Novel oral therapy shows a 3.3% increase in left ventricular ejection fraction, marking a potential breakthrough in treating Duchenne muscular dystrophy cardiomyopathy
In a positive development for the Duchenne muscular dystrophy (DMD) community, Cumberland Pharmaceuticals (Nashville, TN, USA) has announced positive top-line results from its phase II FIGHT DMD trial (A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study with an Open-Label Extension to Determine the Safety, Pharmacokinetics and Efficacy of Oral Ifetroban in Subjects with Duchenne Muscular Dystrophy; ClinicalTrials.gov identifier: NCT03340675). This is the first successful phase II study targeting the cardiac complications of DMD, the leading cause of death in patients with DMD.1,2
Ifetroban is a once-daily oral medication that works by blocking the thromboxane receptor.2 This receptor is thought to play a role in promoting inflammation and fibrosis, processes that contribute to the progression of heart disease in patients with DMD.3 By inhibiting the receptors activity, ifetroban could help reduce cardiac inflammation and fibrosis, thereby preserving and potentially improving heart function in patients.
Study design and methodology
The FIGHT DMD trial was a 12-month, double-blind, randomized, placebo-controlled study involving 41 patients with DMD. Participants received either a low dose of ifetroban (150 mg/day), a high dose of ifetroban (300 mg/day) or a placebo. The study focused on the primary endpoint of improving left ventricular ejection fraction (LVEF), a key measure of heart function.
Results at a glance2
- High-dose ifetroban led to an overall 3.3% improvement in LVEF.
- Specifically, the high-dose group saw a 1.8% increase in LVEF, contrasting with a 1.5% decline in the placebo group.
- When compared to propensity-matched natural history controls, the high-dose group demonstrated a striking 5.4% overall improvement, as control patients experienced a 3.6% decline.
- Both doses were well-tolerated, with no serious drug-related adverse events reported.
Conclusions and future directions
These results could point to ifetroban’s potential to not just preserve but improve cardiac function in patients with DMD. “The improvement in cardiac function observed with ifetroban, particularly in the high-dose group, offers hope for these patients and their families,” noted Dr. Larry W Markham, principal investigator of the FIGHT DMD trial.2
While these findings are promising, additional studies will be critical to confirm the long-term benefits and safety of ifetroban. Nevertheless, these results could mark a milestone in the quest to address a critical unmet need in DMD care.
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References
- Soslow JH, Xu M, Slaughter JC, et al. Cardiovascular Measures of All-Cause Mortality in Duchenne Muscular Dystrophy. Circ Heart Fail. 2023; 16:e010040. doi: 10.1161/CIRCHEARTFAILURE.122.010040.
- Cumberland Pharmaceuticals. Cumberland Pharmaceuticals Announces Breakthrough Results from the Phase 2 FIGHT DMD Trial in Duchenne Muscular Dystrophy Heart Disease. [Press release]. 2024 Available at: https://investor.cumberlandpharma.com/news-releases/news-release-details/cumberland-pharmaceuticals-announces-breakthrough-results-phase (accessed 7 February 2025).
- West JD, Galindo CL, Kim K, et al. Antagonism of the Thromboxane-Prostanoid Receptor as a Potential Therapy for Cardiomyopathy of Muscular Dystrophy. J Am Heart Assoc. 2019;8:e011902. doi: 10.1161/JAHA.118.011902.
Disclosures: This article was created by the touchCARDIO team utilizing AI as an editorial tool (ChatGPT (GPT-4o) [Large language model]. https://chat.openai.com/chat.) The content was developed and edited by human editors. No funding was received in the publication of this article.
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Editor: Gina Furnival, Senior Editorial Director
Support: No funding was received in the publication of this short article.
Cite: Ifetroban improves heart function in Duchenne muscular dystrophy: Promising phase II trial results from FIGHT. touchCARDIO, February 10, 2025.
