NEW YORK, March 16 Intercept Pharmaceuticals Inc said on Sunday its experimental liver disease drug was effective in a third late-stage clinical trial and that the results set the stage for the company to file for marketing approval.
The drug, obeticholic acid (OCA), is designed to treat primary biliary cirrhosis, a disease in which bile ducts in the liver become damaged, allowing harmful substances to build up and scar liver tissue.
Symptoms include dry eyes, fatigue, jaundice, pain in the abdomen, swollen feet and ankles, fatty deposits on the skin around eyes or in the creases of palms, soles, elbows and knees. It is thought to be an autoimmune disorder, in which the body's immune system mistakenly attacks its own cells.
The findings come roughly two months after a clinical trial of the drug in patients with nonalcoholic steatohepatitis, a disease characterized by a buildup of fat in the liver, was halted early because the drug was working better than expected. The news sent the company's shares up 281 percent.
The latest trial, known as POISE, indicates "that OCA clearly produced clinically meaningful improvements," said Professor Frederik Nevens, chairman of the department of hepatology at the University of Leuven in Belgium and the lead investigator on the trial.
Nevens said in a statement that a significant proportion of patients fail to be adequately helped by existing treatments and that new therapies are needed to prevent their disease from progressing to cirrhosis and liver failure.
"I believe that the POISE data indicate OCA will provide a meaningful clinical improvement in these patients," he said.