Mast To Present New Data Supporting Vepoloxamer (MST-188) In Sickle Cell Disease At 9th Annual Sickle Cell Disease Research And Education Symposium And 38th National Sickle Cell Disease Scientific Meeting

- Vepoloxamer shown to improve properties of sickle red blood cells
- Reduced fragility / hemolysis
- Reduced adhesiveness
- Company also presenting at Investigational Drug Symposium and at the Technology Session with an overview of its mobile app for the sickle cell community, VOICE Crisis Alert™

SAN DIEGO, April 9, 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 announced that its lead compound (vepoloxamer) was shown in a nonclinical study to result in dose-dependent and statistically significant reductions in the adhesive properties and fragility of red blood cells from patients with sickle cell disease. The results were obtained from a microfluidics-based assessment of blood samples from 12 sickle cell patients. Data from this study will be presented at the 9th Annual Sickle Cell Disease Research & Educational Symposium and 38th National Sickle Cell Disease Scientific Meeting taking place April 10-13, 2015 at the Westin Diplomat in Hollywood, Florida.  

Dr. R. Martin Emanuele, Senior Vice President, Development, said: "These results are encouraging since hemolysis and adhesiveness of sickle red blood cells are thought to be causal pathologies underlying vaso-occlusive crisis. Of note, we observed a strong concentration-related response for both parameters, which supports the veracity of the drug effect. Also, with regard to the inhibition of cell adhesion, the maximal response in this study was observed over the range of blood concentrations we are targeting in our ongoing Phase 3 study. As such, this data is supportive of effects we expect to see in our Phase 3 EPIC study."

Additionally, the Company's CEO, Brian M. Culley, will participate with a presentation entitled, "Epic Considerations for the EPIC Clinical Trial," in the Investigational Drug Symposium, taking place on Sunday, April 12, 2015, chaired by Susan Perrine, M.D., Professor of Medicine, Pediatrics and Pharmacology and Experimental Therapeutics at Boston University School of Medicine.

The Company also will participate in the Symposium's Technology Session entitled, "Striving for a Unified mHealth Strategy for Research and Clinical Care in Sickle Cell Disease," with a presentation by Gregory D. Gorgas, Senior Vice President, Commercialization, that will feature the free mobile app VOICE Crisis Alert™, which was developed by the Company for individuals with sickle cell disease to track and communicate their crises with their healthcare providers and caregivers.

Abstract Information:

  • An abstract entitled, "Evaluation of purified poloxamer-188 (vepoloxamer) on sickle red blood cell (RBC) adhesion and membrane fragility utilizing microfluidic-based flow adhesion bioassays," by Patrick C. Hines M.D., Ph.D., Jennell White, Ph.D., Xiufeng Gao, M.D., and Ke Liu, Ph.D. of Functional Fluidics and R. Martin Emanuele, Ph.D. of Mast Therapeutics, Inc. summarizes the study data to be presented.
  • The abstract is available on the Company's website at

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).

Vepoloxamer is 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 to evaluate whether vepoloxamer 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 (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 relating to prospects for successful development of vepoloxamer in sickle cell disease, including success with the EPIC study, and anticipated timing of commencement of other clinical studies. 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

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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