SAN DIEGO, Oct. 26, 2015 /PRNewswire/ -- Mast Therapeutics, Inc. (NYSE MKT: MSTX), a clinical-stage biopharmaceutical company leveraging its molecular adhesion and sealant technology (MAST) platform to develop novel therapies for sickle cell disease, heart failure, and stroke, announced that it has initiated its Phase 2 study of vepoloxamer for the treatment of patients with chronic heart failure.
Dr. Edwin L. Parsley, Chief Medical Officer of the Company, said: "Previously-announced positive results from multiple randomized, placebo-controlled studies of vepoloxamer in a well-established model of chronic heart failure, including a repeat-treatment study, as well as recommendations from medical experts in the field, strongly support clinical development of vepoloxamer in this setting. If the promising results observed in non-clinical studies translate to patients with chronic heart failure, vepoloxamer may offer a novel way of directly improving left ventricle contractile function by restoring cardiomyocyte membrane integrity and increasing cardiomyocyte survival. Significantly, in this Phase 2 study, vepoloxamer will be administered over 3 hours in an outpatient setting, as opposed to the 49-hour administration in our sickle cell disease studies, which will help demonstrate its practical utility for chronic heart failure patients. We intend to collect echocardiographic data on parameters such as ejection fraction as well as biomarkers associated with clinical outcomes such as ultra-high sensitivity troponin I and NT-proBNP, as these metrics were improved in a statistically significantly manner in previously-announced heart failure models."
Brian M. Culley, Chief Executive Officer of the Company, added: "We are pleased to have initiated this 150-patient Phase 2 study, which, notably, is testing a new formulation of vepoloxamer. We designed the new formulation to be more suitable for heart failure patients and have filed a provisional patent application. We now have multiple pending patent applications, including a new formulation application and a new composition of matter application, which would provide market protection for vepoloxamer in the setting of heart failure. In addition, the new formulation is distinct from our sickle cell formulation, which may provide important commercial advantages."
About the Phase 2 Chronic Heart Failure Study
The Phase 2 study is a randomized, double-blind, placebo-controlled, multicenter study (both within and outside the U.S.) of a new formulation of vepoloxamer in ambulatory patients, ages 18 to 74, diagnosed with chronic heart failure (New York Heart Association functional Class II or III) who are stable and well-managed, on an established guideline-based medication regimen for at least four weeks, have a documented left ventricular ejection fraction (LVEF) ≤35% as the most recent value within the last 12 months, and have an elevated cardiac troponin I (cTnI) blood level. Approximately 150 patients will be randomized into one of three approximately 50-patient study arms and receive one of two dose levels of vepoloxamer or the placebo control, administered in addition to standard medical therapy as a single intravenous infusion over three hours in an outpatient setting or short-stay inpatient unit depending on local practice and resource availability. The purpose of this clinical study is to evaluate whether vepoloxamer can provide both a functional and biochemical benefit to damaged heart muscle cells, which will be evaluated by measurement of echocardiograms and blood-based laboratory markers, and to evaluate the safety and pharmacokinetics of vepoloxamer in chronic heart failure patients compared to placebo control.
About Mast Therapeutics
Mast Therapeutics, Inc. is a publicly traded biopharmaceutical company headquartered in San Diego, California. The Company is leveraging its MAST platform, derived from over two decades of clinical, nonclinical and manufacturing experience with purified and non-purified poloxamers, to develop vepoloxamer (also known as MST-188), its lead product candidate, for serious or life-threatening diseases and conditions typically characterized by impaired microvascular blood flow and damaged cell membranes. The Company is also developing AIR001, a sodium nitrite solution for inhalation via nebulizer, for the treatment of heart failure with preserved ejection fraction (HFpEF).
Vepoloxamer is an investigational new drug being tested in a pivotal Phase 3 study called EPIC for the treatment of vaso-occlusive crisis in patients with sickle cell disease and in a Phase 2 study for the treatment of patients with chronic heart failure. The Company also plans to initiate clinical development of vepoloxamer in ischemic stroke in 2016. AIR001 is an investigational drug being tested in two institution-sponsored Phase 2a studies in patients with HFpEF. More information can be found on the Company's web site at www.masttherapeutics.com. (Twitter: @MastThera)
Mast Therapeutics™ and the corporate logo are trademarks of Mast Therapeutics, Inc.
Forward Looking Statements
Mast Therapeutics cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements that are based on the Company's current expectations and assumptions. Such forward-looking statements may include, but are not limited to, statements relating to prospects for successful development and commercialization of, and patent protection for, the Company's investigational drugs, vepoloxamer and AIR001, and anticipated timing of achievement of development milestones, such as commencement and completion of clinical studies or regulatory activities, and of announcement of study data. Among the factors that could cause or contribute to material differences between the Company's actual results and the expectations indicated by the forward-looking statements are risks and uncertainties that include, but are not limited to: the uncertainty of outcomes in ongoing and future studies of the Company's product candidates and the risk that its product candidates, including vepoloxamer, may not demonstrate adequate safety, efficacy or tolerability in one or more such studies, including EPIC and the Phase 2 study of vepoloxamer in chronic heart failure; delays in the commencement or completion of clinical studies, including as a result of difficulties in obtaining regulatory agency agreement on clinical development plans or clinical study design, opening trial sites, enrolling study subjects, manufacturing sufficient quantities of clinical trial material, being subject to a "clinical hold," and/or suspension or termination of a clinical study, including due to patient safety concerns or lack of funding; the risk that, even if planned clinical studies are successful, the FDA or other regulatory agencies may determine they are not sufficient to support a new drug application; the potential that, even if clinical studies of a product candidate in one indication are successful, clinical studies in another indication may not be successful; the potential for additional nonclinical or clinical studies to be required prior to initiation of a planned clinical study; the Company's reliance on contract research organizations (CROs), contract manufacturing organizations (CMOs), and other third parties to assist in the conduct of important aspects of development of its product candidates, including clinical studies, manufacturing, and regulatory activities for its product candidates, and that such third parties may fail to perform as expected; the risk that the Company may be required to repay its outstanding debt obligations at a time that could be detrimental to its financial condition, operations and/or business strategy; the Company's ability to obtain additional funding on a timely basis or on acceptable terms, or at all; the potential for the Company to delay, reduce or discontinue current and/or planned development activities, including clinical studies, partner its product candidates at inopportune times or pursue less expensive but higher-risk and/or lower return development paths if it is unable to raise sufficient additional capital as needed; the risk that, even if the Company successfully develops a product candidate in one or more indications, it may not realize commercial success and may never achieve profitability; the risk that the Company is not able to adequately protect its intellectual property rights, through patents or otherwise, and prevent competitors from duplicating or developing equivalent versions of its product candidates or that the use or manufacture of its products or product candidates infringe the proprietary rights of others; and other risks and uncertainties more fully described in the Company's press releases and periodic filings with the Securities and Exchange Commission. The Company's public filings with the Securities and Exchange Commission are available at www.sec.gov.
You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date when made. Mast Therapeutics does not intend to revise or update any forward-looking statement set forth in this press release to reflect events or circumstances arising after the date hereof, except as may be required by law.
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SOURCE Mast Therapeutics
Ioana C. Hone (ir@mastthera.com), 858-552-0866 Ext. 303
