Amylyx Pharmaceuticals is dedicated to developing innovative treatments for neurodegenerative diseases and conditions with significant unmet medical needs. One of their key products, AMX0035, is a fixed-dose combination of sodium phenylbutyrate and taurursodiol, designed to target critical pathways involved in neurodegeneration, specifically endoplasmic reticulum stress and mitochondrial dysfunction. This combination aims to mitigate neuronal cell death, which is a common issue in diseases like amyotrophic lateral sclerosis (ALS) and Wolfram syndrome. The company has reported positive results from clinical trials, including the Phase 2 HELIOS trial, which demonstrated improvements in pancreatic function and other health metrics in patients with Wolfram syndrome.

In addition to AMX0035, Amylyx has acquired Avexitide, a first-in-class GLP-1 receptor antagonist that has received FDA Breakthrough Therapy Designation. This drug is being developed for post-bariatric hypoglycemia and is expected to enter Phase 3 trials in early 2025. The company emphasizes a commitment to transparency and collaboration with the communities they serve, aiming to create meaningful moments for patients and their families while advancing research in neurodegenerative diseases. Through their innovative approaches and ongoing clinical studies, Amylyx Pharmaceuticals seeks to improve outcomes for individuals affected by these challenging conditions.

United States

Amylyx Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, engages in developing various therapeutics for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. The company’s product pipeline includes AMX0035, a dual UPR-Bax apoptosis inhibitor composed of sodium phenylbutyrate and taurursodiol for the treatment of amyotrophic lateral sclerosis. It is also developing AMX0035 for other neurodegenerative diseases. The company was founded in 2013 and is headquartered in Cambridge, Massachusetts.

Nervous System Diseases

Amylyx Pharmaceuticals

Amyotrophic lateral sclerosis

RELYVRIO™ (sodium phenylbutyrate and taurursodiol) is an oral, &nbsp;fixed-dose medication approved to treat amyotrophic lateral sclerosis &nbsp;(ALS) in adults in the U.S. and approved with conditions as ALBRIOZA™ for the treatment of ALS in Canada. Additionally, the European Medicines Agency (EMA) is reviewing the Company’s Marketing Authorisation Application for AMX0035 for the treatment of ALS in Europe. AMX0035 is being explored for the potential treatment of other neurodegenerative diseases.

Relyvrio (AMX0035)

Progressive supranuclear palsy

AMX0035 is an investigational product comprised of two complementary active agents, sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine), which were combined in a co-formulation to reduce neuronal death and dysfunction. AMX0035 is designed to target the endoplasmic reticulum and mitochondrial-dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases.

AMX0035 

Relyvrio (AMX0035) 

Congenital Hyperinsulinism

Hyperinsulinism

Post-Bariatric Hypoglycemia

Avexitide is an investigational, first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist that has been evaluated in five clinical trials for post-bariatric hypoglycemia (PBH) and has also been studied in congenital hyperinsulinism (HI), two indications characterized by hyperinsulinemic hypoglycemia. The U.S. Food and Drug Administration (FDA) has granted avexitide Breakthrough Therapy Designation for both indications, Rare Pediatric Disease Designation in congenital HI, and Orphan Drug Designation for the treatment of hyperinsulinemic hypoglycemia (which includes PBH and congenital HI).
 
Avexitide is designed to bind to the GLP-1 receptor on pancreatic islet beta cells and block the effect of GLP-1 to mitigate hypoglycemia by decreasing insulin secretion and stabilizing glucose levels. In PBH, excessive GLP-1 can lead to the hypersecretion of insulin and subsequent severe hypoglycemic events, including autonomic and neuroglycopenic symptoms if left unaddressed.

Amylyx Pharmaceuticals

Company Details