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New Analyses of Data From POISE — the First Phase 3 Trial in PBC in Two Decades — Examine the Efficacy, Safety and Tolerability Profile of OCA
Intercept Pharmaceuticals (Nasdaq:ICPT), a clinical stage biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic liver and intestinal diseases, announced today new analyses of data from clinical trials on obeticholic acid (OCA) in patients with primary biliary cirrhosis (PBC). Six posters, including posters with new analyses of data from POISE – the first Phase 3 trial in PBC in two decades – are being presented at today’s poster session at the American Association for the Study of Liver Disease (AASLD) Annual Meeting.
OCA, Intercept’s lead product candidate, is a bile acid analog and first-in-class agonist of the farnesoid X receptor (FXR) in development for PBC, nonalcoholic steatohepatitis (NASH), primary sclerosing cholangitis (PSC) and other chronic liver diseases.
In March 2014, Intercept announced that OCA met the primary endpoint of the POISE trial with high statistical significance (p < 0.0001). The posters to be presented at AASLD provide the following information relating to OCA in PBC:
– Poster 318 provides new subgroup analyses of the treatment effect for OCA across a range of patient characteristics associated with greater risk of adverse clinical outcomes, including age at diagnosis, duration of PBC and baseline alkaline phosphatase (ALP).
– Poster 295 presents analyses on the effects of OCA treatment versus placebo on markers of cholestasis (ALP, bilirubin) and hepatobiliary damage (GGT, AST, and ALT), as well as markers of inflammation (C-reactive protein or CRP) and apoptosis (cytokeratin-18 or CK-18).
– Poster 309 presents POISE results in relation to the proportion of patients achieving a response defined by different response criteria, other than the POISE endpoint, such as Paris I, Paris II and Rotterdam.
– Posters 305 and 310 cover new information relating to the titration arm of POISE, where approximately half the patients in the titration arm had their OCA dose increased from 5 mg to 10 mg after six months of treatment based on clinical response. These posters present new data regarding the potential utility of titration as a dosing strategy in the context of managing pruritus, the primary tolerability issue of OCA treatment, while investigating the effects of titration on the efficacy of OCA treatment.
– Poster 319 describes the long-term safety and efficacy results from open-label treatment of OCA for over four years as part of the long-term safety extension trial on patients who participated in a Phase 2 PBC trial that evaluated OCA monotherapy.
Linie Moore, President of the PBCers patient advocacy group, commented on the new PBC research being presented at AASLD: "In the time since I was first diagnosed with PBC almost 20 years ago, there have been no new medications introduced to treat my disease, so it is both gratifying and exciting to see so much progress being made with OCA as a potential new PBC therapy. We hope the PBC community's efforts to encourage research and awareness will continue to inspire new advances in the years to come."
The POISE trial studied the safety and efficacy of a once-daily treatment with OCA in PBC patients with an inadequate therapeutic response to, or who are unable to tolerate, ursodiol. The primary endpoint of the 12-month double-blind portion of the trial, completed in March 2014, was the achievement of both an ALP level < 1.67x ULN with a ≥ 15% reduction from baseline and a normal bilirubin level, as compared to placebo. Patients with ALP and bilirubin levels below these thresholds have been shown in long-term clinical studies to have a significantly lower risk of progressing to liver transplant and death. There were 217 patients randomized to one of three groups in the trial: placebo, 10 mg OCA, or 5 mg OCA for six months titrated to 10 mg OCA based on clinical response; 216 patients were dosed.
Patients completing the double-blind phase had the option to continue in an open-label, long-term safety extension (LTSE) phase for another five years, during which all patients receive OCA treatment with daily doses starting at 5 mg and potentially titrating up to 25 mg a day, as clinically indicated. Of the 198 patients who completed the double-blind phase, more than 95% continued in the LTSE phase of the trial.
