Catabasis plans Phase 3 Duchenne trial

Catabasis Pharmaceuticals announced plans for the Phase 3 POLARIS DMD trial with edasalonexent in patients with Duchenne muscular dystrophy, or DMD. Catabasis plans to initiate the global POLARIS DMD trial in the second half of 2018 with top-line results expected in Q2 of 2020. The POLARIS DMD trial will evaluate the efficacy and safety of edasalonexent in patients with DMD and is intended to support an application for commercial registration of edasalonexent. The trial design was informed by discussions with the FDA as well as input from treating physicians and families of boys affected by Duchenne. The randomized, double-blind, placebo-controlled POLARIS DMD trial has many key elements in common with the Phase 2 MoveDMD trial, including the patient population and functional endpoints. Catabasis anticipates enrolling approximately 125 patients between the ages of 4 and 7 regardless of mutation type who have not been on steroids for at least 6 months. Boys on a stable dose of eteplirsen may be eligible to enroll. The primary efficacy endpoint will be change in the North Star Ambulatory Assessment score after 12 months of treatment with edasalonexent compared to placebo. Key secondary endpoints are planned to include the age-appropriate timed function tests time to stand, 4-stair climb and 10-meter walk/run. Assessments of growth, cardiac and bone health are also planned to be included. Two boys will receive edasalonexent for every boy that receives placebo and after 12 months, all boys are expected to receive edasalonexent in an open-label extension

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