Trophos SA, a clinical stage pharma company developing innovative therapeutics from discovery to clinical validation for under-served medical needs in neurology and cardiology, announced  the completion of the interim analysis of the pivotal efficacy study of olesoxime in the rare neurodegenerative condition spinal muscular atrophy (SMA).
 
The independent Data Monitoring Committee (DMC) has reviewed the treatment effect at one year on the primary outcome measure of efficacy, change in motor function using the MFM scale, together with the latest safety report including electrocardiogram traces, periodic lab findings, haemostatic parameters and serious adverse events listings for all participants. Based on the trial stopping criteria as defined in the protocol as well as no safety concerns related to olesoxime treatment, their recommendation is to continue the study as planned.
An interim analysis of efficacy, as included in the study protocol, has been conducted after all participants have been treated for one year. Over 160 patients have been recruited into the trial between October 2010 and September 2011. Following the recommendations of the DMC, the study will continue until all participants have been treated for two years with the last patient out scheduled for September 2013. Top line results are expected by the end of 2013.
 
"The approval from the DMC to continue the trial based on the interim analysis for this innovative treatment of SMA is perfect news at this stage. We confirmed the good safety of olesoxime treatment and we look forward to completing the two year treatment period to report efficacy in these SMA patients," said Rebecca Pruss, chief scientific officer at Trophos. "SMA is a progressive and disabling neuromuscular disease. Treatments are desperately needed that slow down or prevent the loss of neuromuscular function in SMA patients. This study of olesoxime has been conducted successfully so far due to the enormous commitment of patients and clinicians to find a treatment for SMA. We believe the results due in fourth quarter 2013 could be an historic moment in the development of treatments for SMA."
 
"The recommendation by the DMC to proceed confirms that olesoxime is a safe molecule and we look forward to quickly analysing results on the efficacy outcome of the trial after it reaches the planned conclusion," said principle investigator Dr Enrico Bertini. "Other interesting potential outcomes that will surely emerge from the results of this trial will be related to the issue of biomarker analysis and also to the reliability, variability and validity of electrophysiological data which have been collected longitudinally in patients. All the information from this study will be valuable for future clinical multi-centre trials in SMA. In the meantime we hope that olesoxime will have a therapeutic impact on the natural history of SMA."
 
"Thanks to the donations to the French telethon, we have been supporting the development of olesoxime since the first screening up to and including the ongoing clinical trial. This is an important clinical study and brings hope for a first potential treatment for SMA patients," said Christian Cottet, CEO, AFM-Telethon.
 
"Trophos and the AFM have been working together for over a decade. This crucial clinical study is the latest step in our long standing partnership," said Christine Placet, CEO, Trophos. "Olesoxime has a promising profile as a potential treatment for SMA. We are very hopeful that the results of this study will bring a much needed treatment option and new hope to SMA patients and their families. Trophos has a history of commitment to developing breakthrough therapies for rare and serious neurodegenerative diseases."
 
The study is a 24-month randomized, parallel group, double-blind, placebo controlled trial comparing olesoxime against placebo in non-ambulant type II and type III SMA patients aged from 3 to 25 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.