Intercept Pharmaceuticals (NASDAQ:ICPT) announces more data from the Phase 3 REGENERATE clinical trial evaluating obeticholic acid (OCA) in NASH patients with liver fibrosis. The results are being presented at The International Liver Congress in Vienna.
As reported in February, REGENERATE met one co-primary endpoint (a statistically significant proportion of patients achieving at least a one-stage improvement in fibrosis with no worsening of NASH at month 18) but failed to meet the other (a statistically significant proportion of patients achieving NASH resolution with no worsening of liver fibrosis). Per FDA agreement, only one needed to be met for study success.
Additional analyses based on the per-protocol population (n=668) showed that 13.3% patients receiving 25 mg of OCA each day for at least 15 months experienced at least a two-stage improvement in fibrosis compared to 4.5% for placebo (p=0.0008).
In patients who experienced at least a one-stage change in fibrosis, 38.0% of those receiving OCA 25 mg once-daily improved versus 13.1% who worsened. In the control group, 23.2% improved compared to 20.9% who worsened. 65.6% of treated patients (OCA 25 mg) with elevated ALT (enzyme biomarker of liver stress/damage) at baseline achieved normal levels compared to 37.3% for placebo. The proportions of patients with elevations in another liver enzyme, AST, who achieved normal levels were 54.7% for OCA and 29.3% for control.
On the safety front, adverse events (AEs) were mainly mild or moderate. The rates of serious AEs were 14% in the OCA 25 mg arm, 11% in the OCA 10 mg arm and 11% in the control arm. There were three deaths (two in the control arm and one (glioblastoma) in the OCA 25 mg arm), none considered treatment-related. 3% of patients in the OCA 25 mg arm experienced gallstones or gallbladder inflammation (cholecystitis) compared to 1% in the OCA 10 arm and less then 1% in the control arm. The company says there was no pattern attributable to OCA.
The company will host a conference call today at 11:45 am ET to discuss the results.
Shares are down 1% premarket on light volume.
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