WARRINGTON, Pa., Dec. 10, 2015 /PRNewswire/ -- Discovery
Laboratories, Inc. (NASDAQ: DSCO) today announced that data from
the AEROSURF® Phase 2a clinical program in premature infants 29 to
34 weeks gestational age (GA) with respiratory distress syndrome
(RDS) were presented at the 2015 Hot Topics in Neonatology Annual
Meeting held December 6 - 9 in
Washington D.C. The presentation,
titled "Aerosolized Surfactant – Safety and Tolerability
of Lucinactant for Inhalation in Preterm Infants", was made by
Neil Finer, M.D, Professor Emeritus,
Division of Neonatology, University of
California, San Diego, and Chairman of the AEROSURF Steering
Committee. Discovery Labs had previously released top-line results
of the AEROSURF Phase 2a clinical program in premature infants 29
to 34 weeks GA in November 2015.
The AEROSURF phase 2a clinical program in premature infants 29
to 34 weeks GA consisted of two multicenter, randomized,
open-label, controlled studies in a total of 80 premature infants
receiving nasal continuous positive airway pressure (nCPAP) for
RDS. The studies were designed to evaluate the safety and
tolerability of aerosolized KL4 surfactant administered in five
dose groups (15, 30, 45, 60 and 90 minutes), compared to infants
receiving nCPAP alone. All key objectives of the program were
achieved:
- Overall, the safety and tolerability profile of the AEROSURF
group in this phase 2a program was generally comparable to the
control group.
- Data suggest that AEROSURF may be reducing the incidence of
nCPAP failure, which occurs when premature infants initially
treated with nCPAP alone, require delayed surfactant therapy by
means of invasive endotracheal intubation. Through 72 hours
after the start of AEROSURF treatment, the AEROSURF-treated
patients, predominantly receiving a single dose, had lower rates of
nCPAP failure compared to control in each of the last three dose
groups studied. nCPAP failure rates were 53% in the control
group compared to 38%, 14%1 (excluding one patient who
was inappropriately enrolled) and 38% in the 45, 60 and 90 minute
AEROSURF dose groups, respectively.
The Company has focused its planning for the phase 2b clinical
trial on the 45 and 60 minute dose groups. The combined data
for these groups indicate that, through 72 hours after the start of
AEROSURF treatment, AEROSURF-treated patients had a 27%1
nCPAP failure rate compared to 53% in the control group. This
represents a 26% absolute reduction or a 49% relative reduction in
nCPAP failure compared to control.
"Premature infants with RDS are born with immature lungs and may
require surfactant therapy to sustain them in the first hours of
life and for up to 48 to 72 hours. Currently in the United States surfactant therapy is
administered via endotracheal intubation. Given the invasive
requirements of surfactant administration, the current trend in
neonatology is to use less- or non-invasive methods to treat RDS"
commented Dr. Finer. "The AEROSURF phase 2a data thus far are
encouraging because we are seeing signals suggesting that AEROSURF
may be reducing nCPAP failures and intubations. If this
result is confirmed by future clinical data, AEROSURF could
represent a significant medical advancement in neonatology."
The Hot Topics in Neonatology Annual Meeting is an
internationally recognized medical meeting dedicated to advancing
the practice of neonatology and is attended by more than 1,000
neonatologists and perinatologists each year.
1 The Company previously reported top-line data that
stated nCPAP failure rates in AEROSURF-treated patients were 29%
for the 60 minute dose group and 33% for the 45 and 60 minute dose
groups combined. These rates have been updated based on
finalization of the review of data.
About AEROSURF®
Premature infants with
severe RDS currently are treated with surfactants that can only be
administered by endotracheal intubation supported with mechanical
ventilation, invasive procedures that may each result in serious
respiratory conditions and other complications. To avoid such
complications, many neonatologists treat infants with less severe
RDS by less invasive means, typically nCPAP. Unfortunately, a
significant number of premature infants on nCPAP will respond
poorly (an outcome referred to as nCPAP failure) and may require
delayed surfactant therapy. Since neonatologists currently
cannot predict which infants will experience nCPAP failure,
neonatologists are faced with difficult choices in treating infants
with less severe RDS. This is because the medical outcomes
for those infants who experience nCPAP failure and receive delayed
surfactant therapy may be less favorable than the outcomes for
infants who received surfactant therapy in the first hours of
life.
AEROSURF is a novel, investigational drug/device product that
combines the Company's proprietary KL4 surfactant and its
aerosolization technologies. AEROSURF is being developed to
potentially reduce or eliminate the need for endotracheal
intubation and mechanical ventilation in the treatment of premature
infants with respiratory distress syndrome (RDS). With
AEROSURF, neonatologists may potentially administer aerosolized KL4
surfactant to premature infants supported by nCPAP, without
subjecting them to invasive endotracheal intubation and mechanical
ventilation (each of which can result in serious respiratory
conditions and other complications), which are currently required
to administer surfactant therapy to premature infants. By
enabling delivery of aerosolized KL4 surfactant using less invasive
procedures, AEROSURF, if approved, has the potential to
address a serious unmet medical need, provide transformative
clinical and pharmacoeconomic benefits, and enable the treatment of
a significantly greater number of premature infants with RDS who
could benefit from surfactant therapy but are currently not
treated.
Currently in the U.S., the Company estimates that approximately
120,000 to 150,000 premature infants could benefit from surfactant
therapy. However, due to the risks associated with
endotracheal intubation and mechanical ventilation, only
approximately 50,000 to 60,000 of these infants currently are
treated with surfactants as the initial therapy for severe
RDS. The remaining infants with less severe RDS are usually
supported with nCPAP alone. However, a large percentage of
these infants are not adequately supported with nCPAP alone (an
outcome referred to as nCPAP failure) and thereafter may require
delayed surfactant therapy administered by endotracheal intubation
and mechanical ventilation
About Discovery Labs
Discovery Laboratories, Inc. is a
biotechnology company focused on developing aerosolized KL4
surfactant therapies for respiratory diseases. Surfactants
are produced naturally in the lung and are essential for normal
respiratory function and survival. If surfactant deficiency
or degradation occurs, the air sacs in the lungs can collapse,
resulting in severe respiratory diseases and disorders.
Discovery Labs' technology platform includes a novel synthetic
peptide-containing (KL4) surfactant, that is structurally similar
to pulmonary surfactant, and proprietary drug delivery technologies
being developed to enable efficient delivery of aerosolized KL4
surfactant. Discovery Labs believes that its proprietary
technology platform makes it possible, for the first time, to
develop a significant pipeline of aerosolized surfactant products
to address a variety of respiratory diseases for which there
frequently are few or no approved therapies.
For more information, please visit the Company's website at
www.Discoverylabs.com.
Forward-Looking Statements
To the extent
that statements in this press release are not strictly historical,
all such statements are forward-looking, and are made pursuant to
the safe harbor provisions of the Private Securities Litigation
Reform Act of 1995. These forward-looking statements are
subject to certain risks and uncertainties that could cause actual
results to differ materially from the statements made. Examples of
such risks and uncertainties, including those affecting Discovery
Labs' ability successfully to complete its development programs and
realize the potential benefits of its RDS product portfolio, are
described in Discovery Labs' filings with the Securities and
Exchange Commission, including the most recent reports on Forms
10-K, 10-Q and 8-K, and any amendments thereto. Any forward-looking
statement in this release speaks only as of the date on which it is
made. Discovery Labs assumes no obligation to update or revise any
forward-looking statements.
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SOURCE Discovery Laboratories, Inc.