Intercept Receives Breakthrough Therapy Designation

Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) (Intercept), recently announced that its investigational product obeticholic acid (OCA) has received “breakthrough therapy designation” from the US FDA for the treatment of patients with nonalcoholic steatohepatitis (NASH) with liver fibrosis. This indication constitutes a population of patients with a serious and life-threatening condition reflected by a higher risk of progression to cirrhosis and liver failure. OCA is currently being developed for the treatment of several chronic liver diseases, including primary biliary cirrhosis (PBC), NASH and primary sclerosing cholangitis (PSC).

“We are very pleased to have received breakthrough therapy designation for NASH with liver fibrosis, as it will enable us to work closely with FDA to finalize the design of our Phase III program,” said Mark Pruzanski, MD, President and CEO of Intercept. “This designation underscores a recognition of the urgent need to bring novel treatments to NASH patients who have developed liver fibrosis, which is expected to make this serious disease the leading cause for liver transplant in just the next few years. As a first-in-class FXR agonist, we believe OCA has the potential to be an important treatment option for patients with no currently approved medicines.”

Breakthrough therapy designation for OCA was based on clinical efficacy and safety data from two placebo-controlled, Phase II clinical trials of OCA. A Phase II trial in 64 patients with nonalcoholic fatty liver disease (NAFLD) assessed the impact of OCA treatment on insulin sensitivity (published in Gastroenterology in June 2013). The FLINT Phase II trial in 283 patients with NASH assessed the impact of OCA treatment on liver histology and fibrosis (published in The Lancet in November 2014).

The breakthrough therapy designation was created by the FDA to speed the availability of new therapies for serious or life-threatening conditions. Drugs qualifying for this designation must show credible evidence of a substantial improvement on a clinically significant endpoint over available therapies, or over placebo if there is no available therapy. The designation confers several benefits, including intensive FDA guidance and discussion and eligibility for submission of a rolling NDA.

NASH is a serious chronic liver disease caused by excessive fat accumulation in the liver that induces chronic inflammation which leads to progressive fibrosis (scarring) that can lead to cirrhosis, eventual liver failure, and death. There are currently no drugs approved for the treatment of NASH. Studies have shown that 21% to 26% of NASH patients will develop cirrhosis over 8.2 years of follow-up and that liver-related mortality due to this disease is ten-fold that of the general population. According to recent epidemiological studies, it is estimated that more than 10% of the US adult population has NASH with more than 60% of patients (potentially more than 14 million in total) believed to have liver fibrosis or cirrhosis due to progression of the disease. The proportion of liver transplants attributable to NASH has increased rapidly in past years, and by 2020, the disease is projected to become the leading indication for liver transplant ahead of chronic hepatitis C and alcoholic liver disease. NASH patients with fibrosis are at greater risk of progressing to cirrhosis, liver failure and cancer.

Intercept is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat orphan and more prevalent liver and intestinal diseases utilizing its expertise in bile acid chemistry. For more information, visit www.interceptpharma.com.