Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX) announced top-line results from its Phase 2a ASSIST-SSc trial evaluating oral ifetroban in patients with systemic sclerosis (SSc), a rare autoimmune disease affecting approximately 100,000 people in the U.S.

The 12-month pilot trial enrolled 41 subjects, with 31 receiving ifetroban and 10 receiving a placebo. Patients had either diffuse cutaneous SSc or SSc-associated pulmonary arterial hypertension. The trial's primary objective was to evaluate safety, not efficacy.

Ifetroban was generally well tolerated. Gastrointestinal adverse events occurred more frequently in the ifetroban group than in the placebo group, though most were mild and resolved. A modest increase in bleeding-related adverse events was also observed, consistent with the drug's mechanism as a thromboxane prostanoid receptor antagonist. No serious adverse event during the 12-month treatment period was assessed as related to the study drug.

The majority of subjects in both groups showed limited disease progression during the study, which the company said reduced the trial's ability to detect a treatment difference. Cumberland noted the trial was not designed or powered to evaluate efficacy, and that the sample size and exploratory analytic approach preclude efficacy conclusions.

As next steps, Cumberland plans to submit the clinical study report to the U.S. Food and Drug Administration and complete correlative biomarker studies using specimens collected during the trial. The company will also evaluate cardiac imaging from patients continuing on the open-label extension. Those findings will inform Cumberland's decision on further development of ifetroban in SSc.

Cumberland is also evaluating ifetroban in separate Phase 2 trials for Duchenne Muscular Dystrophy, Idiopathic Pulmonary Fibrosis, and Cancer Metastasis.