Amylyx Pharmaceuticals

Amylyx Pharmaceuticals, Inc. is a biotechnology company focused on the discovery and development of therapies for diseases with significant unmet medical need, with an emphasis on endocrine and neurodegenerative disorders. 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 four investigational therapies across several 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, AMX0114 in amyotrophic lateral sclerosis (ALS), and AMX0318 in PBH and other rare diseases. 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 and AMX0318 is a novel GLP-1 receptor antagonist.