Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX) shares began Tuesday sharply higher. Cumberland, a specialty pharmaceutical company with development efforts focused on new products for rare diseases, today announced positive top-line results from its Phase 2 FIGHT DMD trial. The study evaluated ifetroban, a novel oral therapy for Duchenne muscular dystrophy (DMD) heart disease – the leading cause of death in DMD patients. It marks a breakthrough for these patients, as it’s the first successful Phase 2 study specifically targeting the cardiac complications of their condition.
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, highlighting a critical unmet medical need.
A previous study conducted at Vanderbilt University Medical Center demonstrated that ifetroban is protective against cardiomyopathy in several preclinical models of muscular dystrophy. The results of that study were published in the Journal of the American Heart Association (West 2019). Based on those promising results, Cumberland Pharmaceuticals became the first recipient of an FDA Office of Orphan Products Development clinical trial grant for DMD, funding the development of this Phase 2 clinical trial.
Shares in CPIX gathered 25 cents, or 11.7%, Tuesday to $2.38.
