On Tuesday, Cumberland Pharmaceuticals Inc. CPIX released topline 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.

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.

Also Read: Why Is Cumberland Pharmaceuticals Stock Trading Over 100% on Tuesday?

Patients with DMD slowly lose muscle function, resulting in the inability to walk, difficulty breathing, and heart failure.

The trial enrolled 41 patients who received either low-dose ifetroban (150 mg per day), high-dose ifetroban (300 mg per day), or placebo.

The study’s primary endpoint was an improvement in the heart’s left ventricular ejection fractions (LVEF), a key indicator of how well the heart is pumping blood. 

Key findings include:

• High-dose ifetroban treatment resulted in an overall 3.3% improvement in LVEF.
• The high-dose ifetroban group showed an increase in 1.8% in LVEF, while the study placebo group showed an expected decline in LVEF of 1.5%
• The difference was even more pronounced when compared with propensity-matched natural history controls, with the high-dose treatment providing a significant 5.4% overall improvement in LVEF, while the control patients experienced a 3.6% decline.
• Both doses of ifetroban were well-tolerated, with no serious drug-related events.

Ifetroban has received both Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA. If approved, ifetroban would be the first therapy specifically indicated for DMD-related heart disease.

Price Action: CPIX stock is up 23.01% at $2.62 at the last check Tuesday.

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