Cumberland Pharmaceuticals Inc. a specialty pharmaceutical company with development efforts focused on rare diseases, announced that results from its Phase 2 FIGHT DMD clinical trial were selected for a late-breaking presentation at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Dallas. The trial demonstrated significant cardiac benefits for patients with Duchenne muscular dystrophy (DMD), potentially addressing the leading cause of death in this devastating disease.

The presentation, delivered today by Dr. John Jerry Parent, highlighted the breakthrough findings from Cumberland's study of ifetroban in DMD heart disease. For the abstract presented at the meeting, please see the MDA website.  

The Phase 2 FIGHT DMD trial demonstrated that high-dose ifetroban treatment resulted in a 3.3% improvement in left ventricular ejection fraction (LVEF) compared to placebo. When compared with propensity-matched natural history controls, the difference was even more pronounced, with high-dose treatment providing a significant 5.4% overall improvement in LVEF, as control patients experienced a 3.6% decline in LVEF. This improvement in cardiac function could translate to meaningful benefits in quality of life and survival for DMD patients. The complete presentation slides are now available on Cumberland's website.

"The enthusiastic response from MDA conference attendees reinforces the importance of our work in targeting DMD-related heart disease," said John Jerry Parent, MD, Associate Professor of Clinical Pediatrics and Medical Director of the Pediatric Heart Transplant at Indiana University School of Medicine and Site Investigator at Riley Children's Hospital for the FIGHT DMD trial. "These findings represent a potential paradigm shift in how we approach cardiac complications in DMD patients."

"We are honored that the MDA recognized the significance of our FIGHT DMD trial by selecting it for a late-breaking presentation," said A.J. Kazimi, Chief Executive Officer of Cumberland Pharmaceuticals. "This platform allowed us to share our promising results with the global DMD community, including leading researchers, clinicians and patient advocates who are working tirelessly to improve outcomes for those affected by this devastating disease."

"These results offer hope to thousands of families affected by DMD," said Sharon Hesterlee, PhD, Chief Research Officer, MDA. "While current therapies focus on preserving muscle function, addressing the cardiac complications of DMD remains an urgent unmet need. Cumberland's work with ifetroban represents a critical step forward in potentially extending and improving the lives of DMD patients."

Ifetroban, an oral thromboxane receptor antagonist, has received both Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA for DMD-related heart disease.

Cumberland has secured a growing portfolio of patents with claims associated with the product for this DMD indication. Next steps include further data analysis and completion of a full study report in preparation for an end-of-Phase-2 meeting with the FDA to determine next steps associated with the product's development and commercialization.