Intercept plots a big Phase III NASH trial with room for an early approval

Intercept Pharmaceuticals, striving to launch the world's first treatment for a pervasive liver disease, spelled out its plans for a sizable Phase III study designed to secure an accelerated approval and help the company retain pole position in a potentially lucrative field.

The company's drug is a bile analog called obeticholic acid, or OCA, for NASH, a form of fatty liver disease whose prevalence in the developed world has galvanized a major R&D push among biopharma companies. Intercept's candidate is the industry's most advanced NASH treatment, and, following some impressive mid-stage efficacy data, the biotech is pressing ahead with a 2,500-patient Phase III effort slated to begin enrollment next quarter.

Following discussions with the FDA and European Medicines Agency, Intercept has designed a late-stage trial that will test two doses of OCA against placebo in NASH patients with stage 2 or stage 3 liver fibrosis, or scarring. The study's primary goals are improving fibrosis by at least one stage without worsening NASH scores and resolving NASH symptoms without exacerbating scarring, the company said.

But Intercept doesn't plan on waiting for final data before making its case to regulators. The biotech has scheduled a 72-week interim analysis in which investigators will take a look at tissue samples from about 1,400 patients to chart how well OCA is doing against its primary endpoints, and Intercept expects that data to fuel its FDA and international regulatory filings.

Intercept is banking on an early approval in part thanks to the FDA's breakthrough-therapy designation, a distinction bestowed on OCA that promises access to top agency officials and a speedy review process. The biotech hopes to lock down conditional OKs in the U.S. and Europe with the 72-week results before handing in final outcomes data to support permanent approval.

With that timeline, Intercept remains in first place among companies working to develop treatments for NASH, which stands for nonalcoholic steatohepatitis. Behind it, French biotech Genfit is blueprinting a Phase III trial for a similar treatment, while Gilead Sciences ($GILD), Shire ($SHPG), Enanta Pharmaceuticals ($ENTA) and a host of other advance NASH therapies of their own.

- read the statement (PDF)