Catabasis Pharmaceuticals has announced the end of the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy. Edasalonexent is a novel oral investigational drug which inhibits NF-kappa B, a pathway activated in DMD patients from infancy. The trial has not met its primary endpoint to measure the change in North Star Ambulatory Assessment (NSAA) over one year of edasalonexent treatment compared to placebo. The secondary endpoints also did not show statistically significant improvements where between edasalonexent and placebo, there were no significant differences in muscle enzymes and heart rate. Catabasis are deeply disappointed that the trial did not meet the primary or secondary endpoints.
Overall, Edasalonexent was well-tolerated by participants in the edasalonexent program. The majority of adverse events were mild in nature and there were no treatment-related serious adverse events and no dose reductions. The most common treatment-related adverse events were diarrhea, vomiting, abdominal pain and rash.
Catabasis Pharmaceuticals is greatly appreciative of the boys that participated in the trial and their families and caregivers. The company plans to work with external advisors to explore and evaluate strategic options going forward.
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