PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy completes enrollment.-Catabasis Pharma

Catabasis Pharmaceuticals announced the completion of enrollment for the Phase III PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD). The target enrollment of 125 boys was exceeded due to strong interest from our 40 clinical sites in 8 countries and the support of patient advocacy organizations.

The PolarisDMD trial enrolled 130 boys ages 4 to 7 (up to 8th birthday) with any mutation type and who had not been on steroids for the past 6 months. The trial is a randomized, double-blind, placebo-controlled trial with 2 to 1 randomization such that two boys receive edasalonexent for each boy that receives placebo. At the completion of 52 weeks, all boys and their eligible siblings are expected to have the option to enroll in GalaxyDMD, an open-label extension study designed to assess the long-term safety of edasalonexent. Boys can begin or continue treatment with an approved exon skipping therapy in the GalaxyDMD trial, which has a streamlined schedule with visits to trial sites every six months. Top-line results from the Phase III PolarisDMD trial are expected in the fourth quarter of 2020 and the trial is anticipated to support an NDA filing in 2021.