Additional cardiac biomarker analyses further demonstrate ifetroban's cardioprotective effect in DMD patients

NASHVILLE, Tenn., June 26, 2026 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (NASDAQ:CPIX), an innovation-focused biopharmaceutical company committed to developing new products for rare diseases, today shared updated results from its Phase 2 FIGHT DMD trial evaluating ifetroban, a novel oral therapy for Duchenne muscular dystrophy (DMD) heart disease, at the annual Parent Project Muscular Dystrophy (PPMD) conference in Orlando, Florida. The presentation highlighted key updates to the primary findings in the FIGHT DMD trial, reinforcing ifetroban's potential to address the leading cause of death in patients with DMD.

The presentation was delivered by Chet R. Villa, MD, a pediatric cardiologist at Cincinnati Children's Hospital Medical Center and one of the leaders of the muscular dystrophy committee in Advanced Cardiac Therapies Improving Outcomes Network (ACTION) as part of the conference program.

"Cardiomyopathy is the leading cause of mortality in patients with Duchenne muscular dystrophy, and there are currently no approved treatments that specifically target the underlying cardiac disease," said Dr. Villa. "As a site investigator on the FIGHT DMD trial, I have seen the importance of identifying therapies that protect cardiac function in these young patients. The updated findings being presented today include the reduction in markers of heart muscle injury alongside the previously reported improvement in left ventricular ejection fraction. These demonstrate that ifetroban slows ongoing heart damage in patients with DMD compared to natural history studies, addressing a critical unmet need, and sharing these results with the PPMD community is particularly meaningful."

The complete set of slides presented at the PPMD conference is now available on Cumberland's website.

The 12-month Phase 2 FIGHT DMD trial (NCT03340675) previously demonstrated that high-dose ifetroban treatment resulted in a significant 5.4% improvement in left ventricular ejection fraction (LVEF) compared to a control group composed of placebo-treated patients combined with propensity score-matched natural history patients. The study findings also included reductions in cardiac damage markers (NT-proBNP and cardiac troponin I) in the high-dose group. All patients who completed the 12-month study opted to continue in the open-label extension.

New findings from the Phase 2 FIGHT DMD trial, supported by long-term safety data from the ongoing open-label extension, further characterize the cardioprotective effect exerted by ifetroban in DMD cardiomyopathy. Through 36 months of treatment across both the Phase 2 trial and the open-label extension, ifetroban maintained a favorable safety profile in DMD patients with no treatment-related serious adverse events or new safety concerns. Evaluation of novel blood biomarkers identified a 30% reduction in MYL3 and a 50% reduction in MYOD1 (circulating markers of heart muscle and cell damage), alongside a 2.4-fold increase in FGF16 and a 2.1-fold increase in TSPAN7 (cardioprotective and tissue repair proteins) with ifetroban treatment compared with placebo. These confirmatory findings further support the potential therapeutic mechanisms for ifetroban in DMD cardiomyopathy.

"The results being shared at this year's PPMD conference continue to strengthen our confidence in ifetroban's potential to address the cardiomyopathy that affects all patients with Duchenne muscular dystrophy. The improvement in cardiac function we observed in the 12-month study is supported by additional biological evidence that ifetroban can help protect the heart from ongoing injury," said A.J. Kazimi, Cumberland CEO.

Pat Furlong, founding president and chief executive officer of Parent Project Muscular Dystrophy, said, "For more than three decades, we have worked to ensure that cardiac care receives the attention it deserves in Duchenne muscular dystrophy. Cardiomyopathy affects nearly every patient with DMD, and the absence of treatments designed specifically for this aspect of the disease has been a critical gap in care for the families we serve. The updated results from the FIGHT DMD trial being shared at this year's conference are encouraging and bring families closer to having a therapy that targets the underlying cardiac disease. We thank Cumberland for its continued investment in this work, and we recognize the contributions of the investigators and the families who took part in the trial."

Ifetroban is a once-daily oral medication that works by blocking the thromboxane receptor, which plays a key role in inflammation and fibrosis. The drug has received Orphan Drug Designation, Rare Pediatric Disease Designation and Fast Track Designation from the U.S. Food and Drug Administration (FDA) for the indication of cardiomyopathy associated with DMD. There is currently no approved treatment specifically targeting DMD heart disease, highlighting the critical unmet medical need in this patient population where cardiac complications are universal and represent the leading cause of death.

Cumberland has secured a growing portfolio of patents protecting the product for this DMD heart disease indication. Next steps include the completion of long-term treatment analyses, and the conduct of additional supportive studies.

More information regarding the FIGHT DMD Trial is available at www.fightdmdtrial.com

About Duchenne Muscular Dystrophy (DMD)

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.

About Parent Project Muscular Dystrophy (PPMD)

Parent Project Muscular Dystrophy is a grassroots, parent-led advocacy group with the mission to end DMD. Since its founding in 1994, PPMD has helped to accelerate treatments through research funding, to provide access to optimal DMD care for families, and to affect legislation through advocacy to improve the lives of children with DMD and their families. PPMD also hosts an annual conference which is the largest, most comprehensive, annual international conference focused entirely on DMD. This conference connects families across the world to share their stories and serves as a forum to highlight the progress in ending DMD.

About FIGHT DMD

FIGHT DMD is a community-based organization founded by Terry and Sonya Marlin with the mission of advancing research into DMD heart disease. The organization has provided crucial early-stage funding for multiple research projects focused on understanding and treating cardiac complications in DMD patients. Their support has been instrumental in advancing preclinical research that led to FDA grant funding for clinical trials, demonstrating the power of family-driven advocacy in catalyzing medical innovation.

About Cumberland Pharmaceuticals

Cumberland Pharmaceuticals Inc. is the largest biopharmaceutical company founded and headquartered in Tennessee and is focused on developing innovative products that improve the quality of patient care. The company is advancing a clinical pipeline of late-stage product candidates across multiple therapeutic areas with significant unmet medical needs.

Cumberland's Phase 2 clinical programs are evaluating ifetroban in patients with Duchenne Muscular Dystrophy, Systemic Sclerosis, and Idiopathic Pulmonary Fibrosis.

For more information, please visit www.cumberlandpharma.com.

Forward-Looking Statements

This press release contains forward-looking statements, which are subject to certain risks and reflect Cumberland's current views on future events based on what it believes are reasonable assumptions. No assurance can be given that these events will occur. Forward-looking statements include, among other things, statements regarding the Company's intent, belief or expectations, and can be identified by the use of terminology such as "may," "will," "expect," "believe," "intend," "plan," "estimate," "goal", "should," "seek," "anticipate," "look forward" and other comparable terms or the negative thereof. As with any business, all phases of Cumberland's operations are subject to factors outside of its control, and any one or combination of these factors could materially affect Cumberland's operation results. These factors include risks and uncertainties related to the strategic transaction, risks related to our ability to develop our pipeline of new product candidates, macroeconomic conditions, including changes in interest rates, inflation, tariffs, competition, an inability of manufacturers to produce Cumberland's products on a timely basis, failure of manufacturers to comply with regulations applicable to pharmaceutical manufacturers, natural disasters, public health epidemics, maintaining an effective sales and marketing infrastructure, and other events beyond the Company's control as more fully discussed in its most recent annual report on Form 10-K as filed with the U.S. Securities and Exchange Commission ("SEC"), as well as the Company's other filings with the SEC from time to time. There can be no assurance that results anticipated by the company will be realized or that they will have the expected effects. Readers are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date hereof. The Company does not undertake any obligation to publicly revise these statements to reflect events after the date hereof.

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SOURCE Cumberland Pharmaceuticals Inc.

Cumberland Pharmaceuticals Shares Updated FIGHT DMD Trial Results at the Parent Project Muscular Dystrophy Annual Conference