Originally published on November 5, 2013.

Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for indications with under-served needs in neurology and cardiology, has completed its efficacy study of olesoxime in the rare neurodegenerative condition Spinal Muscular Atrophy (SMA).

Outcome data are expected to be available towards the end of 2013. If positive, Trophos will file olesoxime for market authorization in the EU and the US for the treatment of SMA with the aim of having the drug on the market by 2015.

The phase II/III pivotal study evaluated the efficacy and safety of Trophos’ olesoxime – a novel mitochondria pore modulator with neuroprotective and nerve repair properties. Substantial funding for this study came via the partnership of Trophos with the Association Francaise contre les Myopathies (AFM-Telethon).

The study enrolled 165 SMA patients in a 24-month randomized, parallel group, double-blind, placebo controlled trial comparing olesoxime against placebo in type II and non-ambulant type III SMA patients aged from three to twenty five years old. Olesoxime is administered at the dose of 10 mg/kg/day using a specially developed liquid formulation. Patients were randomized to receive olesoxime in a 2:1 ratio versus placebo. This trial protocol and its data analysis plan went through the EMA protocol advice procedure.

“This study will be a landmark for the SMA community. Olesoxime is a promising therapy for patients living with SMA. It gives hope that they will maintain function and autonomy,” said Dr Enrico Bertini. “The longitudinal data collected from 165 patients for two years comparing functional outcome measures with potentially predictive and innovative biomarkers will make a valuable contribution to future clinical trial designs for SMA or other neuromuscular diseases.”