CAMBRIDGE, Mass. (BUSINESS WIRE)–Amylyx Pharmaceuticals, Inc., a clinical-stage pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, today announced that the last participant has completed the planned 24 weeks of study treatment in CENTAUR, a clinical trial assessing the safety and efficacy of AMX0035 in people with ALS.

“This trial has been an important partnership between industry and academia, and we look forward to analyzing the results gathered in this study,” said the study Principal Investigator Dr. Sabrina Paganoni, investigator at the Healey Center for ALS at Mass General and Assistant Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital. “Thank you to the participants and site study teams who have made this work possible.”

The CENTAUR study was the recipient of the ALS ACT grant, and is supported by ALS Finding a Cure®,-a program of The Leandro P. Rizzuto Foundation, The ALS Association, the Northeast ALS ConsortiumMassachusetts General Hospital Neurology Clinical Research Institute, and was funded in part by the ALS Ice Bucket Challenge.

ALS 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. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost. With muscle action progressively affected, patients in the later stages of the disease may become paralyzed and unable to breathe normally. AMX0035 is Amylyx’s proprietary two-drug combination therapy in development to prevent nerve cell death and degeneration.

Patrick Yeramian, MD, chief medical officer of Amylyx, said, “We’re thankful to everyone involved, and we are working hard to bring topline results to the community as quickly as possible over the coming months.”

CENTAUR is a 24-week, randomized, double-blind, placebo-controlled Phase II clinical trial in people 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 of neuronal death. There is also an open label extension to the trial allowing enrolled participants to continue to receive AMX0035. More information on the CENTAUR trial can be found at or, NCT03127514.

In 2017, AMX0035 was granted orphan drug status by the U.S. Food and Drug Administration (FDA) as a potential medicine for ALS. Amylyx reached its recruitment goal for the CENTAUR study in March 2019.

About AMX0035

AMX0035 is a proprietary 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 demonstrated a synergistic effect between the two compounds in preventing nerve cell death and degeneration.

About Amylyx Pharmaceuticals

Amylyx Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company developing a novel therapeutic for Amyotrophic Lateral Sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases. This press release and further information about Amylyx can be found at:


Merissa Muller
Finn Partners
(617) 778-6633