One year ago today the “new” Esperion re-entered the public markets with an initial public offering that raised $80 million and with a singular focus: to rapidly advance the development of ETC-1002, a unique, first-in-class, oral, once-daily low-density lipoprotein cholesterol (LDL-C) lowering therapy for patients with hypercholesterolemia who are unable to tolerate a statin. Since our re-introduction to public investors, we have gained tremendous momentum toward the accomplishment of our goals. The coming year promises to be even more eventful!
ETC-1002 represents a new and exciting approach to lowering LDL-C and reducing cardiovascular disease risk in patients with hypercholesterolemia who are unable to tolerate a statin. Millions of patients in the U.S. have significantly elevated levels of LDL-C and yet are intolerant to statins – meaning they are unable to take statins because of muscle-related side effects. These patients and their physicians need alternative therapeutic options – ideally a therapy that can be given as a pill. Over the past year, ETC-1002 has demonstrated very promising efficacy and tolerability results that suggest it could be just the therapy needed for treatment of this patient population. For example, ETC-1002 has shown consistent and significant reductions in LDL-C of almost 40 percent and similar reductions in levels of high sensitivity C-reactive protein (hsCRP), a key marker of inflammation associated with cardiovascular disease. No other non-statin therapy has ever demonstrated the ability to lower both LDL-C and hsCRP to this degree.
Over the past year, Esperion received recognition from the scientific and medical communities with a number of our preclinical and clinical studies accepted for publication or presentation at major medical meetings and/or in prestigious journals. In particular, I want to highlight a paper in the Journal of Lipid Research that demonstrated, for the first time, the effectiveness of ETC-1002 in reducing chronic inflammation in preclinical models of inflammation. Finally, we completed nonclinical safety studies of ETC-1002, for which there were no unexpected findings and the reports will be submitted to FDA this month.
During the first half of 2014, we strengthened our Board of Directors with the addition of three new board members: two of whom are highly experienced lipid experts whose vast experience will serve us well as we continue to develop ETC-1002. Antonio M. Gotto, M.D., co-chairman of the Board of Overseers of the Joan and Sanford I. Weill Medical College of Cornell University and vice president of Cornell University, joined in January and has extensive experience in atherosclerosis and lipid regulation, and prior experience working with us at the original Esperion. Mark E. McGovern, M.D. joined in February and is a board-certified cardiologist with more than 20 years of successful experience in the development and regulatory approval of lipid regulating therapies. Gilbert Omenn, M.D., Ph.D. joined the board this week and is Professor of Computational Medicine & Bioinformatics, Internal Medicine, Human Genetics and Public Health, and Director of the Center for Computational Medicine and Bioinformatics at the University of Michigan. Dr. Omenn has a range of experience in the healthcare community in Michigan and around the world, and will provide Esperion with key scientific and policy insights as we advance development of ETC-1002.
Esperion also received recognition from the investment community over the past year, presenting at some of the most well-respected healthcare investment banking conferences, and being added to six equity indexes including the NASDAQ Biotechnology Index (NASDAQ: NBI), the Russell Global, 3000, 2000 and Microcap indices, and the MSCI Global Micro Cap indices.
Most recently, we relocated our headquarters back to Ann Arbor, MI into a technologically advanced, open-design space allowing for increased collaboration, connectivity and idea creation – features that align well with our core values.
With the resources from our IPO we are in an excellent position to continue to rapidly advance ETC-1002. In a few short months we expect to report top-line results from our first Phase 2b clinical study in patients with hypercholesterolemia who are unable to tolerate a statin. Additional clinical and nonclinical results will follow soon after and mark the end of a very exciting Phase 2b development program that will position Esperion for an end-of-Phase 2 meeting with FDA in 2015.
Patients with hypercholesterolemia who are statin intolerant are waiting – they need new, better-tolerated and convenient therapies to lower their LDL-C and possibly reduce their risk of cardiovascular disease. We are committed to bringing ETC-1002 to market to meet the needs of the millions of patients with hypercholesterolemia who need more treatment options.
Results of ETC-1002 Phase 2a Clinical Studies
ETC-1002-003 — Published full results online in the Journal of the American College of Cardiology. Findings from this randomized, double-blind, placebo-controlled, multicenter, parallel-group study in patients with hypercholesterolemia showed that ETC-1002 significantly lowered LDL-C levels up to 27 percent across a broad range of baseline triglyceride levels and was well tolerated.
ETC-1002-005 — Published full safety and efficacy results in the journal Arteriosclerosis, Thrombosis and Vascular Biology. This study in patients with hypercholesterolemia and Type 2 diabetes met its primary endpoint, with results demonstrating that ETC-1002 lowered LDL-C by up to 43 percent and was well tolerated. ETC-1002 also was associated with improvements in other cardiometabolic risk factors in this high-risk, difficult-to-treat patient population.
ETC-1002-006 — Presented full results at the 2013 American Heart Association (AHA) Scientific Sessions. This randomized, double-blind, placebo-controlled, multicenter, proof-of-concept clinical study in patients with hypercholesterolemia and a history of intolerance to two or more statins met its primary endpoint. Results demonstrated that ETC-1002 lowered LDL-C by an average of 32 percent and was well tolerated.
ETC-1002-007 — Reported positive top-line results. This randomized, double-blind, placebo-controlled, multicenter study in patients with elevated levels of LDL-C demonstrated that oral, once-daily ETC-1002 achieved incremental LDL-C lowering of 22 percent at eight weeks, compared with 0 percent in the placebo group, when added to 10 mg of atorvastatin (p<0.0001). ETC-1002 was well tolerated over eight weeks of treatment when added to a statin and no serious adverse events were reported.
We reported results from these Phase 2a clinical studies in three scientific poster presentations at the 2014 National Lipid Association (NLA) Annual Scientific Sessions. The posters provided additional information from previously-presented Phase 2 studies of ETC-1002 and new analyses regarding blood pressure lowering in patients with both hypercholesterolemia and mildly elevated blood pressure.