New Data from the Interim Analysis of REGENERATE Show OCA Improved Noninvasive Measures of Fibrosis in a Subgroup of High-Risk Patients with Fibrosis Due to NASH
NEW YORK, Nov. 16, 2020 (GLOBE NEWSWIRE) — 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 multiple new analyses supporting the use of routine noninvasive tests (NITs) to identify patients with advanced fibrosis due to NASH and measure obeticholic acid (OCA) treatment response. The new analyses, which include an oral presentation of REGENERATE interim analysis data showing that OCA helped patients achieve marked improvements in key noninvasive measures of liver fibrosis, are being presented at The Liver Meeting Digital Experience™, the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD), which will be held virtually from November 13, 2020 to November 16, 2020.
“NITs are rapidly replacing liver biopsy for identifying and monitoring patients with advanced fibrosis due to NASH in routine clinical practice, and this year’s Liver Meeting features a wealth of new data reinforcing the value of noninvasive strategies to manage patients treated with OCA,” said Naim Alkhouri, M.D., Chief of Transplant Hepatology, and Director of the Fatty Liver Program at Arizona Liver Health in Phoenix. “Using a simple, sequential algorithm with two common NITs, we were able to identify a higher-risk subgroup of patients with fibrosis due to NASH and evaluate their treatment response noninvasively; these patients achieved marked reductions in measures of liver biochemistry and liver stiffness as assessed by transient elastography through 18 months of treatment.”
As previously reported, once-daily OCA 25 mg met the primary composite endpoint of fibrosis improvement (≥1 stage) with no worsening of NASH at the planned 18-month interim analysis of the Phase 3 REGENERATE study with high statistical significance (p=0.0002 vs. placebo). The new post hoc analysis being presented at The Liver Meeting evaluated the NIT-based efficacy of OCA in patients from the intent-to-treat population of the REGENERATE interim analysis who had Fibrosis-4 (FIB-4) and transient elastography data available at baseline. FIB-4 and transient elastography were applied sequentially to categorize patients’ fibrosis severity; patients with possible advanced fibrosis (indeterminant status) or advanced fibrosis were pooled (OCA 25 mg, n=266; placebo, n=277). At month 18, OCA reduced mean alanine aminotransferase (ALT) scores and median transient elastography scores by 50.1% and 25.6%, respectively; reductions for placebo were 30.2% and 4.2%, respectively, suggesting that such noninvasive assessments can be utilized to monitor fibrosis improvement in OCA-treated patients.
Additional Analyses Support the Role of NITs for Management of Advanced Fibrosis Due to NASH
Multiple additional analyses being presented at the virtual Liver Meeting reinforce the value of noninvasive strategies for managing patients with advanced fibrosis due to NASH:
- In an oral presentation (Abstract 56), an analysis of more than 4,000 patients screened for the REGENERATE study found that application of two sequential NITs improved the accuracy of identification and reduced misclassification of disease as compared to two simultaneous NITs. The authors concluded that sequential NIT strategies may decrease the need for liver biopsy, while maintaining the accuracy of diagnosis in patients with advanced fibrosis due to NASH.
- An analysis (Abstract 1589) comparing FIB-4, liver stiffness measurement by transient elastography, and liver biopsy to predict the incidence of liver-related outcomes (e.g., cirrhosis complications and/or hepatocellular carcinoma) in patients with nonalcoholic fatty liver disease concluded that the predictive accuracy of FIB-4 and transient elastography is similar to that of liver biopsy for predicting liver-related events.
- A review (Abstract 1576) of data from a large U.S. claims database that included approximately 21,500 patients diagnosed with NASH who met the study’s inclusion criteria found that only 11% had a liver biopsy, underscoring the fact that liver biopsy is infrequently performed in the real world clinical practice setting.
“Strong collaboration among patient groups, academic centers and industry, coupled with large datasets from Phase 3 clinical trials have accelerated our ability to identify and validate noninvasive alternatives to biopsy for our patients with fibrosis due to NASH,” said Jerome Boursier, M.D., Ph.D., professor of Medicine, Hepato-Gastroenterology Department of Angers University Hospital, and head of HIFIH laboratory, Angers University in France. “The new NIT data from the interim analysis of the REGENERATE study being presented at the Liver Meeting represent a major step forward. Clearly, the field is coalescing around a sequential testing strategy that combines two commonly used NITs; this approach addresses the major limitations of liver biopsy because it is both scalable and patient-friendly without appearing to sacrifice predictive accuracy. Sequential use of NITs starting with a simple test confirmed by a specialized one will also help to organize and optimize the patient pathway.”
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 …