Intercept Ends Roller Coaster Weekend With Phase 3 Victory

3/16/14Follow @benthefidler

Intercept Pharmaceuticals gave investors a scare on Friday night, but the New York-based company bounced back on Sunday by revealing that its experimental liver drug, obeticholic acid (OCA), aced a late-stage clinical trial.

Intercept (NASDAQ: ICPT) said that OCA met all of its goals in a Phase 3 study testing the drug in people with primary biliary cirrhosis (PBC), a rare liver disease in which the immune system ends up gradually destroying the bile ducts. Investigators found that OCA lowered patients’ elevated levels of serum alkaline phosphatase—a hallmark of the disease—in a statistically significant way. OCA also met other secondary goals measuring certain liver function statistics, like bilirubin levels, according to Intercept. The company now plans to file a new drug application with the FDA and seek regulatory approval of the drug.

The data release was a quick response to breaking news on Friday that spooked investors. In its annual report, Intercept shared data revealing an unexpectedly high incidence of adverse cardiac events in patients in the OCA study. Shares tumbled more than 16 percent in after-hours trading once investors got wind of the news.

In the PBC study, meanwhile, Intercept enrolled 217 patients with the disorder that don’t respond to, or can’t tolerate ursodeoxycholic acid, the only FDA-approved treatment for people with the disorder. Patients were given either a placebo, a 10 milligram dose of OCA, or a 5 mg dose that was gradually increased to 10 mg over the course of the study. The goal was to see at least a 15 percent reduction in those patients’ serum alkaline phosphatase levels, and normal levels of bilirubin, after a year of treatment with OCA. Intercept succeeded: 47 percent of those on the high dose and 46 percent on the low dose achieved those results, compared to just 10 percent of the patients in the placebo group.

The 10 mg group saw their serum alkaline phosphatase levels drop 39 percent over the course of the trial, compared to 33 percent in the low dose group and 5 percent in those who got a placebo. The most common side effect attributed to OCA was mild to moderate itching, which caused eight patients to drop out of the study. Intercept also said that while there were more serious adverse events reported among those who got its drug than those who didn’t, none were considered drug-related. The company didn’t detail those events—they’ll be revealed when Intercept discloses all the data at a medical meeting in April.

Intercept became a biotech darling in early 2014 when OCA showed such obvious benefits for patients with nonalcoholic steatohepatitis (NASH)—a leading cause of cirrhosis and liver failure—in a mid-stage clinical trial that the company stopped it early. Investors clamored into the stock, sending it to around $240 per share from just over $70 in a single day. Intercept’s stock has since doubled from there. Shares closed on Friday at $462.26 apiece.

Still, the party came to a screeching halt in after-hours trading Friday. Intercept hasn’t yet detailed all of the data from the NASH study, including its safety findings, but the company revealed in a 10-K filing with the Securities and Exchange Commission that seven patients in that trial—some in the placebo group and others dosed with its drug—suffered a total of 10 “severe cardiovascular adverse events.” Though Intercept didn’t shed light on all of these cases specifically, it did reveal that two of the patients treated with its drug died later on. Shares plummeted more than 16 percent late Friday.

Intercept tried to assuage investors’ concerns by noting that there wasn’t a statistically significant difference in such serious events between those on placebo and those dosed with OCA; that investigators have concluded that “none [of the events] were considered probably or definitely related” to treatment with OCA; and that the two patients that died had other serious medical conditions that might have been to blame. Still, the full extent of those details won’t be known until Intercept releases the full results from that study, named “FLINT,” at a medical meeting in November.

The company will hold a conference call to discuss the results of the PBC study, meanwhile, on Monday morning.

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com Follow @benthefidler

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