SAN DIEGO, April 13, 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 its wholly-owned subsidiary,
Aires Pharmaceuticals, Inc., has entered into an agreement with
Duke University to provide support for
a multicenter, randomized, double-blind, placebo-controlled Phase 2
clinical study of the Company's product candidate AIR001 in
patients with heart failure with preserved ejection fraction
(HFpEF). This 100-patient study, known as the Inorganic Nitrite
Delivery to Improve Exercise Capacity in HFpEF (INDIE-HFpEF) study,
will be sponsored by Duke Clinical Research Institute (DCRI) as the
Coordinating Center for the Heart Failure Clinical Research Network
(HFN) and conducted at premier clinical centers in the United States that are part of the
HFN.
HFpEF is a common form of heart failure that has not responded
as well to previously tested treatments as heart failure with
reduced ejection fraction. AIR001, a sodium nitrite solution that
is administered by way of intermittent inhalation via nebulization,
was obtained by Mast Therapeutics in 2014 through the acquisition
of then privately-held Aires Pharmaceuticals. The Company
recently reported positive top-line results from a blinded and
randomized Phase 2a clinical study of AIR001 in HFpEF patients
conducted at Mayo Clinic.
The HFN was established by the 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 National Heart, Lung, and Blood Institute
(NHLBI), part of the National Institutes of Health (NIH).
Under the agreement with the HFN Coordinating Center, as previously
anticipated, the Company will provide test materials, nebulizers,
and regulatory, technical and additional financial support.
The Chair of the HFN Executive Committee is Dr. Eugene Braunwald, Distinguished Hersey Professor
of Medicine at Harvard Medical School. The collaborative
scientific leadership for the study will include Dr. Barry Borlaug, the Principal Investigator, and
other investigators from the HFN Regional Coordinating
Centers. The HFN Coordinating Center will be led by Dr.
Adrian Hernandez and Dr.
Kevin Anstrom of the DCRI.
"We appreciate the NIH's, NHLBI's, and HFN's recognition of the
study of AIR001 in heart failure as an appropriate area of
investigation," stated Brian M.
Culley, Chief Executive Officer of Mast Therapeutics, Inc.
"This study will accelerate efforts to define the potential
efficacy of AIR001 for patients who have heart failure with
preserved ejection fraction. This is an area of significant unmet
need. There are more than 1 million heart failure hospitalizations
each year in the U.S., about half are patients with HFpEF, and,
currently, there are no proven effective therapeutic agents
available for this large patient population."
"The investigator initiated Investigational New Drug Application
(IND) has been submitted and DCRI anticipates that the study will
begin recruiting patients in the third quarter of this year,"
continued Mr. Culley. "We look forward to supporting DCRI and the
HFN on this study."
About the INDIE-HFpEF Study
The Inorganic
Nitrite Delivery to Improve Exercise
Capacity in HFpEF (INDIE-HFpEF) study is a randomized,
double-blind, placebo-controlled crossover study to assess 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 evaluation of whether
AIR001 improves (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 conducted at Mayo Clinic, 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 in
February 2016. 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 a Phase 2a study of AIR001 in patients with HFpEF is ongoing and
AIR001 was recently selected by the Heart Failure Clinical Research
Network for evaluation in a 100-patient, multicenter, randomized,
double-blind, placebo-controlled, Phase 2 study 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 the Company's investigational drugs, including
AIR001, and anticipated timing of achievement of development
milestones, such as commencement of clinical studies or progress
with regulatory activities. 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: that the Company is not the sponsor of the INDIE-HFpEF
study and has no control over the protocol for or conduct of the
study, including whether the study will commence or 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, or may incur significant
unanticipated expenses in connection with procuring sufficient
quantities; the uncertainty of outcomes in ongoing and future
studies of the Company's product candidates and that its product
candidates, including AIR001, may not demonstrate adequate safety,
efficacy, or tolerability in one or more such studies, including
INDIE-HFpEF; the Company's ability to obtain and maintain effective
patent coverage and other market exclusivity protections for its
products without infringing on the proprietary rights of others;
that the Company may be required to repay its outstanding debt
obligations on an accelerated basis and/or 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 Company's
ability to complete development of and successfully commercialize
its product candidates and 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.