Discovery Labs to present lucinactant Phase 2a clinical study results at NACFC Conference

Discovery Laboratories, Inc. (Nasdaq:DSCO) today announces that the Phase 2a clinical study of aerosolized KL4 surfactant (lucinactant) in patients with cystic fibrosis (CF) has completed and the results from the study will be presented at the upcoming North American Cystic Fibrosis Conference (NACFC; October 21-23, 2010). The trial was conducted as an investigator-initiated study under the direction of Dr. Scott H. Donaldson at The University of North Carolina (UNC) with support from PARI Pharma, and was funded by Cystic Fibrosis Foundation Therapeutics, Inc., a nonprofit affiliate of the Cystic Fibrosis Foundation. Preliminary observations from this trial include the determination that delivery of aerosolized KL4 surfactant to CF patients is feasible and that aerosolized KL4 surfactant was generally safe and well tolerated in this trial. Additionally, no serious adverse events (SAEs) were reported following treatment with aerosolized KL4 surfactant. The trial results, including assessment of exploratory effectiveness of aerosolized KL4 surfactant in CF patients, are embargoed until the NACFC presentation.

Dr. Thomas F. Miller, Chief Operating Officer of Discovery Labs, commented, "We are pleased that the outcome of this trial demonstrated feasibility of delivering aerosolized KL4 surfactant to patients with CF, and potentially to patients with other respiratory disorders entailing compromised mucociliary function. We are fortunate to be able to collaborate with leaders in the CF academic community such as Dr. Donaldson and his team at UNC. We look forward to continued collaboration with the UNC team to define next steps and to the presentation of the results from this trial at NACFC later this month."

This study was the first-of-its-kind double-blind, randomized crossover Phase 2a study to evaluate whether Discovery Labs' aerosolized KL4 surfactant, delivered by an investigational eFlow Nebulizer System (PARI Pharma GmbH. Munich, Germany),  is safe and well tolerated in patients with mild to moderate CF lung disease, compared with aerosolized saline control.  In addition, the short-term effectiveness of aerosolized KL4 surfactant was assessed. CF is caused by a genetic mutation that leads to the production of thick, viscous mucus that is difficult to clear from the airways of the lung. The abnormal mucus allows for chronic airway infections that lead to airway destruction, decreased lung function, and ultimately, death. Previous preclinical and exploratory clinical studies suggest that therapeutic surfactants may improve mucociliary clearance, thereby potentially preventing further compromise of lung function. KL4 surfactant may be a uniquely advantaged new therapeutic approach in this regard, since it has been demonstrated to be safe and well tolerated in several previous clinical trials and it can be effectively aerosolized.