Company: Amylyx Pharmaceuticals
Drug Type: Small Molecule
Conditions: ALS, Alzheimer’s disease, Parkinson’s disease.
Mechanism Type: Neuroprotection
Mechanism: AMX0035 a proprietary combination of two drugs, TUDCA and sodium phenylbutyrate, that acts by reducing cell death and neuronal inflammation in response to oxidative insult.
U.S. Status for ALS: Phase II
 Tauroursodeoxycholic acid in the treatment of patients with amyotrophic lateral sclerosis. Elia, AE et al. Eur J Neurol. 2016 Jan;23(1):45-52.
 Phase 2 study of sodium phenylbutyrate in ALS. Cudkowicz, ME et al. Amyotroph Lateral Scler. 2009 Apr;10(2):99-106.
 AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) (CENTAUR) Clinicaltrials.gov, June 19, 2017.
Last updated June 22nd, 2017