Discovery Laboratories, Inc., a biotechnology company focused on developing aerosolized KL4 surfactant therapies for respiratory diseases, today announced that it has initiated its AEROSURF® phase 2b clinical trial in premature infants 26 to 32 weeks gestational age (GA) receiving non-invasive nasal continuous positive airway pressure (nCPAP) for respiratory distress syndrome (RDS). The trial is designed to evaluate premature infants receiving aerosolized KL4 surfactant (including potentially repeat doses) compared to nCPAP alone. Two dose groups will be evaluated. The primary objective of the trial is to demonstrate evidence of efficacy and, if successful, inform the design of a phase 3 clinical program.
"Currently, surfactant therapy is primarily administered to premature infants with RDS via invasive endotracheal intubation. The trend in neonatology is to use less- or non-invasive methods to treat RDS but these preclude the administration of surfactant therapy in a timely manner. AEROSURF allows for non-invasive delivery of aerosolized KL4 surfactant to premature infants receiving nCPAP," commented Steve Simonson, M.D., Discovery Labs' Chief Development Officer. "The initiation of the phase 2b trial is an important next step to build upon the results observed in our recently completed phase 2a clinical trials in premature infants 29 to 34 weeks GA. Data from these trials are encouraging and suggest that AEROSURF may reduce the incidence of nCPAP failure and the need for intubation and delayed surfactant therapy. If this result is confirmed as we advance our development program, AEROSURF could represent a significant medical advancement in neonatology."
The AEROSURF phase 2b trial is a multicenter, randomized, controlled study with masked treatment assignment in premature infants 26 to 32 weeks GA receiving nCPAP for RDS. The trial is designed to evaluate aerosolized KL4 surfactant administered in two dose groups (25 and 50 minutes), including the ability for infants to receive repeat doses, compared to premature infants receiving standard care of nCPAP alone. The following endpoints will be evaluated: time to nCPAP failure (the need for intubation and delayed surfactant therapy), incidence of nCPAP failure and physiological parameters indicating the effectiveness of lung function. The trial will enroll up to 250 premature infants at up to 50 sites in the United States, Canada, Europe and Latin America. Enrollment in the phase 2b trial is starting with premature infants 29 to 32 weeks GA, followed by premature infants 26 to 28 weeks GA after completion of the ongoing phase 2a trial in this age group.
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.
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 the timing of Discovery Labs' planned clinical trials and its ability successfully to complete its development programs, secure regulatory approval of its product candidates in the U.S. and in markets outside the U.S., 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.