Business description of Esperion-Therapeutics-Inc from last 10-k form


 Forward-Looking Statements

        This Annual Report on Form 10-K contains forward-looking statements that involve substantial risks and uncertainties. All statements other than statements of historical facts contained in this Annual Report on Form 10-K, including statements regarding our strategy, future operations, future financial position, future revenue, projected costs, prospects, plans, objectives of management and expected market growth, are forward-looking statements. These statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements.

        The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These forward-looking statements include, among other things, statements about:

    •
    our ability to obtain regulatory approval for ETC-1002;

    •
    the timing and outcome of our Phase 2 clinical studies of ETC-1002;

    •
    the timing and outcome of our Phase 3 clinical program of ETC-1002, including two Phase 3 clinical studies and one long-term safety study;

    •
    our ability to replicate positive results from a completed clinical study in a future clinical study;

    •
    our ability to fund our development programs with existing capital or our ability to raise additional capital in the future;

    •
    the potential benefits, effectiveness or safety of ETC-1002, including as compared to statins, the standard of care for LDL-C lowering therapies, other currently available therapies or therapies in development;

    •
    our ability to respond and adhere to changes in regulatory requirements, including any requirement to conduct additional, unplanned clinical studies, such as a cardiovascular outcomes study in connection with our pursuit of ETC-1002 as an LDL-C lowering therapy in the statin intolerant or other patient populations;

    •
    the progress, timing and amount of expenses associated with our development of ETC-1002;

    •
    guidelines relating to LDL-C levels and cardiovascular risk that are generally accepted within the medical community, including recent changes and any future changes to such guidelines;

    •
    reimbursement policies, including any future changes to such policies or related government legislation, and their impact on our ability to sell ETC-1002, if approved;

    •
    the accuracy of our estimates of the size and growth potential of the statin intolerant market and the rate and degree of ETC-1002's market acceptance, if it is approved;

    •
    our ability to obtain and maintain intellectual property protection for ETC-1002 without infringing on the intellectual property rights of others;

    •
    the loss of any of our key scientific or management personnel;

    •
    our intention to seek to establish strategic relationships or partnerships; and

    •
    our ability to compete with other companies that are, or may be, developing or selling products that may compete with ETC-1002, if approved.

        These forward-looking statements are only predictions and we may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, so you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements we make. We have based these forward-looking statements largely on our current expectations and projections about future events and trends that we believe may affect our business, financial condition and operating results. We have included important factors in the cautionary statements included in this Annual Report on Form 10-K, particularly in Item 1.A. Risk Factors, that could cause actual future results or events to differ materially from the forward-looking statements that we make. Our forward-looking statements do not reflect the potential impact of any future acquisitions, mergers, dispositions, joint ventures or investments we may make.

        You should read this Annual Report on Form 10-K and the documents that we have filed as exhibits to the Annual Report on Form 10-K with the understanding that our actual future results may be materially different from what we expect. We do not assume any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by applicable law.


PART I

        All brand names or trademarks appearing in this report are the property of their respective holders. Unless the context requires otherwise, references in this report to "Esperion" the "Company," "we," "us," and "our" refer to Esperion Therapeutics, Inc.

Item 1.    Business

Overview

        We are a clinical stage biopharmaceutical company focused on developing and commercializing first-in-class, oral, low-density lipoprotein cholesterol (LDL-C) lowering therapies for the treatment of patients with hypercholesterolemia and other cardiometabolic risk markers. ETC-1002, our lead product candidate, is a unique, first-in-class, orally available, once-daily small molecule designed to lower LDL-C levels and avoid the side effects associated with other LDL-C lowering therapies currently available. ETC-1002 is being developed primarily for patients intolerant of statins with elevated levels of LDL-C. Phase 2b clinical trials for ETC-1002 are currently underway and build upon a successful and comprehensive Phase 1 and Phase 2 program. We own the exclusive worldwide rights to ETC-1002 and our other product candidates.

        Statins are the current standard of care for LDL-C lowering for approximately 34 million patients in the United States. However, it is estimated that 2 - 7 million U.S. adults are intolerant of statin therapy due to muscle pain or weakness associated with statin therapy. We believe that ETC-1002, if approved, has the potential to become the preferred once-daily, oral therapy for patients who are unable to tolerate statin therapy. We also believe, because symptoms of muscle pain or weakness occur in up to 20% of patients on statin therapy in clinical practice, that the size of the statin intolerant market is poised to grow as effective non-statin therapies become available.

        In October 2013, we initiated our Phase 2b clinical study in hypercholesterolemic patients with or without statin intolerance (ETC-1002-008), the first clinical study in our Phase 2b program. The ETC-1002-008 study is a 12-week Phase 2b study in approximately 322 patients who are either statin intolerant or statin tolerant. Patients enrolled in the ETC-1002-008 study will complete a five week placebo run-in period and will then be randomized to one of five arms: 1) 120 mg dose of ETC-1002, 2) 180 mg dose of ETC-1002, 3) an active comparator, 10 mg dose of ezetimibe, 4) a combination of 120 mg of ETC-1002 and ezetimibe, or 5) a combination of 180 mg of ETC-1002 and ezetimibe. This Phase 2b clinical study is a parallel dose design with a 12-week duration. The primary objective is to assess the LDL-C lowering efficacy of ETC-1002 monotherapy versus ezetimibe monotherapy in patients with elevated LDL-C levels with or without statin intolerance. In addition, the study will assess the LDL-C lowering efficacy of ETC-1002 in combination with ezetimibe versus ezetimibe monotherapy. We expect to complete the study by the end of 2014.

        We were founded in January 2008 by former executives of and investors in the original Esperion Therapeutics, Inc., a biopharmaceutical company, which was primarily focused on the research and development of therapies to regulate high-density lipoprotein cholesterol, or HDL-C. After successfully completing a Phase 2a clinical study with its synthetic HDL therapy, the original Esperion was acquired by Pfizer Inc. in 2004. ETC-1002 was first discovered at the original Esperion and we subsequently acquired the rights to the product from Pfizer in 2008.

Our Strategy

        Our objective is to be a leader in the discovery, development and commercialization of novel therapies for the treatment of patients with hypercholesterolemia and intolerance to statin therapy. The core elements of our strategy include:

    •
    Rapidly advance the clinical development of ETC-1002 as a novel, first in class, orally available, once-daily, small molecule therapy for hypercholesterolemic patients who are statin intolerant.  In November 2013 at the Scientific Sessions of the American Heart Association, we presented efficacy and safety results from ETC-1002-006, our Phase 2a clinical study in patients with elevated LDL-C and a history of intolerance to two or more statins. We initiated a Phase 2b clinical study in approximately 322 statin intolerant and statin tolerant patients in October 2013 and expect to report top-line results by the end of 2014. This Phase 2b clinical study includes a comparison with Zetia® (ezetimibe), which we believe is currently the most prescribed non-statin LDL-C lowering therapy. Zetia's worldwide sales total more than $2.5 billion, approximately half of which are estimated to be for the treatment of statin intolerant patients. While we have not yet completed any comparative clinical studies, Zetia has reported LDL-C lowering of up to an average of 18% in two pivotal clinical studies and ETC-1002 has demonstrated LDL-C lowering up to an average of 43% in clinical studies to date. Because of its superior LDL-C lowering and an attractive safety and tolerability profile, we believe that ETC-1002, if approved, has the potential to become the preferred orally available, once-daily LDL-C lowering small molecule therapy for hypercholesterolemic patients who are unable to tolerate statin therapy.

    •
    Demonstrate ETC-1002's potential as an add-on therapy for hypercholesterolemic patients who cannot achieve their LDL-C goals despite the use of statin therapy.  In September 2013, we announced top-line safety, tolerability, pharmacokinetics and efficacy results from ETC-1002-007, our Phase 2a clinical study using increasing doses of ETC-1002 as an add-on to atorvastatin calcium. In March 2014, we expect to initiate a Phase 2b clinical study (ETC-1002-009) in approximately 132 patients with hypercholesterolemia who will also be taking a statin. Patients in our Phase 2b clinical study will receive two dose strengths of ETC-1002 as an add-on to low to moderate doses of the four most commonly prescribed statins, which include, atorvastatin, pravastatin, simvastatin and rosuvastatin.

    •
    Develop ETC-1002 for LDL-C lowering in targeted patient populations, and develop our other product candidates to treat other cardiometabolic risk markers in additional patient populations.  We may initiate additional clinical studies to explore ETC-1002 as a potential therapy for patients with multiple cardiometabolic risk markers, including elevated levels of hsCRP, blood glucose, and blood pressure. In addition, we may advance the clinical development of two early-stage product candidates to which we own the exclusive worldwide rights: ESP41091, a small molecule oral therapy; and 4WF, a synthetic apoA-I mimetic targeted for patients with acute coronary syndrome.

    •
    Maintain flexibility in commercializing and maximizing the value of our development programs.  We may enter into strategic relationships with biotechnology or pharmaceutical companies to optimize the value of ETC-1002 or our other earlier-stage development programs. For ETC-1002, we may enter into one or more strategic relationships to access broader geographic markets, pursue broader LDL-C lowering indications and populations or pursue indications outside of LDL-C lowering.