ICPT - Intercept Announces New Findings from Long-term Extension of Landmark POISE Trial in PBC Showing Importance of Biomarkers Beyond ALP at AASLD The Liver Meeting® 2023 | Benzinga
New analysis demonstrates the impact of OCA on achievement of GGT <3.2×ULN and ALP <1.5×ULN
Findings suggest that OCA has the potential to reduce the liver biomarker GGT, in addition to well-known effects on ALP, below thresholds that predict improved outcomes, including survival, in PBC
Data to be featured in poster presentation on Monday, November 13
MORRISTOWN, N.J., Nov. 10, 2023 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc., a biopharmaceutical company and wholly-owned subsidiary of Alfasigma S.p.A. focused on the development and commercialization of novel therapeutics to treat rare and serious liver diseases, today announced new data from a sub-analysis of the landmark Phase 3 POISE trial evaluating the impact of obeticholic acid (OCA) on achievement of gamma-glutamyl transferase (GGT) <3.2×upper limit of normal (ULN) and alkaline phosphatase (ALP) <1.5×ULN for the treatment of primary biliary cholangitis (PBC). This analysis suggests OCA's potential to reduce GGT, in addition to the well-known effects on ALP levels, below the biochemical thresholds that are prognostic for worsening clinical outcomes. These data will be presented on Monday, November 13, 2023, at the American Association for the Study of Liver Diseases' (AASLD) The Liver Meeting® in Boston.
"These new data demonstrate the potential of OCA to reduce GGT, in addition to the already established effect on ALP levels, which have been shown to be predictive of risk of liver transplantation or death in patients with PBC," said M. Michelle Berrey, M.D., M.P.H., President of Research & Development and Chief Medical Officer of Intercept. "With the increasing evidence of improved event-free survival for patients treated with OCALIVA across different studies, it was important to improve our understanding of OCALIVA's impact on different biochemical markers and how that correlates with improved outcomes. We are pleased to join clinicians, researchers and industry peers at The Liver Meeting® 2023 to share these data that reaffirm the need for comprehensive assessment of treatment response beyond ALP when managing patients with PBC."
In the study, patients with PBC who were receiving a stable dose of ursodeoxycholic acid (UDCA) or who were unable to tolerate UDCA were randomized to receive placebo, OCA 5-10 mg (OCA titration), or OCA 10 mg daily. For the OCA 5-10 mg group, OCA 5 mg was titrated to 10 mg at 6 months based on tolerability and biochemical response. A 12-month double-blind (DB) period was followed by a 5-year optional open-label extension (OLE), in which all patients were initially treated with OCA 5 mg daily for the first 3 months; every 3 months thereafter, patients had the option to increase the dose up to 10 mg. Serum GGT and ALP levels were assessed at baseline and every 3 months through the 5-year follow-up.
Hepatocyte injury causes GGT to be released into the blood. Elevated GGT in the setting of elevated ALP and other liver enzyme abnormalities is a marker for hepatobiliary disorder. The goal of this sub-analysis was to evaluate the proportion of patients receiving OCA who achieved and sustained GGT <3.2×ULN and ALP <1.5×ULN. Results include:
- In the double-blind (DB) intent-to-treat population (N=203), the proportion of responders was significantly greater at each time point in both OCA cohorts compared with placebo, with the highest responder rates observed in the OCA 10 mg group.
- In the OCA titration group, 17% (11/66) were responders at DB Months 9 and 12. In the OCA 10 mg group, the highest responder rate was observed at DB Month 9 (31% [21/68]), followed by DB Month 12 (26% [18/68]).
- In the open-label extension (OLE) intent-to-treat population (N=119), the proportion of responders generally increased over time, ranging from 18% (22/119) at OLE Month 3 to 38% (35/91) at OLE Month 51. At OLE Month 60, 37% (17/46) were responders.
"A recent study from the Global PBC study group showed that GGT ?3.2xULN and ALP?1.5xULN increase the risk of liver transplantation or death in patients with PBC," said Robert G. Gish, ...