Esperion had an incredible year in 2013 with more successes, both clinically and financially, than in any other year since we were founded in April 2008 as the new Esperion. We executed on all of our milestones – all toward our goal of bringing ETC-1002 to market. A unique, first-in-class, oral, once-daily investigational small molecule, ETC-1002 is designed to lower levels of low-density lipoprotein cholesterol (LDL-C) and avoid the side effects associated with existing LDL-C lowering therapies. Our goal is to develop ETC-1002 for the millions of patients in the United States with hypercholesterolemia who are statin intolerant (i.e., they can’t take statins because of muscle-related side effects) or who have a less-than-anticipated therapeutic response with or without a statin.
Among our many 2013 highlights:
We got the band back together. I re-joined Roger Newton, our founder, executive chairman and chief scientific officer, and other talented Esperion colleagues and advisors to make ETC-1002 and the new Esperion team a player in the evolving LDL-C lowering space.
- Completed a $33 million private financing in April.
- Completed an $80 million initial public offering (IPO) in June.
- Added to the NASDAQ Biotechnology Index, the Russell Global, Russell 3000, Russell 2000 and Russell Microcap Indices, and the MSCI Micro Cap Indices.
We published or presented multiple Phase 2a clinical trial results that, in aggregate, included data from 242 patients treated with ETC-1002. Results demonstrated consistent and significant reductions in LDL-C as high as 43 percent, and statin-like reductions in levels of high sensitivity C-reactive protein (hsCRP), a key marker of inflammation associated with cardiovascular disease.
- Published 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.
- Published full results from our ETC-1002-003 study in patients with hypercholesterolemia in the Journal of the American College of Cardiology. Findings from this randomized, double-blind, placebo-controlled, multicenter, parallel-group study showed that doses of ETC-1002 up to 120 mg significantly lowered LDL-C levels up to 27 percent and was well tolerated.
- Presented full results from our ETC-1002-005 study in patients with hypercholesterolemia and Type 2 diabetes in an oral session at the Arteriosclerosis, Thrombosis and Vascular Biology Scientific Sessions. The study met its primary endpoint, with results showing that ETC-1002 lowered LDL-C by up to 43 percent compared with placebo 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.
- Presented full results from our ETC-1002-006 study in patients with hypercholesterolemia and a history of statin intolerance in an oral presentation at the 2013 Scientific Sessions of the American Heart Association in Dallas. The study met its primary endpoint, demonstrating that ETC-1002 significantly lowered LDL-C compared with placebo by an average of 32 percent and was well tolerated.
- Reported positive top-line results from our ETC-1002-007 study of ETC-1002 as an add-on to statin therapy in patients with hypercholesterolemia. Results showed that oral, once-daily ETC-1002 was well tolerated and 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.
Following completion of our ETC-1002 Phase 2a clinical development program, we started our Phase 2b program.
- Initiated the first clinical study in our Phase 2b program — our ETC-1002-008 study in patients with hypercholesterolemia and a history with or without statin intolerance (to two or more statins due to muscle-related adverse events). The goals of this study are to compare the LDL-C lowering efficacy of ETC-1002 with ezetimibe — a common therapy for statin intolerance — and to characterize tolerability.
New LDL-Cholesterol Guidelines
In November, the American College of Cardiology and the American Heart Association published new guidelines on the treatment of blood cholesterol to reduce atherosclerotic cardiovascular disease (ASCVD) risk in adults. Since we are developing ETC-1002 to lower LDL-C levels, we were pleased to see in the new guidelines that LDL-C was positioned as the most important modifiable lipid risk factor for cardiovascular disease. The guidelines refocus attention on LDL-C as a key independent modifiable risk factor for ASCVD; reinforce the importance and primacy of LDL-C lowering – rather than triglycerides and HDL-C; and highlight the role of LDL-C levels and percent reduction to assess response to therapy and adherence.
We also were happy to see that statin intolerance was recognized for the first time. Patients who are fully intolerant to statins cannot take a statin at all, and the new guidelines emphasize the need for alternatives for these patients and their physicians.
Finally, the guidelines acknowledge that some patients are unable to tolerate a less-than-recommended intensity of a statin and that it is important to recognize and manage other cardiovascular risk factors beyond just LDL-C, including blood glucose and blood pressure. We are eager to observe the evolution of the new guidelines and how physicians, patients and payors alike respond to and adopt them.
2014 and Beyond
We are building a great and enduring company and believe that the best is definitely yet to come! We are looking forward to delivering on a number of value-enhancing milestones including:
- Completing our ETC-1002-008 Phase 2b study and reporting top-line data by the end of 2014.
- Initiating our ETC-1002-009 Phase 2b clinical study in the first quarter of 2014 and reporting top-line results by the end of 2014. This study — of parallel doses of ETC-1002 over 12 weeks added on to statin therapy in patients with hypercholesterolemia — is designed to demonstrate the ability of ETC-1002 to achieve incremental LDL-C lowering in statin resistant patients with elevated levels of LDL-C.
- Reporting results from a broad range of non-clinical studies.
We strongly believe in the importance of patients with hypercholesterolemia having more and better options to lower their LDL-C, and we are developing ETC-1002 as one of those options. We believe that ETC-1002, if approved, could provide a major contribution by reducing elevated LDL-C levels, beneficially impacting other cardiometabolic biomarkers and meeting the unmet needs of millions of patients.
We look forward to keeping you updated in the New Year.