REGENERATE, a pivotal phase 3 study of obeticholic acid (OCA) by Intercept Pharmaceuticals, Inc., has demonstrated positive results in patients with liver fibrosis from nonalcoholic steatohepatitis (NASH).
The primary efficacy analysis indicated that treatment with once-daily OCA 25 mg met the primary endpoint of fibrosis improvement (≥ 1 stage) with no worsening of NASH after 18 months.
NASH is set to become a leading cause of liver transplants by 2020 and there are no approved medications for treatment. REGENERATE’s topline data indicates that OCA could become the first approved medicine for patients with liver fibrosis resulting from NASH.
Patients who have significant fibrosis as a result of NASH are at the greatest risk of severe liver-related complications, and fibrosis is the strongest predictor of liver-related mortality in such patients. To date, OCA is the only investigational drug to receive an FDA breakthrough therapy designation for NASH with fibrosis.
The most common adverse event reported was dose-related pruritus (19% in placebo, 28% in OCA 10 mg and 51% in OCA 25 mg), with the majority of pruritus events being mild to moderate. Treatment with OCA was also associated with increased LDL cholesterol, with a peak increase of 22.6 mg/dL at 4 weeks, which then reversed and approached baseline at month 18.
REGENERATE will continue through clinical outcomes to confirm clinical benefit. The end-of-study analysis will evaluate the effect of OCA on mortality and liver-related clinical outcomes, and its long-term safety.
Source: Intercept Pharmaceuticals, Inc., February 19, 2019