Amylyx Pharmaceuticals, Inc.

Amylyx Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company focused on developing new therapies for communities with significant unmet medical needs. The company has preclinical and clinical development programs in both endocrine conditions and neurodegenerative diseases. Amylyx's lead investigational asset is avexitide, a first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist. Avexitide has been studied as a treatment for post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism (Congenital HI), both conditions characterized by hyperinsulinemic hypoglycemia. The U.S. Food and Drug Administration (FDA) has granted avexitide Breakthrough Therapy Designation for both PBH and HI, Rare Pediatric Disease Designation in Congenital HI, and Orphan Drug Designation for the treatment of hyperinsulinemic hypoglycemia. Post-bariatric hypoglycemia (PBH) is a condition estimated to affect approximately 8% of people in the U.S. who have undergone sleeve gastrectomy and Roux-en-Y gastric bypass, which is about 160,000 people in the U.S. This condition is believed to result from an excessive GLP-1 response, leading to low blood sugar (hypoglycemia) and reduced quality of life. PBH can cause severe hypoglycemic events where the brain doesn't get enough glucose, potentially leading to impaired thinking, loss of consciousness, and seizures. PBH is also linked to significant disability that can disrupt independent living. Currently, there are no approved therapies for PBH. Avexitide is designed to attach to the GLP-1 receptor on pancreatic islet beta cells, blocking the effect of GLP-1 to reduce hypoglycemia by decreasing insulin secretion and stabilizing blood glucose levels.