Intercept Pharmaceuticals, Inc. (NASDAQ:ICPT) announced results from CONTROL, a placebo-controlled trial to prospectively characterize the lipid metabolic effects of obeticholic acid (OCA) and concomitant statin administration in patients with nonalcoholic steatohepatitis (NASH) with fibrosis or cirrhosis. The CONTROL trial met its primary objective by showing that newly initiated treatment with atorvastatin rapidly reversed OCA-associated increases in LDL to below baseline levels. Most of the effect was observed four weeks after initiation of the lowest available dose of atorvastatin and was sustained throughout the study period.
CONTROL is a 16-week double-blind, placebo-controlled, dose-ranging study of 84 NASH patients with fibrosis and compensated cirrhosis, followed by a two-year long term safety extension (LTSE) open label phase which is currently ongoing. Lipid changes were assessed every four weeks over the course of the double-blind phase. Details of the study design are as follows:
- Statin-naïve or washout patients were randomized to receive one of three doses of OCA (5 mg, 10 mg or 25 mg) or placebo.
- At week four, the lowest approved dose of atorvastatin (10 mg) was added in all patients.
- At week eight, patients were titrated to the next highest prescribed dose of atorvastatin (20 mg).
- At week 12, further titration of atorvastatin (up to 40 mg) was permitted at investigators’ discretion.
The study was designed to measure treatment differences within each group relative to baseline. The intent-to-treat (ITT) analysis is shown below and includes all patients who received at least one dose of study medication.
At week four, mean LDL levels increased in each of the OCA treatment groups, while remaining relatively unchanged in the placebo group. The addition of 10 mg of atorvastatin rapidly reversed mean LDL to below baseline levels in all OCA treatment groups at the first assessed time point (week eight), and this effect was sustained through week 16. The observed mean LDL reductions in the OCA groups were approximately 40 — 45 mg/dL while placebo was 48 mg/dL.
Lipid sub-fraction analysis showed that OCA-related increases in LDL were primarily driven by an increase in large buoyant LDL particles rather than small dense LDL particles. Changes in other lipid parameters were similar to those previously reported with OCA therapy in patients with NASH.
Mild to moderate pruritus was the most common adverse event in patients treated with OCA, occurring in 5%, 5%, 10% and 55% in placebo, 5 mg, 10 mg and 25 mg OCA groups, respectively. Two patients discontinued treatment in the 25 mg OCA treatment arm due to pruritus. Co-administration of atorvastatin and OCA was generally well tolerated and did not result in any unexpected safety observations.
The proportion of patients completing the double-blind period was similar across treatment groups (100%, 95%, 90% and 91% for placebo, OCA 5 mg, OCA 10 mg and OCA 25 mg, respectively). Of these patients, 77 of 79 (97%) chose to participate in the LTSE phase.
During the ongoing LTSE phase, there has been one patient death due to acute renal and liver failure. While Intercept determined it could not be ruled out that this was possibly related to treatment, the principal investigator and the independent Data Safety Monitoring Committee determined the death was unlikely related to OCA.
“The majority of NASH patients in CONTROL were statin eligible according to AHA treatment guidelines, and statins are recommended for patients with NASH in both AASLD and EASL treatment guidelines,” said David Shapiro, M.D., Chief Medical Officer of Intercept. “In CONTROL, we have shown that statin therapy can have an important role in managing LDL when co-administered with OCA in NASH patients with fibrosis and cirrhosis.”
Shares of Intercept Pharmaceuticals are down $129.50, down $5.53 or -4.10%. ICPT has a 1-year high of $177.93 and a 1-year low of $96.63. The stock’s 50-day moving average is $125.38 and its 200-day moving average is $118.26.
On the ratings front, Intercept has been the subject of a number of recent research reports. In a report issued on July 21, BMO analyst Ian Somaiya reiterated a Buy rating on ICPT, with a price target of $218, which implies an upside of 61% from current levels. Separately, on July 17, Oppenheimer’s Jay Olson maintained a Buy rating on the stock and has a price target of $200.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Ian Somaiya and Jay Olson have a yearly average return of 12.8% and 13.8% respectively. Somaiya has a success rate of 61% and is ranked #259 out of 4627 analysts, while Olson has a success rate of 68% and is ranked #553.
Overall, one research analyst has rated the stock with a Sell rating, 2 research analysts have assigned a Hold rating and 5 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 $181.86 which is 34.7% above where the stock closed on Friday.
Intercept Pharmaceuticals 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.