FDA Strikes Out Intercept Pharma's Obeticholic Acid Application For NASH Patients, Focus Shifts To Rare Liver Diseases

FDA issued a Complete Response Letter to Intercept Pharmaceuticals Inc's ICPT marketing application seeking approval for obeticholic acid (OCA) for pre-cirrhotic fibrosis due to NASH.

The FDA stated that any resubmission would require successful completion of the long-term outcomes phase of the REGENERATE study.

Intercept has decided to discontinue all NASH-related investment and restructure its operations to focus on rare and serious liver diseases, aiming for profitability in 2024.

The company will reduce its workforce by approximately one-third and prioritize a fixed-dose combination of OCA and bezafibrate. 

Related: What Now After FDA AdComm Rebuke for Intercept Pharmaceutical? Analyst Says Approval Of Intercept's 'OCA In NASH May Never Occur.

Concurrently, Intercept announced new results from a planned interim analysis of its ongoing Phase 2 study 747-213 assessing improvements in serum biomarkers of hepatic function, cholestasis, and inflammation in patients with primary biliary cholangitis (PBC) after treatment with an investigational combination of OCA and bezafibrate. 

Results showed that OCA 5-10 mg + bezafibrate 400 mg combo effectively normalized multiple biochemical markers associated with PBC-induced liver damage.

Biochemical remission, defined as normalization of associated biomarkers, was induced in 58% of patients on OCA5-10/B400 at 12 weeks vs. 7% (bezafibrate 200 mg), 27% (bezafibrate 400 mg), and 31% (OCA5-10/B200) in the other treatment arms. 

Further, patients in the OCA5-10/ bezafibrate 400 arm experienced consistently high levels of normalization of individual biomarkers. 

Patients in the OCA5-10/B400 arm demonstrated the highest reduction rates from baseline in biomarkers, with a clear dose-response in markers shown to predict transplant-free survival.

Intercept has lowered FY2023 adjusted operating expense guidance to $350–$370 million, including restructuring costs. It reiterates the 2023 Ocaliva net sales guidance of $310–$340 million.

The company expects a net reduction in annual adjusted operating expenses of approximately $140 million – relative to the updated 2023.

Price Action: ICPT shares are up 4.76% at $12.10 during the premarket session on the last check Friday.