SAN DIEGO, April 21, 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 arterial disease, today reported that patient enrollment in its pivotal EPIC study of its lead product candidate, vepoloxamer (MST-188), in sickle cell disease has surpassed the halfway point. Consistent with prior guidance, the Company anticipates reporting top-line results in the first quarter of 2016.
"We are extremely pleased to report that, as of yesterday, 205 patients had been randomized to the EPIC study," stated Brian M. Culley, Chief Executive Officer. "Consistent with projections we set for ourselves more than 18 months ago, we remain on track to announce top-line data in the first quarter of 2016. Our clinical operations team has opened more than 70 sites in ten countries, with more than two-thirds of those sites located in the U.S. In the next few months, we will be opening new sites in at least four additional countries."
"We have cultivated relationships with the sickle cell medical and patient communities and continued to add new sites, which has increased awareness about our study. As a result, we are seeing consistent growth in enthusiasm and enrollment," stated Dr. Edwin L. Parsley, the Company's Chief Medical Officer. "Of note, more than 70% of the EPIC sites have enrolled at least one subject and 75% of those sites have enrolled two or more subjects. A majority of patients have come from our U.S. sites and we believe that at the conclusion of EPIC, the majority of the study population will have come from the U.S."
"Notably, although EPIC is open to patients aged 4-65, the average age of randomized patients is 14. We believe the potential benefits of vepoloxamer are not limited to age, but that younger patients may experience a more distinct crisis, providing the opportunity to assess differences accross age groups. In additon, approximately 20% of the randomized patients are 18 and over. Exposures to vepoloxamer across a wide age range will provide safety and efficacy data supportive of a broad label," continued Dr. Parsley.
Mr. Culley added, "There has never been a drug approved to treat an ongoing sickle cell crisis, however, there have been hundreds of clinical administrations with vepoloxamer even prior to this Phase 3 and it is years ahead of other new treatments being developed for sickle cell disease, so I am confident the sickle cell community is hopeful and excited for a positive outcome from this important study."
About the EPIC Study
The EPIC study is a randomized, double-blind, two-arm, placebo-controlled, Phase 3 clinical trial of vepoloxamer in patients with sickle cell disease. The primary objective of the study is to demonstrate that vepoloxamer reduces the duration of vaso-occlusive crisis. Vaso-occlusive crises are recurring episodes of severe pain and the leading cause of hospitalization for patients with sickle cell disease. A total of 388 patients, ages four to 65, will be enrolled in EPIC. The study also will compare the rates of re-hospitalization for vaso-occlusive crisis and occurrence of acute chest syndrome between the treatment and control groups.
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 (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).
In addition to EPIC, vepoloxamer is being tested in a Phase 2 study to evaluate whether it improves the effectiveness of recombinant tissue plasminogen activator therapy in patients with acute limb ischemia. The Company plans to initiate a Phase 2 study of vepoloxamer in chronic heart failure in the third quarter of this year. AIR001 is being tested in multiple 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 include, but are not limited to, statements concerning the Company's anticipated product development activities, such as expected clinical study commencement and completion and announcement of study results, as well as prospects for and anticipated timing of successful development, approval and commercialization of our product candidates, including vepoloxamer in sickle cell disease. 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; 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 potential for additional nonclinical or clinical studies to be required prior to initiation of a planned clinical study; the risk that, even if 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 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 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 and prevent competitors from duplicating or developing equivalent versions of its product candidates; 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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