Cumberland Pharmaceuticals’ ifetroban improves heart function in Phase 2 DMD trial
CPIX•A 12-month Phase 2 trial found a 5.4% absolute improvement in left ventricular ejection fraction versus combined placebo and natural-history controls. Cumberland Pharmaceuticals plans to seek FDA approval, but the agency requested additional analyses and suggested a randomized, placebo-controlled Phase 3 trial.
1. Trial results and FDA feedback
Cumberland Pharmaceuticals said its 12-month Phase 2 trial of ifetroban for cardiomyopathy associated with Duchenne muscular dystrophy showed a 5.4% absolute improvement in left ventricular ejection fraction versus combined placebo and matched natural-history controls (p=0.002). The FDA questioned whether the proposed evidence would establish efficacy, requested additional analyses and suggested a randomized, placebo-controlled Phase 3 trial. The agency granted rolling review, and the company said ifetroban was generally well tolerated through 36 months, with no treatment-related serious adverse events or new safety concerns.
