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Amylyx Pharmaceuticals, Inc. is a commercial-stage biopharmaceutical company. The Company is engaged in the discovery and development of treatments for neurodegenerative diseases. The Company is focused on the development and commercialization of its product candidate, AMX003 (sodium phenylbutyrate and taurursodiol, also known as ursodoxicoltaurine) for the treatment of amyotrophic lateral sclerosis (ALS).
The Company is developing AMX0035 for other neurodegenerative diseases by leveraging knowledge and relationships in the neurodegenerative space. AMX0035 is a dual unfolded protein response (UPR)-Bax apoptosis inhibitor composed of PB and TURSO (also known as TUDCA). Its development of antisense oligonucleotides (ASOs), including lead ASO AMX0114, which targets calpain-2 (CAPN2), a gene encoding calcium-dependent proteolytic enzyme which has been implicated in the pathogenesis of ALS and other neurodegenerative diseases. .