Savara Announces Manuscript on Long-Term Outcomes With Molgramostim Inhalation Solution (Molgramostim) in Autoimmune Pulmonary Alveolar Proteinosis (aPAP) Published in ERJ Open Research
31 January 2025 - 12:39AM
Business Wire
-- Real World, Retrospective Outcomes Data
Suggest Molgramostim Addresses the Underlying Pathophysiology of
aPAP Resulting in Improved Lung Function, Decreased Disease Burden,
Restored Patient Functionality, and Reduction of Clinical
Symptoms--
Savara Inc. (Nasdaq: SVRA) (the Company), a clinical stage
biopharmaceutical company focused on rare respiratory diseases,
today announced a manuscript titled, “Long-Term Outcomes in Five
Patients with Autoimmune Pulmonary Alveolar Proteinosis Treated
with Molgramostim Inhalation Solution" was published online in the
journal ERJ Open Research. The outcomes data presented suggest that
treatment with molgramostim addresses the underlying
pathophysiology of aPAP, resulting in improved lung function,
decreased disease burden, restored patient functionality, and
reduction of clinical symptoms, and may enable resumption of daily
life activities.
This case series retrospectively evaluated five aPAP patients
who received molgramostim through European single-patient access,
supplied by Savara. Following treatment with molgramostim (mean
duration of 4.2 years), improvements in disease severity were shown
across pulmonary gas transfer, measured by percent predicted
diffusing capacity of the lung for carbon monoxide (DLco) and
alveolar-arterial oxygen gradient (A-aDO2), as well as activities
of daily living. Additionally, surfactant burden was reduced as
indicated by high-resolution computed tomography scans taken before
and after molgramostim treatment. Furthermore, while four of the
five patients had at least one whole lung lavage (WLL) prior to
treatment with molgramostim, none of the five patients required WLL
after more than one year on treatment, suggesting molgramostim may
reduce the need for WLL. No reported serious adverse events
occurred, and treatment was well tolerated.
“Long-term outcomes data from these case studies are encouraging
and support our belief that treatment with molgramostim may address
the root cause of aPAP, resulting in improved and sustained patient
benefit,” said Matt Pauls, Chair and Chief Executive Officer,
Savara. “We are committed to our goal of providing the aPAP
community with the first and only pharmaceutical treatment option
in the U.S. and Europe and look forward to completing the rolling
Biologics License Application (BLA) submission in the U.S. by the
end of 1Q25 and the Marketing Authorization Application (MAA) in
Europe by the end of 2025.”
About Autoimmune PAP
Autoimmune PAP is a rare lung disease characterized by the
abnormal build-up of surfactant in the alveoli (or air sacs) of the
lungs. Surfactant consists of proteins and lipids and is an
important physiological substance that lines the alveoli to prevent
them from collapsing. In a healthy lung, excess surfactant is
cleared and digested by immune cells called alveolar macrophages.
Alveolar macrophages need to be stimulated by
granulocyte-macrophage colony-stimulating factor (GM-CSF) to
function properly in clearing surfactant, but in aPAP, GM-CSF is
neutralized by antibodies against GM-CSF, rendering macrophages
unable to adequately clear surfactant. As a result, an excess of
surfactant accumulates in the alveoli, causing impaired gas
exchange, resulting in clinical symptoms of shortness of breath,
often with cough and frequent fatigue. Patients may also experience
episodes of fever, chest pain, or coughing up blood, especially if
secondary lung infection develops. In the long-term, the disease
can lead to serious complications, including lung fibrosis and the
need for a lung transplant.
About Savara
Savara is a clinical stage biopharmaceutical company focused on
rare respiratory diseases. Our lead program, molgramostim, is a
recombinant human granulocyte-macrophage colony-stimulating factor
(GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar
proteinosis (aPAP). Molgramostim is delivered via an
investigational eFlow® Nebulizer System (PARI Pharma GmbH)
specifically developed for inhalation of a large molecule. Our
management team has significant experience in rare respiratory
diseases and pulmonary medicine, identifying unmet needs, and
effectively advancing product candidates to approval and
commercialization. More information can be found at
www.savarapharma.com, X: @SavaraPharma and LinkedIn.
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 regarding the suggestion that treatment with
molgramostim addresses the underlying pathophysiology of aPAP and
our belief that it may address the root cause of aPAP, resulting in
improved and sustained patient benefit, and statements related to
the anticipated timing of our BLA and MAA submissions. 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, the risks
associated with our ability to successfully develop, complete the
requisite regulatory submissions and obtain regulatory approval
for, and commercialize molgramostim for aPAP; the risks and
uncertainties related to the impact of widespread health concerns
or changing economic or geopolitical conditions; the ability to
project future cash utilization and reserves needed for contingent
future liabilities and business operations; and the availability of
sufficient financial and other resources for Savara’s operations
and to conduct or continue planned clinical development programs.
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, Form 10-K and Form 10-Q. 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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version on businesswire.com: https://www.businesswire.com/news/home/20250130634843/en/
Media and Investor Contact: Savara Inc. Temre Johnson,
Executive Director, Corporate Affairs ir@savarapharma.com
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