Results of AEROSURF® Phase 2a Clinical Program in Premature Infants 29 to 34 Weeks Gestational Age Presented at the 2015 Hot Topics in Neonatology Annual Meeting

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.

SOURCE Discovery Laboratories, Inc.

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