{"id":8850,"date":"2019-10-01T09:55:15","date_gmt":"2019-10-01T09:55:15","guid":{"rendered":"https:\/\/laforcedmd.com\/?p=8850"},"modified":"2022-05-03T18:09:57","modified_gmt":"2022-05-03T18:09:57","slug":"the-phase-3-polarisdmd-trial-of-edasalonexent-in-dmd-has-exceeded-target-enrollment","status":"publish","type":"post","link":"https:\/\/laforcedmd.com\/en\/the-phase-3-polarisdmd-trial-of-edasalonexent-in-dmd-has-exceeded-target-enrollment\/","title":{"rendered":"PolarisDMD Trial in DMD has exceeded target enrollment"},"content":{"rendered":"

Phase 3, PolarisDMD trial of edasalonexent in DMD has exceeded target enrollment<\/span><\/h2>\n

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La Force is happy to share the\u00a0latest edition of the Catabasis Connection newsletter<\/a><\/span><\/p>\n

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Catabasis Pharmaceuticals, Inc.<\/a> <\/span>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 their 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 a New Drug Application (NDA)<\/a><\/span> filing in 2021. <\/strong><\/p>\n

PRESS RELEASE<\/a><\/span><\/strong><\/p>\n

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Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis<\/strong> \u201cWe 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. 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.\u201d<\/p><\/blockquote>\n

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The PolarisDMD trial enrolled 130 boys ages 4 to 7 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 a 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. Read more about these studies here.<\/a><\/span><\/p>\n

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About Edasalonexent (CAT-1004)<\/h4>\n

Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Our ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the open-label extension trial GalaxyDMD. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit\u00a0www.catabasis.com<\/a>.<\/span><\/p>\n

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About Phase 3 PolarisDMD Trial<\/h4>\n

The global Phase 3 PolarisDMD trial is a one-year, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of edasalonexent in patients with DMD. The trial enrolled patients ages 4 to 7 regardless of mutation type who had not been on steroids for at least 6 months. Boys on a stable dose of eteplirsen were also eligible to enroll. The primary efficacy endpoint is change in the North Star Ambulatory Assessment score after 12 months of treatment with edasalonexent compared to placebo. Key secondary endpoints 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 included as important potential areas of differentiation. For each boy that receives a placebo, two boys are receiving 100 mg\/kg\/day of edasalonexent and after 12 months, all boys are expected to receive edasalonexent in the open-label extension study GalaxyDMD. The PolarisDMD trial design was informed by discussions with regulators as well as input from treating physicians, patient organizations and families of boys affected by Duchenne. Top-line results from the Phase 3 PolarisDMD trial are expected in the fourth quarter of 2020. More information about the Phase 3 PolarisDMD clinical trial is available on\u00a0clinicaltrials.gov<\/a>.<\/span><\/p>\n

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About Catabasis<\/h4>\n

The mission of Catabasis Pharmaceuticals is to bring hope and life-changing therapies to patients and their families. There lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and the Phase 3 trial, please visit www.catabasis.com<\/a>.<\/span><\/p>\n