Plymouth-based Esperion Therapeutics Monday announced the results from a Phase 1 study and four preclinical studies for its drug candidate ETC-1002.
The announcement came at the Arteriosclerosis, Thrombosis and Vascular Biology 2011 Scientific Sessions in Chicago.
In the Phase 1 study, treatment with ETC-1002 was shown to be safe and well tolerated and to significantly reduce levels of LDL-C, the so-called bad cholesterol, in subjects with mildly unhealthy levels of cholesterol.
ETC-1002, the company’s lead product candidate, is a novel small molecule that has demonstrated preclinical and clinical activity as a metabolic regulator of imbalances in lipid and carbohydrate metabolism. It is being developed to treat dyslipidemia and other cardio-metabolic risk factors.
Mechanistic studies indicate that treatment with ETC-1002 increases AMP-kinase phosphorylation, inhibits fatty acid and cholesterol synthesis and also enhances fatty acid oxidation. In the Phase 1 study, treatment with ETC-1002 resulted in consistent, dose-related reductions in LDL-C. The randomized, double-blind, placebo-controlled study included 32 subjects treated with daily doses of ETC-1002 up to 120 mg or placebo for 14 days and 21 subjects treated with 120 mg of ETC-1002 or placebo for 28 days.
“Cardio-metabolic diseases remain the No. 1 cause of death in people around the world, and research indicates that an effective agent to manage both plasma lipids and glucose could offer significant benefit to millions of patients,” said Roger Newton, president and CEO of Esperion. “The results of our Phase 1 study show a statistically significant lowering of LDL-C in mildly dyslipidemic subjects and a favorable safety profile, and serve as a solid foundation for clinical evaluation in patients with other cardio-metabolic abnormalities.”
In addition to the results of the Phase 1 study, results from four separate preclinical studies for ETC-1002 were also presented at ATVB this week. In an oral presentation, results from a study of hyperlipidemic hamsters showed that ETC-1002 was able to lower proatherogenic lipoproteins in plasma, reduce adiposity, decrease body weight gain and improve hepatic steatosis. ETC-1002 was also shown to improve plasma lipid profiles, hepatic triglyceride content and glycemic control in a KKAy mouse model of diabetes. Of key importance, these favorable changes resulted in the inhibition of aortic lipid deposition in a LDL receptor-deficient mouse model of atherosclerosis.
“These preclinical studies provide significant additional support for continuing efforts to develop ETC-1002,” Newton said. “A Phase 2 lipid study for ETC-1002 is underway while an additional Phase 2 study to assess the effects of ETC-1002 on glucose control will begin soon. We are aggressively working to advance this promising therapy to late stage clinical research.”
More at www.esperion.com.