- Intercept Pharmaceuticals has withdrawn an approval application for its liver disease drug in Europe, the latest delay in the biotech's effort to bring the first treatment to market for nonalcoholic steatohepatitis, or NASH.
- Intercept initially sought approval of obeticholic acid in NASH in December 2019 in the U.S. and Europe after reporting positive Phase 3 study results. But the Food and Drug Administration rejected the drug last year and asked for more data. Intercept hasn't collected those results in time to include them in its application in Europe.
- The New York biotech has lost nearly 90% of its market value since seeking those approvals nearly two years ago. It could still bounce back, however, if the updated results from its large study, known as REGENERATE, prove positive. Those findings are expected in early 2022 and could form the foundation of new applications to drug regulators.
Intercept's decision to withdraw an application for obeticholic acid in Europe doesn't necessarily come as a surprise. Top executives hinted such an outcome was possible on a conference call last month.
In a statement Thursday, the company explained the decision as a consequence of timing. The results Intercept has submitted so far weren't enough to secure support from European drug regulators, and the company couldn't stretch the review timeline any further to incorporate new data.
On its face, that means obeticholic acid, or OCA, has now been rejected in the U.S. and won't get to market anytime soon in Europe — an outcome that seemed unlikely in 2019 when Intercept became the first biotech to report positive results in a Phase 3 trial in NASH. The company has since lost much of its market capitalization and last September laid off a quarter of its workforce to save cash. Its longtime CEO, Mark Pruzanski, stepped aside a year ago. And Intercept shares, which one traded at more than $400 apiece, are now worth less than $16.
Intercept may still have a chance to rebound. Multiple other drugmakers have failed to capitalize on Intercept's stumbles, as NASH — a liver disease thought to affect millions of Americans — has proven an elusive target. That's given Intercept time to collect the additional information the FDA has required for a new approval submission.
Those results, which come from a re-analysis of the Phase 3 study REGENERATE, are expected early next year. They'll include 18-month biopsy results from about 500 more patients than were included in its original filing, as well as more safety data.
Intercept also anticipates soon getting findings from a different Phase 3 study, REVERSE, in NASH patients who have cirrhosis but don't yet show symptoms.
Those two results, combined, should provide the "long-awaited clarity on the viability of OCA in NASH," wrote Thomas Smith, an analyst at SVB Leerink, in a note to clients. If they're positive, Intercept could re-file for approvals in the U.S. and Europe next year.
Intercept shares fell about 9% in early trading Thursday.