Shares of Intercept Pharmaceuticals (ICPT - Get Report) have almost tripled early Thursday morning on the announcement of positive results from a mid-stage study of the company's experimental therapy for the chronic liver disease known as nonalcoholic steatohepatitis (NASH).
Intercept shares are up 218% to $230 right before the market opens for regular trading. Wow.
The phase II study of Intercept's obeticholic acid (OCA) was stopped early for efficacy after a planned interim analysis showed the primary endpoint had been met. Here's how Intercept described the results:
The decision to stop FLINT has been based on the recommendation of the Data Safety Monitoring Board (DSMB) which reviewed liver biopsy data from before and at the end of the treatment period in approximately half of the 283 randomized patients, in accordance with a planned interim efficacy analysis. This analysis demonstrated that OCA treatment resulted in a highly statistically significant improvement (p=0.0024 on an intention-to-treat [ITT] basis) in the primary histological endpoint, defined as a decrease in the NAFLD Activity Score (NAS) of at least two points with no worsening of fibrosis, as compared to placebo. Those patients who had not yet completed the trial and therefore did not have a second biopsy were treated as non-responders in the ITT analysis. The pre-defined threshold of statistical significance for stopping FLINT was p < 0.0031.
NASH is a chronic liver disease caused by excess fat accumulation in the liver which leads to inflammation and scarring, or fibrosis. Untreated, NASH progresses to cirrhosis and liver failure. Approximately 12% of the U.S. adult population has NASH, while 2.7%, or more than 6 million patients, have advanced liver fibrosis or cirrhosis due to NASH, Intercept says, citing recent studies.
Intercept is developing OCA for four different liver-related diseases. A phase III study in primary billary cirrhosis is ongoing, with results expected later this year.
One reason for the huge move in Intercept shares this morning might be based on speculation that the company will be able to seek regulatory approval for OCA in NASH based on the positive results of the phase II study along with already compiled safety data.
BMO analyst Jim Birchenough reacted this way to the OCA data:
Results are unexpected, potentially paradigm changing and transformational for ICPT, in our opinion. With an estimated 2.7% of the US population, or ~8 million individuals, with advanced liver fibrosis or cirrhosis due to NASH, we would suggest at a minimum that our current peak sales of $865M in primary biliary cirrhosis could be two-fold higher in NASH, with just 2% market penetration. Magnitude of opportunity and timelines for realizing the opportunity are uncertain at this point but could be many fold higher. With potential broader application to patients with obesity, diabetes, and broader non-alcoholic fatty liver disease (NAFLD), we expect significant large pharma interest in ICPT to follow these results.
Oppenheimer's Akiva Felt calls the Intecept data "overwhelmingly positive" but also throttles back near-term expectations:
What's next in NASH? Following the FDA/AASLD workshop in September, we believe more work needs to be done with the NAS score endpoint before moving into Phase 3. That said, OCA is now the leading candidate in development for this large unmet medical need with no approved therapies.
Intercept is holding a conference call at 4:30 pm ET today.
-- Reported by Adam Feuerstein in Boston.
Follow Adam Feuerstein on Twitter.