Amylyx Pharmaceuticals

Amylyx Pharmaceuticals, Inc. is a commercial-stage biotechnology company, which engages in the provision of therapies for neurodegenerative diseases and amyotrophic lateral sclerosis (ALS). The company is headquartered in Cambridge, Massachusetts and currently employs 136 full-time employees. The company went IPO on 2022-01-07. The firm is focused on three investigational therapies across several neurodegenerative and endocrine diseases. Its lead investigational asset is avexitide, an investigational, first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist. Avexitide has been evaluated as a treatment for Post-Bariatric Hypoglycemia (PBH) and congenital HI, two indications characterized by hyperinsulinemic hypoglycemia. In addition to avexitide, it is advancing AMX0035, an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol in Wolfram syndrome and progressive supranuclear palsy (PSP), and AMX0114 in amyotrophic lateral sclerosis (ALS). AMX0035 is designed to mitigate neurodegeneration by targeting endoplasmic reticulum stress and mitochondrial dysfunction, two cellular processes central to neuronal cell death and neurodegeneration. AMX0114 is an investigational antisense oligonucleotide.