By: Adam Feuerstein | 01/09/14 - 09:30 AM EST
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.
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.
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.
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