Therefore, the reversal of steatohepatitis with no evidence of progression to advanced fibrosis (stage 3 or 4), may be an acceptable surrogate endpoint suitable both for phase 2b and 3 trials that enroll patients with NASH and evidence of early fibrosis."
Challenges and opportunities in drug and biomarker development for nonalcoholic steatohepatitis: Findings and recommendations from an American Association for the Study of Liver Diseases (AASLD) - Food and Drug Administration (FDA) joint workshop
Intercept Pharmaceuticals is up 10% Monday because FDA appears copacetic with the company's proposal to use a surrogate efficacy endpoint in the planned pivotal phase III study of its NASH drug OCA.
Baird analyst Brian Skorney comments:
We think this reflects a very favorable assertion from an article co-authored by an FDA clinical reviewer. We believe the FLINT study demonstrated that OCA can effectively hit this endpoint in a large Phase 3 study...
We think we could see Intercept's announcement of a Phase 3 program in the near future and believe an FDA sign-off to move forward with a study that could support accelerated approval based on biopsy will be a major derisking event for the stock here.
Good news for Intercept, but Skorney also notes concerns about the potential for a heart safety risk tied to OCA remains.