Phase 2 Study of Obeticholic Acid (INT-747) for Primary Biliary Cirrhosis

Intercept Pharmaceuticals announced positive results from its Phase 2 study of obeticholic acid (OCA or INT-747) as monotherapy for the treatment of primary biliary cirrhosis (PBC). In this placebo-controlled, double-blind clinical trial, 59 patients with elevated alkaline phosphatase (AP) received either 10mg or 50mg of OCA or placebo. The primary efficacy endpoint was a reduction in AP. At the end of the 12-week treatment period, both doses of OCA produced significant reductions in AP (10mg: -45%; 50mg: -38%; placebo: 0%; p<0.0001 both doses vs. placebo). There were also significant improvements in other liver enzymes, including gamma-glutamyl transferase. In addition, serum markers of inflammation and immunity also improved with significant reductions of C reactive protein (CRP), and immunoglobulin M (IgM), which is closely associated with the autoimmune dysfunction in PBC.

OCA is a potent, first-in-class farnesoid X receptor (FXR) agonist derived from the primary human bile acid chenodeoxycholic acid, the natural endogenous FXR agonist. FXR is a nuclear receptor serving as a bile acid sensor that controls bile acid synthesis and flow in the liver. This nuclear receptor has so far been shown to be integral to the following functions: bile homeostasis, lipid metabolism, glucose metabolism, and inflammation/immune response.

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