Amylyx Pharmaceuticals Announces Health Canada Accepts for Review the New Drug Submission for AMX0035 for the Treatment of ALS

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Amylyx Pharmaceuticals, Inc. today announced that Health Canada has accepted for review the New Drug Submission (NDS) for AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS).

“We are pleased that our submission has been accepted for review by Health Canada and we are committed to serving those affected by ALS in this country,” said Chris Aiello, General Manager and Head of Canada at Amylyx. “Today’s news marks another milestone in our efforts to bring a potential new treatment option to people with ALS. We look forward to working closely with Health Canada and the broader ALS community in Canada as we press ahead.”

CENTAUR was a Phase 2, multicenter study in 137 participants with ALS encompassing a 6-month randomized placebo-controlled phase and an open-label long-term follow-up phase. As previously reported, the study met its primary efficacy endpoint of slowing ALS as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). In the study, AMX0035 showed numerical benefits on secondary outcomes including measures of muscle strength, breathing, and hospitalizations. While there were similar rates of adverse events and discontinuations recorded in the AMX0035 and placebo groups during the 24-week randomized phase, GI events occurred with greater frequency (≥2%) in the AMX0035 group. Across all randomized participants from CENTAUR, those who started on AMX0035 had a 44% lower risk of death compared to those who started on placebo during a follow up period of up to three years. Median survival duration was 25.0 months (95% CI, 19.0-33.6 months) in the group that started on AMX0035 and 18.5 months (95% CI, 13.5-23.2 months) in the group that started on placebo, a 6.5-month difference.

Amylyx plans to submit a marketing application to the European Medicines Agency’s (EMA) by the end of 2021 and continues to discuss AMX0035 with health authorities around the world, including the U.S. Food and Drug Administration (FDA), to determine the most appropriate path forward in each territory and will continue to keep the global ALS community updated. Additionally, Amylyx plans to initiate a Phase 3 clinical trial in Europe and the United States by Q3 2021.

About AMX0035

AMX0035 is an investigational product comprised of two complementary active agents, sodium phenylbutyrate (PB) and ursodoxicoltaurine (TURSO; also known as taurursodiol), which were combined in a co-formulation 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.