Cumberland Pharmaceuticals Shares Phase 2 FIGHT DMD Trial Results at the Parent Project Muscular Dystrophy Annual Conference
- Significant 5.4% improvement in left ventricular ejection fraction compared to control group
- Reduced blood levels of cardiac damage markers while placebo group showed increases
- All patients who completed the 12-month study chose to continue with open-label extension
- Drug received both Orphan Drug and Rare Pediatric Disease Designations from FDA
- Well-tolerated safety profile with acceptable pharmacokinetics
- Higher dosing requirements needed compared to typical adult doses
- Further clinical trials and FDA discussions needed before potential approval
- No immediate path to commercialization yet determined
Insights
Cumberland's Phase 2 DMD heart drug shows promising cardiac benefits with significant LVEF improvement and reduced biomarkers of heart damage.
The Phase 2 FIGHT DMD trial for ifetroban demonstrates compelling evidence of cardioprotective effects in Duchenne muscular dystrophy patients. The high-dose treatment group showed a
What's particularly significant is the dual evidence of efficacy: functional improvement in heart performance coupled with biochemical evidence of reduced cardiac damage. The reduction in cardiac biomarkers (NT-proBNP and troponin I) in the treatment group, while these markers increased in placebo patients, strongly suggests ifetroban is preventing ongoing heart injury - exactly what you want to see in a cardioprotective therapy.
The pharmacokinetic data addresses an important clinical question: DMD patients required higher doses than typical adults to achieve similar plasma levels, with no evidence of drug accumulation. This supports the 300mg daily dosing used in the high-dose group and demonstrates an acceptable safety profile despite the higher dosing requirements.
That all patients opted to continue in the open-label extension speaks volumes about the treatment's tolerability and perceived benefit. With heart disease being the leading cause of death in DMD and no currently approved treatments specifically for DMD-related cardiac complications, ifetroban addresses a critical unmet need with a mechanism targeting inflammation and fibrosis through thromboxane receptor blockade.
The dual regulatory designations (Orphan Drug and Rare Pediatric Disease) provide significant development advantages and market protections if approved. Cumberland's growing patent portfolio further strengthens their commercial position for this indication.
New pharmacokinetic and cardiac biomarker data further demonstrate ifetroban's potential to protect the heart and reduce cardiac damage in DMD patients
The trial results were presented this afternoon by FIGHT DMD Trial Principal Investigator Larry W. Markham, MD, Professor of Pediatrics and Medicine at Riley Children's Hospital and the Indiana University School of Medicine, as part of the "Therapies that Slow Progression" conference session. "PPMD has had a focus on heart disease and has been a part of this trial since its inception. We are proud to share the latest promising findings from the FIGHT DMD Trial with a group that has consistently supported us throughout our efforts to develop an effective treatment for DMD heart disease," said Dr. Markham. The complete slide presentation is now available on Cumberland's website.
"Seeing these promising results validates our belief that targeted heart therapies can make a meaningful difference for our children," said Terry Marlin, President and Founder of FIGHTDMD, a community-based organization with the mission of advancing DMD research. "This trial represents what's possible when families refuse to accept the status quo and invest in advancing medical research."
FIGHTDMD helped co-fund the preclinical studies at Vanderbilt's Monroe Carell Jr. Children's Hospital which formed the foundation for the FDA grant funding this trial. The trial was named The FIGHT DMD Trial in honor of their organization's early support. The Marlin family also provided input into the clinical trial design and contributed personal photos for the trial's patient brochure.
The 12-month Phase 2 FIGHT DMD trial (NCT03340675) demonstrated that high-dose ifetroban treatment resulted in a significant
Critically, high-dose ifetroban treatment was associated with reduced blood levels of cardiac damage markers (NT-proBNP and cardiac troponin I), while these markers of heart damage increased in placebo-treated patients, suggesting ifetroban could help prevent ongoing cardiac injury. This biochemical evidence of reduced cardiac damage, combined with functional heart improvement, provides compelling evidence of ifetroban's cardioprotective effects.
The study also revealed pharmacokinetic insights, showing that DMD patients receiving higher doses than typical adults achieved similar plasma levels, with no evidence of drug accumulation, supporting the 300 mg daily dosing used in the high-dose group. Despite the higher dosing requirements, ifetroban was well-tolerated with an acceptable pharmacokinetic profile in patients with DMD.
The observed improvement in cardiac function together with the reduction in cardiac damage biomarkers suggest a clinically significant impact for ifetroban in both heart disease and potential to impact overall survival in DMD. Demonstrating confidence in the treatment, all patients who completed the 12-month study opted to continue with the open-label extension.
"These new results reinforce our conviction that ifetroban has the potential to address the leading cause of death in DMD patients," said A.J. Kazimi, Cumberland CEO. "The improvement in heart function observed, combined with the reduction in cardiac damage markers, suggests we may be able to meaningfully impact the progressive heart disease that affects virtually all DMD patients. What's particularly encouraging is that there is both functional improvement and biochemical evidence of reduced cardiac injury - exactly what you'd want to see in a therapy designed to protect the heart. We're deeply grateful to both Terry Marlin and FIGHT DMD for their early vision and funding and to PPMD for their longstanding support and partnership throughout this journey – as both organizations have been instrumental in making this breakthrough possible. We're optimistic about taking the next steps toward bringing this much-needed treatment to DMD patients and their families."
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 both Orphan Drug Designation Rare Pediatric Disease Designation, from the FDA for the indication of DMD heart disease. 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 analysis of long-term treatment results and discussions with the FDA to determine the regulatory pathway forward based on these encouraging results.
More information regarding The FIGHT DMD Trial can be found here: 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
- Acetadote® (acetylcysteine) injection, for the treatment of acetaminophen poisoning;
- Caldolor® (ibuprofen) injection, for the treatment of pain and fever;
- Kristalose® (lactulose) oral, a prescription laxative, for the treatment of constipation;
- Sancuso® (granisetron) transdermal, for the prevention of nausea and vomiting in patients receiving certain types of chemotherapy treatment;
- Vaprisol® (conivaptan) injection, to raise serum sodium levels in hospitalized patients with euvolemic and hypervolemic hyponatremia; and
- Vibativ® (telavancin) injection, for the treatment of certain serious bacterial infections including hospital-acquired and ventilator-associated bacterial pneumonia, as well as complicated skin and skin structure infections.
The company also has a series of Phase II clinical programs underway evaluating its ifetroban product candidate in patients with cardiomyopathy associated with Duchenne Muscular Dystrophy, Systemic Sclerosis and Idiopathic Pulmonary Fibrosis.
For more information on Cumberland's approved products, including full prescribing information, please visit the individual product websites, which can be found on the company's website 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," "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 macroeconomic conditions, including rising interest rates and inflation, 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
View original content to download multimedia:https://www.prnewswire.com/news-releases/cumberland-pharmaceuticals-shares-phase-2-fight-dmd-trial-results-at-the-parent-project-muscular-dystrophy-annual-conference-302487504.html
SOURCE Cumberland Pharmaceuticals Inc.
FAQ
What were the key results of Cumberland Pharmaceuticals' Phase 2 FIGHT DMD trial for ifetroban?
How does Cumberland's ifetroban work in treating DMD heart disease?
What FDA designations has CPIX's ifetroban received for DMD treatment?
What is the current treatment landscape for DMD heart disease?
What are Cumberland Pharmaceuticals' next steps for ifetroban in DMD treatment?
