra-rare genetic form of the disease that robs them of time with loved ones," said Eric Tse, General Manager of Biogen Canada. "For over a decade, Biogen has been committed to advancing ALS research to provide a deeper understanding of this devastating disease. We are passionate about doing what we can to make a difference and are deeply committed to the rare disease community." According to the ALS Society of Canada, ALS currently impacts an estimated 4,000 Canadians, with approximately 1,000 new Canadians diagnosed each year.2 The NDS is based on results from the Phase 3 VALOR trial, a 28-week randomized, double-blind, placebo-controlled clinical study, which assessed the safety and efficacy of tofersen in patients with weakness attributable to ALS and a confirmed SOD1 mutation. A regulatory decision on the New Drug Submission is expected in early 2025. About tofersen Tofersen is an antisense oligonucleotide (ASO) designed to bind to SOD1 mRNA to reduce SOD1 protein production. The U.S. Food and Drug Administration granted accelerated approval for QALSODY™ (tofersen) to treat amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with tofersen. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).3 Tofersen also received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommending its marketing authorization under exceptional circumstances for the treatment of adults with ALS associated with a mutation in the SOD1 gene.4 A decision on a marketing authorization in the European Union is expected in the second quarter of 2024. Biogen licensed tofersen from Ionis Pharmaceuticals, Inc. under a collaborative development and license agreement. Tofersen was discovered by Ionis. In addition to an ongoing open label extension (OLE) of VALOR, tofersen is being studied in the Phase 3, randomized, placebo-controlled ATLAS study to evaluate whether tofersen can delay clinical onset when initiated in presymptomatic individuals with a SOD1 genetic mutation and biomarker evidence of disease activity (elevated plasma NfL). More details about ATLAS (NCT04856982) can be found at clinicaltrials.gov. About Amyotrophic Lateral Sclerosis and SOD1-ALS Amyotrophic lateral sclerosis (ALS) is a rare, progressive, and fatal neurodegenerative disease that results in the loss of motor neurons in the brain and the spinal cord that are responsible for controlling voluntary muscle movement. People with ALS experience muscle weakness and atrophy, causing them to lose independence as they steadily lose the ability to move, speak, eat, and eventually breathe. Average life expectancy for people with ALS is three to five years from time of symptom onset.5 Multiple genes have been implicated in ALS. Genetic testing helps determine if a person's ALS is associated with a genetic mutation, even in individuals without a known family history of the disease. More than 15 percent of people with ALS are thought to have a genetic form of the disease;6 however, they may not have a known family history of the disease.1 SOD1-ALS is diagnosed in approximately two percent of all ALS cases.1 In people with SOD1-ALS, mutations in their SOD1 gene cause their bodies to create a toxic misfolded form of SOD1 protein. This toxic protein causes motor neurons to degenerate, resulting in progressive muscle weakness, loss of function, and eventually, death.6 Biogen's Continuous Commitment to ALS For over a decade, Biogen has been committed to advancing ALS research to provide a deeper understanding of all forms of the disease. The company has continued to invest in and pioneer research despite making the difficult decision to discontinue a late-stage ALS asset in 2013. Biogen has applied important learnings to its portfolio of assets for genetic and other forms of ALS, with the goal of increasing the probability of bringing a potential therapy to patients in need. These applied learnings include evaluating genetically validated targets in defined patient populations, pursuing the most appropriate modality for each target and employing sensitive clinical endpoints. In addition to tofersen, the company has a pipeline of investigational drugs being evaluated in ALS, including BIIB105. About Biogen Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patient lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment, to deliver long-term growth. Biogen has been proudly serving Canadian patients for more than 25 years. For information about Biogen Canada, please visit . SOURCE Biogen Canada

None  Welcome to this week's Chutes & Ladders, our roundup of hirings, firings and retirings throughout the industry. Please send the good word—or the bad—from your shop to Max Bayer or Gabrielle Masson, and we will feature it here at the end of each week. Voyager returns to Biogen’s orbit, plucking another executive Voyager Therapeutics You can always count on Al Sandrock to have his finger on the pulse of Biogen. The 21-year vet of the neuroscience pharma has returned (metaphorically speaking) to his old haunting grounds, leaving with fresh talent. Voyager announced it was hiring Toby Ferguson, M.D., as its chief medical officer, effective March 25. Ferguson was most recently vice president and head of neuromuscular development at Biogen, where he joined in 2013. He started as a medical director and advanced to executive medical director, before taking his current position.  His claim to fame was leading the team that produced SOD1 ALS med Qalsody, which received FDA accelerated approval based on the reduction of a disease biomarker. In light of Amylyx’s recent late-stage failure, Qalsody is arguably the most significant ALS treatment advancement in recent years. Voyager is currently working on a SOD1 gene therapy that’s entering IND-enabling studies.  Before joining Biogen, Ferguson was an assistant professor of neurology at Temple University in Philadelphia. Release Pfizer names new CSO for anti-infectives research unit  Pfizer  Despite Pfizer making its oncology ambitions clear, the New York pharma does in fact have additional assets elsewhere, including anti-infectives. Much of that work centers on COVID treatment Paxlovid, with additional candidates aimed at RSV infection and complicated urinary tract infections.  Now, the company is bolstering that team, hiring Isabel Najera, Ph.D., as chief scientific officer of the anti-infective research unit. She previously was the head of research for infectious disease at Janssen, before the pharma dismantled and divested from the work altogether.  Najera cut her teeth at Roche, where she spent a total of 19 years across a number of different positions. She started in 1999 as a research scientist working on hepatitis C virology and spent the last four years there as the deputy head of discovery and head of clinical microbiology for inflammation, immunology and infectious diseases. Rigel hires ex-Sangamo CMO for the same role  Rigel Pharmaceuticals Lisa Rojkjaer, M.D., is joining Rigel as executive vice president and chief medical officer, the biotech announced this week. She joins from Sangamo where she held the same role. Rojkjaer at Sangamo for less than a year, from May 2023 to March 2024. She spent more than six-and-a-half years at two prior CMO gigs, with Viracta Therapeutics and Nordic Nanovector, respectively. She’s also spent more than six years across two stints at Novartis. Release > FibroGen has tapped Deyaa Adib, M.D., to serve as the oncology company’s SVP and chief medical officer. Before joining FibroGen, Adib was Triumvira Immunologics’ CMO and previously held roles at Rain Therapeutics, Blueprint Medicines, Baxalta, ARIAD Pharmaceuticals, Aventis, Sanofi and Astellas Pharma. Release > Jullian Jones, Ph.D., has joined MOMA Therapeutics to serve as the platform company’s chief business officer. Jones comes from Monte Rosa Therapeutics, where she also held the CBO title and oversaw the biotech’s recent partnership with Roche Pharmaceuticals. Release > Capsida Biotherapeutics has promoted Rob Murphy from his role as VP of technical operations to chief manufacturing and quality officer. Murphy joined the biotech in 2020 and helped design, build and staff Capsida’s state-of-the-art gene therapy manufacturing site. Release > Apellis Pharmaceuticals is appointing Philip Ferrone, M.D., to serve as the company’s chief medical retina adviser, effective March 18. Ferrone currently is, and will continue to be, the director of clinical research at Vitreoretinal Consultants in New York. Release > Tim Clackson, Ph.D., is taking the top spot at IDRx, replacing founding CEO and co-founder Ben Auspitz, while Brad Dahms will step on as chief financial officer and chief business officer. Clackson and Dahms both join IDRx from Theseus Pharmaceuticals, where they served as CEO and CFO, respectively. Release > GSK’s head of biologics and devices manufacturing Bill Dawson has been appointed the new chair of the Medicines Manufacturing Industry Partnership, an organization representing medicines manufacturers in the U.K. Dawson has been with GSK for nearly 8 years, and worked at Novartis before that, according to his LinkedIn. Release > Corinne Le Goff has resigned from her position as IMUNON’s president and CEO, exiting to pursue another business opportunity. Executive Chair Michael Tardugno, who served as CEO before Le Goff, will assume daily leadership responsibilities until a successor is selected. Release > VC 4BIO Capital has promoted Natalie Johnston, Ph.D., to principal, while appointing Abbas Hussain and Leszek Lisowski, Ph.D., to its advisory board. Previously, Johnston served as an investment associate for 4BIO, while Hussain most recently worked at Vifor Pharma and Lisowski helped establish two gene therapy companies: Exigen Biotherapeutics and Sendatu Therapeutics. Release > Y-mAbs Therapeutics EVP, Chief Financial Officer, Secretary and Treasurer Bo Kruse is resigning, effective as of the day a successor is appointed or July 31, 2024. Kruse has been with the company for nearly nine years, according to his LinkedIn profile. Release > Endeavor BioMedicines is bringing on two new leaders, appointing Enoch Kariuki as president and Vishaal Turakhia as chief financial officer. Kariuki most recently served as CEO of Lengo Therapeutics (acquired by Blueprint Medicines), while Turakhia was previously managing director at Piper Sandler’s healthcare investment banking group. Release > Joseph Bertelsen is joining U.K.-based biotech Nuclera as chief commercial officer. He previously worked at the Institute for Protein Innovation, a research site in Massachusetts, where he was the director of commercialization. Release

LEXINGTON, Mass., March 13, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced the appointment of Toby Ferguson, M.D., Ph.D., as Chief Medical Officer (CMO), effective March 25, 2024. Ferguson is a skilled and experienced healthcare executive with a proven record in advancing portfolios of novel therapies across diverse indications in central nervous system (CNS), neuromuscular, and rare diseases. He most recently served as Vice President, Head of Neuromuscular Development Unit at Biogen, Inc. “Voyager is advancing rapidly toward the clinic, with one IND filing targeted each year through 2026 for our wholly-owned programs,” said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager. “Toby brings an exceptional set of skills in the design and running of clinical programs, particularly in neurological diseases, and we believe his expertise will be immediately beneficial as we work to file our first IND with VY-TAU01, our antibody targeting pathological tau in Alzheimer’s disease, in the first half of this year.” Joining Biogen in 2013, Ferguson held positions of increasing responsibility, including as Medical Director, Senior Medical Director, Executive Medical Director, and culminating his time at Biogen as Vice President, Head of Neuromuscular Development Unit. During his tenure, he built and developed teams focused on neuromuscular and movement disorders, overseeing strategy for these areas across Biogen R&D. Notably, he led the team that developed QALSODY® (tofersen), the first genetically targeted therapy for SOD1 amyotrophic lateral sclerosis (ALS); this therapeutic received the first accelerated approval based on reduction of neurofilament, a surrogate biomarker pioneered by Ferguson, his team, and the broader ALS community. Overall, his teams at Biogen were responsible for nine successfully executed INDs and three proof-of-concept clinical trials and filings. Prior to joining Biogen, Ferguson was Assistant Professor of Neurology, Shriners Pediatric Research Center and Temple University School of Medicine. He received his M.D. and Ph.D. from University of Florida College of Medicine and his B.S. from University of Florida, Gainesville. He also completed a residency in neurology and a neuromuscular fellowship at the University of Pennsylvania. “I am excited to join Voyager, a company leading the development of neurogenetic medicines through its innovative approach to developing novel AAV capsids and delivering therapies across the blood-brain barrier,” said Ferguson. “In my career, I have always been driven to seek out new modalities that can profoundly impact patients’ lives. Voyager represents for me an extraordinary opportunity to fulfill this mission.” About Voyager TherapeuticsVoyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and multiple other diseases of the central nervous system. Many of our programs are derived from our TRACER™ AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc.; and Sangamo Therapeutics, Inc. For more information, visit www.voyagertherapeutics.com. Voyager Therapeutics® is a registered trademark, and TRACER™ is a trademark, of Voyager Therapeutics, Inc. Forward-Looking StatementsThis press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “will,” “expect,” “continue,” “anticipate,” or “potential,” and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding the timing of the effective date of the appointment of Ferguson, the contributions Ferguson may make in his role with the Company, and Voyager’s ability to advance its AAV-based gene therapy programs and tau antibody program, including expectations for Voyager’s achievement of preclinical and clinical development milestones for its potential development candidates such as IND filings, are forward looking. All forward-looking statements are based on estimates and assumptions by Voyager’s management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, the ability of Ferguson to commence his role and perform his duties successfully; the continued development of Voyager’s technology platforms, including Voyager’s TRACER platform and its antibody screening technology; the initiation, timing, conduct and outcomes of Voyager’s preclinical studies; and the sufficiency of cash resources to fund its operations and pursue its corporate objectives. These statements are also subject to a number of material risks and uncertainties that are described in Voyager’s most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. ContactsTrista Morrison, NACD.DC, tmorrison@vygr.com Investors: Adam Bero, Ph.D., abero@kendallir.com Media: Brooke Shenkin, brooke@scientpr.com