Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, and the Sean M. Healey & AMG Center for ALS at Mass General today announced that AMX0035 demonstrated a significant treatment benefit for people with ALS in the CENTAUR study.

In the study, participants taking AMX0035 had a statistically significant slowing of ALS disease progression as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) compared to placebo (p<0.05), the primary outcome of the trial.

Detailed results from CENTAUR will be submitted for peer-reviewed publication and presentation at a future medical congress.

"Today marks a significant step forward in the fight to develop new treatments for ALS," said Dr. Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR study, investigator at the Healey Center for ALS at Mass General and Assistant Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital. "The study results highlight AMX0035 as a potentially beneficial new treatment for people with ALS, and the design and execution of the CENTAUR trial are a testament to true collaboration across the many stakeholders in this fight. Thank you to everyone who made this possible, and I look forward to presenting the full study results in the coming months."

"We are honored and humbled to have reached this milestone after working nearly seven years on the development of AMX0035," said Joshua Cohen, CEO, Chairman, and Co-Founder of Amylyx. "Thank you to each and every participant, family, physician, nurse, coordinator, vendor, and advisor who has and continues to work with us to better the lives of people with ALS."

"With these results, Amylyx now has a responsibility to move ahead as efficiently as possible, as people living with ALS don’t have time to wait," said Justin Klee, President and Co-Founder of Amylyx. "We will work closely with the FDA and the ALS community, including the wonderful Northeast ALS Consortium leadership and member sites that conducted the CENTAUR trial, to decide next steps and will continue to keep everyone informed."

Participants in CENTAUR were given the option after the trial to enroll in an open-label extension study to receive treatment with AMX0035. Nearly 90 percent of participants who completed CENTAUR elected to enroll in the extension study. Interim data from the ongoing extension study will be presented in 2020.

In addition, the company will provide an update on regulatory plans and further details on expanded access plans in early 2020.

"ALS Finding a Cure is proud to have catalyzed and supported the CENTAUR study, and I am encouraged by what the results mean for people living with ALS. Our team at the Mass General Neurological Clinical Research Institute is proud of this collaboration with Amylyx and our colleagues in the Northeast ALS Consortium on this important study," commented Dr. Merit Cudkowicz, Chief Medical Officer from ALS Finding a Cure, Director of the Healey Center for ALS, Chief of Neurology at Mass General, and the Julieanne Dorn Professor of Neurology at Harvard Medical School. "We look forward to advancing this research and what it might mean for those living with ALS."

Dr. Neil Thakur, executive vice president for mission strategy at The ALS Association, added, "We are proud to have supported AMX0035 and Amylyx from an early stage and are very excited about what AMX0035 may accomplish for people with ALS. This company and study team have focused on the patient perspective during the design and conduct of this study and we are happy to work with and innovate together with them. We are excited to continue to collaborate on this therapy in the future."

Dr. Rudolph Tanzi, Ph.D., Kennedy Professor of Neurology, Massachusetts General Hospital, chair of the Cure Alzheimer’s Fund Research Leadership Group and chair of the Amylyx SAB, shared, "The positive results from the CENTAUR ALS study demonstrate that the novel mechanism of AMX0035 may represent a new treatment approach for not only ALS, but for Alzheimer’s disease. I am very excited about the demonstrated benefit of AMX0035 in people with ALS, and look forward to the results from the ongoing PEGASUS trial for people with Alzheimer’s disease."

ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. The progressive neurodegeneration in ALS eventually leads to the death of motor neurons and loss of the ability of the brain to initiate and control muscle movement. With muscle action progressively affected, patients in the later stages of the disease may become paralyzed and unable to breathe normally.

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