Data Presented in Oral Presentation Session at AHA's Scientific Sessions
"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
"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
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.
About Statin Intolerance
According to the USAGE survey, an academic study of approximately 10,000
current and former statin users published in 2012 in the
About the ETC-1002 Clinical Development Program
ETC-1002 is a unique, first-in-class, orally available, once-daily small molecule designed to lower levels of LDL-C and to avoid side effects associated with existing LDL-C lowering therapies. ETC-1002 has a unique dual mechanism of action that works by inhibiting ATP citrate lyase (ACL), a key enzyme in the cholesterol biosynthetic pathway, and activating a complementary enzyme, 5-adenosine monophosphate-activated protein kinase (AMPK). Both enzymes are known to play significant roles in the synthesis of cholesterol and glucose in the liver. By inhibiting cholesterol synthesis in the liver, ETC-1002 causes the liver to take up LDL particles from the blood, which reduces LDL-C levels.
In seven completed Phase 1 and 2 clinical studies, involving more than 300 patients who received ETC-1002, consistent and clinically meaningful reductions in LDL-C cholesterol and high sensitivity C-reactive protein (hsCRP), a key marker of inflammation associated with cardiovascular disease, have been observed. Across all completed clinical studies, ETC-1002 has been well tolerated. To date, one serious adverse event, considered unrelated to ETC-1002, has been observed in 317 patients treated with ETC-1002 at doses of up to 240 mg and up to 12 weeks in duration.
Last month, Esperion initiated ETC-1002-008, the Company's first Phase 2b clinical study. The study is evaluating parallel doses of ETC-1002 over 12 weeks as monotherapy or in combination with ezetimibe in approximately 322 patients with hypercholesterolemia and a history with or without statin intolerance (to two or more statins due to muscle-related adverse events). The goals of this study are to compare the LDL-C lowering efficacy of ETC-1002 with ezetimibe, a common therapy for statin intolerance, and to characterize tolerability. Top-line results are expected in late 2014.
Webcast Briefing Details
Esperion will host a reception on
Forward Looking Statements
This press release contains forward-looking statements that are made
pursuant to the safe harbor provisions of the federal securities laws,
including statements regarding the therapeutic potential of ETC-1002.
Any statements contained in this press release that are not statements
of historical fact may be deemed to be forward-looking statements.
Forward-looking statements involve risks and uncertainties that could
cause Esperion's actual results to differ significantly from those
projected, including, without limitation, the risk that unanticipated
developments could interfere with the development (and
commercialization) of ETC-1002, as well as other risks detailed in
Esperion's filings with the
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