Benchmarking clinical value in NASH will require more than the topline data reported this week by Intercept Pharmaceuticals Inc. The readout gained Ocaliva obeticholic acid the title of first NASH therapy to meet in Phase III, but lingering efficacy and safety questions leave the field still lacking a clear picture of what meaningful benefit looks like in the indication.
In topline data announced Feb. 19 from the Phase III REGENERATE trial, the highest dose of Ocaliva met one of two primary endpoints, and the consensus among physicians is that it was the more important one. Still, the number of patients who benefited from the therapy was small, and the study stoked existing concerns about elevated LDL levels and pruritus.
Gaining a better understanding of the types of patients who respond best to the therapy and whether they should receive statins to lower LDL will require detailed analyses from REGENERATE, plus data from Intercept’s next Phase III trial REVERSE, which is enrolling a population with more advanced fibrosis at