Pharmaxis (ASX:PXS) has today revealed its recently completed international Phase 3 trial of Bronchitol® (mannitol) in adults with cystic fibrosis (CF) met its primary endpoint.
VIDEO INTERVIEW: Gary Phillips, chief executive officer, Pharmaxis
Phillips commented: "The study results reinforce the body of clinical trial evidence for Bronchitol and I’d like to thank both the global CF community and the Pharmaxis team who have delivered a very high quality study with low rates of patient withdrawal in both treatment arms."
Pharmaxis has partnered its work on Bronchitol for the United States with Chiesi Group, a global pharmaceutical company headquartered in Parma, Italy.
Chiesi USA, the American affiliate of Chiesi Group is responsible for completing and filing the updated Bronchitol NDA with the US Food and Drug Administration (FDA).
Key outcomes
The study recruited adult CF patients with all grades of disease that were already on the best standard of care.
Importantly, the study demonstrated the superiority of Bronchitol versus the comparator on the primary endpoint (FEV1 change from baseline over 26 week treatment period), with an effect of 54 ml (p=0.020), corresponding to a 2.2% relative change (p=0.025).
The improvement in lung function was less than that seen in the adult CF population in previously reported phase 3 studies.
The company added that no statistically significant differences between treatment groups in secondary endpoints were recorded, although a trend was observed in favour of Bronchitol for another lung function parameter (FVC).
Bronchitol had a good safety profile with similar rates of adverse events seen compared to control.
FDA resubmission is expected in 2018.