- In a decision that has hiked up the company's share price, the Food and Drug Administration has agreed Intercept Pharmaceuticals can almost halve its recruitment target for the 2019 interim analysis of its study in non-alcoholic steatohepatitis (NASH) patients with liver fibrosis as well as change the endpoints.
- The aim is now to recruit 750 rather than 1,400 patients for the Phase 3 REGENERATE study, with a change in definition of improvement of NASH and an endpoint switch from both improvement in fibrosis and resolution of NASH to realizing one or the other.
- Intercept has also clarified the definition of NASH resolution; this was previously left open in earlier protocols. The trial is testing the safety and efficacy of its drug, obeticholic acid (OCA).
"The study was originally designed with both endpoints based on the FLINT study, which showed that OCA improved all pathological features of non-alcoholic steatohepatitis (NASH) patients," explained Mark Pruzanski, Intercept Pharmaceuticals CEO and president in an investor conference call. "None of our competitors have data supporting efficacy for dual endpoints. This gives us two shots at the goal," Pruzanski added.
The change brings Intercept's endpoints into line with other companies' studies and will allow development of OCA to remain on track. The company originally expected to complete recruitment for the interim analysis by mid-2017 and should still be able to come in on time due to the lower enrollment targets.
As obesity levels continue to increase, the NASH market could be worth $35 billion in 2025. It's currently an area of unmet need, but competition is starting to heat up as drugmakers push into mid- to late-stage clinical trials. The data from REGENERATE, according to Pruzanski, has potential to differentiate OCA from other drugs in late-stage development especially if both endpoints are reached.
Gilead Sciences, for example, has four NASH candidates in its pipeline in Phase 1 and 2, including selonsertib and simtuzumab. And Irish biopharma Allergan has been busy in the NASH market, acquiring two NASH-focused companies: Tobira Therapeutics' cenicriviroc is in Phase 2, with a pivotal study planned this year, and Akarna Therapeutics' AKN-083 is in preclinical development.
Despite a Phase 2a miss last fall, Galectin Therapeutics is still pinning its hopes on a larger NASH study due to read out in December 2017. Regulus and AstraZeneca's RG-125 (AZD4076) are also in Phase 2, as is Cempra's antibacterial solithromycin.