Intercept Pharmaceuticals has submitted its top candidate to the FDA for approval in primary biliary cirrhosis, a rare disease that results in serious liver damage.
Intercept Pharmaceuticals, striving to launch the world's first treatment for a pervasive liver disease, spelled out its plans for a sizable Phase III study designed to secure an accelerated approval and help the company retain pole position in a potentially lucrative field.
Late last week investors wound up using Intercept Pharmaceuticals as a punching bag after new data on its NASH drug for fatty liver disease raised some troubling questions about its safety and efficacy. Its stock was hit hard, forced down 30% in hours as analysts mulled the implications of potential trouble.
After crunching the data on Intercept's clinical study of OCA for nonalcoholic steatohepatitis, investigators say they tracked some distinct improvements for patients suffering from the liver disease. But they also fretted about some troubling safety issues as well as inadequate efficacy endpoints that will have to be carefully considered in follow-up studies, tempering some of the high excitement that has been stoked by some analysts and the biotech's investors ahead of a pivotal Phase III study.
Still riding high on some strong early-year data for its liver disease treatment, Intercept Pharmaceuticals' shares shot up once more as its lead drug came through in Phase IIb, dismissing some earlier safety worries and setting the table for late-stage study.
In recent years it's been the big biotechs in the U.S. which have registered approvals for the drugs most likely to succeed on the market. But in reviewing EvaluatePharma's recent picks for top Phase III drugs, it's interesting to see some prominent positions among the Big Pharma crowd. Read the full report >>
Investigators for Intercept Pharmaceuticals spelled out the impact of its lead therapy on rare cases of a liver disease called primary biliary cirrhosis (PBC), fleshing out the results of a late-stage study that it will use to seek U.S. and European approvals later in the year.
Intercept Pharmaceuticals delivered a bad news-good news punch over the weekend about its lead drug.
On Friday night Intercept Pharmaceuticals managed to rattle investors with a 10-K filing flagging a collection of serious cardiac adverse events recorded in a study of its lead drug OCA. But come Sunday morning, the biotech balanced out that bitter taste with an announcement that its Phase III trial had ended with positive data, setting up a regulatory filing for approval.
Shares of Intercept Pharmaceuticals went into overdrive this morning, soaring a remarkable 125% on the surprise news that a Phase IIb clinical study for its lead drug funded largely by the NIH ended early after achieving the primary endpoint.