Catabasis reaches target enrollment for Part B of MoveDMD trial
Catabasis Pharmaceuticals announced that target enrollment of 30 patients has been reached for Part B of the MoveDMD trial, a 12-week trial to assess the safety and efficacy of edasalonexent, CAT-1004, in Duchenne muscular dystrophy. Edasalonexent is an oral small molecule that the Company believes has the potential to be a disease-modifying therapy for DMD patients, regardless of their underlying dystrophin mutation. Catabasis has previously reported positive safety, tolerability, pharmacokinetics and biomarker results from Part A of the trial. Based on scheduling of the enrolled patients as of September 30, 2016, the Company expects to report top-line safety and efficacy results from this Phase 2 trial in the first half of Q1 2017. In the first portion of the MoveDMD trial (Part A), 17 ambulatory boys between ages 4 and 7 with a genetically confirmed diagnosis of DMD across a range of dystrophin mutations received edasalonexent. The boys were steroid naive or had not used steroids for at least six months prior to the trial. This portion of the trial was conducted at three sites in the U.S., and assessed the safety, tolerability and pharmacokinetics of edasalonexent in patients at three dosing levels during seven days of dosing. Sixteen of the boys who participated in the first part of the MoveDMD trial are participating in Part B. Phase 2 of the MoveDMD trial is a randomized, double-blind, placebo-controlled trial of approximately 30 boys to evaluate the safety and efficacy of edasalonexent in DMD over a 12-week period at five clinical trial sites in the U.S. at two dosing levels, 67 mg/kg/day and 100 mg/kg/day. The open-label extension was initiated in July and includes dosing with edasalonexent for 36 weeks beyond the 12-week placebo-controlled portion of the trial and will evaluate longer term safety and efficacy with the same clinical end points. PPMD and the Muscular Dystrophy Association are providing funding to support participant travel for the MoveDMD trial.