Windtree Therapeutics has announced that its Phase 2b clinical trial of Aerosurf aerosolized KL4 surfactant for the treatment of respiratory distress syndrome (RDS) in premature infants receiving nasal continuous positive airway pressure (nCPAP) failed to meet its primary endpoint, reduction in nCPAP failure at 72 hours.
The study enrolled 221 infants that received either nCPAP alone or nCPAP plus either a 25 minute dose or a 50 minute dose. nCPAP failure rates in 25 minute and 50 minute Aerosurf dose groups were similar to those in babies receiving nCPAP alone.
The company blames the failure of the trial to meet the endpoint on disruptions in the delivery of Aerosurf to the patients due to disposable cartridge filters that clogged in almost a quarter of the cases.
According to Windtree data from those patients in the 50 minute dose group for whom the delivery of surfactant was uninterrupted show an nCPAP failure rate of 31% compared to 44% for nCPAP alone.
The company said that Aerosurf was well tolerated, with a comparable incidence of adverse events and serious adverse events in all groups, and that babies that received Aerosurf and still experienced nCPAP failure appeared to require less time on mechanical ventilation than babies that experienced nCPAP failure in the control group.
Windtree Chief Medical Officer Steve Simonson commented, “While the results did not meet the planned top-line analysis, we are very encouraged to see that, when dose is delivered as intended, the 50 minute dose exhibited a positive treatment effect with a safety profile comparable to that of nCPAP and consistent with the results we obtained in our previously completed phase 2a open-label clinical trial in similar gestational age infants.”
CEO Craig Fraser added, “While analysis of data is ongoing, we believe we have achieved a number of important clinical objectives including providing evidence of a treatment effect in the higher dose and the potential to replicate desired results. We look forward to finalizing a regulatory and clinical plan to incorporate our next generation ADS, along with other learning’s from this trial, as we pursue a path that will allow us to advance Aerosurf into phase 3 development.”
Read the Windtree Therapeutics press release.
