SAN DIEGO, Sept. 8, 2014 /PRNewswire/ -- Mast
Therapeutics, Inc. (NYSE MKT: MSTX) today reported top-line results
from a Phase 2 study of AIR001 (sodium nitrite) inhalation solution
for the treatment of pulmonary arterial hypertension (PAH).
Mast obtained the rights to the AIR001 program through its
acquisition of privately-held Aires Pharmaceuticals, Inc. earlier
this year. In the primary efficacy analysis of the Phase 2 study,
all doses showed improvement in median pulmonary vascular
resistance (PVR). In the secondary efficacy analysis, all doses
showed improvements in the median distances obtained in the
6-minute walk test, including clinically-meaningful improvements at
the highest dose level. Additionally, AIR001 was
well-tolerated, with no treatment related serious adverse
events. In particular, methemoglobin levels remained normal
(< 1.5%), which distinguishes AIR001 from safety concerns
associated with intravenously-administered nitrite.
Edwin L. Parsley, D.O., interim
Chief Medical Officer, stated: "These results are promising
and consistent with earlier findings of AIR001 as an agent that can
have a positive effect on hemodynamic parameters in a PH
population. To date, more than 120 individuals have received
AIR001, including patients who have received repeat administration
for as long as 12 months, patients who were treatment naive, and
patients on PAH disease-specific background therapy. Given
the hemodynamic improvements observed, we feel AIR001 may be
uniquely suited to address the serious unmet need facing the large
number of patients with pulmonary hypertension associated with left
heart disease. Consequently, we will be pursuing clinical
development of AIR001 in that indication and plan to support
multiple, institution-sponsored Phase 2a studies that will evaluate
1) acute hemodynamic effects, 2) acute effects versus
placebo on maximum oxygen consumption and exercise hemodynamics,
and 3) inhaled versus intravenous administration of nitrite,
as well as the safety of multiple doses of AIR001, in patients with
PH associated with left heart disease."
Brian M. Culley, Chief Executive
Officer, stated: "We are encouraged by the results seen in the
Phase 2 study of AIR001 and believe they further validate our
acquisition of Aires. The data from the study show benefits
consistent with prior studies and support further development of
AIR001. We look forward to proceeding with the Phase 2a studies in
PH associated with left heart disease and anticipate reporting
preliminary study results as early as the second half of 2015."
About the Phase 2 Study (AIR001-CS05)
The Phase 2
study was a multi-center, open-label, randomized, parallel-dose
study to determine the safety and efficacy of AIR001 in subjects
with PAH. Subjects were randomized into one of three
treatment arms and treated with AIR001 for 16 weeks: 80 mg
once daily after a 2-week "run-in" period of 46 mg once daily; 46
mg four times daily after a 2-week run-in period of 46 mg four
times daily; or 80 mg four times daily after a 2-week run-in period
of 46 mg four times daily. The primary objective of the study
was to evaluate the efficacy of inhaled nebulized AIR001 as
determined by change in pulmonary vascular resistance (PVR) from
baseline to week 16, measured immediately post-completion of AIR001
nebulization. Secondary endpoints included change from
baseline to week 16 in: 6‑Minute Walk Distance (6MWD) assessed
immediately post-completion of AIR001 nebulization (peak), but no
more than 40 minutes after completion of AIR001 nebulization;
hemodynamic measurements of cardiac output, mean right atrial
pressure and pulmonary capillary wedge pressure at peak; N-Terminal
Pro-Brain Natriuretic Peptide (NT-proBNP); hemodynamics and 6MWD at
trough; and quality of life measures.
The study was powered to enroll 90 patients, however, prior to
its acquisition by Mast, Aires discontinued the study due to
capital constraints. Data is available from 29 patients who
enrolled in the study.
About AIR001
AIR001 (sodium nitrite) inhalation
solution, also known as Aironite®, is an
intermittently nebulized formulation of nitrite. Under hypoxic
conditions, AIR001 is converted to nitric oxide. Nitrite mediated
nitric oxide formation has several beneficial effects, including
dilation of blood vessels and reduction of inflammation and
undesirable cell growth.
AIR001 has been granted orphan drug status by the U.S. Food and
Drug Administration and the European Medicines Agency for the
treatment of pulmonary arterial hypertension.
About Mast Therapeutics
Mast Therapeutics, Inc. is a
publicly traded biopharmaceutical company headquartered in
San Diego, California. The
Company is leveraging the MAST (Molecular Adhesion and Sealant
Technology) platform, derived from over two decades of clinical,
nonclinical and manufacturing experience with purified and
non-purified poloxamers, to develop 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 enrolling subjects in EPIC, a pivotal Phase 3
study of MST-188 in sickle cell disease, and in a Phase 2 study to
evaluate whether MST-188 improves the effectiveness of recombinant
tissue plasminogen activator therapy in patients with acute limb
ischemia. The Company also is planning to initiate a Phase 2
clinical study of MST-188 in patients with acute decompensated
heart failure in the first half of 2015 and to announce details of
the study's design later this year. In addition, the Company
is developing AIR001, which it acquired in February 2014, in patients with pulmonary
hypertension associated with left heart disease. 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. Aironite® is a trademark of Aires
Pharmaceuticals, Inc., a wholly-owned subsidiary of Mast
Therapeutics.
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 clinical development plans and anticipated
timing of achievement of development milestones for the Company's
product candidates, including AIR001, such as commencement and
completion of clinical studies and announcement of study
results. 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 AIR001, may not demonstrate adequate safety,
efficacy or tolerability in one or more such studies; 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 institutional review boards or the FDA or other
regulatory agencies to require additional nonclinical or clinical
studies prior to initiation of a planned clinical study of a
product candidate; 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
with its products and may never generate revenue sufficient to
achieve profitability; the risk that the Company is not able to
adequately protect its intellectual property rights relating to the
MAST platform and MST-188 or AIR001 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.
Logo -
http://photos.prnewswire.com/prnh/20120612/LA22456LOGO-a
SOURCE Mast Therapeutics, Inc.