Discovery Laboratories initiates AEROSURF phase 2b clinical trial in premature infants with RDS

Dec 29 2015

Discovery Laboratories, Inc. (NASDAQ: DSCO), a biotechnology company focused on developing aerosolized KL4 surfactant therapies for respiratory diseases, today announced that it has initiated its AEROSURF® phase 2b clinical trial in premature infants 26 to 32 weeks gestational age (GA) receiving non-invasive nasal continuous positive airway pressure (nCPAP) for respiratory distress syndrome (RDS). The trial is designed to evaluate premature infants receiving aerosolized KL4 surfactant (including potentially repeat doses) compared to nCPAP alone. Two dose groups will be evaluated. The primary objective of the trial is to demonstrate evidence of efficacy and, if successful, inform the design of a phase 3 clinical program.

"Currently, surfactant therapy is primarily administered to premature infants with RDS via invasive endotracheal intubation. The trend in neonatology is to use less- or non-invasive methods to treat RDS but these preclude the administration of surfactant therapy in a timely manner. AEROSURF allows for non-invasive delivery of aerosolized KL4 surfactant to premature infants receiving nCPAP," commented Steve Simonson, M.D., Discovery Labs' Chief Development Officer. "The initiation of the phase 2b trial is an important next step to build upon the results observed in our recently completed phase 2a clinical trials in premature infants 29 to 34 weeks GA. Data from these trials are encouraging and suggest that AEROSURF may reduce the incidence of nCPAP failure and the need for intubation and delayed surfactant therapy. If this result is confirmed as we advance our development program, AEROSURF could represent a significant medical advancement in neonatology."

The AEROSURF phase 2b trial is a multicenter, randomized, controlled study with masked treatment assignment in premature infants 26 to 32 weeks GA receiving nCPAP for RDS. The trial is designed to evaluate aerosolized KL4 surfactant administered in two dose groups (25 and 50 minutes), including the ability for infants to receive repeat doses, compared to premature infants receiving standard care of nCPAP alone. The following endpoints will be evaluated: time to nCPAP failure (the need for intubation and delayed surfactant therapy), incidence of nCPAP failure and physiological parameters indicating the effectiveness of lung function. The trial will enroll up to 250 premature infants at up to 50 sites in the United States, Canada, Europe and Latin America. Enrollment in the phase 2b trial is starting with premature infants 29 to 32 weeks GA, followed by premature infants 26 to 28 weeks GA after completion of the ongoing phase 2a trial in this age group.