Intercept Pharmaceuticals (NASDAQ:ICPT) stock fell ~18% on Sept. 30 after the company said obeticholic acid (OCA) failed to show improvement in fibrosis with no worsening of a liver disease, thus not meeting the main goal of a phase 3 trial.
The study, dubbed REVERSE, was evaluating OCA against placebo in patients with compensated cirrhosis (scarring, or fibrosis of liver) due to nonalcoholic steatohepatitis (NASH). A total 919 patients received either once-daily placebo, OCA 10 mg, or OCA 10 mg for the first three months with titration as per protocol up to OCA 25 mg for the remaining study period.
The trial did not meet its main goal of a ≥ 1-stage histological improvement in fibrosis with no worsening of NASH following up to 18 months of therapy, the company said in a Sept. 30 press release.
Intercept noted that, 11.1% of those on OCA 10 mg and 11.9% of those in the OCA 10mg-to-25 mg group achieved a ≥1-stage improvement in fibrosis with no worsening of NASH after up to 18 months of therapy, compared with 9.9% of those on placebo.
The company, however, noted that a positive impact on liver stiffness was seen across both drug dose groups.
Intercept added that there was a numerical increase in the number of liver safety events for the OCA-treated arms; most were mild in severity and related to biochemical changes.
The company noted that there were no severe or fatal adjudicated hepatic safety events in any treatment arm.
Safety was evaluated in 916 patients. The most common Treatment-emergent adverse events (TEAE) was itching (31% in placebo, 41% in OCA 10 mg and 57% in OCA 10-to-25 mg) and itching pruritus was the most common reason for treatment discontinuation, according to the company.
TEAEs and serious TEAEs and deaths were balanced across all treatment groups in the REVERSE study, the company added.
In addition, Intercept said that serious gallbladder-related events were balanced across arms and consistent with the known mechanism of action of FXR-agonists, the OCA 10-to-25 mg group had a higher incidence of gallstones.
The company noted that its planned new drug application (NDA) for its lead indication of liver fibrosis due to NASH will be supported by positive data from a phase 3 trial called REGENERATE and is unaffected by the efficacy results of REVERSE.
(Editor’s note: There was error in the earlier version of the article where the words 'after three months' were added at the start of the 4th paragraph. This has been removed. The treatment duration was up to 18 months which was also included in the original article and has been kept.)
This was corrected on 10/01/2022 at 6:24 AM. There was error in the earlier version of the article where the words 'after three months' were added at the start of the 4th paragraph. This has been removed. The treatment duration was up to 18 months which was also included in the original article and has been kept.