ETC-1002 is a novel, first-in-class, orally available, once-daily LDL-C lowering 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 has the potential to regulate both lipid and carbohydrate metabolism. ETC-1002 appears to work 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.

To date, Esperion has studied ETC-1002 in eight completed clinical trials.

ETC-1002 Phase 1 and 2 Clinical Studies*

Total Subjects: 888 / Treated: 654

ETC-1002 Phase 1 and 2 Clinical Studies Listing for Website 033015


*As of March 2015

Phase 2A Clinical Study Results

Our completed Phase 2a clinical studies have demonstrated significant average LDL-C and hsCRP reductions by up to 40%. High sensitivity C-reactive protein, or hsCRP, is a key marker of inflammation associated with cardiovascular disease.

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 566 patients treated with ETC-1002 at doses of up to 240 mg and up to 12 weeks in duration.

Initially, we intend to seek approval of ETC-1002 for patients with elevated levels of LDL-C who are unable to tolerate statin therapy due to muscle pain or weakness. Subsequently, we expect that we will seek approval of ETC-1002 as an add-on to a statin in a broader population of patients who are unable to achieve their LDL-C goals, despite being on a statin regimen and therefore remain at an increased risk for cardiovascular disease. We believe ETC-1002 could provide a major contribution to reducing risk associated with elevated LDL-C by meeting the unmet needs of millions of patients.