Cumberland Pharmaceuticals Announces Breakthrough Results from the Phase 2 FIGHT DMD Trial in Duchenne Muscular Dystrophy Heart Disease
Cumberland Pharmaceuticals (CPIX) announced breakthrough results from its Phase 2 FIGHT DMD trial evaluating ifetroban for Duchenne muscular dystrophy (DMD) heart disease. The 12-month study enrolled 41 DMD patients, testing low dose (150mg) and high dose (300mg) ifetroban against placebo.
The trial showed significant positive outcomes: The high-dose group demonstrated a 3.3% overall improvement in left ventricular ejection fraction (LVEF), with a 1.8% increase compared to a 1.5% decline in the placebo group. When compared to natural history controls, the difference was even more pronounced, showing a 5.4% overall improvement versus a 3.6% decline in controls.
Ifetroban, a once-daily oral thromboxane receptor antagonist, was well-tolerated with no serious drug-related events. The drug has received both Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA. If approved, it would be the first therapy specifically indicated for DMD-related heart disease.
Cumberland Pharmaceuticals (CPIX) ha annunciato risultati innovativi dal suo studio di Fase 2 FIGHT DMD che valuta l'ifetroban per la cardiopatia correlata alla distrofia muscolare di Duchenne (DMD). Lo studio di 12 mesi ha arruolato 41 pazienti con DMD, testando dosi basse (150mg) e alte (300mg) di ifetroban rispetto a un placebo.
La sperimentazione ha mostrato risultati positivi significativi: il gruppo ad alta dose ha dimostrato un 3,3% di miglioramento complessivo nella frazione di eiezione del ventricolo sinistro (LVEF), con un aumento dell'1,8% rispetto a un declino dell'1,5% nel gruppo placebo. Rispetto ai controlli della storia naturale, la differenza era ancora più marcata, mostrando un 5,4% di miglioramento complessivo contro una diminuzione del 3,6% nei controlli.
L'ifetroban, un antagonista orale del recettore del trombossano da assumere una volta al giorno, è stato ben tollerato senza eventi avversi gravi legati al farmaco. Il farmaco ha ricevuto sia la Designazione di Farmaco Orfano che la Designazione di Malattia Pediatrica Rara dalla FDA. Se approvato, sarebbe la prima terapia specificamente indicata per la cardiopatia correlata alla DMD.
Cumberland Pharmaceuticals (CPIX) anunció resultados innovadores de su ensayo de Fase 2 FIGHT DMD que evalúa el ifetroban para la enfermedad cardíaca relacionada con la distrofia muscular de Duchenne (DMD). El estudio de 12 meses incluyó a 41 pacientes con DMD, probando dosis bajas (150mg) y altas (300mg) de ifetroban frente a un placebo.
El ensayo mostró resultados positivos significativos: el grupo de dosis alta demostró una mejora general del 3,3% en la fracción de eyección del ventrículo izquierdo (LVEF), con un aumento del 1,8% en comparación con una disminución del 1,5% en el grupo placebo. En comparación con los controles de la historia natural, la diferencia fue aún más pronunciada, mostrando una mejora general del 5,4% frente a una disminución del 3,6% en los controles.
El ifetroban, un antagonista del receptor de tromboxano oral que se toma una vez al día, fue bien tolerado sin eventos adversos graves relacionados con el medicamento. El fármaco ha recibido tanto la Designación de Medicamento Huérfano como la Designación de Enfermedad Pediátrica Rara de la FDA. Si es aprobado, sería la primera terapia específicamente indicada para la enfermedad cardíaca relacionada con la DMD.
컴벌랜드 제약(CPIX)이 듀셋 근이영양증(DMD) 심장 질환에 대한 이페트로반의 2상 FIGHT DMD 임상 시험에서 혁신적인 결과를 발표했습니다. 12개월간 진행된 연구에는 41명의 DMD 환자가 등록되었으며, 이페트로반 저용량(150mg) 및 고용량(300mg)을 위약과 비교하여 테스트했습니다.
임상 시험은 의미 있는 긍정적인 결과를 보여주었습니다: 고용량 그룹은 좌심실 박출률(LVEF)의 3.3% 전반적인 개선을 보여주었고, 이는 위약 그룹에서 1.5% 감소와 비교하여 1.8% 증가한 수치입니다. 자연적 역사 통제와 비교했을 때, 차이는 더욱 뚜렷하여 5.4%의 전반적인 개선을 보여주었고, 이는 통제군에서 3.6% 감소입니다.
이페트로반은 하루에 한 번 복용하는 구강용 트롬복산 수용체 길항제로서 잘 견뎌졌으며 심각한 약물 관련 사건은 없었습니다. 이 약물은 FDA로부터 고아약 지정 및 희귀 소아 질병 지정을 받았습니다. 승인된다면 DMD 관련 심장 질환에 대해 특별히 지시된 최초의 치료제가 될 것입니다.
Cumberland Pharmaceuticals (CPIX) a annoncé des résultats révolutionnaires issus de son essai de Phase 2 FIGHT DMD évaluant l'ifetroban pour la cardiopathie liée à la dystrophie musculaire de Duchenne (DMD). L'étude de 12 mois a recruté 41 patients atteints de DMD, testant des doses faibles (150 mg) et élevées (300 mg) d'ifetroban par rapport à un placebo.
L'essai a montré des résultats positifs significatifs : le groupe à dose élevée a présenté une amélioration globale de 3,3 % de la fraction d'éjection du ventricule gauche (LVEF), avec une augmentation de 1,8 % par rapport à une diminution de 1,5 % dans le groupe placebo. Comparé aux contrôles de l'histoire naturelle, la différence était encore plus marquée, montrant une amélioration globale de 5,4 % contre une diminution de 3,6 % chez les contrôles.
L'ifetroban, un antagoniste oral du récepteur de thromboxane à prendre une fois par jour, a été bien toléré sans événements indésirables graves liés au médicament. Ce médicament a reçu à la fois la désignation de médicament orphelin et la désignation de maladie pédiatrique rare de la FDA. En cas d'approbation, ce serait la première thérapie spécifiquement indiquée pour les maladies cardiaques liées à la DMD.
Cumberland Pharmaceuticals (CPIX) hat bahnbrechende Ergebnisse aus seiner Phase-2-Studie FIGHT DMD zur Evaluierung von Ifetroban bei Duchenne-Muskeldystrophie (DMD) Herzkrankheit bekannt gegeben. Die 12-monatige Studie rekrutierte 41 DMD-Patienten und testete niedrige Dosen (150 mg) und hohe Dosen (300 mg) von Ifetroban gegen ein Placebo.
Die Studie zeigte signifikante positive Ergebnisse: Die Hochdosisgruppe zeigte eine 3,3%ige Gesamterhöhung der linksventrikulären Ejektionsfraktion (LVEF) mit einem Anstieg um 1,8% im Vergleich zu einem Rückgang von 1,5% in der Placebogruppe. Im Vergleich zu natürlichen Kontrollgruppen war der Unterschied noch ausgeprägter, wobei eine 5,4%ige Gesamtverbesserung gegenüber einem Rückgang von 3,6% in den Kontrollen festgestellt wurde.
Ifetroban, ein oral einzunehmender Thromboxan-Rezeptor-Antagonist, wurde gut vertragen, ohne schwerwiegende medikamentenbezogene Zwischenfälle. Das Medikament erhielt sowohl die Orphan Drug Designation als auch die Rare Pediatric Disease Designation von der FDA. Im Falle einer Genehmigung wäre es die erste Therapie, die speziell für DMD-bezogene Herzkrankheiten angezeigt ist.
- First successful Phase 2 study targeting DMD heart disease
- High-dose ifetroban showed 3.3% improvement in LVEF vs placebo
- 5.4% LVEF improvement compared to natural history controls
- No serious drug-related adverse events reported
- Received both Orphan Drug and Rare Pediatric Disease Designations
- None.
Insights
The Phase 2 FIGHT DMD trial results represent a significant milestone in the treatment of Duchenne muscular dystrophy (DMD) cardiac complications. The 3.3% improvement in left ventricular ejection fraction (LVEF) with high-dose ifetroban is particularly noteworthy, as DMD patients typically experience progressive cardiac decline. The 5.4% difference compared to natural history controls further validates the drug's potential efficacy.
From a market perspective, this breakthrough positions Cumberland strategically in the lucrative rare disease space. DMD affects approximately 1 in 3,500 male births worldwide, with cardiac complications being the primary cause of mortality. The lack of approved therapies for DMD-related heart disease creates a substantial market opportunity, potentially commanding premium pricing typical of orphan drugs.
The dual regulatory designations (Orphan Drug and Rare Pediatric Disease) provide significant advantages, including:
- 7 years of market exclusivity upon approval
- Tax credits for clinical trial costs
- Potential priority review voucher
- Reduced FDA fees
Cumberland's growing patent portfolio for ifetroban in DMD strengthens their market position and potential for commercialization. The company's focus on rare diseases, combined with their established commercial infrastructure for other approved products, positions them well for the eventual launch.
Looking ahead, key considerations include the Phase 3 trial design, which will likely require larger patient populations and longer duration to confirm these promising results. The clear efficacy signal and safety profile from Phase 2 suggest a higher probability of success in Phase 3, though timeline to market is likely 2-3 years at minimum, assuming positive results and standard regulatory review times.
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.
A previous study conducted at Vanderbilt University Medical Center demonstrated that ifetroban is protective against cardiomyopathy in several preclinical models of muscular dystrophy. The results of that study were published in the Journal of the American Heart Association (West 2019). Based on those promising results, Cumberland Pharmaceuticals became the first recipient of an FDA Office of Orphan Products Development clinical trial grant for DMD, funding the development of this Phase 2 clinical trial.
"These results represent a significant milestone in DMD cardiomyopathy," said Larry W. Markham, MD, Professor of Pediatrics and Medicine, Indiana University School of Medicine, Division Chief of Pediatric Cardiology at Riley Children's Hospital and Principal Investigator of the FIGHT DMD trial. "We are seeing evidence that there is an opportunity to potentially alter the course of heart disease in DMD patients. The improvement in cardiac function observed with ifetroban, particularly in the high-dose group, offers hope for these patients and their families."
The FIGHT DMD trial (NCT03340675), is a 12-month, double-blind, randomized, placebo-controlled study evaluating ifetroban, an oral thromboxane receptor antagonist. The trial enrolled 41 DMD patients who received either low dose ifetroban (150 mg per day), high dose ifetroban (300 mg per day), or placebo. The study's primary endpoint was an improvement in the heart's left ventricular ejection fractions (LVEF).
Key findings include:
- High dose ifetroban treatment resulted in an overall
3.3% improvement in LVEF. - The high dose ifetroban group showed an increase in
1.8% in LVEF, while the study placebo group showed an expected decline in LVEF of1.5% - When compared with propensity matched natural history controls, the difference was even more pronounced, with the high dose treatment providing a significant
5.4% overall improvement in LVEF, as the control patients experienced a3.6% decline in LVEF - Both doses of ifetroban were well-tolerated, with no serious drug-related events.
"The cardiac imaging data from this trial is compelling," noted Jonathan Soslow, MD, Professor of Pediatrics, Vanderbilt University, pediatric cardiologist and cardiac imaging expert. "The preservation and even improvement in cardiac function seen with ifetroban treatment stands in stark contrast to the expected decline we typically observe in untreated DMD patients."
"This trial represents hope for our Duchenne community," said Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy. "Heart disease remains one of the most devastating aspects of Duchenne, and these results suggest we may finally have a therapeutic option that could make a meaningful difference in the lives of patients and families."
"These impressive results represent a pivotal moment for Cumberland Pharmaceuticals and, more importantly, for the DMD community," said A.J. Kazimi, Chief Executive Officer of Cumberland Pharmaceuticals. "As the first company to receive FDA Orphan Products Development funding for a DMD clinical trial, we're honored to be advancing a potential breakthrough therapy for DMD-related heart disease. These results validate our commitment to developing innovative treatments for rare diseases and underscore the importance of collaborative partnerships between industry, academia, and regulatory agencies in addressing critical unmet medical needs."
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 and Rare Pediatric Disease Designation from the FDA, highlighting its potential significance in treating this devastating condition. If approved, ifetroban would be the first therapy specifically indicated for DMD-related heart disease.
Cumberland has secured a growing portfolio of patents with claims associated with the product for this DMD indication. Next steps include further data analysis and completion of a full study report in preparation for an end of Phase 2 meeting with the FDA to determine next steps associated with the product's development and commercialization.
More information regarding the FIGHT DMD trial can be found here: www.fightdmdtrial.com
References:
Soslow JH, Xu M, Slaughter JC, et al. Cardiovascular Measures of All-Cause Mortality in Duchenne Muscular Dystrophy. Circ Heart Fail. 2023 Aug;16(8):e010040. doi: 10.1161/CIRCHEARTFAILURE.122.010040. Epub 2023 Jun 8. PMID: 37288563; PMCID: PMC10524475.
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(21):e011902. doi: 10.1161/JAHA.118.011902.
About Cumberland Pharmaceuticals
Cumberland Pharmaceuticals Inc. is the largest biopharmaceutical company founded and headquartered in
The company's portfolio of FDA-approved brands includes:
- 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.
In addition to this Duchenne muscular dystrophy program, the company also has Phase 2 clinical studies underway evaluating its ifetroban product candidate in patients with 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
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FAQ
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