Savara Announces First Patient Dosed in Pivotal Phase 3 Autoimmune Pulmonary Alveolar Proteinosis (aPAP) Trial
June 30 2021 - 10:47AM
Business Wire
- The IMPALA-2 trial will evaluate the efficacy and safety of
molgramostim nebulizer solution (molgramostim) in aPAP, a rare lung
disease with no approved pharmacological treatment options
- Key learnings from the Phase 2/3 IMPALA trial informed the
Phase 3 IMPALA-2 trial design
Savara Inc. (Nasdaq: SVRA), a clinical stage biopharmaceutical
company focused on rare respiratory diseases, today announced that
the first patient has been dosed in the pivotal IMPALA-2 clinical
trial. IMPALA-2 is a Phase 3 trial designed to evaluate the
efficacy and safety of molgramostim compared to placebo.
Molgramostim is an inhaled formulation of recombinant human
granulocyte-macrophage colony-stimulating factor (GM-CSF). The
trial is expected to be conducted at ~50 sites across the U.S.,
Canada, Japan, South Korea, and various countries in Europe and is
anticipated to enroll ~160 patients with aPAP.
“Autoimmune PAP is caused by abnormal accumulation of surfactant
sediment in the alveoli, leading to impaired gas exchange between
the lungs and blood,” said Bruce Trapnell, M.D., IMPALA-2
International Coordinating Investigator, Professor of Medicine and
Pediatrics, University of Cincinnati College of Medicine. “The
disease has a meaningful impact on a patient’s quality of life,
causing them to become increasingly breathless, often with cough
and frequent fatigue. With an unpredictable yet progressive
clinical course, aPAP can include serious secondary infections,
respiratory failure or pulmonary fibrosis requiring a lung
transplantation. Research has demonstrated that treatment with
inhaled GM-CSF can improve the clinical signs and symptoms of aPAP,
which makes sense as it’s a logical replacement of a protein that
has been neutralized by autoantibodies.”
“With the first patient dosed in IMPALA-2, we hit a critical
milestone in-line with our guidance for the trial,” said Badrul
Chowdhury, M.D., Ph.D., Chief Medical Officer, Savara. “Our highest
priority is to continue activating sites and working with the
global aPAP community to enroll patients. Data from the IMPALA
trial gave us confidence that molgramostim has the potential to
address a significant unmet need in aPAP and we look forward to
building on those supportive data to advance the development of
molgramostim as the first potential pharmacological treatment for
this debilitating disease.”
Initiation of IMPALA-2 is based on results from the Phase 2/3
IMPALA clinical trial which were published in the New England
Journal of Medicine in September 2020. While the IMPALA trial did
not meet the primary endpoint of alveolar-arterial oxygen gradient
(A-aDO2), the totality of data showed that multiple key secondary
and exploratory endpoints either achieved nominal statistical
significance or trended in favor of the active drug arms, and
results from the open-label period demonstrated a sustained
treatment effect, or continued improvement, after longer term
exposure to molgramostim. In December 2019, the U.S. Food and Drug
Administration (FDA) granted Breakthrough Therapy Designation for
molgramostim in aPAP based on data from the double-blind treatment
period of IMPALA.
IMPALA-2 is a Phase 3, 48-week, randomized, double-blind,
placebo-controlled clinical trial designed to compare the efficacy
and safety of molgramostim 300 mcg administered once daily by
inhalation with matching placebo in patients with aPAP. The primary
efficacy variable is change from baseline in percent predicted
diffusing capacity for carbon monoxide (DLCO), a gas exchange
measure. Three secondary efficacy variables evaluate clinical
measures of direct patient benefit: St. George’s Respiratory
Questionnaire (SGRQ) Total Score, SGRQ Activity Component Score,
and exercise capacity using a treadmill test. The primary time
point for efficacy assessment will be at week 24, however, efficacy
will be assessed through week 48 to show durability of effect.
Safety will be assessed through week 48. Following the 48-week
double-blind treatment period, patients will roll-over to a 48-week
open-label period and will receive molgramostim 300 mcg
administered once daily.
More information on the IMPALA-2 trial (NCT04544293) can be
found at clinicaltrials.gov.
About Savara
Savara is a clinical stage biopharmaceutical company focused on
rare respiratory diseases. Our lead program, molgramostim nebulizer
solution, is an inhaled granulocyte-macrophage colony-stimulating
factor (GM-CSF) in Phase 3 development for autoimmune pulmonary
alveolar proteinosis (aPAP). 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 https://savarapharma.com/. (Twitter: @SavaraPharma,
LinkedIn: www.linkedin.com/company/savara-pharmaceuticals/).
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 expected number and location of sites
participating in the IMPALA-2 trial, the anticipated enrollment,
that our highest priority is to continue activating sites and
working with the global aPAP community to enroll patients, that
molgramostim has the potential to address a significant unmet need
in aPAP, and that we look forward to building on the supportive
data from IMPALA to advance the development of molgramostim as the
first potential pharmacological treatment for this debilitating
disease. 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. 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 and
uncertainties relating to the impact of the COVID-19 pandemic on
our business and operations, the outcome of our ongoing and planned
clinical trials, 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 ability to successfully develop molgramostim,
the risks associated with the process of developing, obtaining
regulatory approval for and commercializing drug candidates such as
molgramostim that are safe and effective for use as human
therapeutics, and the timing and ability of Savara to raise
additional capital as needed to fund continued operations. 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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Savara Inc. IR & PR Anne Erickson
(anne.erickson@savarapharma.com) (512) 851-1366
Savara (NASDAQ:SVRA)
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