Intercept Pharmaceuticals Rockets as Lead NASH Drug Snags Breakthrough Status From the FDA

January 30, 2015
By Krystle Vermes, BioSpace.com Breaking News Staff

Biopharmaceutical company Intercept Pharmaceuticals , has seen its stock grow more than 28 percent since Jan. 29 when it announced that the U.S. Food and Drug Administration (FDA) had given Breakthrough Therapy Designation to its nonalcoholic steatohepatitis drug, obeticholic acid. The company has been testing OCA for the treatment of NASH in patients with liver fibrosis, according to Bidness ETC.

NASH is a disease that is caused by chronic fat accumulation in the liver, leading to chronic inflammation. This can eventually result in liver scarring, liver failure and death.

The Breakthrough Therapy Designation came after the FDA reviewed data from two placebo-controlled, Phase II clinical trials.

"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, president and chief executive officer of Intercept Pharmaceuticals. "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."

Planning for the Road Ahead
On Jan. 12, Intercept Pharmaceuticals released cumulative information on its performance in 2014 and mapped out some of its plans for 2015. The company specified that it had ended the year with nearly $240 million in cash and investments. It expects its operating expenses to total around $200 million in 2015, and much of this money will go toward products in its pipeline.

In addition to announcing its projected expenses, Intercept Pharmaceuticals noted that it intends to move forward with its NASH Phase III clinical program in the first half of 2015.

Intercept Pharmaceuticals primarily focuses on developing therapeutics to treat liver and intestinal diseases, and OCA is currently its lead product. OCA is a bile acid analog and agonist of the farnesoid X receptor. Some of the conditions that OCA is being developed to treat aside from NASH include biliary cirrhosis and primary sclerosing cholangitis.

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