Shares of Intercept Pharmaceuticals (ICPT - Free Report) plunged 39.7% after it announced the receipt of a Complete Response Letter (CRL) regarding the New Drug Application (NDA) for obeticholic acid (OCA) seeking approval for the treatment of fibrosis due to nonalcoholic steatohepatitis (NASH).
Based on the data reviewed by the FDA, the agency issued a CRL as it determined that the predicted benefit of OCA based on a surrogate histopathologic endpoint remains uncertain and does not sufficiently outweigh the potential risks to support accelerated approval for the treatment of patients with liver fibrosis due to NASH.
Moreover, the agency recommended Intercept to submit additional post-interim analysis efficacy and safety data from the ongoing REGENERATE study in support of potential accelerated approval and continue the long-term outcomes phase of the study.
The news was disappointing as Intercept’s OCA was a frontrunner in receiving approval for the treatment of NASH.
Per Intercept, the FDA has progressively increased the complexity of the histologic endpoints, creating a very high bar that only OCA has so far met in a late-stage study. Intercept disagreed with the agency and believes that the agency’s decision was based on an apparently incomplete review.
The agency had earlier postponed a tentatively scheduled advisory committee meeting (Adcom), and hence a delay in approval was in the cards. However, the CRL was disappointing.
NASH is a serious progressive liver disease caused by excessive fat accumulation in the liver that induces chronic inflammation, resulting in progressive fibrosis (scarring) that can lead to cirrhosis, eventual liver failure, cancer and death.
A potential approval would have significantly boosted Intercept’s prospects, given the market potential of NASH. Importantly, OCA would have been one of the first therapies approved for the treatment of NASH.
Intercept’s shares have declined 62.3% in the year so far against the industry’s growth of 10.5%.
While the NASH market promises potential, it is quite challenging as well. Developing a treatment for NASH is a complex affair and there have been quite a few failures so far in this space.
Last month, France-based Genfit (GNFT - Free Report) announced disappointing results from an interim analysis of the RESOLVE-IT phase III study evaluating once-daily, 120mg of elafibranor in adults with NASH. The trial did not meet the predefined primary endpoint of NASH resolution without worsening of fibrosis in the ITT population of 1,070 patients.
Meanwhile, Viking Therapeutics is enrolling in the phase IIb VOYAGE study evaluating VK2809 in biopsy-confirmed NASH and fibrosis. Clinical-stage biopharmaceutical company, Galmed Pharmaceuticals Ltd. (GLMD - Free Report) is also developing Aramchol, a liver targeted, oral SCD1 modulator for patients with NASH and fibrosis.
Intercept currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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