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Esperion Therapeutics Mendelian Randomization Study Results to Be Presented at ACC

Monday, March 6, 2017   (0 Comments)
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Esperion Therapeutics, Inc. (NASDAQ: ESPR) announced Brian A. Ference, M.D., M.Phil., M.Sc., F.A.C.C., Associate Professor of Medicine, Wayne State University School of Medicine, will present results from Mendelian randomization studies conducted to evaluate the effect of lower LDL-C mediated by genetic variants in the ATP Citrate Lyase (ACL) gene on the risk of cardiovascular events. The abstract, “Genetic Target Validation for ATP-Citrate Lyase Inhibition” will be presented in an oral presentation during the American College of Cardiology (ACC) 66th Annual Scientific Session on Sunday, March 19, 2017 at 8:51 a.m. Eastern Time at the Walter E. Washington Convention Center, Washington, DC in Room 147A. The abstract for this presentation is available on the ACC website at:

The importance of genetic validation of targets for LDL-C lowering and cardiovascular disease risk reduction through Mendelian randomization studies was highlighted at the Proprotein Convertase Subtilisin Kexin 9 (PCSK9) inhibitor Endocrine-Metabolism Advisory Committee meetings in June 2015. Mendelian randomization studies have previously provided genetic target validation for several LDL-receptor-mediated LDL-C lowering therapies including 3-Hydroxy-3-Methyl-Glutaryl-Coezyme A (HMG-CoA) reductase (the enzyme target of statins), Niemann-Pick C1 Like 1 (NPC1L1) (the enzyme target for ezetimibe) and PCSK9is.

In early 2016, Esperion partnered with Dr. Ference to design and conduct Mendelian randomization studies to determine the genetic validity of inhibition of ACL, the enzyme target of bempedoic acid, for LDL-C lowering and the potential for cardiovascular disease risk reduction. The Mendelian randomization studies conducted on ACL by Dr. Ference, as with previous studies of other LDL-C lowering therapies mediated by the LDL receptor, identified a genetic score consisting of independently inherited variants in the ACL gene, which mimic the effect of ACL inhibition. Results from the studies demonstrated that these genetic variants have the same effect on the risk for cardiovascular events per unit change in LDL-C as variants that mimic the effects of statins, PCSK9is and ezetimibe. Dr. Ference and colleagues concluded that an ACL inhibitor such as bempedoic acid should reduce the risk of cardiovascular events by the same amount as statins per unit reduction in LDL-C.


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