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.
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.
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.
SOURCE Discovery Laboratories, Inc.