Intercept Pharmaceuticals, Inc. (ICPT - Free Report) initiated a phase III trial, REVERSE, on obeticholic acid ("OCA").
The randomized phase III study will evaluate the efficacy and safety of OCA, in subjects with compensated cirrhosis due to non-alcoholic steatohepatitis (“NASH”) in approximately 540 patients with a biopsy-confirmed diagnosis of cirrhosis.
The primary endpoint of the study is the percentage of subjects with histological improvement in fibrosis by at least one stage using the NASH Clinical Research Network (“CRN”) scoring system after 12 months of treatment. Patients enrolled in the trial are being randomized in a 1:1:1 ratio to one of the three treatment arms — once-daily dosing of OCA 10 mg, once-daily OCA 10 mg with titration to 25 mg at three months, or placebo. The patients who successfully complete the double-blind phase of REVERSE will be enrolled in an open-label extension phase for up to 12 additional months.
Intercept expects to submit marketing authorization for OCA as a treatment option for NASH patients with compensated cirrhosis, in the United States and international markets on the basis of the positive results from the study. A subsequent outcome trial is also in the cards to confirm the clinical benefit on a post-marketing basis in a broader population of NASH patients with cirrhosis.
We note that OCA is already approved as Ocaliva in combination with ursodeoxycholic (“UDCA”), for the treatment of primary biliary cholangitis (“PBC”) in adults with an inadequate response to UDCA or as a monotherapy in adults unable to recuperate with UDCA. The initial uptake of Ocaliva has been encouraging. However, there has been a lot of debates regarding the drug’s safety. Prescription demand softened following the Dear Health Care Provider letter and the FDA safety communication on Ocaliva.
Intercept is also evaluating OCA to treat a variety of other non-viral progressive liver diseases such as NASH, primary sclerosing cholangitis and biliary atresia.
Intercept’s stock has lost 47.9% in the last six months compared with the industry’s decline of 5%. In particular, the company’s shares have plunged significantly due to safety issues regarding the approved drug Ocaliva.
The company initiated a phase III study (REGENERATE) on Ocaliva for the treatment of non-cirrhotic NASH in patients with advanced liver fibrosis. Enrollment for the interim analysis cohort in the REGENERATE trial was completed. CONTROL study is being conducted to evaluate the effect of Ocaliva in combination with statin therapy on lipid metabolism in patients with NASH. Results from AESOP revealed that OCA met the primary endpoint of statistically significant reduction in alkaline phosphatase while results from CONTROL showed that the company achieved its objective in demonstrating that the lowest available dose of atorvastatin rapidly reverses OCA associated LDL changes to below baseline levels in NASH patients with fibrosis or cirrhosis.
Given the challenges faced by the drug in recent times, the label expansion of OCA will boost the growth prospects.
Per estimates, NASH is expected to surpass hepatitis C as the leading reason for liver transplants in the United States and Europe. NASH market has huge potential and a tentative approval will boost Ocaliva’s prospects. However, bigwigs like Novartis AG (NVS - Free Report) and Gilead Sciences (GILD - Free Report) have FXR agonists in phase II or earlier stages of clinical or preclinical development that can be used to treat PBC, NASH and the other liver diseases.
Zacks Rank & Key Pick
Intercept currently carries a Zacks Rank #3 (Hold).
A better-ranked stock in the healthcare sector is Exelixis (EXEL - Free Report) which currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Exelixis’ earnings per share estimates increased from 72 cents to 77 cents for 2018 over the last 60 days.
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