Drug Name: AMX0035
Company: Amylyx Pharmaceuticals
Location: US-Massachusetts
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

[1] Tauroursodeoxycholic acid in the treatment of patients with amyotrophic lateral sclerosis. Elia, AE et al. Eur J Neurol. 2016 Jan;23(1):45-52.
[2] Phase 2 study of sodium phenylbutyrate in ALS. Cudkowicz, ME et al. Amyotroph Lateral Scler. 2009 Apr;10(2):99-106.
[3] AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) (CENTAUR), June 19, 2017.

Last updated June 22nd, 2017

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