PLYMOUTH (WWJ) — The Plymouth pharma startup Esperion Therapeutics Inc. (Nasdaq: ESPR), Wednesday announced the first patient in a Phase 2b clinical study had received the first dose of ETC-1002, its lead anti-cholesterol drug.
The company said the goal of the study are to compare how well ETC-1002 lowers low-density lipoprotein cholesterol, the so-called bad cholesterol, with a drug called ezetimibe, a drug commonly given to patients who can’t tolerate today’s statin cholesterol-lowering drugs such as Lipitor.
ETC-1002 is an orally administered drug taken once a day designed to reduce bad cholesterol in the blood without the side effects some patients experience with statins.
“We believe ETC-1002 can specifically address the unmet medical needs of these patients, who are at elevated risk of cardiovascular disease,” said Esperion president and CEO Tim Mayleben. “We look forward to reporting top-line results from this study by the end of 2014.”
The randomized, double-blind, multicenter study is evaluating parallel doses of ETC-1002 as stand-alone therapy or in combination with ezetimibe in approximately 322 patients. The primary objective of the study is to assess the LDL-C lowering efficacy of ETC-1002 therapy versus ezetimibe therapy in patients with high cholesterol whether or not they have statin intolerance treated for 12 weeks. Secondary objectives include assessing the effect of ETC-1002 on additional blood fats and other compounds associated with heart health; characterizing the how well tolerated and safe ETC-1002 is; and assessing the dose range of ETC-1002 to inform final Phase 3 trials. The trial is being conducted at approximately 75 clinical sites in the United States.
In June 2013, Esperion announced positive top-line results from a Phase 2a proof-of-concept clinical study of ETC-1002 in patients with hypercholesterolemia with a history of intolerance to two or more statins. The results of that study were used to inform the design of the latest study, which met its goals — demonstrating that ETC-1002 lowered LDL-C by an average of 32 percent compared with an LDL-C reduction of 3 percent in the placebo group (p<0.0001) and was well tolerated.
According to studies, about 12 percent of patients on statins stop therapy, with 62 percent of them citing side effects as the reason for discontinuation. More than 86 percent of patients who discontinued therapy because of side effects cited muscle pain as the primary reason for discontinuing their statin. Based on these data, it is estimated that approximately 6 percent of statin users, or more than 2 million adults in the United States, ceased therapy because of pain and are therefore considered to be statin intolerant. Poor statin adherence can be associated with worse cardiovascular outcomes.
ETC-1002 has shown consistent and clinically significant cholesterol reduction effects in seven earlier studies.