WARRINGTON, Pa., May 20, 2015 /PRNewswire/ -- Discovery
Laboratories, Inc. (NASDAQ: DSCO), a specialty biotechnology
company focused on developing aerosolized KL4 surfactant therapies
for respiratory diseases, today reported that the first patient has
been enrolled in the next phase of its phase 2a clinical evaluation
of AEROSURF®, which is designed to evaluate the
safety and tolerability of higher and repeat doses of aerosolized
KL4 surfactant administered to premature infants 29 to 34 weeks
gestational age (GA) who are receiving nasal continuous positive
airway pressure (nCPAP) for respiratory distress syndrome (RDS),
compared to infants receiving nCPAP alone. This phase is
intended to expand upon the knowledge gained in the recently
completed phase 2a clinical trial, the results of which were
reported in mid-May 2015. The Company expects to complete
this study in the fourth quarter of 2015.
The recently completed phase 2a clinical trial was
designed to assess the safety and tolerability of three escalating
single doses of aerosolized KL4 surfactant in premature infants 29
to 34 weeks gestational age who are receiving nCPAP for RDS,
compared to infants receiving nCPAP alone. All key objectives
were achieved, including the primary objective of demonstrating a
favorable safety and tolerability profile of AEROSURF, establishing
proof of concept for the Company's proprietary technology platform
based on physiological data suggesting that aerosolized KL4
surfactant is being delivered into the lung of premature infants,
and acceptable performance of the novel capillary aerosol generator
(CAG) technology in the neonatal intensive care unit (NICU).
This next phase 2a dose expansion study is a multicenter,
randomized, open-label, controlled study and is designed to
evaluate the safety and tolerability of aerosolized KL4 surfactant
administered in higher and repeat doses in premature infants 29 to
34 weeks GA who are receiving nCPAP for RDS, compared to infants
receiving nCPAP alone. In addition to safety and
tolerability, another objective of the expansion study is to
continue to evaluate the physiological effects of higher and repeat
dosing of aerosolized KL4 surfactant. The results of this
expansion trial are expected to provide further insight and data to
support the design of the planned AEROSURF phase 2b clinical
program.
"The initiation of the 2a dose expansion phase is an important
next step to build upon the promising results observed in our
initial phase 2a clinical trial. We believe that, with higher
and/or repeat doses, AEROSURF may have the potential to reduce the
need for invasive intubation due to nCPAP failure in this fragile
patient population. Our objective for this phase is to gain
additional knowledge that can be incorporated into the design of
our phase 2b clinical trial," commented Steve Simonson, M.D., Discovery Labs' Chief
Development Officer.
In addition, the Company plans the following phase 2 clinical
assessments going forward: (1) A phase 2a clinical trial assessing
the safety and tolerability of AEROSURF of escalating doses
administered to 26 to 28 week gestational age infants, with an
ability to administer repeat doses. The Company anticipates
completing this trial in the fourth quarter of 2015; and (2) A
phase 2b clinical trial to determine the optimal dose and define
the expected efficacy margin of AEROSURF treatment. The Company
anticipates completing this trial in mid 2016.
About AEROSURF®
AEROSURF (lucinactant) for inhalation 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 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 nasal continuous positive airway pressure
(nCPAP), without subjecting them to invasive 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 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 intubation and
mechanical ventilation
About Discovery Labs
Discovery Laboratories, Inc. is a specialty 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
Securities Litigation Reform Act of 1995. These
forward-looking statements are subject to 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.