Intercept Pharmaceuticals Announces Pivotal Phase 3 Clinical Trial of Obeticholic Acid in NASH

21-May-2015 - USA

Intercept Pharmaceuticals, Inc. announced its plans for an international Phase 3 trial of obeticholic acid (OCA), the company's lead FXR agonist, in patients with non-cirrhotic nonalcoholic steatohepatitis (NASH) with liver fibrosis. OCA has received breakthrough therapy designation in this patient population from the U.S. Food and Drug Administration (FDA). In accordance with harmonized advice from the FDA and European Medicines Authority (EMA), the Randomized Global Phase 3 Trial to Evaluate the Impact on NASH with Fibrosis of Obeticholic Acid Treatment (REGENERATE) has been designed as a double-blind, placebo-controlled pivotal Phase 3 clinical trial expected to enroll up to approximately 2,500 patients and assess the potential benefit of OCA treatment on liver-related clinical outcomes. The trial will include a pre-planned interim histology analysis after 72 weeks of treatment in approximately 1,400 patients which is intended to serve as the basis for seeking U.S. and international marketing approvals of OCA for the treatment of NASH patients with liver fibrosis. Intercept will hold a conference call and audio webcast today at 8:00 a.m. ET to discuss aspects of the planned Phase 3 trial. Conference call details are provided below.

The REGENERATE trial will be conducted at approximately 250 centers in North America, Europe and other regions and trial initiation is anticipated in 3Q 2015. Patients will be randomized 1:1:1 to one of placebo, 10 mg of OCA, or 25 mg of OCA, taken once daily. The study population will primarily be comprised of NASH patients with stage 2 or stage 3 advanced liver fibrosis who will be evaluated on the primary efficacy endpoints in the 72-week interim analysis and subsequently. In addition, a relatively small group of NASH patients with stage 1 early liver fibrosis with an increased risk of rapid progression due to concomitant diabetes, obesity or active liver inflammation indicated by elevated ALT will also be enrolled, but not included in the primary endpoint analyses.

The interim analysis after 72 weeks of treatment in approximately 1,400 patients will be conducted on the following co-primary endpoints, which are intended to serve as the basis for seeking U.S. and international marketing approvals: (i) the proportion of OCA-treated patients relative to placebo achieving at least one stage of liver fibrosis improvement with no worsening NASH; and (ii) the proportion of OCA-treated patients relative to placebo achieving NASH resolution with no worsening of liver fibrosis. Additional supportive histologic endpoints and non-invasive markers of liver fibrosis and steatohepatitis will also be evaluated. The REGENERATE trial will remain blinded after the interim analysis and continue to follow patients until the occurrence of a pre-specified number of adverse liver-related clinical events, including progression to cirrhosis, to confirm clinical benefit on a post-marketing basis.

https://www.bionity.com/en/news/152932/intercept-pharmaceuticals-announces-pivotal-phase-3-clinical-trial-of-obeticholic-acid-in-nash.html