Intercept Pharma Announces Additional Positive Data From POISE Trial

(RTTNews) - Intercept Pharmaceuticals Inc. (ICPT) announced efficacy and safety results from the Phase 3 POISE trial of obeticholic acid or OCA for the treatment of primary biliary cirrhosis or PBC to be presented in a late-breaker session at the International Liver Congress of the European Association for the Study of the Liver or EASL.

OCA, Intercept's lead product candidate, is a bile acid analog and first-in-class agonist of the farnesoid X receptor or FXR in development for PBC, nonalcoholic steatohepatitis or NASH and other liver and intestinal diseases.

As previously reported, the POISE trial met its primary endpoint with high statistical significance (p < 0.0001), demonstrating OCA's efficacy on a composite liver biochemical endpoint which has been shown to strongly correlate with improved clinical outcomes. The proportion of patients meeting the POISE primary endpoint was: 10% in the placebo group, 47% in the 10 mg OCA group and 46% in the 5-10 mg OCA group (both dose groups p < 0.0001 vs placebo).

The company said that New data in today's presentation show that OCA treated patients achieved a highly statistically significant reduction in alkaline phosphatase or ALP as early as two weeks, with a peak effect achieved by six months.

Pruritus, generally mild to moderate, was the most frequently reported adverse event in the POISE trial. However, only one (1%) of the patients in the OCA 5-10 mg titration group discontinued therapy due to pruritus after moving up to the 10 mg dose, as compared to seven (10%) of the patients in the 10 mg OCA group. Additional data presented today show that the incidence and severity of OCA related pruritus diminishes with time on therapy. Specifically, pruritus scores were no different from placebo in both OCA treatment groups during the second half of the trial.

Separately, in a poster presentation at the International Liver Congress, researchers presented a retrospective analysis of two Phase 2 trials of OCA for the treatment of PBC in a total of 224 patients. Pooled results from these trials were evaluated using the Phase 3 POISE trial endpoint.

This analysis showed that overall 43% of patients receiving 10 mg, 25 mg or 50 mg of OCA met the POISE primary endpoint, compared with 8% who received placebo (p < 0.0001), corroborating the Phase 3 results. Moreover, a strong OCA treatment effect was observed regardless of whether OCA was administered as monotherapy or as an add-on to existing ursodiol. OCA treatment was generally well-tolerated, with pruritus, primarily mild or moderate, being the most common adverse event.

Intercept said it plans to submit data from the Phase 3 POISE trial and the two Phase 2 trials of OCA for the treatment of PBC as part of a New Drug Application to the U.S. Food and Drug Administration and a Marketing Authorization Application to the European Medicines Agency. These regulatory filings are currently anticipated at the end of 2014.