Amylyx drug for ALS fails at Phase 3


United States

A confirmatory Phase 3 trial of a drug for patients with amyotrophic lateral sclerosis (ALS) failed to meet its primary and secondary endpoints, raising the possibility that the therapy, originally approved in 2022, could be withdrawn from the market. The developer, Amylyx Pharmaceuticals Inc, announced the trial results on 8 March, saying that a voluntary market withdrawal had not been ruled out. The therapy, AMX0035, is a combination of two small molecules, sodium phenylbutyrate and taurursodiol, which each target different neurodegenerative pathways.