Savara Inc. (Nasdaq: SVRA), an orphan lung disease company, today
announced interim results from OPTIMA, a Phase 2a clinical study
evaluating its lead product candidate Molgradex, an inhaled
formulation of recombinant human granulocyte-macrophage
colony-stimulating factor (GM-CSF), for the treatment of
nontuberculous mycobacterial (NTM) lung infection. The ongoing
study is evaluating treatment of both Mycobacterium avium complex
(MAC) infection, and the more difficult-to-treat Mycobacterium
abscessus (MABSC) infection. Savara believes microbiological data
from this early analysis demonstrate an encouraging efficacy
signal, with a favorable safety profile.
The interim analysis focused on efficacy, as assessed by
microbiological results, in 14 patients who completed the 24-week
treatment period and had culture results available up to at least
the 16-week timepoint. Ten of the evaluable patients have MAC
infection and four have MABSC infection. Of the patients with MAC
infection, eight are in treatment Group 1 (on anti-mycobacterial
treatment) and two are in treatment Group 2 (not on
anti-mycobacterial treatment). The four evaluable MABSC patients
are evenly split between both treatment groups. Safety and
tolerability was assessed for all 32 patients enrolled in the
study.
Summary of Microbiological DataThe data show
that among the 10 patients with MAC infection, four experienced a
consistent sputum smear conversion to negative by week 24, and
three experienced a negative sputum culture at weeks 16 and 20,
with culture results pending for the week 24 timepoint. Sputum
smear or sputum culture conversions were not observed in the four
patients with MABSC infection. Due to the generally slow growth of
nontuberculous mycobacteria on culture media, a sputum sample can
be determined negative only after eight weeks of observation,
causing a corresponding lag time in the assessment of the culture
data.
“These early signals of potential efficacy are encouraging, as
is the good tolerability and safety profile of Molgradex in
patients with refractory NTM lung infection,” said study
investigator Rachel Thomson, MBBS PhD., Associate Professor,
University of Queensland, Australia. “Importantly, the
microbiological responses seem to be associated with improvements
in clinical signs and symptoms, and I am keen to see if these
responses can be sustained over a longer period of time, and if we
will see more of them. Antibiotics currently used with these
patients are often poorly tolerated, frequently fail to eradicate
the infection and are associated with a high recurrence rate. Based
on this interim data review, I am optimistic that Molgradex may
help combat NTM infection in a unique way by stimulating the innate
immune system in the lungs.”
Summary of Safety InformationSafety was
assessed in the total study population. Among the 32 patients, six
(19%) experienced serious adverse events (SAEs), including one
patient who died by suicide. The death was considered unrelated to
treatment. The majority of SAEs consisted of hospitalizations due
to pulmonary exacerbations or worsening of NTM infection, of which
one was considered possibly treatment-related. GM-CSF was generally
well tolerated, with nine patients (28%) reporting mostly mild,
potentially treatment-related respiratory adverse events.
Respiratory adverse events were defined as shortness of breath,
chest tightness or wheeze. A total of three patients (9%)
discontinued treatment due to adverse events.
Consistent with the systemic pharmacological effect of GM-CSF on
white blood cells, 17 patients (53%) experienced increased levels
of blood eosinophils. The increase generally peaked at the week 4
timepoint, and levels decreased or plateaued at subsequent
visits.
Based on the microbiological data and safety profile, which
provides the basis to continue treating patients for a longer
period of time, the duration of the OPTIMA study is extended from
24 to 48 weeks. This increases the ability to observe a more robust
anti-infective effect, including culture conversions. Final results
from OPTIMA are now expected by the first quarter of 2020.
About the OPTIMA Clinical Study OPTIMA is an
open-label, non-controlled, multi-center, Phase 2a clinical study
of Molgradex in 32 subjects (≥18 years of age) with persistent
pulmonary NTM lung infection. OPTIMA enrolled subjects with chronic
Mycobacterium avium complex (MAC) infection or Mycobacterium
abscessus (MABSC) infection, with all patients having either
antibiotic refractory infection or intolerance to standard NTM
antibiotics. Patients with cystic fibrosis were not enrolled. The
study comprises a 48-week treatment period and a 12-week follow up
period. Two groups of subjects were recruited into the OPTIMA
study. Group 1 consists of patients who remained sputum culture
positive while currently on a multidrug NTM guideline-based
anti-mycobacterial regimen, which had been ongoing for at least six
months prior to the baseline visit. Group 2 consists of patients
who remained sputum culture positive, but either stopped a
multidrug NTM guideline-based anti-mycobacterial regimen at least
28 days prior to screening due to lack of response or intolerance,
or never started such treatment.
The primary endpoint in the study is sputum culture conversion,
defined as at least three consecutive sputum samples without growth
of nontuberculous mycobacteria. Secondary endpoints include: (i)
the number of patients with sputum smear conversion to negative,
defined as at least three consecutive negative acid-fast bacilli
(AFB) stained sputum smears on microscopy among patients who were
smear positive at baseline, (ii) the number of patients with
durable sputum culture conversion, defined as sputum culture
conversion at or before week 48 and culture still negative for
growth of nontuberculous mycobacteria at 12-week follow up, (iii)
the number of patients with durable sputum smear conversion,
defined as sputum smear conversion at or before week 48 and AFB
stained smear still negative for nontuberculous mycobacteria at
12-week follow up among patients who were smear positive at
baseline, and (iv) other microbiological indicators, exercise
capacities and patient reported outcomes.
About NTM Lung InfectionNTM lung infection is a
rare and serious lung disorder associated with increased rates of
morbidity and mortality. Nontuberculous mycobacteria are
naturally-occurring organisms and NTM lung infection can occur when
an individual inhales the organism from the 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, 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.
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,
or aPAP, in Phase 2a development for nontuberculous mycobacterial,
or NTM, lung infection, and in preparation for Phase 2a development
in cystic fibrosis, or CF, affected individuals with chronic NTM
lung infection; and AeroVanc, a Phase 3 stage inhaled vancomycin
for treatment of persistent methicillin resistant staphylococcus
aureus, or 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. The most recent acquisition is aerosolized
amikacin/fosfomycin, a Phase 2-ready, proprietary combination
antibiotic, which has demonstrated potent and broad-spectrum
antibacterial activity against highly drug resistant pathogens.
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,
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 Savara’s belief that microbiological data from this early
analysis demonstrate an encouraging efficacy signal with a
favorable safety profile, that the early signals of potential
efficacy are encouraging, as is the good tolerability and safety
profile of Molgradex in patients with refractory NTM lung
infection, that the microbiological responses seem to be associated
with improvements in clinical signs and symptoms, statements
regarding the interest in seeing if these responses can be
sustained over a longer period of time and if we will see more of
them, statements regarding optimism that based on this interim data
review Molgradex may help combat NTM infection in a unique way by
stimulating the innate immune system in the lungs, that the
microbiological data and safety profile provides a basis to
continue treating patients for a longer period of time, statements
regarding the duration of the OPTIMA study, that the increase in
the duration of the OPTIMA study increases the ability to observe a
more robust anti-infective effect, including culture conversions,
statements regarding the timing of final results from the OPTIMA
study, 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. 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 and
planned clinical trials for our product candidates (including the
OPTIMA clinical study), 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 the OPTIMA clinical study), the
ability to obtain the necessary patient enrollment for our product
candidates in a timely manner, the ability to successfully identify
product acquisition candidates, 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, AeroVanc and amikacin/fosfomycin
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, Inc. IR: Ioana C. Hone (ir@savarapharma.com) (512)
961-1891
Savara, Inc. PR and Media:Anne Erickson
(anne.erickson@savarapharma.com) (512) 851-1366
For IR: Solebury Trout Gitanjali Jain Ogawa
(Gogawa@troutgroup.com) (646) 378-2949
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