Intercept Pharmaceuticals Inc (NASDAQ:ICPT) announced its plans for an international Phase 3 trial of obeticholic acid (OCA), the company’s lead FXR agonist, in patients with non-cirrhotic nonalcoholic steatohepatitis (NASH) with liver fibrosis. OCA has received breakthrough therapy designation in this patient population from the U.S. Food and Drug Administration (FDA). In accordance with harmonized advice from the FDA and European Medicines Authority (EMA), the Randomized Global Phase 3 Trial to Evaluate the Impact on NASH with Fibrosis of Obeticholic Acid Treatment (REGENERATE) has been designed as a double-blind, placebo-controlled pivotal Phase 3 clinical trial expected to enroll up to approximately 2,500 patients and assess the potential benefit of OCA treatment on liver-related clinical outcomes. The trial will include a pre-planned interim histology analysis after 72 weeks of treatment in approximately 1,400 patients which is intended to serve as the basis for seeking U.S. and international marketing approvals of OCA for the treatment of NASH patients with liver fibrosis. Intercept will hold a conference call and audio webcast today at 8:00 a.m. ET to discuss aspects of the planned Phase 3 trial. Conference call details are provided below.
The REGENERATE trial will be conducted at approximately 250 centers in North America, Europe and other regions and trial initiation is anticipated in 3Q 2015. Patients will be randomized 1:1:1 to one of placebo, 10 mg of OCA, or 25 mg of OCA, taken once daily. The study population will primarily be comprised of NASH patients with stage 2 or stage 3 advanced liver fibrosis who will be evaluated on the primary efficacy endpoints in the 72-week interim analysis and subsequently. In addition, a relatively small group of NASH patients with stage 1 early liver fibrosis with an increased risk of rapid progression due to concomitant diabetes, obesity or active liver inflammation indicated by elevated ALT will also be enrolled, but not included in the primary endpoint analyses.
The interim analysis after 72 weeks of treatment in approximately 1,400 patients will be conducted on the following co-primary endpoints, which are intended to serve as the basis for seeking U.S. and international marketing approvals: (i) the proportion of OCA-treated patients relative to placebo achieving at least one stage of liver fibrosis improvement with no worsening NASH; and (ii) the proportion of OCA-treated patients relative to placebo achieving NASH resolution with no worsening of liver fibrosis. Additional supportive histologic endpoints and non-invasive markers of liver fibrosis and steatohepatitis will also be evaluated. The REGENERATE trial will remain blinded after the interim analysis and continue to follow patients until the occurrence of a pre-specified number of adverse liver-related clinical events, including progression to cirrhosis, to confirm clinical benefit on a post-marketing basis.
“We are very pleased to announce the key aspects of our planned Phase 3 REGENERATE trial today, a culmination of our analyses of the FLINT trial results in OCA-treated NASH patients with advanced liver fibrosis and extensive collaboration with U.S. and European regulatory agencies,” said Mark Pruzanski, M.D., President and Chief Executive Officer. “There are no approved medicines available to treat the disease which is why NASH is predicted to become the leading indication for liver transplant within the next few years.”
“Recently presented data analyses in patients with NASH and liver fibrosis from the FLINT trial suggest that in addition to OCA’s apparent ability to resolve NASH, OCA appears to meaningfully improve liver fibrosis, particularly in those patients at higher risk of progressing to cirrhosis,” said Vlad Ratziu, M.D., Ph.D., Professor of Hepatology at the Pierre and Marie Curie University, Paris. “A benefit on liver fibrosis is important since studies have consistently shown it to be the best histological predictor of both liver-related complications and all-cause mortality.” (Original Source)
Shares of Intercept Pharmaceuticals closed yesterday at $313.98 . ICPT has a 1-year high of $349.08 and a 1-year low of $128.50. The stock’s 50-day moving average is $277.99 and its 200-day moving average is $214.73.
On the ratings front, Intercept Pharmaceuticals has been the subject of a number of recent research reports. In a report issued on May 11, RBC analyst Michael Yee reiterated a Buy rating on ICPT, with a price target of $490, which implies an upside of 56.1% from current levels. Separately, on the same day, Janney Montgomery Scott’s Chiara Russo maintained a Buy rating on the stock and has a price target of $363.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Michael Yee and Chiara Russo have a total average return of 13.0% and 28.8% respectively. Yee has a success rate of 75.5% and is ranked #194 out of 3606 analysts, while Russo has a success rate of 75.0% and is ranked #674.
In total, 2 research analysts have assigned a Hold rating and 11 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $313.98 which is 32.9% above where the stock closed yesterday.
Intercept Pharmaceuticals Inc is a biopharmaceutical company. The Company is engaged in the development and commercialization of novel therapeutics to treat chronic liver disease utilizing its proprietary bile acid chemistry.