Biotech Values

[DewDiligence]

CNAT—why is it that biopsies were not done to measure efficacy...?

This was a biomarker trial; showing clinical efficacy in NAFLD/NASH would require a different kind of trial that would include before-and-after biopsies.

Are they going to start phase-3 based on surrogate-marker endpoints?

That’s the plan, although it’s not yet clear which kinds of liver-disease patients will be enrolled in this trial. The results of the other phase-2 trials reading out in 2015 will guide this decision.

Will FDA approve such a trial?

The FDA recently published guidelines (in conjunction with AASLD) on acceptable surrogate markers for NASH, so the idea of seeking accelerated approval based on a set of such biomarkers is consistent with the FDA’s current thinking.