Amylyx Pharmaceuticals Plans Regulatory Filing of AMX0035 for the Treatment of ALS in Canada

- Marketing Application in Canada (New Drug Submission) Planned for First Half of 2021
- Amylyx Also Plans to Pursue Early Access Options in Canada

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s Disease and other neurodegenerative diseases, today announced plans to submit a New Drug Submission (NDS) for AMX0035 for the treatment of ALS with Health Canada in the first half of 2021. The decision to submit is based on the positive clinical data from the CENTAUR trial and feedback from Health Canada. In addition, Amylyx will explore options for early access to AMX0035 in Canada, including a potential special access program (SAP) in collaboration with the Canadian ALS Research Network, and aims to finalize plans by the end of the second quarter of 2021.

“Results from CENTAUR demonstrated that AMX0035 has both functional and long-term survival benefits which were seen on top of baseline use of approved ALS therapies,” said Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR trial, investigator at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and Assistant Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital. “As ALS is a serious disease that progresses rapidly, this trial provides important information in the quest to be able to offer viable treatments – and hope – for people living with ALS.”

"Two of the things we value most at Amylyx are transparency and patient-centricity,” said Joshua Cohen, Co-CEO, Chairman and Co-Founder of Amylyx. “In that spirit, we are pleased to announce our plans to submit for review and to pursue early access options in Canada and will work to submit the NDS as swiftly as possible because we know that every day matters to people with ALS and their families." Justin Klee, Co-CEO and Co-Founder of Amylyx, added, "Thank you to the entire ALS community who has helped bring this forward; your partnership is paramount. We will keep everyone closely informed as we proceed."

CENTAUR was a 24-week placebo-controlled study of 137 participants with ALS that evaluated the safety and efficacy of AMX0035. As previously reported, the study met its primary efficacy endpoint of slowing ALS as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). Similar rates of adverse events were seen in the AMX0035 and placebo arms.

Participants who completed CENTAUR were eligible to enroll in an open-label extension (OLE) and receive AMX0035. In a nearly three-year overall survival analysis of all randomized participants from CENTAUR, those originally randomized to AMX0035 had a 44% lower risk of death compared to those originally randomized to placebo.

“The CENTAUR clinical trial was led by the Healey & AMG Center at Mass General Hospital with participants enrolled at centers that are part of the Northeast ALS Consortium. ALS Finding a Cure and the ALS Association provided key support. This trial is the best example of how close partnership between people living with ALS, ALS physicians and scientists, trial networks, foundations and pharma can accelerate progress to develop potentially effective and safe treatments,” said Merit Cudkowicz, MD, co-principal investigator of the CENTAUR trial, Director of the Healey & AMG Center for ALS and Chair of Neurology at Massachusetts General Hospital and the Julieanne Dorn Professor of Neurology at Harvard Medical School.

“These positive clinical trial results are encouraging for the approximately 3,000 Canadians living with ALS and their families who are desperately looking for effective treatment options,” said Angela Genge, M.D., Director of the Clinical Research Unit at the Montreal Neurological Institute in Quebec.

“Amylyx’s decision to pursue regulatory approval and a special access program is a promising milestone for the Canadian ALS community,” said Tammy Moore, CEO, ALS Society of Canada. “There is an urgent need for people living with ALS to have access to proven, effective therapies and we hope to see the momentum continue in meaningful ways that will make a difference to Canadians living with the disease.”

In addition to the planned submission in Canada, Amylyx continues to discuss AMX0035 with health authorities around the world to determine the most appropriate path forward in each territory. Amylyx will update the community as soon as possible.

About Amylyx Pharmaceuticals

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

Merissa Muller
Finn Partners
(617) 778-6633
Merissa.Muller@finnpartners.com