Savara Provides Update on Case Reports of Inhaled Granulocyte-Macrophage Colony Stimulating Factor for the Treatment of Nontu...
May 29 2018 - 8:00AM
Savara, Inc. (NASDAQ:SVRA), an orphan lung disease company, today
provided an update on two case reports exploring the use of
aerosolized granulocyte-macrophage colony stimulating factor
(GM-CSF) for the treatment of Mycobacterium abscessus (M.
abscessus), a species of multidrug-resistant nontuberculous
mycobacteria (NTM), in individuals living with cystic fibrosis
(CF). Notably, culture conversion has now been achieved in both
patients, along with meaningful clinical improvement.
The two case reports were initially published in
the European Respiratory Journal (ERJ) by Mark E. Wylam, M.D.,
pulmonologist and critical-care specialist at the Mayo Clinic
College of Medicine, and his coworkers. In the published case
reports, inhaled GM-CSF eradicated M. abscessus infection in the
first subject and precipitated a strong microbiological response in
the second subject. Subsequent to the publication, the second
subject has now achieved culture conversion without NTM antibiotic
treatment.
“This is a remarkable result given that this
second subject, unlike the first one, was not treated with NTM
antibiotics,” stated Mark E. Wylam, M.D., the senior author of the
ERJ publication. “Both subjects remain on aerosolized GM-CSF and
have experienced clinically meaningful improvements in weight gain
and lung function. I believe these case studies strongly reinforce
the scientific rationale for the treatment of NTM lung infection
using inhaled GM-CSF, and I look forward to the results of Savara’s
ongoing OPTIMA study.”
In Q1 2018 Savara initiated the OPTIMA study, a
30-patient, multi-center, open-label Phase 2a clinical trial to
investigate the efficacy and safety of Molgradex for the treatment
of chronic NTM lung infection. Subjects with either M. abscessus or
Mycobacterium avium complex (MAC) infection are being enrolled,
with all subjects having either antibiotic refractory infection or
intolerance to standard NTM antibiotics. The trial consists of 24
weeks of Molgradex treatment, followed by a 12-week observational
period. The primary endpoint is sputum culture conversion defined
as at least three consecutive negative sputum cultures. Secondary
endpoints include other microbiological indicators, exercise
capacity and patient-reported outcomes.
Patient enrollment in the OPTIMA study is on
track to complete in Q3 2018 and topline results are expected in H1
2019. However, as OPTIMA is an open-label study, depending on
enrollment and other factors, interim results from the study may be
provided in 2018.
About NTM Lung InfectionNTM
lung infection is a rare and serious lung disorder associated with
increased rates of morbidity and mortality. NTM are
naturally-occurring organisms and NTM lung infection can occur when
an individual inhales the organism from their environment and
develops a slowly progressive and destructive lung disease. NTM
lung infection is typically characterized by cough, fatigue and
weight loss. NTM infection often becomes chronic and requires long
courses of multiple antibiotics, and despite aggressive treatment
regimens, treatment failure rates are high, and recurrence of
infection common. Chronic NTM lung infection can have a significant
impact on quality of life. There are approximately 50,000 to 80,000
individuals affected by NTM lung infection in the U.S., the most
common types involving MAC and M. abscessus. There have been
few advancements in new systemic treatments for NTM lung infection.
However, in a recent Phase 3 clinical trial by Insmed, local
delivery of an inhaled form of amikacin directly to the lung was
shown to be effective in approximately one third of treatment
refractory patients with pulmonary MAC infection, suggesting
administration of high local concentrations of drug directly at the
site of infection provides an attractive new avenue to improve
clinical outcomes in this and other difficult-to-treat chronic lung
infections.
About MolgradexMolgradex is an
inhaled formulation of recombinant human GM-CSF, in Phase 3
development for autoimmune pulmonary alveolar proteinosis and in
Phase 2a development for NTM lung infection. Molgradex is delivered
via an investigational eFlow® Nebulizer System (PARI Pharma GmbH).
Molgradex has been granted Orphan Drug Designation for the
treatment of PAP in the United States and the European Union.
About Savara Savara is an
orphan lung disease company. Savara's pipeline comprises:
Molgradex, an inhaled granulocyte-macrophage colony-stimulating
factor, or GM-CSF, in Phase 3 development for autoimmune pulmonary
alveolar proteinosis and in Phase 2a development for NTM lung
infection; and AeroVanc, a Phase 3 stage inhaled vancomycin for
treatment of MRSA lung infection in CF. 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,
identifying unmet needs, developing 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 the belief that the Mayo Clinic case reports strongly
reinforce the scientific rationale for treatment of NTM lung
infection using inhaled GM-CSF and looking forward to the results
of the ongoing OPTIMA study, that patient enrollment in the OPTIMA
study is on track to complete in Q3 2018, that topline results are
expected in H1 2019, that interim results from the OPTIMA study may
be provided in 2018, that in a recent Phase 3 clinical trial
by Insmed, local delivery of an inhaled form of amikacin
directly to the lung was shown to be effective in approximately one
third of treatment refractory patients with pulmonary MAC
infection, suggesting administration of high local concentrations
of drug directly at the site of infection provides an attractive
new avenue to improve clinical outcomes in this and other difficult
to treat chronic lung infections and Savara’s strategy. 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 our ongoing clinical
trials for our product candidates (including the Phase 2a study of
Molgradex for NTM), 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 (including our Phase 2a study of Molgradex for NTM), the
ability to obtain the necessary patient enrollment for our product
candidates in a timely manner (including our Phase 2a study of
Molgradex for NTM), the ability to successfully develop our product
candidates, the risks associated with the process of developing,
obtaining regulatory approval for and commercializing drug
candidates such as Molgradex and AeroVanc that are safe and
effective for use as human therapeutics and the timing and ability
of Savara to raise additional equity 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.
Contacts:
Savara: Ioana C. Hone (ir@savarapharma.com) (512) 961-1891
For IR: Solebury Trout Gitanjali Jain Ogawa
(Gogawa@troutgroup.com) (646) 378-2949
For Media: Neon InteractivePatrick Wallace
(patrick@neoninteractive.com) (619) 200-7856
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