Based on the positive top-line results from Phase I and Phase II studies, Esperion Therapeutics announced in October, 2016, that the global pivotal Phase III program of bempedoic acid (ETC-1002) will include hypercholesterolemia patients on any statin at any dose. The Phase III studies will include hypercholesterolemic patients (i) with/without atherosclerotic cardiovascular disease (ASCVD) and who are statin intolerant (ii) with ASCVD and/or heterozygous familial hypercholesterolemia (HeFH) not adequately treated with current lipid-modifying therapies (iii) with/without ASCVD and in combination with ezetimibe. Esperion’s Phase III strategy could lead to a broad label for bempedoic acid, and provide stiff competition to the much hyped PCSK9 inhibitors.

"I think it's actually got a lot of potential. It is kind of a super ezetimibe."

-Thought Leader, North America

Bempedoic acid is a first-in-class adenosine triphophate citrate lyase (ACL) inhibitor that is being developed as a once-daily, orally administered therapy for lowering LDL-cholesterol. In clinical studies it has shown to reduce LDL-cholesterol by up to 43% as a monotherapy, and up to 48% in combination with ezetimibe. The market opportunity for this drug lies as an add-on therapy for patients on maximally tolerated statins and/or ezetimibe, and those with statin intolerance. However, if approved, it may face tough competition from the highly efficacious PCSK9 inhibitors, Amgen’s Repatha (evolocumab) and Sanofi/Regeneron Pharmaceuticals’ Praluent (alirocumab), and Merck’s CETP inhibitor, anacetrapib (see blogs from my colleague here and here for insights on the market potential of these therapies).

Despite its moderate efficacy, we believe there is a considerable market opportunity for bempedoic acid to fit in as third-line therapy for dyslipidemia as the PCSK9 inhibitors are highly priced injectables and there has been a decline in enthusiasm among physicians towards the novel CETP inhibitor class due to multiple discontinuations. However, competition could be more challenging should anacetrapib substantially reduce cardiovascular (CV) risks in the REVEAL outcomes trial.

Given the current safety, efficacy, and dosing profile of bempedoic acid, we anticipate positive results in a wide range of patients in the CLEAR Phase III program, including CV outcomes benefits, and expect this drug to edge out anacetrapib as third-line treatment option for patient with hypercholesterolemia. More importantly, it will offer an important alternative for patients with statin intolerance. These factors added together suggest that bempedoic acid will gain enough patient share to realize substantial revenues for Esperion.

 

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