SAN DIEGO, Dec. 6, 2016 /PRNewswire/ -- Mast Therapeutics, Inc. (NYSE MKT: MSTX) today reported
that the first patient has been enrolled in an investigator-sponsored Phase 2 study of the Company's lead product candidate,
AIR001, for the treatment of heart failure with preserved ejection fraction (HFpEF). The Inorganic Nitrite to Amplify the
Benefits and Tolerability of Exercise Training (INABLE-TRAINING) in HFpEF study will evaluate AIR001's potential to improve the
clinical responses to exercise training in individuals with HFpEF. The INABLE-TRAINING study is expected to enroll approximately
68 patients who will undergo 12 weeks of cardiac rehabilitation, including exercise training, and will be randomized to receive
either AIR001 or placebo inhalation solution through the training period. The primary endpoint of the study will be the change in
exercise capacity as measured by peak oxygen consumption.
"We are pleased to report that patient enrollment is underway in this Phase 2 study of AIR001 for the treatment of HFpEF,"
stated Brian M. Culley, Chief Executive Officer of Mast Therapeutics, Inc. "There are now three
Phase 2 studies actively enrolling patients to evaluate AIR001 for the treatment of HFpEF and we remain excited about its
potential in this indication. Data from this and other studies will help guide our clinical and regulatory strategy in this area
of significant unmet need. Currently, HFpEF affects approximately half of the more than 5 million people in the U.S. diagnosed
with heart failure and has no proven effective treatment. Heart failure is a leading cause of morbidity and mortality among the
elderly worldwide and is the primary diagnosis in more than 1 million hospitalizations each year, with medical costs projected to
rise to more than $50 billion in the U.S. alone by 2030."
About the INABLE-TRAINING Study
This is a Phase 2 randomized, double-blind, parallel-group placebo-controlled clinical trial testing whether inhaled AIR001
(sodium nitrite solution), as compared to inhaled placebo, can enhance the benefits from chronic exercise training (ET) in
subjects with HFpEF. All subjects will undergo 12 weeks of ET. Participants will be randomized to receive inhaled AIR001 three
times daily or inhaled sodium chloride (placebo) three times daily during the study period. Study subjects will wear
accelerometry devices to track daily activity levels at home. After 12 weeks of ET as part of standard cardiac rehab, subjects
will repeat the assessment of cardiovascular function and exercise capacity as performed at study entry to assess efficacy at a
final visit.
The Phase 2 study has 2 aims. First, determine whether treatment with inhaled AIR001 in addition to ET for 12 weeks improves
exercise capacity and hemodynamic reserve in HFpEF. Expired gas analysis, inert gas (C2H2) rebreathe, and echocardiography will
be performed during rest and exercise to measure oxygen consumption (VO2), CO, and hemodynamics before and after completion of 12
weeks of ET with inhaled NO2- vs ET with inhaled placebo. Second, determine whether treatment with inhaled AIR001 in addition to
ET for 12 weeks increases daily activity levels and quality of life (QOL), and reduces symptoms of effort intolerance during ET.
Subjects will use externally worn accelerometer devices to track daily physical activity. Tolerability of ET will be assessed by
Borg perceived effort and dyspnea scores. Large and small vessel endothelial function (brachial and digital arteries) and QOL
will also be assessed. Secondary endpoints include cardiac output reserve, peak exercise workload, rest and exercise hemodynamics
assessed by echocardiography, Borg dyspnea and fatigue scores recorded during ET, endothelial function assessed by tonometry and
brachial artery flow mediated dilation, QOL assessed by the Kansas City Cardiomyopathy Questionnaire. (ClinicalTrials.gov
Identifier: NCT02713126)
About AIR001
AIR001 is a sodium nitrite solution for intermittent inhalation via nebulization. Nitrite is a direct vasodilator and can be
recycled in vivo to form nitric oxide (NO) independent of the classical NO synthase (NOS) pathway. Nitrite mediated NO
formation has several beneficial effects, including dilation of blood vessels and reduction of inflammation and undesirable cell
growth. Generation of NO from sodium nitrite is not dependent upon endothelial function and is enhanced in the setting of tissue
hypoxia and acidosis, conditions in which NOS activity typically is depressed. In early clinical studies, AIR001 demonstrated
positive hemodynamic effects with reductions observed in right atrial pressure and pulmonary capillary wedge pressure, as well as
improvements in mean pulmonary artery pressures, cardiac output, and exercise tolerance as measured by six-minute walk distance.
In a randomized, double-blind, placebo-controlled Phase 2a study of AIR001 in patients with heart failure with preserved ejection
fraction (HFpEF) (n=26), the AIR001 treatment group showed a statistically significant decrease in pulmonary capillary wedge
pressure during exercise compared to the control group and AIR001 was generally well-tolerated.
About Mast Therapeutics
Mast Therapeutics, Inc. is a publicly traded biopharmaceutical company headquartered in San Diego,
California. The Company has two clinical-stage investigational new drugs, AIR001 and vepoloxamer. AIR001, a sodium
nitrite solution for intermittent inhalation via nebulization, is in Phase 2 clinical development for the treatment of heart
failure with preserved ejection fraction (HFpEF). More information can be found on the Company's web site at www.masttherapeutics.com. Mast Therapeutics™ and the
corporate logo are trademarks of Mast Therapeutics, Inc.
Forward Looking Statements
Mast Therapeutics cautions you that statements in this press release that are not a description of historical fact are
forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements
may be identified by the use of words referencing future events or circumstances such as "expect," "intend," "plan,"
"anticipate," "believe," and "will," among others. Examples of forward-looking statements in this press release include
statements relating to AIR001's potential to treat HFpEF, the timing of completion of any clinical studies, and the Company's
development plans for AIR001. Forward-looking statements should not be read as guarantees of future performance or results
because they involve the Company's beliefs and assumptions based on currently available information and are subject to
significant known and unknown risks and uncertainties that may cause actual performance and results to differ materially from
expectations indicated by the forward-looking statements. Some of the factors that could cause actual performance or results to
differ include, without limitation: the Company's need for additional funding and the risk that it may not be able to obtain
sufficient additional funding as needed; risks associated with the Company's ability to manage operating expenses; uncertainty
related to the Company's ability to continue to operate as a going concern; risk of an event of default under the Company's debt
facility that could result in the Company being required to repay its outstanding debt obligation and related fees on an
accelerated basis and/or at a time that could be detrimental to the Company's financial condition, operations and/or business
strategy; the impact of significant reductions in the Company's operations on its ability to develop its product candidates or
maintain compliance with laws and regulations relating to public companies; the Company's ability to maintain compliance with
NYSE MKT continued listing standards and policies and to maintain the listing and trading of its common stock on a national
securities exchange; uncertainties inherent in the conduct of clinical studies and the risk that the Company's product candidates
may not demonstrate adequate safety, efficacy or tolerability in one or more clinical studies for approval by regulatory
authorities; the Company's lack of control over investigator-sponsored clinical studies of AIR001, including whether any of the
studies will commence or be completed on anticipated timelines, or at all; the potential for the Company to sell or license part
or all of its assets; the potential for significant delays, reductions, or discontinuation of current and/or planned development
activities if the Company is unable to raise sufficient additional capital as needed; the Company's dependence on third parties
to assist with important aspects of development of the Company's product candidates, including the conduct of clinical and
nonclinical studies, the manufacture and supply of clinical trial material, including drug delivery devices, and the conduct of
regulatory activities, and the risk that such third parties may fail to perform as expected leading to delays in product
candidate development and additional costs; the risk that the Company is not able to obtain or maintain effective patent coverage
or other market exclusivity protections for its products, if approved, or that the use or manufacture of the Company's products
may infringe the proprietary rights of others; and other risks and uncertainties more fully described in the Company's press
releases and its reports filed 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, Inc.