Amylyx Pharmaceuticals (NASDAQ:AMLX) is a clinical-stage biopharmaceutical company founded in 2013 and headquartered in Cambridge, Massachusetts. The company is dedicated to developing therapies for debilitating neurodegenerative and neuromuscular diseases, leveraging novel small-molecule combinations to target cellular stress pathways. Since its inception, Amylyx has focused on translating pioneering research into late-stage clinical programs aimed at addressing high unmet medical needs in amyotrophic lateral sclerosis (ALS) and other neurological disorders.
The company’s lead product, AMX0035 (marketed as Relyvrio in the United States), is a fixed-dose oral combination of sodium phenylbutyrate and taurursodiol. AMX0035 is designed to protect neurons by reducing endoplasmic reticulum and mitochondrial stress. After positive Phase 2 trial results, Amylyx secured FDA approval for Relyvrio in 2022, making it one of the first disease-modifying treatments available for ALS patients in the U.S. In parallel, Amylyx is advancing exploratory studies of AMX0035 in early-stage Alzheimer’s disease and other indications, as well as investigating next-generation assets in migraine and rare neurological conditions.
Amylyx operates in North America and has established partnerships to support global clinical trials and regulatory filings. The company’s research and development activities are conducted at its Cambridge headquarters, with additional facilities in Vancouver, Canada, where early discovery and biomarker development efforts are based. By collaborating with international academic centers, patient advocacy groups and contract research organizations, Amylyx ensures broad geographic reach and efficient trial execution.
Leadership at Amylyx includes co-founders Joshua L. Cohen, who serves as Chief Executive Officer, and Justin M. Klee, the company’s Chief Scientific Officer. Under their guidance, the executive team has expanded to include experienced professionals in clinical development, regulatory affairs and commercial operations. Together with a board of directors comprised of industry veterans and scientific advisors, Amylyx continues to build a robust pipeline aimed at delivering innovative treatments for patients living with life-threatening neurological diseases.
