Olesoxime

Olesoxime (TRO19622) is the lead compound of Trophos' proprietary cholesterol-oxime compound family of mitochondrial pore modulators.

It is a molecule that has a cholesterol-like structure and displays strong 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. Starting 2010, olesoxime is undergoing a phase II clinical trial as treatment for spinal muscular atrophy.