Significant Cardiac Benefit Seen With Ifetroban in Patients With Duchenne Muscular Dystrophy

Treatment with ifetroban, a potent and selective thromboxane-prostanoid receptor antagonist, resulted in significant cardiac benefit over 12 months in patients with Duchenne muscular dystrophy (DMD), according to the results of the phase-2 FIGHT DMD trial presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference.

According to an abstract by John Jerry Parent, MD, of Indiana University School of Medicine, and colleagues, the leading cause of death in patients with DMD is cardiomyopathy. This phase-2 trial was designed to evaluate the safety and efficacy of ifetroban over a 12-month period in 41 patients with DMD.

Patients were randomly assigned to placebo, low-dose ifetroban, or high-dose ifetroban daily for 12 months. Treatment with the high-dose ifetroban resulted in an overall 3.3% improvement in left ventricular ejection fraction (LVEF) at 12 months; treatment with high-dose ifetroban yielded an increase in 1.8% in LVEF compared with a decrease of 1.5% for placebo.

When compared with propensity matched natural history controls, the difference was more pronounced; patients assigned the high-dose treatment had a significant 5.4% overall improvement in LVEF, compared with a 3.6% decrease in LVEF in those assigned placebo.

The researchers noted that transcriptomic profiling identified potential biomarkers that changed with ifetroban treatment and corresponded with LVEF improvement.

Commenting in a press release, study researcher Jonathan Soslow, MD, Professor of Pediatrics, Vanderbilt University, said, “the cardiac imaging data from this trial is compelling. The preservation and even improvement in cardiac function seen with ifetroban treatment stands in stark contrast to the expected decline we typically observe in untreated DMD patients.”

The Food and Drug Administration has granted ifetroban Orphan Drug Designation and Rare Pediatric Designation for the treatment of cardiomyopathy associated with DMD.

Reference

Parent JJ, Markham LW, Soslow JH, et al. Oral Thromboxane Receptor Antagonist Demonstrates Significant Cardiac Benefit in 12-Month Phase 2 Trial in Duchenne Muscular Dystrophy Patients. LB440. Presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference.