Intercept Pharmaceuticals’ Ocaliva failed to treat patients with compensated cirrhosis due to nonalcoholic steatohepatitis better than a placebo in a Phase III trial, the company announced Friday.
Intercept Pharmaceuticals’ obeticholic acid (Ocaliva or OCA) failed to treat patients with compensated cirrhosis due to nonalcoholic steatohepatitis (NASH) better than a placebo in a Phase III trial, the company announced Friday.
Results of the failed trial showed that 11.1% of patients who took 10 mg daily of OCA and 11.9& taking 25 mg daily met the test’s endpoints. This was not considered a statistically significant improvement over the 9.9% of patients who met the endpoints with a placebo.
Generally, a liver transplant is the only treatment for patients with this type of advanced cirrhosis. Doctors and the FDA measure liver fibrosis in 14 stages and the failed trial was testing OCA in Stage 4 patients who have extensive liver scarring.
The FDA has set a high bar for approving a medicine to treat this type of NASH, according to M. Michelle Berrey, M.D., president of research & development and chief medical officer at Intercept.
RBC Capital Market analyst Brian Abrahams said the results were expected.
“(This) does not come as a great surprise, as demonstrating histological benefits in cirrhotic fibrosis stage 4 NASH has been historically challenging,” Abrahams said in a note to clients.
Despite the low expectations, Intercept’s stock plummeted more than 15 percent in heavier-than-usual trading on Friday. Intercept trades on the Nasdaq Composite Market under the symbol ICPT.
Intercept reported it is still on track with the development of OCA for treatment of patients with more serious liver fibrosis due to NASH. The company is focused on refiling a New Drug Application based on re-analyzing results from a Phase III trial the company dubbed REGENERATE, Berrey said.
The FDA rejected the company’s NDA for this indication in 2020. Intercept discovered positive results from REGENERATE when it considered the analysis of three pathologist experts as a consensus panel, the company reported in July. Under the new analysis, 22.4% of patients met the endpoint compared to 9.6% on placebo.
OCA has been approved to treat a rare type of cirrhosis in chronic liver disease patients known as primary biliary cholangitis, or PBC.
