On Tuesday, Cumberland Prescribed drugs Inc. CPIX launched topline outcomes from its Part 2 FIGHT DMD trial.
The research evaluated ifetroban, a novel oral remedy for Duchenne muscular dystrophy (DMD) coronary heart illness – the main reason behind dying in DMD sufferers.
DMD is a uncommon and incurable pediatric illness attributable to mutations within the gene encoding dystrophin, a protein vital for muscle perform, together with the guts.
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Sufferers with DMD slowly lose muscle perform, ensuing within the lack of ability to stroll, issue respiration, and coronary heart failure.
The trial enrolled 41 sufferers who obtained both low-dose ifetroban (150 mg per day), high-dose ifetroban (300 mg per day), or placebo.
The research’s main endpoint was an enchancment within the coronary heart’s left ventricular ejection fractions (LVEF), a key indicator of how properly the guts is pumping blood.
Key findings embody:
- Excessive-dose ifetroban remedy resulted in an total 3.3% enchancment in LVEF.
- The high-dose ifetroban group confirmed a rise in 1.8% in LVEF, whereas the research placebo group confirmed an anticipated decline in LVEF of 1.5%
- The distinction was much more pronounced compared with propensity-matched pure historical past controls, with the high-dose remedy offering a big 5.4% total enchancment in LVEF, whereas the management sufferers skilled a 3.6% decline.
- Each doses of ifetroban had been well-tolerated, with no critical drug-related occasions.
Ifetroban has obtained each Orphan Drug Designation and Uncommon Pediatric Illness Designation from the FDA. If authorized, ifetroban could be the primary remedy particularly indicated for DMD-related coronary heart illness.
Value Motion: CPIX inventory is up 23.01% at $2.62 on the final test Tuesday.
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