Discovery Labs Receives FDA Clearance of IND to Initiate AEROSURF® Phase 2 Clinical Program

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 clinical program.  

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.

About AEROSURF^®

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.

About SURFAXIN^®

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 surfactants.

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 status.

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.

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 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.