Many years ago, as an attending physician in an academic emergency department, I was surprised to see a number of patients presenting with advanced cardiovascular disease (CVD). What made this so unusual was that these patients had no known “traditional” CVD risk factors, such as smoking or high blood pressure, but were all relatively young–in their 30s or 40s–and obese. Their CVD had progressed beyond our ability to reverse years of damage. Oftentimes, I felt helpless and wanted to do something to help prevent people from reaching this stage of disease, so I switched careers and began working in the pharmaceutical industry on the development of new cardiometabolic medicines.
Fast forward to 2013, where CVD remains the number one cause of death and disability in the United States. Among the leading risk factors for CVD is an elevated level of LDL (“bad”) cholesterol, which is also known as hypercholesterolemia.
For the past 13 years I have been working to develop safe and effective drug therapies that can help lower CVD risk in patients — including by lowering elevated LDL-C levels. There is incontrovertible evidence that lowering LDL-C reduces risk for patients with hypercholesterolemia. Specifically, for every 1.6 mg/dL decrease in LDL-C, CVD risk is reduced by 1%. At Esperion we are focused on reducing this risk with ETC-1002, a novel, first-in-class, orally available, once-daily investigational drug.
Based on early findings from our initial clinical studies, I am very excited about the potential for ETC-1002. In seven completed clinical studies, involving more than 300 patients who received our product candidate, ETC-1002 has shown consistent and clinically meaningful reductions in LDL-C.
Our most recent trial, a Phase 2a clinical study, focused on evaluating ETC-1002 in patients with an elevated LDL-C level who were already taking statin therapy. The objective of this study was to see whether adding ETC-1002 to 10 mg of atorvastatin would be well-tolerated and safe. We also wanted to see how effective ETC-1002 was at lowering LDL-C on top of a statin after eight weeks. Of 58 patients in total, 42 were randomized to ETC-1002 and atorvastatin, while 16 took a placebo and atorvastatin. Results of the study showed ETC-1002 to be well-tolerated with no serious adverse events. We also found that ETC-1002 added on to atorvastatin reduced LDL-C by an additional 22 percent, whereas patients in the placebo + atorvastatin group experienced no reduction in their LDL-C.
We plan to study the potential of ETC-1002 further in a larger Phase 2b study of patients with hypercholesterolemia who are already taking a statin but are not yet at their LDL-C goal. These patients will receive multiple dose strengths of ETC-1002 as an add-on to atorvastatin in the study, which is expected to begin in early 2014.
Our goal is for ETC-1002 to be approved by the U.S. Food and Drug Administration for individuals who aren’t able to meet their LCL-C lowering goal–with or without statins. Early signs look promising…and I can’t wait to see what we learn next!