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 it has submitted a New Drug Submission (NDS) to Health Canada for AMX0035 (sodium phenylbutyrate (PB)-taurursodiol (TURSO)) for the treatment of ALS.
“The submission of the NDS in Canada represents a significant milestone in our efforts to develop a new treatment option for people living with ALS who have no time to wait,” said Joshua Cohen, Co-CEO, Chairman and Co-Founder of Amylyx. “We’d like to thank the people who participated in CENTAUR, trial investigators, the ALS community and our partners and team, who have worked tirelessly to help us achieve this milestone,” added Justin Klee, Co-CEO and Co-Founder of Amylyx.
CENTAUR was a 24-week placebo-controlled study of 137 participants with ALS that evaluated the safety and efficacy of AMX0035. As previously reported, the study met its primary efficacy endpoint of slowing ALS as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). The rates of adverse events were similar between the AMX0035 and placebo arms of the study. However, discontinuations related to adverse events occurred more frequently in the AMX0035 arm than in the placebo arm. In a nearly three-year overall survival analysis of all randomized participants from CENTAUR, those who started on AMX0035 had a 44% lower risk of death compared to those who started on placebo. 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.
“There is a critical and urgent need for people living with ALS to have equitable, timely and affordable access to proven therapies,” said Tammy Moore, CEO ALS Society of Canada. “Amylyx’ decision to pursue regulatory approval of AMX0035 is an important step forward for this community who continues to face a devastating disease.”
“Amylyx is committed to bringing AMX0035 through the regulatory approval process as quickly as possible in Canada,” said Chris Aiello, Head of Canada and General Manager at Amylyx. “We will continue to work closely with Health Canada as we ramp up to potentially bring AMX0035 to people living with ALS.”
In addition to seeking approval of AMX0035 for the treatment of ALS in Canada, Amylyx recently announced plans to submit a Marketing Authorization Application (MAA) for AMX0035 to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) by the end of 2021 and to initiate a Phase 3 clinical trial in Europe and the United States by Q3 2021. Amylyx continues to discuss AMX0035 with health authorities around the world to determine the most appropriate path forward in each territory and will continue to keep the global ALS community updated.
AMX0035 is an investigational product comprised of two complementary active agents, sodium phenylbutyrate (PB) and taurursodiol (TURSO), which were combined in a co-formulation to reduce neuronal death and dysfunction. AMX0035 was specifically co-formulated and manufactured by Amylyx to ensure proper absorption, exposure, and quality. 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.