European Medicines Agency Grants Orphan Drug Designation to Amylyx Pharmaceuticals’ AMX0035 for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has recommended that AMX0035 be designated as an orphan medicinal product for the treatment of ALS. AMX0035 is an investigational, first-in-class, disease-modifying therapy designed to reduce neuronal death and dysfunction.

The EMA grants Orphan Drug Designation status for products intended for the treatment, prevention or diagnosis of rare, life-threatening or chronically debilitating conditions where the product may represent a significant benefit over existing treatments.

“ALS patients worldwide are in need of new therapies,” said Joshua Cohen, Co-CEO, Chairman, and Co-Founder of Amylyx, “We’re excited about today’s news and look forward to working with the European Medicines Agency on the next steps for AMX0035.”

In late 2019, Amylyx announced that the CENTAUR clinical trial of AMX0035 in patients with ALS met its primary endpoint, demonstrating that patients who received AMX0035 showed a statistically significant, longer retention of function as measured by the ALSFRS-R as compared to patients who received placebo. Amylyx is working towards publishing these results in a medical journal as quickly as possible.

The CENTAUR clinical trial was a pivotal, six month, parallel-group, randomized, double-blind, placebo-controlled clinical trial that enrolled 137 patients randomized to placebo or AMX0035. Upon completion of the 24-week treatment, patients were eligible to enroll in an open label extension study in which all patients were given access to AMX0035.

Amylyx’ open-label extension study (CENTAUR-OLE) remains ongoing. Amylyx is also working towards publication of interim data from this extension study. More information on the open label extension can be found at www.clinicaltrials.gov, NCT03488524.

About AMX0035

AMX0035 is Amylyx’ first in class investigational therapy designed to reduce neuronal death and dysfunction. AMX0035 targets mitochondrial and endoplasmic reticulum dependent neuronal degeneration pathways.

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.