We are a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic liver diseases, such as primary biliary cirrhosis and nonalcoholic steatosis, utilizing our expertise in bile acid chemistry. Our product candidates have the potential to treat orphan and more prevalent liver and intestinal diseases for which there currently are limited therapeutic solutions.
Our Lead Product Candidate
Our lead product candidate, obeticholic acid, or OCA, is a bile acid analog, a chemical substance that has a structure based on a naturally occurring human bile acid. OCA is a first-in-class product candidate that selectively binds to and induces activity in the farnesoid X receptor, or FXR, which we believe has broad liver-protective properties. We are developing OCA initially for primary biliary cirrhosis, or PBC, as a second line treatment for patients who have an inadequate response to or who are unable to tolerate standard of care therapy and therefore need additional treatment. PBC is a chronic autoimmune liver disease that, if inadequately treated, may eventually lead to cirrhosis, liver failure and death. We are conducting a Phase 3 clinical trial of OCA in PBC, which we call the POISE trial, that we anticipate will serve as the basis for seeking regulatory approval in the United States and Europe. In December 2012, we completed enrollment of the POISE trial with 217 patients, exceeding the originally targeted number of patients by approximately 20%. In December 2013, the last patient follow-up visit was completed, marking the conclusion of the double-blind phase of the POISE trial. Of the 217 patients randomized, 19 patients (approximately 9%) discontinued early, including seven patients (approximately 3%) who did so due to pruritus. Top-line results from the double-blind phase of the POISE trial are expected to be available in the second quarter of 2014.We currently expect results from the POISE trial to be available in the second quarter of 2014. OCA has received orphan drug designation in the United States and Europe for the treatment of PBC.
We are also developing OCA for the treatment of nonalcoholic steatohepatitis (NASH). The Farnesoid X Receptor Ligand Obeticholic Acid in Nonalcoholic Steatohepatitis Treatment (FLINT) trial has been sponsored and conducted by the National Institute of Diabetes & Digestive & Kidney Diseases (NIDDK). FLINT enrolled 283 adult NASH patients at eight US centers comprising the NIDDK's NASH clinical research network. Patients were randomized to receive either a 25 mg dose of OCA or placebo for 72 weeks. In January 2014, the treatment phase of the FLINT trial was stopped early for efficacy based on a planned interim analysis showing that the primary endpoint of the trial had been met (p=0.0024).
We own worldwide rights to OCA outside of Japan and China, where we have exclusively licensed the compound to Dainippon Sumitomo Pharma, or DSP, and granted it an option to exclusively license OCA in certain other Asian countries.
Patents covering the composition of matter for OCA expire in 2022, before any patent term adjustments or patent term extensions. Our current plan is to commercialize OCA in the United States and Europe ourselves for the treatment of PBC by targeting a limited and focused group of specialist physicians.