Amylyx Pharmaceuticals Receives FDA Orphan Drug Designation for AMX0035 for the Treatment of Amyotrophic Lateral Sclerosis

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Amylyx Pharmaceuticals, Inc., announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AMX0035, an oral therapeutic in clinical development for the treatment of amyotrophic lateral sclerosis (ALS). The Company recently began a Phase 2 clinical study, the CENTAUR trial, to evaluate the safety and efficacy of AMX0035 in ALS patients.

The U.S. Orphan Drug Act provides incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the U.S. Orphan drug designation by the FDA conveys up to seven years of marketing exclusivity if the compound receives regulatory approval, and offers various development incentives, including tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design.

Background on AMX0035
AMX0035 is a combination of two small molecules, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). Each compound has demonstrated strong efficacy in several cellular and animal models of ALS. When individually tested in ALS clinical trials, PB and TUDCA have both shown safety, tolerability, and preliminary signs of efficacy. In preclinical trials, Amylyx has demonstrated a synergistic effect between the two compounds, suggesting that the combination may have improved efficacy compared to the individual agents.

About the CENTAUR Trial
CENTAUR is a 24-week, randomized, double-blind, placebo-controlled Phase II clinical trial in 132 participants with ALS. The trial's primary objectives are to evaluate the safety and tolerability of AMX0035 and assess the drug's impact on disease progression as measured by the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) over the 24-week study period. The trial will also evaluate the impact of AMX0035 on isometric strength as measured by ATLIS, respiratory function, and exploratory biomarkers. There will also be an open label extension to the trial so that all enrolled patients will have the option of continuing treatment once the 24-week treatment period concludes. More information on the CENTAUR trial can be found at or, NCT03127514. Please contact (855)-437-4823 or email if you are interested in participating. Contact information for trial sites will be made available on

About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company currently developing a novel therapeutic for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases. The company's therapeutic, AMX0035, targets the neuroinflammation and nerve cell death that characterize these diseases. AMX0035 is a proprietary combination of existing compounds that have worked synergistically to prevent cell death and neurotoxic inflammation in multiple preclinical models. AMX0035 entered a Phase 2 clinical trial (CENTAUR) in ALS patients in June 2017. Learn more at:


Amylyx Pharmaceuticals Inc.
Josh Cohen, 617-682-0917
Steinerman Biomedical Communications
Peter Steinerman, 516-641-8959


Source: Amylyx Pharmaceuticals, Inc.

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