by: Parent Project Muscular Dystrophy
Catabasis announced that the Phase 3 PolarisDMD trial for edasalonexent in Duchenne has completed enrollment and exceeded the target enrollment.
PPMD is proud that The Duchenne Registry was able to play a role in helping the company reach this exciting milestone. We look forward to additional updates from Catabasis.
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Special edition: September 2019
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Catabasis Pharmaceuticals Announces The Phase 3 PolarisDMD Trial Of Edasalonexent In Duchenne Muscular Dystrophy Has Exceeded Target Enrollment
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today the completion of enrollment for the Phase 3 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. Top-line results from the Phase 3 PolarisDMD trial are expected in the fourth quarter of 2020 and the trial is anticipated to support an NDA filing in 2021.
“We are thrilled to reach this important milestone. The interest and feedback from families and trial sites has been overwhelmingly positive. At a time when there are multiple trials for Duchenne, we are very pleased that physicians and families chose the Phase 3 PolarisDMD trial for edasalonexent,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis. “Edasalonexent has the potential to be a foundational therapy, providing benefit to boys, regardless of their underlying mutation, with the potential to benefit muscle function, as well as cardiac function and bone health. We look forward to completing the trial next year and are working diligently toward the goal of making edasalonexent available to patients.”
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.
