SAN DIEGO, Aug. 1, 2016 /PRNewswire/ -- Mast
Therapeutics, Inc. (NYSE MKT: MSTX), a biopharmaceutical company
developing novel, clinical-stage therapies for sickle cell disease
and heart failure, today reported that the first patient has been
enrolled in a multicenter, randomized, double-blind,
placebo-controlled Phase 2 clinical study of AIR001 in patients
with heart failure with preserved ejection fraction (HFpEF). The
100-patient study, known as the Inorganic Nitrite Delivery to
Improve Exercise Capacity in HFpEF (INDIE-HFpEF) study, is
sponsored by Duke Clinical Research Institute (DCRI) as the
Coordinating Center for the Heart Failure Clinical Research Network
(HFN) and is being conducted at premier clinical centers that are
part of the HFN.
HFpEF is a common form of heart failure. Earlier this year, the
Company reported positive top-line results from a blinded and
randomized Phase 2a clinical study of AIR001 in HFpEF patients as
well as positive interim results from an ongoing Phase 2a clinical
study of AIR001 in patients with pulmonary hypertension associated
with HFpEF.
"AIR001, a sodium nitrite solution administered via inhalation,
has potential as an effective treatment for heart failure, and we
are pleased that patient enrollment is underway," stated
Brian M. Culley, Mast Therapeutics'
CEO. "This study will advance our efforts to characterize the
efficacy of AIR001 for patients who have heart failure with
preserved ejection fraction. There are more than 1 million heart
failure hospitalizations each year in the U.S., about half of which
involve patients with HFpEF. Currently, no proven effective
therapeutic agents are available for this large patient population.
We look forward to continuing to support DCRI and the HFN on this
study."
The HFN was established by the National Heart, Lung, and Blood
Institute (NHLBI) to expedite clinical research on treatments and
strategies to improve the management of acute and chronic heart
failure. The HFN's work is supported by a grant awarded by the
NHLBI, part of the National Institutes of Health (NIH). The
Company's wholly-owned subsidiary, Aires Pharmaceuticals, Inc., is
providing test materials, nebulizers, and regulatory, technical and
additional financial support for the INDIE-HFpEF study. The Chair
of the HFN Executive Committee is Dr. Eugene Braunwald, Distinguished Hersey Professor
of Medicine at Harvard Medical School.
About the INDIE-HFpEF Study
The INDIE-HFpEF study is a
randomized, double-blind, placebo-controlled crossover study to
evaluate the effect of AIR001 on peak exercise capacity as assessed
by cardiopulmonary exercise testing (CPET). Approximately 100
patients with a diagnosis of HFpEF will be enrolled across
approximately 20 clinical centers in the United States. The
primary endpoint will be the peak oxygen consumption
(VO2) after four weeks of treatment with nebulized
inhaled AIR001 or placebo as assessed by CPET performed at peak
drug levels. Secondary objectives include (i) submaximal activity
tolerance chronically, (ii) quality of life, (iii) chronic filling
pressures as assessed by echocardiography and natriuretic peptide
levels, and/or (iv) ventilator efficiency or submaximal exercise
capacity at peak drug levels, and evaluation of the safety and
tolerability of AIR001.
About the Heart Failure Clinical Research Network
(HFN)
The HFN is an NHLBI clinical research network.
The primary goal of the HFN is to conduct multiple clinical trials
to evaluate treatments and strategies to improve management of
acute and chronic heart failure. The HFN provides a unique platform
for collaborative research by bringing together many premier
centers across North America. HFN
is composed of nine Regional Coordinating Centers and their
affiliated sites, whose investigators provide scientific leadership
in the collaborative development of the HFN's scientific agenda.
The HFN is recognized for robust enrollment in heart failure
clinical trials and high scientific productivity. The goal of
partnering with HFN is to accelerate research and medical
innovation, and provide early results that may improve public
health. More information can be found on the HFN's website,
https://www.hfnetwork.org/.
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 recently completed randomized, double-blind,
placebo-controlled Phase 2a study of AIR001 in 30 patients with
HFpEF, the AIR001 treatment group showed a statistically
significant decrease in pulmonary capillary wedge pressure during
exercise compared to the control group and was generally
well-tolerated.
About Mast Therapeutics
Mast Therapeutics, Inc. is a
publicly traded biopharmaceutical company headquartered in
San Diego, California. The Company
is developing two clinical-stage investigational new drugs for
serious or life-threatening diseases and conditions. Vepoloxamer,
the Company's lead product candidate, is in Phase 3 clinical
development for the treatment of vaso-occlusive crisis in patients
with sickle cell disease and in Phase 2 clinical development for
the treatment of patients with heart failure. Enrollment in
the Company's 388-patient Phase 3 study of vepoloxamer in patients
with sickle cell disease, known as the EPIC study, was completed
earlier this year. Enrollment in the Company's Phase 2 study
of vepoloxamer in patients with chronic heart failure is
ongoing. AIR001, the Company's second product candidate, is
in Phase 2 clinical development for the treatment of patients with
heart failure with preserved ejection fraction (HFpEF). Enrollment
in Phase 2 studies of AIR001 in patients with HFpEF are ongoing,
including a 100-patient, multicenter, randomized, double-blind,
placebo-controlled, Phase 2 study in patients with HFpEF being
conducted by the Heart Failure Clinical Research Network.
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
within the meaning of the Private Securities Litigation Reform Act
of 1995 that are based on the Company's current expectations and
assumptions. Such forward-looking statements may be identified by
the use of forward-looking words such as "intend," "plan,"
"anticipate," "believe," "expect," among others, and include, but
are not limited to, statements relating to prospects for successful
development of AIR001 as a treatment for heart failure
patients. There are a number of factors that could cause or
contribute to material differences between actual events or results
and the expectations indicated by the forward-looking statements.
These factors include, but are not limited to: that the
Company is not the sponsor of the INDIE-HFpEF study and has no
control over the conduct of the study, including whether the study
will be completed on anticipated timelines, or at all; the
Company's reliance on third parties for the manufacture and supply
of test material and nebulizer devices for use in the INDIE-HFpEF
study and that the Company may not be able to supply such material
or devices for the study on a timely basis, or at all; the inherent
uncertainty of outcomes in clinical studies of new investigational
drugs, such as AIR001 and vepoloxamer and the risk that these
product candidates may not demonstrate adequate safety, efficacy or
tolerability in ongoing or future clinical studies; the risk that,
even if current and planned clinical studies are successful, the
FDA or other regulatory agencies may determine they are not
sufficient to support a new drug application; the risk that the
Company is not able to obtain and maintain effective patent
coverage or other market exclusivity protections for its products,
if approved, without infringing the proprietary rights of others;
risks associated with the Company's ability to manage
operating expenses and obtain additional capital as needed; the
Company's potential inability to continue as a going concern if it
does not raise additional capital as needed; uncertainty related to
the Company's ability to remain in compliance with the terms and
restrictions under its debt facility and the potential that it may
be required to repay outstanding debt obligations on an accelerated
basis and/or at a time that could be detrimental to the Company's
financial condition, operations and/or business strategy, including
the prepayment of $10 million of the
principal balance if results from the EPIC study are not positive
and/or not available on or before October 14, 2016; the
potential for the Company to significantly delay, reduce or
discontinue current and/or planned development and
commercial-readiness activities or sell or license its assets at
inopportune times if it is unable to raise sufficient additional
capital as needed; the Company's dependence on third parties to
assist with important aspects of development of its product
candidates, including conduct of its clinical studies and supply
and manufacture of clinical trial material, and, if approved,
commercial product, and the risk that such third parties may fail
to perform as expected, leading to delays in product candidate
development or approval or inability to meet market demand for
approved products, if any; 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; 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, Inc.