A recently approved Ipsen drug has the potential to become the first pharmaceutical alternative to liver transplantation for an autoimmune disease that destroys the bile ducts inside and outside the liver, the company said on Thursday.
The company plans to report the results of its mid-stage trial of Irqirvo in patients with primary sclerosing cholangitis at the European Association for the Study of the Liver meeting in Amsterdam next month.
In a 12-week study, 68 patients with PSC received Irqirvo in daily doses of 80 milligrams or 120 mg, or a placebo.
Patients on Irqirvo had significant improvement in liver enzyme levels, starting as early as four weeks after treatment began, with greater effects seen with the higher dose.
Blood tests also showed liver scarring stabilized to a greater degree in the Irqirvo group, and those on the higher dose of the drug also had significant relief from the itchiness characteristic of the disease.
Rates of adverse events were similar in the three groups.
Irqirvo, already approved for treating a similar condition known as primary biliary cholangitis, is thought to act by stimulating molecules in the body that regulate the processing of fatty acids and by inhibiting bile acid synthesis.
The data are encouraging, study leader Dr. Cynthia Levy of the University of Miami Miller School of Medicine said in a statement.
“PSC is a serious liver disease and currently, liver transplantation is the only treatment that can significantly improve the prognosis,” Dr. Levy said, adding that larger trials are needed to further evaluate the potential of Irqirvo in PSC.