Ifetroban

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 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 . 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. SOURCE Cumberland Pharmaceuticals Inc. 21% more press release views with Request a Demo

Positive Results from Phase 2a Clinical Trial Study met its primary endpoint demonstrating safety and tolerability of ifetroban in patients with high-risk solid tumors Trial revealed promising efficacy signals with fewer deaths due to metastasis in the ifetroban arm, than in the placebo arm (p=0.037) Results support development of ifetroban as a candidate for metastasis prevention NASHVILLE, Tenn., June 2, 2026 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company focused on developing new products for rare diseases, and Vanderbilt Health today announce data from a Phase 2a clinical trial of ifetroban to prevent metastasis in high-risk solid tumors. The study's primary safety endpoint was achieved, along with favorable trends in decreased metastasis recurrence and metastasis-free survival. A safe and effective medication that reduces distant metastatic recurrence could transform cancer management and improve the lives of millions of cancer survivors and their families. The randomized, double-blind, placebo-controlled Phase 2a trial evaluated the safety of ifetroban, an investigational thromboxane A2 receptor antagonist, in patients with solid tumors at high risk of early metastatic recurrence. Cancer types included breast, lung, pancreatic, soft tissue, bladder, and renal cancers. The study met its primary endpoint, demonstrating that ifetroban was safe and well-tolerated in this patient population. Rates for adverse events related to treatment were similar between placebo and ifetroban. No serious adverse events (> grade 3) in either group were identified as being related to study treatment. Treatment discontinuation rates were not statistically different between placebo and ifetroban. Although primarily a safety study and intentionally not powered for efficacy, the study compared the percentage of patients with distant metastatic recurrence 12 months after completion of therapy in both groups (10 placebo-treated and 18 ifetroban-treated participants) as a prespecified secondary endpoint. While 50% of participants experienced distant metastatic recurrence in the placebo arm, only 17% of participants experienced distant metastatic recurrence in the ifetroban arm (p=0.091). Three deaths due to distant metastatic disease occurred in the placebo arm, and none occurred in the ifetroban arm (p=0.037). Though metastasis is a primary driver of cancer lethality, most current therapies act on tumor cells directly. Approaches targeting the mechanisms underlying the metastatic process are lacking. Even during clinical remission, microscopic metastases can remain present, leaving many patients at serious risk for metastatic recurrence. The premise of this novel therapy is that antagonizing the thromboxane A2 receptor and blocking platelet activation and aggregation lessens tumor cells' ability to migrate, spread, cluster, invade distal organs, and evade immune detection. This was the first trial evaluating the effects of ifetroban in people with solid tumors with high risk for early recurrence, defined as ≥ 50% chance of recurrence within 5 years of diagnosis. The intervention was given after all cancer-related therapies and surgical procedures had been completed; participants received the intervention for 12 months and were then followed for an additional 12 months. Among 29 participants, 10 received placebo and 19 received ifetroban. "A therapeutic intervention aimed at metastasis prevention for cancer patients with high risk of recurrence that is given during the period of "watchful waiting" could be groundbreaking if proven beneficial in larger scale investigations," said Dr. Ben Ho Park of the Vanderbilt-Ingram Cancer Center. "We look forward to pursuing those pivotal studies as we relentlessly look for treatments to benefit patients living with cancer." This clinical trial translated robust in silico and preclinical data to humans, confirming safety of ifetroban in patients with solid tumors and preliminarily suggesting that ifetroban may target biologic mechanisms involved in distant metastatic recurrence. A phenome-wide association study (PheWAS) was conducted by Vanderbilt Health investigators using the BioVU biorepository, which linked a naturally occurring genetic variant in the thromboxane receptor gene (TBXA2R) to an increased risk of metastatic disease across multiple cancer types. Preclinical studies subsequently published in Molecular Cancer Therapeutics demonstrated that ifetroban reduced metastasis in several animal models without affecting tumor growth, and that the drug's effects appeared to involve strengthening of the vascular endothelial barrier and inhibiting the ability of tumor cells to migrate across blood vessel walls. "The favorable safety profile of ifetroban in this patient population, combined with the efficacy signals observed in this study, supports continued investigation of ifetroban as a candidate for metastasis prevention," said A.J. Kazimi, chief executive officer of Cumberland Pharmaceuticals. "The contributions of the Vanderbilt Health team have been essential to advancing this program." Results of this Phase 2a clinical trial will be used to guide the further clinical development verifying efficacy and further demonstrating safety. About Ifetroban Ifetroban is a potent and selective thromboxane-prostanoid receptor (TPr) antagonist. It exhibits high affinity for TPr on many cell types including platelets, cardiomyocytes, vascular and airway smooth muscle, and fibroblasts, and lacks agonistic activity. Cumberland is also evaluating ifetroban in Phase 2 clinical programs for patients with Duchenne Muscular Dystrophy, Systemic Sclerosis and Idiopathic Pulmonary Fibrosis. Ifetroban has a favorable safety profile as evidenced by multiple completed clinical trials collectively enrolling over 1,400 people. About Cumberland Pharmaceuticals Cumberland Pharmaceuticals Inc. is a Nashville-based specialty pharmaceutical company focused on developing new therapies for rare diseases. The company is advancing a clinical pipeline of investigational candidates, including ifetroban and IV guanfacine, across multiple therapeutic areas with significant unmet medical need. Cumberland's Phase 2 clinical programs are evaluating ifetroban in patients with Duchenne Muscular Dystrophy, Systemic Sclerosis, and Idiopathic Pulmonary Fibrosis, in addition to the oncology program described in this release. For more information, please visit . 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. SOURCE Cumberland Pharmaceuticals Inc. 21% more press release views with Request a Demo