SAN DIEGO, May 16, 2016 /PRNewswire/ -- Mast
Therapeutics, Inc. (NYSE MKT: MSTX), a biopharmaceutical company
developing novel clinical-stage therapies for sickle cell disease
and heart failure today announced positive interim results from an
ongoing Phase 2a study of AIR001 in patients with pulmonary
hypertension (PH) associated with heart failure with preserved
ejection fraction (HFpEF). The interim results were presented
at the American Thoracic Society (ATS) International Conference by
lead investigator Marc A. Simon,
M.D., M.S., F.A.C.C. The ATS International Conference is being held
May 13 – 18, 2016 at the Moscone
Center in San Francisco,
California.
In the 10 patients who had been studied to date, nebulized
inhaled nitrite (AIR001) administration significantly lowered
central pressures, specifically, right atrial, right ventricular
systolic and diastolic, pulmonary artery (PA)
systolic/diastolic/mean, and pulmonary artery occlusion (PAOP)
pressures. Of note, pulmonary artery occlusion and mean pulmonary
artery pressures were markedly decreased from baseline median
values. In addition, there was an observed increase in pulmonary
artery compliance. There was no significant decrease is systemic
blood pressures or change in heart rate. Methemoglobin levels
increased modestly, but remained less than 1.9% and did not meet
stopping criteria of the study, which was 5%. AIR001 was generally
well-tolerated.
"This is the first report of the acute hemodynamic effects of
multiple inhaled nitrite doses in patients with pulmonary
hypertension due to heart failure with preserved ejection
fraction," stated Dr. Simon. "The interim results observed to date
are important as they demonstrate that AIR001 can significantly
lower right atrial pressures, pulmonary artery pressures, and
pulmonary artery occlusion pressures, as well as improve pulmonary
artery compliance."
"These data are consistent with results we saw in a separate
Phase 2a study of AIR001 in HFpEF earlier this year and are a
further step in validating our second asset and establishing the
potential clinical utility of AIR001 in HFpEF," stated Brian M. Culley, Chief Executive Officer of Mast
Therapeutics. "We look forward to advancing AIR001 in this area of
high unmet medical need for which there is no FDA-approved therapy
available."
Poster Information:
- The poster entitled "Efficacy and safety of inhaled sodium
nitrite in pulmonary hypertension associated with heart failure
with preserved ejection fraction" will be presented by Dr. Simon
at 11:00 a.m. PT on May 16, 2016, at
the Moscone Center in San Francisco,
California.
- A copy of the poster will be available after 11:00 a.m.
PT on the Company's website at:
http://www.masttherapeutics.com/technology/publications/.
About the Phase 2a Study
This is an
institution-sponsored, single-center, open label Phase 2a study to
evaluate the effect of AIR001 delivered in a dose escalation manner
on the change in cardiovascular hemodynamics in subjects with
pulmonary hypertension who undergo standard right heart
catheterization. The study will enroll a total of approximately 50
subjects with pulmonary hypertension. Approximately 20 of the
subjects will have a diagnosis of PH associated with HFpEF (WHO
Group II PH). Subjects receive a first dose of 45 mg of
AIR001 via nebulizer, with one subsequent escalation dosage to 90
mg approximately 60 minutes after the first dose, based on safety
and tolerability. During the study, right heart/pulmonary
artery hemodynamics are measured continuously, and cardiac output
is measured at 15 minute intervals, as well as noninvasive systemic
blood pressure and pulse oximetry monitoring. Changes in
hemodynamics and calculated pulmonary systemic vascular
resistances, as well as pulmonary artery compliance will be
performed.
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 http://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 for patients with HFpEF. 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 interim clinical study
results may not be indicative of final study results and complete
study results may not be positive with regard to efficacy or safety
of AIR001 for patients with HFpEF; that the Company is not the
sponsor of the ongoing Phase 2a study of AIR001 in patients with PH
associated with HFpEF and has no control over the protocol for or
conduct of the study, including whether the study will be completed
on anticipated timelines, or at all; 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; the Company's potential inability to continue as a going
concern if it does not raise additional capital as needed, and its
potential inability to obtain additional funding on a timely basis
or on acceptable terms, or at all; the risk 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,
including the prepayment of $10
million of the principal balance of its debt facility if
results from the Company's Phase 3 study of vepoloxamer in sickle
cell disease are not positive; the potential for the Company to
significantly delay, reduce or discontinue current and/or planned
development activities or sell or license its assets at inopportune
times if it is unable to raise sufficient additional capital as
needed; 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;
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.
Logo: http://photos.prnewswire.com/prnh/20120612/LA22456LOGO-a
To view the original version on PR Newswire,
visit:http://www.prnewswire.com/news-releases/positive-interim-results-from-second-phase-2a-study-of-air001-in-patients-with-heart-failure-with-preserved-ejection-fraction-hfpef-presented-at-american-thoracic-society-international-conference-300268818.html
SOURCE Mast Therapeutics, Inc.