Cumberland Pharmaceuticals stock tumbles on trial results

Cumberland Pharmaceuticals Inc (NASDAQ:CPIX) shares fell 11% in after-hours trading Friday following the release of top-line results from its Phase 2a ASSIST-SSc trial evaluating oral ifetroban for patients with systemic sclerosis.

The trial, which enrolled 41 patients across two cohorts, showed ifetroban was generally well tolerated but failed to demonstrate clear efficacy signals. The majority of subjects in both treatment groups showed limited disease progression during the study, which reduced the ability of the trial to detect a treatment difference.

Gastrointestinal adverse events were more frequent in the ifetroban group than in the placebo group, though the majority were mild and resolved. A modest increase in bleeding-related adverse events was also observed with ifetroban, consistent with its mechanism as a thromboxane prostanoid receptor antagonist, and these events were predominantly mild.

The 12-month trial enrolled patients with either diffuse cutaneous or pulmonary arterial hypertension manifestations of systemic sclerosis. A total of 31 subjects received ifetroban and 10 received placebo. The trial was not designed or powered to evaluate efficacy, and the sample size and exploratory analytic approach preclude efficacy conclusions, according to the company.

Cumberland plans to submit the clinical study report to the FDA and 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 the company's decision on further development of ifetroban in systemic sclerosis.

Systemic sclerosis is a rare autoimmune disease affecting approximately 100,000 individuals in the U.S. While approved therapies exist for certain pulmonary manifestations, no therapy is currently approved to treat or prevent the primary cardiac involvement of the disease.