Investors have been waiting for the data from Intercept Pharmaceuticals' (NASDAQ:ICPT) FLINT trial testing obeticholic acid, or OCA, in patients with nonalcoholic steatohepatitis, or NASH, since Intercept Pharmaceuticals announced back in January that the trial was stopped because of positive efficacy.
It was worth the wait.
We already knew the FLINT trial had passed its primary endpoint, defined as a decrease in the NAFLD Activity Score of at least two points with no worsening of fibrosis, because that's the reason the trial was stopped. But we didn't know how big a difference we'd see. Turns out, nearly half (46%) of patients taking OCA achieved the goal, compared to just 21% of those that were treated with placebo.
The secondary endpoints are equally important because the FDA hasn't determined what endpoint is most important for proving that a NASH drug is effective. The phase 3 trial could end up having a different primary endpoint than the one used for the FLINT trial.
Resolution of NASH, for instance, might get used since it's arguably more clinically meaningful than just an improvement on the NAFLD Activity Score. In the FLINT trial, 22% of OCA-treated patients had a NASH resolution, compared to just 13% of patients on placebo. Unfortunately the improvement wasn't statistically significant, but it could presumably reach statistical significance in a phase 3 trial if the magnitude is repeatable in a larger trial.
Improvement in fibrosis, or scarring, also looked good with 35% of patients taking OCA showing an improvement in fibrosis, compared to just 19% of placebo-treated patients, which was statistically significant. It seems that OCA either works for a patient or it doesn't because the average improvement was just a 0.2 points from a baseline of 1.9 points. While that was statistically significant compared to the 0.1 point decline in the placebo group it doesn't seem all that meaningful when you look at the entire population.
The biggest surprise in the top-line data release came from safety date. Intercept previously disclosed 10 cardiovascular events with the number of events higher in patients taking OCA, but it turns out that all the life threatening cardiovascular events were deemed unrelated to treatment.
Solid efficacy, seemingly safe, what's next?
Intercept still has to get the complete data from the group that ran the trial before it can finalize a phase 3 trial with the FDA. Then it has to run the trial. Then it has to get OCA approved.
We're talking about years before OCA is approved for NASH.
But OCA looks like it should get approved for another liver disease, primary biliary cirrhosis before that. With this generally positive data, doctors might be willing to use OCA off label when it's only approved to treat primary biliary cirrhosis. Intercept Pharmaceuticals expects to file for primary biliary cirrhosis with EU and FDA regulators in the first half of next year, putting potential approvals in the latter half of the year.