Cumberland Pharmaceuticals unveils new program to develop Vasculan for systemic sclerosis

The US Food and Drug Administration (FDA) has cleared Cumberland’s investigational new drug application (IND) for Phase II clinical program for Vasculan in patients with systemic sclerosis.

"Cumberland is committed to developing products that address unmet medical needs," said A.J. Kazimi, Chief Executive Officer of Cumberland Pharmaceuticals.

"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."

Systemic sclerosis (SSc), also called scleroderma, is a rare autoimmune disorder that affects the skin and internal organs. It is characterized by vasculopathy, inflammation, and fibrosis. SSc affects 2.5 million people worldwide and does not yet have a proven curative therapy. This disease has a high morbidity and the highest case-specific mortality of any rheumatic disorder with 50% of patients dying or developing major internal organ complications within 3 years of diagnosis.

Although several medications are used to treat the skin disease associated with SSc, there is no universally effective treatment to improve the function of affected internal organs such as the lungs, heart, and gastrointestinal tract, hence the treatment of SSc remains an unmet need.

"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 of the Department of Cardiovascular Medicine at the Vanderbilt University Medical Center and lead investigator for the trial.

"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."

In 2011, Cumberland announced the acquisition of the ifetroban program in collaboration with Vanderbilt University and Cumberland Emerging Technologies (CET).

Last year, Cumberland initiated the clinical development of Boxaban® (ifetroban) oral capsule with a Phase II study for patients with aspirin-exacerbated respiratory disease (AERD), a respiratory disease affecting nearly a million patients in the U.S. Cumberland is also developing an injectable formulation, Hepatoren (ifetroban) in patients with Hepatorenal Syndrome (HRS), a life-threatening condition involving progressive kidney failure. Cumberland completed initial Phase II studies for both Boxaban and Hepatoren in 2015.

About Ifetroban

Ifetroban is a potent and selective thromboxane-prostanoid receptor (TPr) antagonist. Ifetroban exhibits high-affinity for TPr on platelets, vascular and airway smooth muscle and certain other cell types and lacks agonistic activity. Ifetroban also displays anti-platelet, antivasospastic and antibronchospastic activities and is effective in certain preclinical models of vasospasm, thrombosis, reperfusion injury and endothelial dysfunction, including models that are insensitive to aspirin.