US-based pharmaceutical company Cumberland Pharmaceuticals has reported positive top-line outcomes from a Phase II trial of ifetroban as a potential treatment for Duchenne muscular dystrophy (DMD).
A total of 41 subjects with DMD were enrolled in the double-blind, placebo-controlled FIGHT DMD trial, which lasted 12 months.
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Subjects received either a daily dose of either 100mg (low dose) or 300mg (high dose) of the oral thromboxane receptor antagonist ifetroban or a placebo.
Improvement in the heart’s left ventricular ejection fraction (LVEF) was the trial’s primary endpoint.
The results showed that high-dose treatment of the therapy led to a 3.3% overall improvement in LVEF.
The high-dose arm experienced a 1.8% increase in LVEF, against a 1.5% decline in the placebo arm.
Compared with propensity-matched natural history controls, the high-dose treatment showed an overall improvement of 5.4%, as control subjects reported a decline of 3.6% in LVEF.
Both doses of the therapy were observed to be well-tolerated, without any serious drug-related events.
Ifetroban functions by blocking a receptor involved in inflammation and fibrosis, and has received orphan drug and rare paediatric disease statuses from the Food and Drug Administration (FDA).
If approved, the drug is expected to become the first therapy specifically indicated for DMD-related heart disease.
Cumberland Pharmaceuticals CEO AJ Kazimi said: “As the first company to receive FDA orphan products development funding for a DMD clinical trial, we’re honoured to be advancing a potential breakthrough therapy for DMD-related heart disease.
“These results validate our commitment to developing innovative treatments for rare diseases and underscore the importance of collaborative partnerships between industry, academia, and regulatory agencies in addressing critical unmet medical needs.”
Based in Tennessee, Cumberland Pharmaceuticals develops, acquires and commercialises products for hospital acute care, gastroenterology and oncology.
In May 2023, the company announced plans to launch the Phase II FIGHTING FIBROSIS trial of ifetroban for idiopathic pulmonary fibrosis (IPF).
