Discovery Lab enrolls first patient in phase 2a clinical trial of AEROSURF

May 20 2015

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.