ETC-1002 is an innovative, 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 is absorbed rapidly in the small intestine and enters the liver through cell surface receptors different from those transporters that selectively take up statins.

Once in the liver, ETC-1002 inhibits ACL. Pre-clinical studies show that in the liver, ETC-1002 is converted to a derivative coenzyme, or ETC-1002-CoA, which directly inhibits ACL, a key enzyme that supplies substrate for cholesterol and fatty acid synthesis in the liver.

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

ETC-1002 Phase 1 and 2 Clinical Studies**

Total Subjects: 1072 / Treated: 726


*Average LDL-C % change from baseline
**As of July 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 726 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.