Discovery Laboratories, a specialty biotechnology company dedicated to advancing a new standard of respiratory critical care, has announced that the US Food and Drug Administration (FDA) has cleared the company's investigational new drug (IND) application for AEROSURF and the company can initiate its Phase II clinical program.
The Phase II clinical program is expected to include two studies.
The primary goal of the initial study, Phase II-a, 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 II-b, will be informed by the results of the Phase II-a study.
The primary objective of the Phase II-b study will be to determine optimal dose and to estimate the efficacy margin. Results of the Phase II-b study will inform the design of the Phase III efficacy and safety study. Phase II-b is expected to be conducted in multiple centers and completed by mid-2015.
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.