Olesoxime is Trophos' lead compound of a proprietary mitochondrial pore modulator series. 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). Olesoxime has been shown to be active in the SOD1 model of Amyotrophic Lateral Sclerosis (ALS), often referred to as "Lou Gehrig's Disease".

*Actelion and Trophos entered into a binding agreement in July 2010 whereby Actelion obtained an exclusive option to acquire privately-held Trophos SA, a clinical stage pharmaceutical company. The decision on whether Actelion will exercise the option to acquire Trophos will be made upon completion of the ongoing pivotal Phase III study with olesoxime, expected by the end of 2011.

Current status
Olesoxime is currently being evaluated in a pivotal Phase III study conducted by Trophos SA in 15 centers in France, Germany, UK, Belgium and Spain. The study completed enrollment in the first quarter of 2010 with 512 patients diagnosed with ALS between 6 and 36 months before enrollment and receiving standard of care.

The study is an 18-month randomized, parallel group, double-blind, placebo-controlled trial evaluating the efficacy and safety of olesoxime against placebo and has benefited from protocol advice obtained from the European Medical Agency EMA.

The primary endpoint of the study is the overall 18-month survival rate. Secondary endpoints include the ALS Functional Rating Scale, time to assisted ventilation, vital capacity (a measure of respiratory function), Manual Muscular Testing and quality of life.

Available clinical data
Phase I studies in healthy volunteers and Phase Ib studies in ALS patients demonstrated that olesoxime is well-tolerated. These studies also helped to determine the dose regimen used in the pivotal Phase III study.

Milestones
2010 – Actelion and Trophos enter into binding agreement
2010 – Trophos completes patient enrollment in pivotal efficacy Phase III study
2005 – Trophos receives orphan drug designation for olesoxime for the treatment of ALS

Key scientific literature
Bordet et al (2007). Identification and characterization of cholest-4-en-3-one, oxime (TRO19622), a novel drug candidate for amyotrophic lateral sclerosis. Pharmacol Exp Ther 322:709-720.