Cumberland Pharmaceuticals Inc (NASDAQ: CPIX), a US-based specialty pharmaceutical company, on Monday announced 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.

The company says that 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 demonstrating the drug's potential to prevent ongoing heart damage.

The trial results were presented by FIGHT DMD Trial principal investigator Larry W. Markham, MD, Professor of Pediatrics and Medicine at Riley Children's Hospital and the Indiana University School of Medicine, as part of the 'therapies that Slow Progression' conference session.

"PPMD has had a focus on heart disease and has been a part of this trial since its inception. We are proud to share the latest promising findings from the FIGHT DMD Trial with a group that has consistently supported us throughout our efforts to develop an effective treatment for DMD heart disease," said Dr Markham.

FIGHTDMD helped co-fund the preclinical studies at Vanderbilt's Monroe Carell Jr. Children's Hospital which formed the foundation for the FDA grant funding this trial.

According to the company, the 12-month Phase 2 FIGHT DMD trial 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.

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.