– Significantly Lower Long-Term Risk of Death or Permanent Assisted Ventilation and Risk of First Hospitalization Among Participants Originally Randomized to AMX0035 Versus Placebo

– Data were Consistent with CENTAUR and CENTAUR OLE Primary Outcome Measures of Statistically Significant Slowing of Functional Decline and Long-Term Survival Results Associated with the Administration of AMX0035

CAMBRIDGE, Mass. —
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 the presentation of results from the CENTAUR trial demonstrating significantly lower long-term risk of death or permanent assisted ventilation (PAV) and risk of first hospitalization. These data were presented in an oral session at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference held March 15-18, 2021.

CENTAUR was a 24-week placebo-controlled study of 137 participants with ALS that evaluated the safety and efficacy of AMX0035. The study was led by Drs. Sabrina Paganoni and Merit Cudkowicz from the Healey Center for ALS at Mass General in collaboration with the Northeast ALS Consortium. As previously reported, the study met its primary efficacy endpoint of slowing ALS as measured by a statistically significant effect on the ALS Functional Rating Scale-Revised (ALSFRS-R). Similar rates of adverse events were recorded in the AMX0035 and placebo groups during the 24-week randomized period. Additionally, in an overall survival analysis of all randomized participants from CENTAUR and the open label extension (OLE), those originally randomized to AMX0035 had a statistically significant 44% reduction in the risk of death during follow-up compared to those originally randomized to placebo.

“We are strongly encouraged by the results from the CENTAUR trial,” said Dr. Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR study, investigator at the Sean M. Healey & AMG Center for ALS at Mass General and Assistant Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital. “These results show a consistent effect with AMX0035 across endpoints of survival, hospitalization and permanent ventilation. I am enthusiastic about these results which continue to support AMX0035 as a potential therapeutic for ALS.”

“The CENTAUR clinical trial, led by the Healey & AMG Center at Mass General Hospital with participants enrolled through the Northeast ALS (NEALS) Consortium centers, was able to advance thanks to the contributions from ALS Finding a Cure and the ALS Association,” said Merit Cudkowicz, MD, co-principal investigator of the CENTAUR Trial, Director of the Healey & AMG Center for ALS and Chair of Neurology at Mass General and the Julieanne Dorn Professor of Neurology at Harvard Medical School. “These new findings support our continued hopes to bring a safe and effective treatment to our patients with ALS.”

As recently reported, Amylyx 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.  Additionally, Amylyx will be exploring options for early access to AMX0035 in Canada including a potential special access program (SAP) and aims to finalize plans by the end of the second quarter of 2021.

“The results from CENTAUR, which showed a significantly lower risk of death or permanent assisted ventilation, and first hospitalization, are an important step forward in a therapeutic area with a significant unmet need,” said Justin Klee, Co-CEO and Co-Founder at Amylyx. “These data will help support our continued efforts to bring AMX0035 to the ALS community. To that end, we are continuing to work collaboratively with global regulatory agencies to find the most efficient path forward.”

Long-Term Risk of Death or PAV and First Hospitalization Results from CENTAUR:

Participants randomized in the CENTAUR trial were evaluated for the key study events (death, PAV and first hospitalization) through July 2020. Results demonstrated the mean hazard of any key event was 44% lower in those originally randomized to AMX0035 versus placebo. These results supplement the previously reported functional and long-term survival results seen with AMX0035.

A study limitation is that time to death observation was longer than time to PAV and hospitalization. This resulted in a potential for missing data after dropout for hospitalizations and ventilation/tracheostomy events.

Presentation details:
Title: Lower Long-Term Risk of Death or Permanent Ventilation and First Hospitalization Among Participants with ALS Receiving AMX0035 in the CENTAUR Trial
Poster #: 46
Date: Thursday, March 18, 2021
Time: 3:45 – 4:00 p.m. ET

At this time, the MDA conference is only open to researchers; however, Amylyx will publish the data presented at MDA as soon as possible.

“We are excited to share these results from CENTAUR at this year’s virtual MDA Conference,” said Josh Cohen, Co-CEO, Chairman and Co-Founder at Amylyx. “We would like to thank MDA for continuing their commitment to elevate neuroscience research in order to help bring new research and potential treatment options to highly vulnerable patient populations.”

About CENTAUR
CENTAUR was a 24-week, randomized, double-blind, placebo-controlled clinical trial that evaluated the safety and tolerability of AMX0035 and assessed the drug’s impact on disease progression as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) when compared to placebo. The trial 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.

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.

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. More information on the CENTAUR trial can be found at https://amylyx.com/trials/ or www.clinicaltrials.gov, NCT03127514 or NCT03488524.

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.

About AMX0035
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.

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