Intercept (ICPT) Announces New Data from Interim Analysis of REGENERATE Show OCA Helped Patients with Liver Fibrosis Due to NASH Achieve Sustained Improvement

Intercept Pharmaceuticals, Inc. (Nasdaq: ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, today announced additional data indicating that obeticholic acid (OCA) helped patients with nonalcoholic steatohepatitis (NASH) achieve sustained improvements in liver biochemistry and noninvasive markers of liver fibrosis over two years of treatment. The new results based upon a post hoc review of the interim analysis data from the Phase 3 REGENERATE study are being presented at the virtual International Liver Congress™ 2020, the 55th Annual Meeting of the European Association for the Study of the Liver (EASL).

“The primary goal of a medicine to treat patients with advanced fibrosis due to NASH is to halt or reverse the progression to cirrhosis and its devastating complications,” said Rohit Loomba, M.D., director, U.C. San Diego NAFLD Research Center and director of hepatology at U.C. San Diego School of Medicine. “These new noninvasive data from REGENERATE provide further evidence that OCA can help patients achieve this goal. It is encouraging to see a consistent and sustained effect across multiple noninvasive tests that clinicians use in practice every day to monitor and manage their patients. The marked improvement in measurements of liver stiffness observed with OCA therapy was particularly notable. Additionally, these data give us greater confidence that OCA continues to provide meaningful and durable benefit beyond the histologic benefit already established at 18 months.”

As previously reported, once-daily OCA 25 mg met the primary endpoint of fibrosis improvement (≥1 stage) with no worsening of NASH at the planned 18-month interim analysis of REGENERATE with high statistical significance (p=0.0002 vs. placebo). The new analysis included patients from the interim analysis intent-to-treat (ITT) population randomized early enough to have both evaluable Month 18 biopsies (N = 251–263 per treatment arm) and Month 24 data at the time of the interim analysis (N = 120–125 per arm). Changes from baseline to Month 24 in alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma-glutamyl transferase (GGT), serum markers of fibrosis (FIB-4, AST to platelet ratio index [APRI]), and liver stiffness (FibroScan® vibration-controlled transient elastography [VCTE]; subset, N = 64‒70 per arm) were analyzed.

Mean values of transaminases and other serum-based tests improved rapidly in patients treated with OCA and were sustained beyond 18 months of therapy compared with placebo. FibroScan VCTE also demonstrated improvement in liver stiffness in patients treated with OCA versus placebo after 24 months of therapy, with a mean difference of 2.7 kPa between OCA 25 mg and placebo. At baseline, median liver stiffness values were in the advanced fibrosis range; at 24 months, median values of patients treated with OCA 25 mg were below the threshold of 7.9 kPa, and most patients treated with OCA 25 mg had moved from advanced to moderate fibrosis.

Changes in transaminases and noninvasive markers of fibrosis were associated with changes in histologic fibrosis, with the greatest improvements observed in patients who had a ≥1 stage improvement in fibrosis stage at 18 months. Moreover, early changes in these markers were more pronounced in patients who had histologic fibrosis improvement at Month 18. Overall, these noninvasive data suggest that longer treatment duration with OCA will likely result in greater fibrosis reduction beyond 18 months.

The overall adverse event profile of OCA 25 mg in the subgroup of the ITT population with 24 months of follow-up at the time of the interim analysis was generally consistent with that observed in the overall ITT population. The most common adverse event was pruritus. The incidence of serious adverse events was balanced across the placebo and OCA 25 mg groups. Few serious adverse events occurred in more than one patient and no consistent pattern of serious adverse events was observed.

About Liver Fibrosis due to NASH

Nonalcoholic steatohepatitis (NASH) is a serious progressive liver disease caused by excessive fat accumulation in the liver that induces chronic inflammation, resulting in progressive fibrosis (scarring) that can lead to cirrhosis, eventual liver failure, cancer and death. Advanced fibrosis is associated with a substantially higher risk of liver-related morbidity and mortality in patients with NASH. In the United States, NASH is currently the second leading cause for liver transplantation overall, and in females, the leading cause. NASH is anticipated to become the leading indication for liver transplantation in Europe within the next decade. There are currently no medications approved for the treatment of NASH.

About the REGENERATE Study

REGENERATE is a Phase 3, randomized, double-blind, placebo-controlled, multicenter study assessing the safety and efficacy of obeticholic acid (OCA) on clinical outcomes in patients with liver fibrosis due to NASH. A pre-specified 18-month analysis was conducted to assess the effect of OCA on liver histology comparing month 18 biopsies with baseline. REGENERATE has completed target enrollment for the clinical outcomes cohort, with 2,480 adult NASH patients randomized at over 300 qualified centers worldwide, and is expected to continue through clinical outcomes for verification and description of clinical benefit. The end-of-study analysis is designed to evaluate the effect of OCA on all-cause mortality and liver-related clinical outcomes, as well as long-term safety.

You May Also Be Interested In

• TG Therapeutics (TGTX) Announces Additional Data Presentations for BRIUMVI in Multiple Sclerosis
• Inhibikase Therapeutics (IKT) Issues Letter to Shareholders and Provides Update on Development Programs
• Vertex Pharma (VRTX) Announces Advancements of Suzetrigine (VX-548) in Acute and Neuropathic Pain