News & Events zu Cumberland Pharmaceuticals Inc (CPIX)CPIXCumberland Pharmaceuticals Inc

Cumberland Submits NDA for Ifetroban to Treat DMD-Associated Cardiomyopathy

Cumberland provided an update on its ifetroban product candidate in development as a potential treatment for the cardiomyopathy associated with Duchenne muscular dystrophy, or DMD. The company plans to submit a New Drug Application, or NDA, to the FDA for ifetroban as a potential treatment for the cardiomyopathy associated with DMD. Cumberland requested a pre-NDA meeting with FDA, which was held this fall. Following the meeting FDA granted the company's request for a rolling review of the NDA, which allows completed sections of the application to be provided to the FDA for review, prior to the submission of the entire application. FDA suggested a Phase 3 trial and raised several issues regarding the Phase 2 study results. In response, the company provided additional data supporting its efficacy findings, natural history data from a second DMD registry, and documentation of how the statistical analysis plan was developed. The planned submission for Traditional Approval will include data from the company's FIGHT DMD clinical study and its ongoing open-label extension, data from two natural history databases of DMD patients, preclinical data published in the Journal of the American Heart Association, manufacturing information and a safety database that includes more than 1,400 subjects across approximately 30 clinical studies. Ifetroban is proposed as 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 FDA for the indication of cardiomyopathy associated with DMD.

Cumberland Announces Phase 2a Results for Oral Ifetroban

Cumberland announced top-line results from its Phase 2a ASSIST-SSc trial evaluating oral ifetroban for patients with systemic sclerosis, or SSc. These results support the safety and tolerability of ifetroban in this rare disease population. Cumberland will next complete correlative biomarker studies using specimens collected during the trial, along with an evaluation of cardiac imaging from patients continuing on the open-label extension. SSc is a rare autoimmune disease characterized by excess collagen deposition resulting in skin thickening, cardiovascular disease, gastrointestinal problems, and more, and affects approximately 100,000 individuals in the U.S. Current therapies target specific manifestations of SSc, and approved options are available for SSc-associated interstitial lung disease. However, there is no approved therapy directed at the primary cardiac issues associated with SSc. Both cardiac and pulmonary manifestations remain among the leading causes of mortality for patients with the disease. Ifetroban is a once-daily oral medication that works by blocking the thromboxane receptor, which plays a key role in inflammation, vascular dysfunction and fibrosis. In preclinical models of pulmonary arterial hypertension and heart disease, ifetroban treatment was associated with a reduction in fibrosis and provided cardioprotective effects. A delay or inhibition of tissue fibrosis and improved cardiac function could provide significant improvement in quality of life for SSc patients. These preclinical results were foundational to the development of this Phase 2a clinical trial. The 12-month Phase 2a ASSIST-SSc trial was a pilot trial to evaluate the safety of oral ifetroban in patients with SSc. Patients with either diffuse cutaneous or pulmonary arterial hypertension manifestations were randomized to receive ifetroban or matching placebo. A total of 31 subjects received ifetroban and 10 subjects received placebo. Safety was evaluated by the frequency of treatment emergent adverse events between treatment groups. Exploratory efficacy endpoints were evaluated by SSc cohort to assess the effect of ifetroban on multiple aspects of SSc disease, including cardiovascular, pulmonary, skin, activity and quality of life. Ifetroban was generally well tolerated, with a safety profile consistent with prior experience. As in prior ifetroban studies, gastrointestinal adverse events were more frequent in the ifetroban group than in the placebo group; the majority were mild and resolved. A modest increase in bleeding-related adverse events was also observed with ifetroban, in keeping with its mechanism as a thromboxane prostanoid receptor antagonist, and these events were predominantly mild. No serious adverse event during the 12-month treatment period was assessed by the medical monitor as related to study drug. The majority of subjects in both treatment groups showed limited disease progression during the study, which reduced the ability of a trial of this size to detect a treatment difference. This Phase 2a trial was not designed or powered to evaluate efficacy, and the sample size and exploratory analytic approach preclude efficacy conclusions. Cumberland has reviewed the results from both the dcSSc and SSc-PAH cohorts with clinical experts in SSc. As next steps, the Company plans to submit the clinical study report to the FDA and to also complete correlative biomarker studies using specimens collected during the trial, along with an evaluation of cardiac imaging from patients continuing on the open-label extension. Those findings will inform Cumberland's decision on the further development of ifetroban in SSc.

Cumberland Authorizes Up to $5M Share Repurchase Program

In addition to the special dividend, the Board authorized an open-market share repurchase program of up to $5M in Cumberland shares over time. Several Board members will also be establishing new share purchase plans to increase their holdings in the Company.