Discovery Labs Receives FDA Clearance of IND to Initiate AEROSURF® Phase 2
WARRINGTON, Pa., Nov. 12, 2013
WARRINGTON, Pa., Nov. 12, 2013 /PRNewswire/ -- Discovery Laboratories, Inc.
(NASDAQ: DSCO), a specialty biotechnology company dedicated to advancing a new
standard of respiratory critical care, today announced that the U.S. Food and
Drug Administration (FDA) has cleared the Company's investigational new drug
(IND) application for AEROSURF^® and the Company can initiate its phase 2
The phase 2 clinical program is expected to include two studies. The primary
goal of the initial study, phase 2a, is to evaluate the safety and
tolerability of a single exposure of aerosolized KL4 surfactant drug product.
This study is planned as an escalating dose study evaluating three dose
levels of aerosolized KL4 surfactant. The comparator is nCPAP alone. The
study will be conducted in three centers in the U.S. and is expected to be
completed by mid-2014. The design of the second study, phase 2b, will be
informed by the results of the phase 2a study. The primary objective of the
phase 2b study will be to determine optimal dose and to estimate the efficacy
margin. Results of the phase 2b study will inform the design of the phase 3
efficacy and safety study. Phase 2b is expected to be conducted in multiple
centers and completed by mid-2015.
AEROSURF is a novel investigational drug-device combination product being
developed to deliver Discovery Labs' KL4 surfactant in aerosolized form to
premature infants with respiratory distress syndrome (RDS). AEROSURF could
potentially allow for the administration of KL4 surfactant to premature
infants without invasive endotracheal intubation, and may enable the treatment
of a significantly greater number of premature infants who could benefit from
surfactant therapy but are currently not treated.
About Respiratory Distress Syndrome (RDS)
RDS is a condition in which premature infants are born with a lack of natural
lung surfactant and are unable to absorb sufficient oxygen.Premature infants
born prior to 37 weeks gestation have not fully developed their own natural
lung surfactant and therefore need treatment to sustain life.RDS is
experienced in approximately half of the babies born between 26 and 32 weeks
gestational age.The incidence of RDS approaches 100 percent in babies born
less than 26 weeks gestational age.RDS can result in long-term respiratory
problems and death.
Premature infants with RDS currently are treated with surfactants that can
only be administered by endotracheal intubation supported with mechanical
ventilation, both invasive procedures that may result in serious respiratory
conditions and complications. To avoid such adverse results, neonatologists
generally provide surfactants as initial therapy only to premature infants
with severe RDS where the potential benefits of surfactant therapy outweigh
the risks associated with endotracheal intubation and mechanical ventilation.
For infants with less severe RDS, neonatologists will first attempt to provide
respiratory support using a less invasive means, such as nasal continuous
positive airway pressure (nCPAP). Unfortunately, a significant number of
these infants do not respond adequately to nCPAP, an outcome referred to as
nCPAP failure, and require subsequent surfactant administration via intubation
and mechanical ventilation. As it is not possible to ascertain in advance
which infants will experience nCPAP failure, neonatologists are faced with a
dilemma, because the outcome for those infants who experience nCPAP failure
and receive delayed surfactant therapy may not be as favorable as the outcome
for those infants who receive surfactant therapy as initial therapy.
Discovery Labs estimates that, on an annual basis, approximately 160,000
premature infants in the U.S. could potentially benefit from early surfactant
therapy to address surfactant deficiency or insufficiency. However, only
approximately 45,000 will receive surfactants as an initial therapy. More
than 70 percent of surfactant deficient infants (approximately 115,000) do not
receive first-line surfactant therapy and instead receive nCPAP alone.
Discovery Labs believes that the neonatal medical community increasingly
recognizes the potential of a synthetic, peptide-containing surfactant, such
as SURFAXIN^® and, importantly, a less-invasive method of delivering
surfactant, such as AEROSURF, to treat premature infants at risk of suffering
from respiratory disorders.
About Discovery Labs
Discovery Laboratories, Inc. is a specialty biotechnology company focused on
advancing a new standard in respiratory critical care. Discovery Labs'
technology platforms include a novel proprietary KL4 surfactant, a synthetic,
peptide-containing surfactant that is structurally similar to pulmonary
surfactant, and proprietary drug delivery technologies being developed to
enable efficient delivery of aerosolized KL4 surfactant and other inhaled
therapies. Discovery Labs' strategy is initially focused on neonatology and
improving the management of respiratory distress syndrome (RDS) in premature
infants. Discovery Labs believes that its RDS product portfolio has the
potential to become the new standard of care for RDS and, over time, enable
the treatment of a significantly greater number of premature infants who could
benefit from surfactant therapy but are currently not treated.
The U.S. Food and Drug Administration (FDA) approved SURFAXIN^® (lucinactant)
Intratracheal Suspension for the prevention of RDS in premature infants who
are at high risk for RDS. SURFAXIN is the first synthetic, peptide-containing
surfactant approved by the FDA and the only alternative to animal derived
IMPORTANT SAFETY INFORMATION
SURFAXIN is intended for intratracheal use only. The administration of
exogenous surfactants, including SURFAXIN, can rapidly affect oxygenation and
lung compliance. SURFAXIN should be administered only by clinicians trained
and experienced with intubation, ventilator management, and general care of
premature infants in a highly supervised clinical setting. Infants receiving
SURFAXIN should receive frequent clinical assessments so that oxygen and
ventilatory support can be modified to respond to changes in respiratory
Most common adverse reactions associated with the use of SURFAXIN are
endotracheal tube reflux, pallor, endotracheal tube obstruction, and need for
dose interruption. During SURFAXIN administration, if bradycardia, oxygen
desaturation, endotracheal tube reflux, or airway obstruction occurs,
administration should be interrupted and the infant's clinical condition
assessed and stabilized. SURFAXIN is not indicated for use in acute
respiratory distress syndrome (ARDS).
For more information about SURFAXIN, please visit www.surfaxin.com.
Disclosure Note: The RDS population estimates and data included in the
discussion above have been derived from the following sources, among others:
IMS MIDAS data (MAT Sept 2008); CDC National Vital Statistics, 2005, Births by
birth weight (CDC Website); "Annual Summary of Vital Statistics: 2006",
Pediatrics, Martin et al.; Vermont Oxford Network (VON) data, 2006; UNICEF
data, 2005 (website); and our primary market research (2010). Discovery Labs'
estimate of the number of premature infants that could potentially benefit
from early surfactant therapy is derived from data from the foregoing sources,
taking into account several factors, including without limitation, gestational
age of premature infants, treatment rates for nCPAP, rates of nCPAP failure,
and incidence of RDS.
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 include those
related to Discovery Labs' research and development activities generally and,
in particular, Discovery Labs' AEROSURF development program, including, among
other things, (i)time-consuming and expensive pre-clinical studies, clinical
trials and other efforts, which may be subject to potentially significant
delays or regulatory holds, or failure, and (ii) regulatory requirements
relating to development and manufacture of the drug and aerosol delivery
components of Discovery Labs' combination drug/device products, as well as
those risks and uncertainties 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.
SOURCE Discovery Laboratories, Inc.
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