The US Food and Drug Administration (FDA) has cleared Discovery Laboratories' investigational new drug (IND) application for Aerosurf and to initiate phase II clinical programme.

The phase II clinical programme is expected to include two studies. The primary goal of the initial study, phase IIa, 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 US and is expected to be completed by mid-2014. The design of the second study, phase IIb, will be informed by the results of the phase IIa study. The primary objective of the phase IIb study will be to determine optimal dose and to estimate the efficacy margin. Results of the phase IIb study will inform the design of the phase III efficacy and safety study. Phase IIb is expected to be conducted in multiple centres 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.

Discovery Labs believes that the neonatal medical community increasingly recognizes the potential of a synthetic, peptide-containing surfactant, such as SURFAXIN and, importantly, a less-invasive method of delivering surfactant, such as Aerosurf, to treat premature infants at risk of suffering from respiratory disorders.