Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX) announced 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 conference in Las Vegas. The results demonstrate multiple indicators of cardiac benefit and the drug's potential to prevent ongoing heart damage in DMD patients, addressing the leading cause of death in this rare disease population.
Significant Cardiac Function Improvement
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.
"The improvement in heart function observed, combined with the reduction in cardiac damage markers, suggests we may be able to meaningfully impact the progressive heart disease that affects virtually all DMD patients," said A.J. Kazimi, Cumberland CEO.
Biomarker Evidence of Cardioprotection
Critically, high-dose ifetroban treatment was associated with reduced blood levels of cardiac damage markers (NT-proBNP and cardiac troponin I), while these markers of heart damage increased in placebo-treated patients. This biochemical evidence of reduced cardiac damage, combined with functional heart improvement, provides compelling evidence of ifetroban's cardioprotective effects.
The observed improvement in cardiac function together with the reduction in cardiac damage biomarkers suggest a clinically significant impact for ifetroban in both heart disease and potential to impact overall survival in DMD. Demonstrating confidence in the treatment, all patients who completed the 12-month study opted to continue with the open-label extension.
Pharmacokinetic Profile and Safety
The study 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.
Addressing Critical Unmet Medical Need
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 and 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.
Trial Leadership and Community Support
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. "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.
The trial received crucial support from FIGHTDMD, a community-based organization founded by Terry and Sonya Marlin. 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. "Seeing these promising results validates our belief that targeted heart therapies can make a meaningful difference for our children," said Terry Marlin, President and Founder of FIGHTDMD.
Next Steps and Regulatory Pathway
Cumberland has secured a growing portfolio of patents protecting the product for this DMD heart disease indication. Next steps include the analysis of long-term treatment results and discussions with the FDA to determine the regulatory pathway forward based on these encouraging results.
DMD is a rare and incurable pediatric disease caused by mutations in the gene encoding dystrophin, a protein critical for muscle function, including the heart. Patients with DMD slowly lose muscle function, resulting in the inability to walk, difficulty breathing, and heart failure. While current treatments can help manage some DMD symptoms, there are no approved therapies specifically targeting DMD-related heart disease.
