A company that could become the first to win Food and Drug Administration approval for a drug to treat a form of liver disease rapidly growing worldwide on Thursday added to positive interim data announced two months ago at a liver conference underway in Vienna. However, while seen as encouraging, the data also raised questions among investors.
New York-based Intercept Pharmaceuticals announced new data from the interim analysis of the Phase III REGENERATE study of Ocaliva (obeticholic acid) in patients with liver fibrosis due to nonalcoholic steatohepatitis (NASH) at the European Association for the Study of the Liver’s 2019 International Liver Congress.
Shares of Intercept were down more than 13 percent on the Nasdaq Thursday afternoon.
The data showed that in the intent-to-treat population, once-daily treatment with Ocaliva at 25mg met the primary endpoint of improving fibrosis by one stage or more, with no worsening of NASH, in 23.1 percent of patients, compared with 11.9 percent of those receiving placebo. Although a numerically greater proportion of patients receiving the drug at 25mg or 10mg than those receiving placebo achieved another primary endpoint, NASH resolution, that result did not reach statistical significance. Under an agreement between the company and the FDA, the study was required to reach at least one primary endpoint.
In the per-protocol population – a subset of the intent-to-treat population – the percentage of patients achieving an improvement of at least two stages of fibrosis was nearly three times higher among those receiving the drug at 25mg than among those receiving placebo.
Cowen analyst Ritu Baral wrote in a note to clients that while the per-protocol secondary analysis was encouraging, the lack of an intent-to-treat analysis was disappointing to investors, who wanted to see the latter analysis as they feared that it could look “substantially worse.” Nevertheless, Baral wrote, management’s comments at an investor event at the EASL conference could allay some of those concerns, as the executives said the intent-to-treat cuts were in line with the per-protocol analysis, albeit with smaller magnitudes of relative effect. However, there were also additional fears about the safety protocol, as there was an imbalance of gall stone and gallbladder inflammation events, and the company did not have substantive answers to questions about them.
In contrast with the drop in Intercept’s shares Thursday, the company’s stock rose dramatically in February when it announced positive data from REGENERATE’s pre-planned 18-month interim analysis showing the study met its primary endpoint of improvement by at least one stage of fibrosis.
Ocaliva, currently marketed in 5mg and 10mg doses, has accelerated FDA approval for biliary cholangitis.
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