Trophos announces positive interim review in pivotal study of olesoxime in Spinal Muscular Atrophy
Trophos SA 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 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.
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 (see press release of September 8, 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 trial is substantially funded by Trophos' partnership with the Association Francaise contre les Myopathies (AFM-Telethon) (see press release of March 19, 2009). The trial protocol has benefited from the EMA protocol advice procedure.
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