As members of the Esperion team developing ETC-1002—a unique, first-in-class, orally available, once-daily small molecule designed to lower low density lipoprotein cholesterol (LDL-C) levels, particularly in individuals with hypercholesterolemia who are intolerant to statins—we are excited any time there is a specific emphasis on, or a new development in, this underserved patient population. In the U.S., it is estimated that up to 20 percent of adults taking statins for elevated LDL-C are intolerant to the therapy. Most statin intolerance is related to muscle pain or weakness (myalgia). Patients who cannot take a statin to control their hypercholesterolemia have few available treatment options and often have very high LDL-C levels.
At the National Lipid Association (NLA) 2014 Scientific Sessions, which we attended earlier this month in Orlando, the topic of statin intolerance was well represented in the scientific program and throughout the meeting. The NLA is the premier medical organization for physicians and scientists focused on enhancing the practice of lipid management in clinical medicine.
- NLA Devoted a Session to Statin Intolerance
A full session was dedicated to statin intolerance. Speakers and panelists discussed the implementation of appropriate assessment and management techniques for muscle-related adverse events associated with lipid-lowering drugs; a review of the mechanism of statin-drug interactions, including the impact on statin-induced muscle side effects; and the effects of statins on muscle strength or exercise performance.
- NLA’s Statin Intolerance Task Force Provided a Definition and Recommendations
The NLA released draft recommendations for patient-centered treatment of dyslipidemia, which include an LDL-C goal of < 100mg/dL for patients at low, moderate and high risk and an LDL-C goal of < 70mg/dL for patients at very high risk. We were impressed that these guidelines are available for review and comment until May 31. The NLA’s Statin Intolerance Task Force presented a “pragmatic definition of statin intolerance: adverse symptoms, signs and/or laboratory abnormalities attributed by the patient and/or provider to a statin and perceived by the patient to interfere with daily life activities. These include predominately muscle-related symptoms with muscle aches being the most common, but also may include other symptoms, liver enzyme increases and isolated muscle enzyme (creatine kinase) increases.” Importantly, the NLA has included the phrase “real or perceived” in further describing the symptoms.The Task Force concluded that statin intolerance requires a patient-centered approach in practice; reducing the dose of statin, switching to a different statin and alternate regimens such as every other day dosing are recommended for patients with statin intolerance; for patients who cannot tolerate a statin using the above strategies, alternate agents alone or in combination may be considered; and innovative approaches to research on statin intolerance are needed.
- Esperion Presented Data on ETC-1002 in Statin Intolerant Patients
Esperion presented three scientific posters at the NLA including the results of our Phase 2 clinical trial of ETC-1002 in patients who are statin intolerant. Specifically, one poster entitled “ETC-1002 lowers LDL-cholesterol and is well tolerated in hypercholesterolemic patients with statin intolerance,” concluded that ETC-1002 lowered LDL-C by 32 percent and reduced levels of hsCRP, a key marker of inflammation, by 42 percent in patients who had failed two or more statins due to muscle-related side effects. ETC-1002 was well tolerated, and muscle-related adverse events were similar in the ETC-1002 and placebo groups. No patients who received ETC-1002 discontinued the study due to muscle-related adverse events. You can view all three posters presented here: http://investor.esperion.com/events.cfm.
We believe the significant attention paid to statin intolerance at the NLA’s annual meeting validates the need for new and better treatment options for the two to seven million U.S. patients with hypercholesterolemia who cannot tolerate statins. Our primary goal is to develop ETC-1002 for this patient population. We are working toward that objective with our ongoing Phase 2b 008 study, a randomized, double-blind, multicenter trial that is evaluating parallel doses of ETC-1002 over 12 weeks as monotherapy or in combination with ezetimibe in approximately 350 patients with hypercholesterolemia and a history with or without statin intolerance (to two or more statins due to muscle-related adverse events). We expect to report top-line results in the fourth quarter of this year.