Demonstrating that ETC-1002 lowered LDL-C by an average of 32 percent and was well tolerated in patients with hypercholesterolemia and history of statin intolerance
Esperion Therapeutics, Inc. (Nasdaq: ESPR), a clinical-stage biopharmaceutical company focused on developing and commercializing first-in-class, oral, low-density lipoprotein cholesterol (LDL-C) lowering therapies for the treatment of hypercholesterolemia, today announced full results of a Phase 2 clinical study of its lead product candidate ETC-1002 in patients with hypercholesterolemia and a history of statin intolerance. The study, ETC-1002-006, met its primary endpoint, demonstrating that ETC-1002 significantly lowered LDL-C compared to placebo by an average of 32 percent and was well tolerated. The data were presented today in an oral presentation at the 2013 Scientific Sessions of the American Heart Association in Dallas by principal investigator Paul D. Thompson, M.D. Esperion previously announced positive topline results from this study in June 2013.
"To achieve a 32% reduction in LDL cholesterol with a non-statin is very impressive and more reduction than any other currently approved lipid lowering agent except the statins. This drug may have promise for people who have trouble tolerating statins and could be important given that more than 2 million people in the United States are estimated to be statin intolerant," said Dr. Thompson, director of cardiology, at Hartford Hospital, and professor of medicine at the University of Connecticut. "Statin intolerant patients have very few therapeutic options and an oral treatment that significantly lowers LDL-C and is well tolerated could be very useful in this patient population."
"We continue to build on the positive results from this Phase 2 clinical study and recently initiated a large Phase 2b clinical study in statin intolerant and statin tolerant patients," said Tim M. Mayleben, president and chief executive officer of Esperion. "This Phase 2b clinical study will further evaluate the potential of ETC-1002 in statin intolerant patients, a patient population that we believe is underserved by currently available therapies and who remain at elevated risk for cardiovascular disease."
ETC-1002-006 Study Design and Results
This Phase 2a proof-of-concept clinical study was designed to evaluate the LDL-C lowering efficacy, safety and tolerability of ETC-1002 compared with placebo in patients with hypercholesterolemia and a history of intolerance to two or more statins. The study also assessed the ability of patients with a history of statin intolerance to achieve their NCEP ATP-III LDL-C goal. Study participants were dosed for eight weeks starting at 60 mg for two weeks, followed by 120 mg, 180 mg and 240 mg for two weeks each (or placebo only for eight weeks). A total of 56 patients were evaluated in the study, of whom 37 were randomized to receive ETC-1002 and 19 to receive placebo.
Thirty-one ETC-1002 patients and 15 placebo patients completed eight weeks of treatment. Three patients in the placebo group and none in the ETC-1002 group withdrew from the study for muscle-related reasons (e.g., musculoskeletal pain, muscle fatigue, muscle weakness, myalgia).
Final results showed that ETC-1002 lowered LDL-C by statistically significant 32 percent compared with 3 percent in the placebo group (p=0.0001). Approximately two-thirds of patients reached their ATP-III NCEP LDL-C goal. In the ETC-1002 group, high sensitivity C-reactive protein (hsCRP), a recognized marker for inflammation, was significantly reduced after eight weeks by 42 percent (p=0.0022). Levels of ApoB and Non-HDL-C, other important biomarkers, were also significantly reduced.
Overall adverse event rates were comparable between the ETC-1002 and placebo groups, with muscle-related adverse events similar between groups. No serious adverse events (SAE) were observed among placebo patients; one SAE occurred in the ETC-1002 treatment group that was considered unrelated to the study medication.