Drug Name: Dexpramipexole
Company: Biogen
Location: US-Massachusetts
Drug Type: Small Molecule
Conditions: ALS, multiple sclerosis, Alzheimer’s disease, spinal muscular atrophy
Mechanism Type: Mitochondrial bioenergetics
Mechanism: The compound is a small molecule modulator of mitochondrial bioenergetics.
U.S. Status for ALS: Discontinued

[1] Dexpramipexole versus placebo for patients with amyotrophic lateral sclerosis (EMPOWER): a randomised, double-blind, phase 3 trial. Cudkowicz, ME et al. Lancet Neurol. 2013 Nov;12(11):1059-67.
[2] Biogen Idec Reports Top-Line Results from Phase 3 Trial Investigating Dexpramipexole in People with Amyotrophic Lateral Sclerosis (ALS). BusinessWire, 3 Jan 2013. Accessed 9 Mar 2016 from
[3] Phase 3 Extension Study of Dexpramipexole in ALS (ENVISION)., 24 Nov 2014. Accessed 11 Mar 2016 from
[4] A post hoc analysis of subgroup outcomes and creatinine in the phase III clinical trial (EMPOWER) of dexpramipexole in ALS. Bozik, ME et al. Amyotroph Lateral Scler Frontotemporal Degener. 2014 Sep;15(5-6):406-13.

Last updated February 1st, 2016

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