Catabasis Pharmaceuticals Phase 3 PolarisDMD Clinical Trial for Edasalonexent in Duchenne Muscular Dystrophy Progress Update and Additional Trial Sites Open for Enrollment
January 03, 2019 at 06:38 pm IST
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Catabasis Pharmaceuticals, Inc. shared an update on clinical trial sites in the United States and recent international clinical trial application approvals to perform the Phase 3 PolarisDMD trial for edasalonexent (CAT-1004) in Duchenne muscular dystrophy (DMD). PolarisDMD clinical trial sites in the United States are actively enrolling patients. The PolarisDMD trial is evaluating the efficacy and safety of edasalonexent in patients with DMD and is intended to support an application for commercial registration of edasalonexent. Top-line results from the Phase 3 PolarisDMD trial are expected in the second quarter of 2020. A total of nine PolarisDMD clinical trial sites are now open for enrollment across the United States and at least ten additional sites are expected to open shortly. Additionally, Catabasis has received approval of clinical trial applications to conduct the PolarisDMD trial in Canada and multiple countries in Europe. Clinical trial sites are expected to open in Canada this month and sites in Europe as well as Australia and Israel, pending regulatory approval, are expected to open in first quarter of this year. In total, the PolarisDMD trial is expected to include approximately 40 clinical trial sites globally with enrollment expected to be completed in 2019. The global Phase 3 PolarisDMD trial is a one-year, randomized, double-blind, placebo-controlled trial. Catabasis plans to enroll approximately 125 patients ages 4 to 7 (up to 8th birthday) 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 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. Two boys will receive 100 mg/kg/day of edasalonexent for each boy that receives placebo, and, after 12 months, all boys are expected to receive edasalonexent in an open-label extension study. 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. The Phase 3 PolarisDMD trial is designed to further validate the positive efficacy seen in the MoveDMD Phase 2 trial and open-label extension evaluating edasalonexent. In the MoveDMD trial, edasalonexent has been shown to preserve muscle function and substantially slow DMD disease progression across all four assessments of muscle function (the North Star Ambulatory Assessment, time to stand, 4-stair climb and 10-meter walk/run) through 72 weeks of treatment compared to an off-treatment control period. Preclinical data and clinical biomarker data from the MoveDMD Phase 2 trial suggest that edasalonexent could have potential benefits in skeletal muscle, diaphragm and heart. Edasalonexent has been well tolerated through more than 50 patient-years of treatment with no safety signals observed.
Astria Therapeutics, Inc. is a biopharmaceutical company focused on developing therapies for allergic and immunological diseases. The Company's lead program, STAR-0215, is a monoclonal antibody inhibitor of plasma kallikrein in clinical development for the treatment of hereditary angioedema (HAE), a rare, debilitating and potentially life-threatening disease. Its second program, STAR-0310, is a monoclonal antibody OX40 antagonist in preclinical development for the treatment of atopic dermatitis (AD). The Company owns two patent families directed to STAR-0215. The first patent family is directed to the composition of matter of its product candidate STAR-0215 and its use in treating various plasma kallikrein associated disorders including HAE. In the second patent family, the Company owns one International (PCT) patent application directed to methods of treating various plasma-kallikrein associated disorders, including HAE, with specific dosing regimens of the STAR-0215 antibody.
Catabasis Pharmaceuticals Phase 3 PolarisDMD Clinical Trial for Edasalonexent in Duchenne Muscular Dystrophy Progress Update and Additional Trial Sites Open for Enrollment