Intercept Pharmaceuticals Inc (NASDAQ:ICPT) announced additional results of the phase 2 clinical trial with obeticholic acid (OCA), a potent farnesoid X receptor agonist as a potential treatment for patients with primary sclerosing cholangitis (PSC). The results were presented today during a poster presentation at the Liver Meeting 2017, the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD), in Washington, D.C.
AESOP is a 24-week, double-blind, placebo-controlled, dose-ranging trial evaluating the efficacy and safety of OCA compared to placebo in 77 patients with PSC. Patients were randomized to one of three treatment groups: placebo, OCA 1.5-3 mg, and OCA 5-10 mg (with dose titration occurring at the 12-week midpoint).
OCA achieved the primary endpoint of the AESOP trial: patients receiving 5 mg of OCA daily with the option to titrate to 10 mg achieved a statistically significant reduction in alkaline phosphatase (ALP) as compared to placebo at week 24 (p<0.05). The results from this dose-ranging study suggest that 5 mg may be the optimal titrated dose of OCA in this patient population.
Patients in the OCA 1.5-3 mg group also achieved statistically significant reductions in ALP versus placebo as measured by LS mean percent change from baseline at week 24. By week 24, ALP increased 1% in the placebo group and decreased by 22% in both the OCA 1.5-3 mg and OCA 5-10 mg groups (p<0.05).
There are currently no approved medications for PSC. Some patients are treated with ursodeoxycholic acid (UDCA) even though the AASLD treatment guidelines for PSC recommend against its use. In AESOP, a significant proportion of patients used UDCA, with 48%, 48% and 46% of patients on placebo, OCA 1.5-3 mg and OCA 5-10 mg, respectively, receiving UDCA at baseline.
In a post-hoc analysis examining the effects of OCA in the presence and absence of UDCA, ALP reductions were observed with OCA regardless of treatment with UDCA. Patients receiving OCA monotherapy had greater reductions in ALP at week 12 and week 24 as compared to patients who received OCA in addition to UDCA. At week 12, patients in the OCA 5-10 mg group receiving OCA monotherapy achieved a 30% LS mean reduction in ALP as compared to a 16% reduction in patients receiving OCA in combination with UDCA. At week 24, LS mean reductions in ALP in the OCA 5-10 mg group were 25% for patients receiving OCA monotherapy and 14% for patients receiving OCA in combination with UDCA.
Pruritus is a common symptom of PSC and was the most common adverse event observed in AESOP, occurring in 46%, 60% and 67% of patients in the placebo, OCA 1.5-3 mg and OCA 5-10 mg groups, respectively.
A two-year open-label extension of AESOP remains ongoing. Of those patients who completed the double-blind phase of the AESOP trial, 97% chose to participate in the open-label extension phase.
“There is an urgent need for effective therapies in PSC, a rare cholestatic liver disease which can lead to cirrhosis, cholangiocarcinoma and premature mortality,” said Dr. Kowdley. “These proof-of-concept results from the AESOP trial are encouraging and represent an important contribution to the growing momentum in PSC research. Further analyses of the AESOP results will help us better understand the effects of OCA in key subpopulations of interest.”
Corporate presentation July 2017
Shares of Intercept are up nearly 3% in after-hours trading Monday. ICPT has a 1-year high of $139.44 and a 1-year low of $54.98. The stock’s 50-day moving average is $78.92 and its 200-day moving average is $106.95.
On the ratings front, Intercept stock has been the subject of a number of recent research reports. In a report issued on October 20, Needham analyst Alan Carr reiterated a Hold rating on ICPT, without suggesting a price target. On October 6, Seaport Global’s Vernon Bernardino initiated coverage with a Buy rating on the stock and has a price target of $136.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Alan Carr and Vernon Bernardino have a yearly average return of 7.8% and 4.7% respectively. Carr has a success rate of 47% and is ranked #630 out of 4703 analysts, while Bernardino has a success rate of 32% and is ranked #1401.
Sentiment on the street is mostly bullish on ICPT stock. Out of 16 analysts who cover the stock, 8 suggest a Buy rating , 6 suggest a Hold and 2 recommend to Sell the stock. The 12-month average price target assigned to the stock is $138.58, which implies an upside of 107% from current levels.
Intercept is a biopharmaceutical company, which engages in the research, development, and commercialization of novel therapeutics in treating chronic liver diseases. Its product pipeline is OCALIVA which is used for the treatment of primary biliary cholangitis, nonalcoholic steatohepatitis, primary sclerosing cholangitis, and biliary atresia.