Drug Development

Intercept ‘lowers the bar’ on its pivotal PhIII OCA study for NASH

Intercept CEO Mark Pruzanski

Intercept CEO Mark Pruzanski has redrawn the rules on their pivotal Phase III study of obeticholic acid (OCA).

After talks with the FDA, Pruzanski said today that investigators can reduce their target on the number of patients needed for an interim analysis of the FXR agonist in 2019, dropping the target from 1,400 to 750. And the agency has signed off on changing the endpoint from fibrosis and NASH resolution to either fibrosis or NASH while accepting an objective definition of NASH resolution to clarify success.

“We now only need to achieve one endpoint for the trial to be considered successful,” the CEO said on a call with analysts Friday morning, saying that the bar has been lowered on success, increasing their odds of success. And if the drug hits both endpoints, he added, Intercept $ICPT will be able to “clearly differentiate this drug from others.”

“We believe we now have the best case scenario,” he aded, gaining a lower hurdle while keeping their sights set on both endpoints.

“We continue to be confident in both endpoints,” said Pruzanski. And the interim readout on the closely watched study is expected in 2019 remains the same. “We’ve successfully derisked the study” without risking the integrity of the trial, he said. Rivals in the field, he added, have fielded data on one or the other endpoint.

The switch at Intercept could have big implications for Allergan, notes Evercore ISI’s Umer Raffat, which is planning its own Phase III after buying Tobira. And it appears consistent with Gilead’s plan for the “Ph 3 for selonsertib (where primary endpoint will be fibrosis.)”

It’s clear that the change-up is occurring after Intercept’s timeline on recruiting the patients needed for an interim analysis fell well behind schedule. Earlier, Intercept had said that it could enroll all 1,400 patients in the first half of this year. Now it’s saying that the new goal of 750 will be completed in mid-2017, meaning that the switch will help keep them on schedule instead of losing the edge they have with rivals.

Intercept set up the call on the fly this morning, without any explainer in a press release. That move drove its stock into the red ahead of the announcement, but shares snapped back into the green once the news became clear.


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RAPS Regulatory Convergence 2017