Cumberland Pharmaceuticals recently added Vasculan to its clinical portfolio. The company has began the clinical development of Vasculan (ifetroban) oral capsule for the treatment of systemic sclerosis (SSc), also know as scleroderma, an autoimmune, connective tissue disease characterized by thickening of the skin, caused by accumulation of collagen, and by problems the smallest arteries.

The U.S. Food and Drug Administration (FDA) has cleared the company’s investigational new drug application (IND) for the Phase 2 clinical trial of Vasculan for the treatment of systemic sclerosis.

“Cumberland is committed to developing products that address unmet medical needs,” Cumberland Pharmaceuticals CEO A.J. Kazimi said in a news 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 2 patient study.”

Systemic sclerosis affects 2.5 million people around the world. There are two overlapping forms: limited cutaneous scleroderma, which is limited to the skin on the face, hands, and feet; and diffuse cutaneous scleroderma, which covers more of the skin, and is at risk of progressing to visceral organs, including the heart, lungs, kidneys, and gastrointestinal tract.

The prognosis is difficult to predict until the disease differentiates into recognizable subsets. Patients with limited cutaneous scleroderma have a good prognosis, with a 10-year survival of 75 percent, and fewer than 10 percent develop pulmonary arterial hypertension (PAH) after 10 to 20 years. Patients with diffuse cutaneous scleroderma have a 10-year survival of 55 percent.

Although several medications are used to treat the skin pathology associated with scleroderma, currently there is no effective treatment to improve the function of affected internal organs such as the lungs, heart, and gastrointestinal tract.

“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,” Brittain said.

In 2011, Cumberland acquired ifetroban’s development program in partnership with Cumberland Emerging Technologies (CET) and Vanderbilt University. In 2015, the company began the clinical development of Boxaban (ifetroban) oral capsule with a Phase 2 clinical trial for patients with aspirin-exacerbated respiratory disease.

Cumberland is also focused on the clinical development of an injectable drug called Hepatoren (ifetroban) for the treatment of Hepatorenal Syndrome, a disease that causes progressive kidney failure. In 2015, the company completed preliminary Phase 2 trials for both Boxaban and Hepatoren.