Trophos completes enrolment in registration trial for ALS
Marseille – French biotech Trophos has completed enrolment of more than 500 patients suffering from Amyotrophic Lateral Sclerosis (ALS) into a pivotal phase 3 efficacy study of olesoxime (TRO19622). The 18-month registration trial for the orphan drug will be supported by the EUR6m EU fund for the MitoTarget project that seeks to assess the therapeutic potential of Trophos' novel proprietary class of mitochondrial pore modulator in neurological diseases. It will evaluate the efficacy and safety of a 330 mg olesoxime dose per day against placebo in patients who had diagnosed with ALS between 6 and 36 months before enrolment and who are already treated with riluzole at 50 mg twice a day The primary end-point of the study is the overall 18 month survival rate. Secondary end-points include the ALS Functional Rating Scale, time to assisted ventilation, vital capacity (a measure of respiratory function), Manual Muscular Testing and quality of life. Preclinical studies 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; olesoxime has been shown to be active in the SOD1G93A mouse model of ALS (Bordet et al., JPET 322:709-720, 2007). It has been demonstrated to be well-tolerated and showed an excellent safety profile.