olesoxime

Olesoxime (TRO19622) is an orally active, cholesterol-derived small molecule designed as a neuroprotective agent. It specifically targets the outer mitochondrial membrane by binding to two key proteins: the translocator protein (TSPO) and the voltage-dependent anion-selective channel (VDAC). By interacting with these components of the mitochondrial permeability transition pore (mPTP) complex, olesoxime helps maintain mitochondrial integrity, prevents the loss of membrane potential, and inhibits the release of pro-apoptotic factors such as cytochrome c under conditions of cellular stress. Originally developed by the French biotech Trophos (later acquired by Roche), olesoxime was primarily investigated for the treatment of neurodegenerative diseases including Spinal Muscular Atrophy (SMA) and Amyotrophic Lateral Sclerosis (ALS). Despite showing promise in Phase 2 trials for SMA, development was ultimately discontinued by Roche in 2018 following regulatory feedback and strategic considerations in a rapidly evolving SMA therapeutic landscape.

Other names

cholest-4-en-3-one oximecholest4-en-3-one oximecholest 4-en-3-one oxime