Hearts & Minds Blog

ACC.14: Spotlighting the Importance of Finding New Therapies for Statin Intolerant Patients

Roger Newton, PhD, FAHA

Executive Chairman and Chief Scientific Officer

Late last month, the American College of Cardiology (ACC) held its 63rd Annual Scientific Session & Expo with more than 13,000 cardiovascular experts coming together in Washington, D.C. As I have for many years, I traveled to ACC.14 with my Esperion colleagues to hear firsthand the latest research findings in the prevention, diagnosis and treatment of cardiovascular disease (CVD).

I especially enjoyed this year’s conference, given that I have spent my career researching and developing LDL-C lowering treatments, and that our lead product candidate, ETC-1002, is in development for individuals with hypercholesterolemia who are intolerant to statins. It was exciting to see the growing focus on statin intolerance, both in research, clinical results, and even in my informal one-on-one conversations in the hallways of the Convention Center.

I believe that the attention on statin intolerance at ACC.14 signals a new era in the treatment of patients at risk of CVD, and further validates the need to develop new and better treatment options for lowering LDL-C.

Statin intolerance is estimated to affect as many as 20 percent of adults taking statins in the United States, or two to seven million people. This is an extremely important unmet need, as poor adherence to statins can be associated with worse CVD outcomes. That is why our team at Esperion is focusing all of our efforts to further develop ETC-1002. Not only does ETC-1002 work differently from the PCSK9 inhibitors in terms of its mechanism of action, but it would be given as a pill, unlike PCSK9 inhibitors which will be delivered by injection. Another potential benefit is that ETC-1002 doesn’t appear to accumulate in the muscle leading to myalgia in some patients, as statins historically have been known to cause.

Our team has studied ETC-1002 in patients with hypercholesterolemia and a history of statin intolerance and found that it significantly lowered LDL-C by an average of 32 percent compared with placebo. Importantly, no patients receiving ETC-1002 were discontinued from this study for muscle related adverse events.

I strongly believe there is a place for an oral non-statin therapy for patients who cannot tolerate statins, and the Esperion team is committed to continuing to study ETC-1002 in this underserved patient population. The 008 study, a double-blind, randomized, multicenter Phase 2b study, is evaluating parallel doses of ETC-1002 over 12 weeks as monotherapy or in combination with ezetimibe in approximately 322 patients with hypercholesterolemia and a history with or without statin intolerance (to two or more statins due to muscle-related adverse events). I hope to bring ETC-1002 to market for the millions of patients with hypercholesterolemia who cannot tolerate statins.