Amylyx Pharmaceuticals, Inc.

Amylyx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel therapies for diseases with high unmet medical needs, particularly focusing on neurodegenerative diseases and endocrine conditions. The company's mission is to pursue opportunities with urgency, rigorous science, and unwavering commitment to the communities it serves. Headquartered in Cambridge, Massachusetts, Amylyx was founded in 2013 by Joshua Cohen and Justin Klee.

Amylyx's pipeline includes several investigational therapies. Key product candidates include avexitide, which is being developed for post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism (HI), and AMX0035, currently under investigation for Wolfram syndrome and progressive supranuclear palsy (PSP). The company is also advancing AMX0114 for amyotrophic lateral sclerosis (ALS) and AMX0318 for PBH and other rare diseases. Previously, AMX0035 was approved as Relyvrio in the U.S. and Albrioza in Canada for ALS, but it was voluntarily withdrawn from the market in April 2024 following a disappointing Phase 3 trial.

Following the withdrawal of its ALS treatment, Amylyx has shifted its primary focus to its remaining pipeline assets, particularly avexitide. The company acquired avexitide from Eiger BioPharmaceuticals in June 2024 and is currently conducting a pivotal Phase 3 LUCIDITY clinical trial for post-bariatric hypoglycemia, with topline data anticipated in Q3 2026. Amylyx Pharmaceuticals went public in January 2022, trading on the Nasdaq Global Market under the ticker symbol AMLX. Joshua Cohen and Justin Klee serve as Co-Chief Executive Officers. The company reported its Q1 2026 financial results, indicating a cash runway expected to fund operations into 2028.