Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX) and Vanderbilt Health announced results from a Phase 2a clinical trial of ifetroban, an investigational drug designed to prevent cancer metastasis in patients with high-risk solid tumors.

The randomized, double-blind, placebo-controlled study met its primary safety endpoint, demonstrating that ifetroban was safe and well-tolerated in 29 participants with various cancer types including breast, lung, pancreatic, soft tissue, bladder, and renal cancers. Adverse event rates were similar between placebo and ifetroban groups, with no serious adverse events related to study treatment in either group.

While the study was primarily designed to evaluate safety rather than efficacy, secondary endpoint results showed that 17% of ifetroban-treated participants experienced distant metastatic recurrence compared to 50% in the placebo group. Three deaths due to distant metastatic disease occurred in the placebo arm, while none occurred in the ifetroban arm.

The trial evaluated ifetroban, a thromboxane A2 receptor antagonist, in patients with solid tumors at high risk of early metastatic recurrence, defined as having at least a 50% chance of recurrence within five years of diagnosis. Participants received the intervention for 12 months after completing all cancer-related therapies and surgical procedures.

"A therapeutic intervention aimed at metastasis prevention for cancer patients with high risk of recurrence that is given during the period of 'watchful waiting' could be groundbreaking if proven beneficial in larger scale investigations," said Dr. Ben Ho Park of the Vanderbilt-Ingram Cancer Center.

Cumberland Pharmaceuticals is also evaluating ifetroban in Phase 2 clinical programs for patients with Duchenne Muscular Dystrophy, Systemic Sclerosis and Idiopathic Pulmonary Fibrosis. The Nashville-based specialty pharmaceutical company focuses on developing therapies for rare diseases.