Intercept Pharmaceuticals, Inc. (NASDAQ: ICPT) reported its clinical trial results of obeticholic acid in patients with liver fibrosis due to nonalcoholic steatohepatitis (NASH). The Company reported top of the line results, which sent its shares surging by 14% shortly after the opening bell on Tuesday.
NASH is a progressive liver disease caused by excessive fat in the liver. Risk factors include obesity, gastric bypass surgery, high cholesterol, and type 2 diabetes. Over time, patients can incur inflammation and scarring of the liver.
Intercept reported that its once a day OCA 25 mg met the primary endpoint of fibrosis improvement with no worsening of NASH at the planned 18-month interim analysis. In the primary efficacy analysis, a greater number of patients in both OCA treatment compared to placebo achieved the primary endpoint of NASH resolution.
Intercept’s trial showed significant improvement, as 23.1% of patients showed fibrosis improvement compared to 11.9% on placebo. However, the results did not reach statistical significance. As agreed with the U.S. Food and Drug Administration, Intercept had to meet at least two primary endpoints for its trial.
Despite the secondary results, OCA remains the only investigational drug to have received Breakthrough Therapy designation from the FDA.
“We are thrilled to report the first positive registrational Phase 3 study results in patients with NASH, a devastating disease that is on track to become a leading cause of liver transplant in coming years,” said Mark Pruzanski, M.D., President and Chief Executive Officer of Intercept.
“The topline REGENERATE data we are reporting today support our belief that OCA will become the first approved medicine for those living with liver fibrosis due to NASH. We are deeply grateful to the patients, investigators and study staff whose ongoing participation in REGENERATE has brought us one step closer to delivering a much-needed therapeutic option to address the enormous unmet medical need in this population.” concluded Pruzanski.
Based on the results, Intercept intends to file for approval in the U.S. and Europe in the second half of 2019.