Olesoxime (TRO19622) is the lead compound of the Trophos' proprietary cholesterol-oxime compound family of mitochondrial pore modulators. Preclinical studies have demonstrated that olesoxime 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) and olesoxime has been shown to be active in the NSE-Cre F7/F7 model of SMA.
Olesoxime is currently undergoing a pivotal efficacy study in another rare motor neuron disease, Amyotrophic Lateral Sclerosis (ALS), which has recruited 512 patients and for which results are expected in Q4 2011. Olesoxime has successfully completed a phase 1b study in SMA patients, having previously completed phase I/Ib studies in healthy volunteers and ALS patients. These clinical trials demonstrated that the product is well-tolerated and has an excellent safety profile. They also showed that once-a-day oral dosing achieves the predicted exposure level required for efficacy, based on preclinical models.
