CAS Number 22033-87-0 ChemSpider 23350363 Molar mass 399.65 | PubChem CID 21763506 Formula C27H45NO 3D model (Jmol) Interactive image | |
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Olesoxime (TRO19622) is an experimental neuroprotective drug. It is the lead compound of Trophos' cholesterol-oxime compound family of mitochondrial pore modulators.
It is a molecule that has a cholesterol-like structure and displays neuroprotective properties. TRO19622 is as effective as a cocktail of three neurotrophic factors in keeping motor neurons alive in culture.
Preclinical studies have demonstrated that the compound promotes the function and survival of neurons and other cell types under disease-relevant stress conditions through interactions with the mitochondrial permeability transition pore (mPTP).
A 2009–2011 phase 3 clinical trial of olesoxime in amyotrophic lateral sclerosis did not demonstrate a significant increase in survival versus placebo.
The results of a 2011–2013 combined phase 2/3 trial of olesoxime in spinal muscular atrophy (SMA) indicated that the compound prevents deterioration of muscle function.
Since 2015, olesoxime is being developed as a drug for SMA by Hoffmann-La Roche.