AUSTIN, Texas, May 10, 2017 /PRNewswire/ -- Savara Inc. (NASDAQ:
SVRA) today announced that it has received guidance from the U.S.
Food and Drug Administration on the clinical program requirements
for a New Drug Application submission in the U.S. for Molgradex, an
inhaled formulation of recombinant human GM-CSF, for the treatment
of autoimmune pulmonary alveolar proteinosis, or PAP. Based on the
FDA's guidance, Savara will modify the endpoint hierarchy and
statistical analyses of its ongoing IMPALA study, currently
enrolling patients in Europe and
Japan, to qualify the study as a
pivotal Phase 3 study in the U.S. The total number of patients to
be enrolled will be increased from 51 to 90 to support the modified
design. If successful, the amended study would serve as the sole
pivotal study for regulatory submission for marketing authorization
of Molgradex for the treatment of PAP in the U.S. in addition to
Europe and
Japan.
"In the short time since we acquired Molgradex in July 2016 we have made tremendous progress, and
having the opportunity to utilize our ongoing IMPALA study as a
global Phase 3 study is a major win for the company, with the
potential to considerably expedite the approval of the product in
the U.S. by eliminating the need to conduct a separate Phase 3
study," stated Rob Neville, Chief
Executive Officer of Savara. "This is very good news for the PAP
community in the U.S., as we believe Molgradex can offer a game
changing treatment alternative in a disease where the current
standard treatment option is to periodically conduct an invasive
whole lung lavage procedure requiring general anesthesia."
The IMPALA study is a randomized, double-blind,
placebo-controlled study designed to compare the efficacy and
safety of Molgradex with placebo in patients with PAP. The study,
which began enrolling patients in Europe and Japan last year, will be expanded to enroll a
total of 90 patients, half of which have already been enrolled.
Patient enrollment is expected to be completed by the first quarter
of 2018, and top line data is expected to be available by the
fourth quarter of 2018.
"Molgradex is a unique product that directly addresses the
disease mechanism causing PAP – an autoimmune blockade of a
naturally occurring signaling protein, GM-CSF, which is required to
clear excess surfactant from the lungs," said Dr. Bruce Trapnell, M.D., Professor of Medicine and
Pediatrics, Cincinnati Children's Hospital Medical Center, and
Director of the NCATS-NHLBI Rare Lung Diseases Clinical Research
Consortium. "PAP is a debilitating lung disease with a high
clinical need for an effective medicinal treatment to reduce or
eliminate the need for whole lung lavage. Inhaled GM-CSF restores
the ability of lung cells to clear surfactant. Based on an
increasing body of literature and clinical experience of
explorative use of inhaled GM-CSF, I and my colleagues treating PAP
patients worldwide are very optimistic about the potential of
Molgradex as a truly transformative treatment option in PAP."
The primary endpoint in the IMPALA study will remain the
absolute change from baseline in arterial-alveolar oxygen gradient,
a measure of patient's oxygenation status, following 24 weeks of
treatment. In addition, the FDA will focus its review on three key
secondary endpoints that will be assessed to show improvement in
clinical symptoms and function, including six-minute walk distance,
St. George's respiratory
questionnaire, and the time to need of whole lung lavage. Patients
are randomized to receive treatment for up to 24 weeks in one of
three treatment arms: 1) Molgradex 300 µg administered once daily,
2) Molgradex 300 µg and matching placebo administered daily in
7-day intermittent cycles of each, or 3) inhaled placebo
administered once daily.
About Pulmonary Alveolar Proteinosis (PAP)
PAP is a
rare lung disease which is characterized by the build-up of lung
surfactant in the alveoli, or air sacs, of the lungs. The
surfactant consists of proteins and lipids, and is an important
physiological substance that lines the inside of the alveoli to
prevent the lungs from collapsing. In a healthy lung, the old and
inactivated surfactant is cleared and digested by immune cells
called alveolar macrophages. Alveolar macrophages need to be
stimulated by GM-CSF to function properly in clearing surfactant,
but in autoimmune PAP, GM-CSF is neutralized by antibodies against
GM-CSF, rendering the macrophages unable to perform their tasks. As
a result, an excess of surfactant accumulates in the alveoli,
causing obstruction of gas exchange, and patients start to
experience shortness of breath, and decreased exercise tolerance.
Patients may also experience chronic cough, as well as episodes of
fever, chest pain, or coughing 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
lung transplant.
About Molgradex
Molgradex, an inhaled formulation of
recombinant human GM-CSF, is being developed for the treatment of
autoimmune pulmonary alveolar proteinosis, or PAP. Savara is also
pursuing indication expansion, with priority on the development of
Molgradex in rare infectious lung diseases, where stimulation of
the innate immune system has the potential to improve clinical
outcomes. The Company expects to announce details related to its
indication expansion strategy by the third quarter of 2017.
Molgradex has been granted Orphan Drug Designation for the
treatment of PAP in the United
States and the European Union.
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, an inhaled granulocyte-macrophage
colony-stimulating factor, or GM-CSF, in Phase 3 development,
AeroVanc, an inhaled vancomycin in preparation for a Phase 3 study,
and Aironite, an inhaled nebulized sodium nitrite in Phase 2
development. Savara's strategy involves expanding its pipeline of
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
regarding the Molgradex development strategy, potential to
considerably expedite the approval of the product in the U.S., that
Molgradex can offer a game changing treatment alternative, patient
enrollment expected to be completed by the first quarter of 2018,
and top line data expected to be available by the fourth quarter of
2018 and expecting to announce details related to its indication
expansion strategy by the third quarter of 2017. 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 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 in a timely manner, the timing and ability of Savara to
raise additional equity capital to fund continued operations; the
ability to successfully develop Savara's product candidates, and
the risks associated with the process of developing, obtaining
regulatory approval for and commercializing drug candidates that
are safe and effective for use as human therapeutics. Risks and
uncertainties facing Savara are described more fully in Savara's
filings with the Securities and Exchange Commission including the
Form 8-K filed on April 27, 2017,
other filings on Form 8-K, the Annual Report on Form 10-K for the
fiscal year ended December 31, 2016,
the Quarterly Report on Form 10-Q for the quarter ended
March 31 2017 and the Registration
Statement on Form S-4 related to the Mast/Savara merger. 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 any 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.