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 that long-term survival data from the CENTAUR trial of AMX0035 in patients with ALS were selected for an oral presentation in the Contemporary Clinical Issues Plenary Session on April 19 at the 2021 American Academy of Neurology (AAN) Annual Meeting. The meeting is being held virtually on April 17-22.
The abstract, “Long-Term Survival of Participants in the CENTAUR Trial of AMX0035 for ALS,” was one of 26 abstracts awarded the merit of distinction by the American Academy of Neurology and the Science Committee. The distinction is awarded to the abstract deemed to be the top in its topic category in quality of study and interest to the neurologic community.
Prior to the Contemporary Clinical Issues Plenary Session, the data will be highlighted in the Top Science Press Conference being held virtually ahead of the AAN Annual Meeting on Tuesday, April 13, 2021 at 11:00 a.m. ET. The conference is open to registered journalists.
“Each day counts when it comes to progressive and fatal neurodegenerative disorders like ALS, and we remain committed to sharing important learnings from CENTAUR given the functional and overall survival benefits demonstrated for those who received AMX0035 in the trial,” said Josh Cohen, Co-CEO, Chairman and Co-Founder at Amylyx. “We are honored to share findings from CENTAUR with the medical and scientific community at AAN this year and will continue to provide updates regarding AMX0035 as we have them.”
Justin Klee, Co-CEO and Co-Founder of Amylyx, added, “We’re encouraged by the incredible level of support from advocates, academia and the ALS community as we continue to work collaboratively with global regulatory agencies to bring AMX0035 through the regulatory and clinical development process. We have been fortunate to work with extraordinary clinician scientists and would like to thank Dr. Sabrina Paganoni who has been an inspiration to us and the ALS community through her leadership, dedication and contributions to ALS research.”
Top Science Press Conference
Date: April 13, 2021
Time: 11:00 a.m. ET
*Open to registered journalists only
Title: “Long-Term Survival of Participants in the CENTAUR Trial of AMX0035 for ALS”
Session: Contemporary Clinical Issues Plenary Session
Date: Monday, April 19, 2021
Time: 10:00 a.m. to 12:30 p.m. ET
*A live, moderated fireside chat with audience Q&A to follow the Contemporary Clinical Issues Plenary Session from 12:30-1:30 p.m. ET
CENTAUR was a 24-week randomized, double-blind, placebo-controlled clinical trial of 137 participants with ALS that evaluated the safety and efficacy of AMX0035 and assessed the study drug’s impact on disease progression as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) when compared to placebo. CENTAUR also assessed the effects of AMX0035 on other measures that are critical to people with ALS, including muscle strength, lung vital capacity, and biomarkers of neuronal degeneration.
As published in the New England Journal of Medicine (NEJM), the study met its primary efficacy endpoint of slowing ALS progression as measured by the ALSFRS-R. The rates of adverse events were similar between the AMX0035 and placebo arms of the study. Discontinuations related to adverse events occurred more frequently in the AMX0035 arm than in the placebo arm.
CENTAUR enrolled patients 18-80 years old with definite ALS and within 18 months of symptom onset. The trial did not restrict patients from receiving edaravone or riluzole. Participants who completed CENTAUR were given the option after the trial to enroll in an open-label extension study and receive AMX0035 for up to 30 months (132 weeks). 92% of eligible CENTAUR participants elected to enroll in the extension study. In a nearly three-year overall survival analysis of all randomized participants from CENTAUR that was published in Muscle & Nerve, those originally randomized to AMX0035 had a 44% lower risk of death compared to those originally randomized to placebo.
CENTAUR was the recipient of the ALS ACT grant, and is supported by The ALS Association, ALS Finding a Cure, a program of The Leandro P. Rizzuto Foundation, the Northeast ALS Consortium, Sean M. Healey & AMG Center for ALS at Mass General, and was funded in part by the ALS Ice Bucket Challenge.
About Amyotrophic Lateral Sclerosis (ALS)
ALS is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually death. The vast majority of patients with ALS (>90%) have sporadic disease, showing no clear family history. Approximately 6000 people are diagnosed with ALS in the United States every year with an approximately similar number of deaths every year.
AMX0035 is an investigational product designed to reduce neuronal death and dysfunction. AMX0035 targets endoplasmic reticulum and mitochondrial dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases.
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.