Catabasis Pharmaceuticals, Inc. reported new positive efficacy and safety results showing preservation of muscle function and sustained disease-modifying effects in boys with Duchenne muscular dystrophy (DMD) in the MoveDMD trial open-label extension following 48 and 60 weeks of treatment with edasalonexent. Consistent improvements in all assessments of muscle function were observed after more than a year of oral 100 mg/kg/day edasalonexent treatment compared to the rates of change in the pre-specified control period for boys prior to receiving edasalonexent treatment. Additionally, supportive changes in non-effort based measures of muscle health were seen, with significant longer-term reductions in muscle enzymes and C-reactive protein (CRP), supporting the durability of edasalonexent treatment effects. Edasalonexent continued to be well tolerated with no safety signals observed in the trial. These data will be presented on Saturday, February 17, 2018, at 14:15 CET at the XVI International Conference on Duchenne and Becker Muscular Dystrophy in Rome, Italy, and detailed at future scientific conferences.
Read more by clicking here.