Core Insights - Cumberland Pharmaceuticals Inc. released topline results from its Phase 2 FIGHT DMD trial evaluating ifetroban for Duchenne muscular dystrophy (DMD) heart disease, which is a leading cause of death in DMD patients [1][2] - If approved, ifetroban would be the first therapy specifically indicated for DMD-related heart disease, having received both Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA [3] Study Details - The trial enrolled 41 patients who received either low-dose ifetroban (150 mg per day), high-dose ifetroban (300 mg per day), or placebo [2] - The primary endpoint was an improvement in the heart's left ventricular ejection fractions (LVEF), a key indicator of heart function [2] Key Findings - High-dose ifetroban treatment resulted in an overall 3.3% improvement in LVEF, with the high-dose group showing a 1.8% increase compared to a 1.5% decline in the placebo group [5] - Compared to propensity-matched natural history controls, the high-dose treatment provided a significant 5.4% overall improvement in LVEF, while control patients experienced a 3.6% decline [5] - Both doses of ifetroban were well-tolerated, with no serious drug-related events reported [5] Market Reaction - Following the trial results, CPIX stock increased by 23.01%, reaching $2.62 [3]
Cumberland Pharmaceuticals' Duchenne Candidate Shows Improved Heart Function In Patients With Associated Heart Disease