AUSTIN, Texas, July 11, 2017 /PRNewswire/ -- (Savara, Inc.
NASDAQ: SVRA), a clinical-stage specialty pharmaceutical company
focused on the development and commercialization of novel therapies
for the treatment of serious or life-threatening rare respiratory
diseases, today reported positive interim results from an ongoing
50-patient open-label Phase 2 study of Aironite in patients with
pulmonary hypertension (PH) from multiple different etiologies. The
results were presented in an invited lecture at the 4th
Annual Drug Discovery and Development Symposium for Pulmonary
Hypertension on July 10th,
2017 by lead investigator Marc A.
Simon, M.D., M.S., F.A.C.C., Associate Professor of
Medicine, Bioengineering, and Clinical Translational Science and
Director, Heart Failure Research / Clinical Hemodynamics Core
Facility, at the Pittsburgh Heart, Lung, and Blood Vascular
Medicine Institute at the University of
Pittsburgh.
In the 41 patients enrolled to date in the study, administration
of Aironite significantly improved multiple hemodynamic measures,
with most pronounced improvements in patients with pulmonary
hypertension due to heart failure with preserved ejection fraction
(PH-HFpEF). In the 10 PH-HFpEF patients analyzed, Aironite
administration resulted in significant overall decreases in right
atrial pressure, systolic and diastolic right ventricular pressure,
systolic and diastolic pulmonary artery pressure, and pulmonary
capillary wedge pressure. Of note, pulmonary capillary wedge
pressure decreased by 7.5 mmHg (95% CI –9.0 to –6.0; baseline
median value 18mmHg; p < 0.05) and mean pulmonary artery
pressure decreased by 7.9 mmHg (95% CI –9.4 to –6.3; baseline
median value 34 mmHg; p < 0.05). Pulmonary artery compliance
(Cpa) was improved by 35% (+0.97 ml/mmHg, 95% CI +0.25 to +1.68; p
< 0.01). Aironite was generally well-tolerated and no
significant safety concerns were identified, supporting the primary
safety outcome of the study.
"These additional results build upon prior interim data
published last year in the Journal of Clinical Investigation, and
demonstrate that Aironite can significantly improve cardiopulmonary
hemodynamics in HFpEF as well as Group 3 PH patients, both clinical
conditions which are inadequately treated by currently approved
medicinal treatments," stated Taneli Jouhikainen, M.D., Ph.D.,
Chief Operating Officer of Savara. "If the observed short-term
improvements are translated into clinically meaningful functional
improvements in our ongoing placebo-controlled studies in HFpEF
patients, we believe the product will have exciting potential to be
advanced towards Phase 3 studies and hopefully an eventual NDA
filing."
About the Phase 2 Study
This is an
institution-sponsored, single-center, open-label Phase 2 study, the
main objective of which is to investigate the hemodynamic effects
and mechanisms of action of Aironite delivered in a single
escalating dose to PH patients who undergo right heart
catheterization. The study will enroll patients with PH from
different etiologies, comprising 20 patients with advanced PH
associated with heart failure with preserved ejection fraction
(HFpEF-PH), 20 patients with pulmonary arterial hypertension who
may be on standard PH therapy, and 10 patients with PH associated
with advanced lung disease. Subjects receive a first dose of 45 mg
of Aironite via nebulizer, with one subsequent escalation of dosage
to 90 mg approximately 60 minutes after the first dose. During the
study, right heart and 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. Changes in hemodynamics and calculated pulmonary and
systemic vascular resistances, as well as pulmonary artery
compliance are performed utilizing standard formulas.
About Aironite
Aironite is a sodium nitrite solution
for inhalation via nebulization that has demonstrated encouraging
results in Phase 2 clinical trials conducted to date in patients
with heart failure with preserved ejection fraction (HFpEF).
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 and Nitrite-mediated NO formation have
multiple beneficial effects, including dilation of blood vessels,
reduction in hemodynamic perturbations during exercise in the
failing heart, and improvements in skeletal muscle bioenergetics
and mitochondrial respiration, resulting in improved efficiency of
oxygen utilization during submaximal exercise.
Savara is also supporting two placebo-controlled
institution-sponsored Phase 2 studies of Aironite in patients with
HFpEF, referred to as the INDIE and the INABLE studies. The main
objective of the INDIE study is to assess the efficacy of Aironite
on maximum exercise capacity (peakVO2), activity levels, and
quality of life after four weeks of dosing, whereas the main
objective of the INABLE study is assess the efficacy of Aironite on
the same outcome measures when used in conjunction with supervised
cardiac rehabilitation exercise training. Both studies are
currently ongoing, and being conducted at leading U.S. based
medical institutions.
About Savara
Savara Inc. is a clinical-stage
specialty pharmaceutical company focused on the development and
commercialization of novel therapies for the treatment of serious
or life-threatening rare respiratory diseases. Savara's pipeline
comprises Molgradex, a Phase 3 stage inhaled granulocyte-macrophage
colony-stimulating factor (or GM-CSF) for pulmonary arterial
proteinosis (or PAP), AeroVanc, an inhaled vancomycin for MRSA
infection in Cystic Fibrosis in preparation for Phase 3, and
Aironite, an inhaled sodium nitrite in Phase 2 development.
Savara's strategy involves expanding its pipeline of potentially
best-in-class products through indication expansion, strategic
development partnerships and product acquisitions, with the goal of
becoming a leading company in its field. Savara's management team
has significant experience in orphan drug development and pulmonary
medicine, in identifying unmet needs, creating and acquiring new
product candidates, and effectively advancing them to approvals and
commercialization. More information can be found at
www.savarapharma.com. (Twitter: @SavaraPharma)
Forward Looking Statements
Savara 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. Such statements include, but are not limited to, statements
relating to that these additional results demonstrate that Aironite
can significantly improve cardiopulmonary hemodynamics in HFpEF as
well as Group 3 PH patients, our belief that the product will have
exciting potential to be advanced towards Phase 3 studies and
hopefully an eventual NDA filing, and that Aironite has
demonstrated encouraging results in Phase 2 clinical trials
conducted to date. Savara may not actually achieve any of the
matters referred to in such forward looking statements, and you
should not place undue reliance on these forward-looking
statements. Because such statements are subject to risks and
uncertainties, actual results may differ materially from those
expressed or implied by such forward-looking statements. These
forward-looking statements are based upon Savara's current
expectations and involve assumptions that may never materialize or
may prove to be incorrect. Actual results and the timing of events
could differ materially from those anticipated in such
forward-looking statements as a result of various risks and
uncertainties, which include, without limitation, risks and
uncertainties associated with the outcome of ongoing clinical
trials for Aironite and Savara's other product candidates, the
ability to project future cash utilization and reserves needed for
contingent future liabilities and business operations, the
availability of sufficient resources for Savara's operations and to
conduct or continue planned clinical development programs, the
ability to obtain the necessary patient enrollment for Aironite and
other product candidates in a timely manner, the timing and ability
of Savara to raise additional equity capital to fund continued
operations; the ability to successfully develop Aironite and
Savara's other product candidates, and the risks associated with
the process of developing, obtaining regulatory approval for and
commercializing drug candidates such as Aironite that are safe and
effective for use as human therapeutics. All forward-looking
statements are expressly qualified in their entirety by these
cautionary statements. For a detailed description of our risks and
uncertainties, you are encouraged to review our documents filed
with the SEC including our recent filings on Form 8-K, 10-Q and
10-K. You are cautioned not to place undue reliance on
forward-looking statements, which speak only as of the date on
which they were made. Savara undertakes no obligation to update
such statements to reflect events that occur or circumstances that
exist after the date on which they were made, except as may be
required by law.
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SOURCE Savara Inc.