French biopharmaceutical company Trophos SA announced the completion of the interim analysis of the pivotal efficacy study of olesoxime in Spinal Muscular Atrophy (SMA) which showed the molecule to be safe. 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 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 laboratory 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.
"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."
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