Trophos initiates pivotal efficacy study of ALS drug

French drug developer Trophos SA has initiated a pivotal efficacy study of olesoxime in amyotrophic lateral sclerosis. The study is supported by  the European Union MitoTarget project. The trial protocol has benefited  from European Medicines Agency (EMEA) Protocol Advice procedure.  Efficacy results are expected in mid-2011.

Olesoxime (TRO19622) is the lead compound of Trophos' proprietary  cholesterol-oxime compound family of mitochondrial pore modulators.  Preclinical studies have demonstrated that the compounds promote the  function and survival of neurons and other cell types under  disease-relevant stress conditions through interactions with the  mitochondrial permeability transition pore, and olesoxime has been  shown to be active in the SOD1 model of ALS.

Olesoxime has successfully completed Phase I studies in healthy  volunteers and Phase Ib trials in ALS patients. These 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. Drug  interaction studies with riluzole, the only registered treatment for  ALS, showed no interaction of TRO19622 on riluzole  pharmacokinetics.