Amylyx Pharmaceuticals

Amylyx Pharmaceuticals is a clinical-stage pharmaceutical company developing therapies for diseases with high unmet need, with a particular focus on neurodegenerative conditions. Founded in 2013 by Brown University undergraduates, the company is headquartered in Cambridge, Massachusetts, and operates across Canada, EMEA and Japan. Its scientific work spans neuroscience, mitochondrial and endoplasmic reticulum dysfunction, and the development of clinical-stage therapeutics across neurology, rare diseases and metabolic disorders.

The company's lead programme, AMX0035, is a fixed-dose combination of taurursodiol and sodium phenylbutyrate developed for amyotrophic lateral sclerosis (ALS). It received its first approval in 2022 and was withdrawn in 2024 following phase 3 trial results. Amylyx continues to work on ALS beyond AMX0035, alongside investigational programmes in post-bariatric hypoglycemia and Wolfram syndrome.

Amylyx describes its approach as modality-agnostic, matching therapeutic modalities to the diseases it targets rather than adhering to a single platform. The company states its values as audacious, curious, authentic, engaged and accountable, and emphasises a science-first, rigorous research culture oriented towards underserved patient communities.