Company: Roche Pharmaceuticals
Drug Type: Small Molecule
Conditions: ALS, Spinal Muscular Atrophy
Mechanism Type: Mitochondrial protectant
Mechanism: Olesoxime is a cholesterol-oxime compound that targets and preserves mitochondrial integrity and function under conditions of cellular stress by modulating the mitochondrial permeability transition pore, and as a result promotes the function and survival of neurons and other cell types under disease-relevant stress conditions. The compound improved muscle function and reduced astrogliosis and microglial activation in preclinical studies in SOD1 ALS mouse models, but failed Phase III trials in ALS.
U.S. Status for ALS: Discontinued
 Safety and Efficacy of TRO19622 as add-on Therapy to Riluzole Versus Placebo in Treatment of Patients Suffering From Amyotrophic Lateral Sclerosis (ALS) (MITOTARGET) ClinicalTrials.gov, 27 Mar 2012. Accessed 16 Mar 2016 from https://clinicaltrials.gov/ct2/show/NCT00868166.
 A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy. ClinicalTrials.gov, 1 Mar 2016. Accessed 11 Mar 2016 from https://clinicaltrials.gov/ct2/show/NCT02628743.
 A phase II-III trial of olesoxime in subjects with amyotrophic lateral sclerosis. Lenglet, T. et al. Eur J Neurol. 2014 Mar;21(3):529-36.
 Olesoxime delays muscle denervation, astrogliosis, microglial activation and motoneuron death in an ALS mouse model. Sunyach, C et al. Neuropharmacology. 2012 Jun;62(7):2346-52.
Last updated June 21st, 2017