Cumberland Readying a Phase 2 Trial of Oral Drug, Ifetroban, to Treat Scleroderma

Cumberland Pharmaceuticals, Inc., recently announced the addition to its drug pipeline of Vasculan (ifetroban), an oral capsule that soon will enter clinical testing as a treatment of systemic sclerosis (SSc), or scleroderma.

Vasculan is being evaluated for safety and efficacy in a randomized, double-blind and placebo-controlled Phase 2 clinical study in people with diffuse cutaneous systemic SSc or SSc-associated pulmonary arterial hypertension. An Investigational New Drug (IND) application has been cleared by the U.S. Food and Drug Administration (FDA). The trial (NCT02682511) expects to recruit about 34 patients.

“Cumberland is committed to developing products that address unmet medical needs,” A.J. Kazimi, Cumberland’s chief executive officer, said in a press release. “Following the exciting preclinical results demonstrating the potential of ifetroban to impact fibrosis, we are now ready to build on those findings by moving directly to a Phase II patient study.”

Ifetroban is a selective thromboxane-prostanoid receptor (TPr) antagonist that exhibits high affinity for TPs on platelets, vascular and airway smooth muscle, and other cell types, while lacking agonistic activity.  Ifetroban also displays anti-platelet, antivasospastic and antibronchospastic activity, and has shown effectiveness in certain preclinical models of vasospasm, thrombosis, reperfusion injury, and endothelial dysfunction. Cumberland acquired the ifetroban program in 2011, working with Vanderbilt University and Cumberland Emerging Technologies (CET).

“I am excited to investigate ifetroban as a potential therapeutic for scleroderma patients given the significant unmet medical need these patients face,” said Dr. Evan Brittain, the study’s principal investigator with the Department of Cardiovascular Medicine at Vanderbilt University Medical Center. “Cardiac involvement and pulmonary arterial hypertension are major causes of morbidity and mortality in patients with scleroderma.

“In preclinical work, ifetroban prevents cardiac fibrosis in a model of pulmonary arterial hypertension. Evaluating a treatment that may delay or inhibit tissue fibrosis could significantly improve the quality of life for these patients. We are hopeful that ifetroban will prove to be an effective therapeutic for patients suffering from systemic sclerosis or scleroderma with or without pulmonary arterial hypertension,” Dr. Brittain said.

Scleroderma is a rare, autoimmune disorder that affects the skin and internal organs. Generally characterized by vasculopathy, inflammation and fibrosis, it affects nearly 2.5 million people worldwide.