Cumberland Pharmaceuticals a specialty pharmaceutical company committed to developing new products for rare diseases, shared the latest positive results from its Phase 2 FIGHT DMD trial evaluating ifetroban, a novel oral therapy for Duchenne muscular dystrophy (DMD) heart disease, at the annual Parent Project Muscular Dystrophy (PPMD) conference in Las Vegas. These new results highlight multiple indicators of cardiac benefit with ifetroban treatment in DMD heart disease, the leading cause of death in DMD, including previously unreported pharmacokinetic findings and cardiac biomarker data that demonstrate the drug’s potential to prevent ongoing heart damage..
Ifetroban from Cumberland Showed a 5.4% Improvement Over Patients Receiving a Placebo
The 12-month Phase 2 FIGHT DMD trial (NCT03340675) demonstrated that high-dose ifetroban treatment resulted in a significant 5.4% improvement in left ventricular ejection fraction (LVEF) compared to a control group composed of placebo-treated patients combined with propensity score-matched natural history patients. This represents a clinically meaningful difference in a progressive disease where heart function typically declines over time. – Learn More: NCT03340675 –
The study also revealed pharmacokinetic insights, showing that DMD patients receiving higher doses than typical adults achieved similar plasma levels, with no evidence of drug accumulation, supporting the 300 mg daily dosing used in the high-dose group. Despite the higher dosing requirements, ifetroban was well-tolerated with an acceptable pharmacokinetic profile in patients with DMD.
What is Ifetroban?
Ifetroban is a once-daily oral medication that works by blocking the thromboxane receptor, which plays a key role in inflammation and fibrosis. The drug has received both Orphan Drug Designation Rare Pediatric Disease Designation, from the FDA for the indication of DMD heart disease. There is currently no approved treatment specifically targeting DMD heart disease, highlighting the critical unmet medical need in this patient population where cardiac complications are universal and represent the leading cause of death.
