Pharmaxis recently released positive results of its completed international phase 3 trial of Bronchitol in patients with cystic fibrosis (CF). Bronchitol successfully met the primary and key secondary endpoints of the trial.
The primary endpoint of the trial—one of the largest clinical trials conducted on CF—was to assess whether Bronchitol improves lung function as measured by a change in Forced Expiratory Volume in 1 second (FEV1) when administered 400 mg twice per day for 6 months. During the study patients treated with Bronchitol had a statistically significant improvement in lung function from baseline of 6.6% (p=0.001 versus placebo). Lung function improved at week 6 and was sustained through to week 26.
The key secondary endpoint of the trial was to assess whether Bronchitol further improves lung function in patients already being treated with the most commonly used CF therapeutic,
dornase alfa (Pulmozyme™). In patients being treated with concurrent dornase alfa, FEV1 improved after 6 months by 5.2% from baseline (p=0.002 versus placebo).
Over the 6-month treatment period, there was significant lung function improvement for both those patients being treated with Bronchitol and dornase alfa (p=0.008 versus placebo) and those being treated with Bronchitol alone (p=0.015 versus placebo).
Bronchitol was well-tolerated overall in the trial subjects and had a favorable safety profile. There was no difference in adverse events or serious adverse events between the treatment groups. The most common adverse event was cough, which was mild to moderate in most cases and similar between the treatment arms.
The trial included 324 subjects between the ages of 6 and 56 years. The lung function ranges were from 26% to 94% of the predicted FEV1. The randomized, double blind, placebo controlled study was conducted in 40 centers in the United Kingdom, Ireland, Australia, and New Zealand. Pharmaxis plans to file a marketing application later this year.