Enanta Announces Results of INTREPID Study of EDP-305 for the Treatment of Primary Biliary Cholangitis

Enanta Pharmaceuticals, Inc. (NASDAQ:ENTA), a clinical-stage biotechnology company dedicated to creating small molecule drugs for viral infections and liver diseases, today announced topline results from its INTREPID Phase 2 study of EDP-305, a Farnesoid X receptor (FXR), in subjects with primary biliary cholangitis (PBC).

The INTREPID study was a 12-week, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability, pharmacokinetics and efficacy of EDP-305 in subjects with PBC, with or without an inadequate response to ursodeoxycholic acid. The primary endpoint of the study was to evaluate the proportion of subjects with at least 20% reduction in alkaline phosphatase (ALP) from pre-treatment value (ALP response), or normalization of ALP, at week 12.

In the intent-to-treat (ITT) analysis, EDP-305 1 mg and 2.5 mg treatment arms resulted in 45% (n=14/31, p=0.106) and 46% (n=13/28, p=0.063) ALP response, respectively, compared to 11% (n=1/9) in the placebo arm. Absolute changes from baseline in ALP at week 12 in both the EDP-305 1 mg arm (p=0.017) and the 2.5 mg arm (p= 0.021) compared to the change from baseline in the placebo arm were statistically significant. In a post-hoc analysis, the proportions of ALP responders among those who completed treatment with no missing value at week 12 were 50% (n=14/28, p=0.039) and 62% (n=13/21, p=0.011), respectively, compared to 11% in placebo (n=1/9).

In the ITT analysis, key secondary endpoints, which included changes from baseline in liver enzymes alanine aminotransferase (ALT), aspartate aminotransferase (AST) and gamma-glutamyl transferase (GGT) at week 12, were met with a statistically significant difference compared to placebo in both the EDP-305 1 mg arm and the 2.5 mg arm. Absolute changes from baseline at week 12 in these key secondary endpoints in the EDP-305 1 mg and 2.5 mg arms compared to placebo were respectively: ALT (p=0.001, p=0.009), AST (p=0.000, p=0.001) and GGT (p=0.000, p=0.000). A statistically significant difference in the percent change from baseline of these key biomarkers at week 12 was also observed in both EDP-305 arms compared to placebo.