Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX) presented updated results from its Phase 2 FIGHT DMD trial at the Parent Project Muscular Dystrophy annual conference in Orlando, Florida, according to a company press release.

The presentation, delivered by Chet R. Villa, MD, a pediatric cardiologist at Cincinnati Children's Hospital Medical Center, included new biomarker analyses from the trial evaluating ifetroban, an oral thromboxane receptor blocker, in patients with Duchenne muscular dystrophy (DMD) cardiomyopathy.

New findings from the trial showed a 30% reduction in MYL3 and a 50% reduction in MYOD1, both markers of heart muscle and cell damage, in patients receiving ifetroban compared with placebo. The data also showed a 2.4-fold increase in FGF16 and a 2.1-fold increase in TSPAN7, proteins associated with cardioprotection and tissue repair.

These results build on previously reported 12-month trial data showing a 5.4% improvement in left ventricular ejection fraction in high-dose ifetroban patients compared to a control group, along with reductions in cardiac damage markers NT-proBNP and cardiac troponin I.

Through 36 months of treatment across the Phase 2 trial and an ongoing open-label extension, ifetroban showed no treatment-related serious adverse events or new safety concerns.

Ifetroban has received Orphan Drug Designation, Rare Pediatric Disease Designation, and Fast Track Designation from the U.S. Food and Drug Administration for cardiomyopathy associated with DMD. No treatment specifically targeting DMD-related heart disease is currently approved.

Cumberland stated that next steps include completing long-term treatment analyses and conducting additional supportive studies.