Pharmaxis reports positive results from Phase III cystic fibrosis trial

Australian pharmaceutical company Pharmaxis has reported positive results of its recently completed international Phase III trial of Bronchitol in people with cystic fibrosis.

The primary endpoint of the trial was to assess whether Bronchitol improves lung function as measured by a change in forced expiratory volume in one second (FEV1) when administered 400mg twice per day for six months. The clinical trial comfortably met this endpoint. 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 six and was sustained through to week 26, said Pharmaxis.

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 cystic fibrosis (CF) therapeutic, dornase alfa (Pulmozyme). This endpoint was also successfully achieved. For patients being treated with concurrent dornase alfa, FEV1 improved after six months by 5.2% from baseline (p=0.002 versus placebo), the company said.

Over the six 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).

Alan Robertson, CEO of Pharmaxis, said: We are delighted that Bronchitol performed so well in this important long term study and we now know that it can change the therapeutic landscape for many of the 75,000 people with this disease. In a trial which recruited a wide range of patients with varying disease severity, Bronchitol showed significant health benefits.

As the first dry powder formulation to publish positive results in cystic fibrosis it promises convenience for patients who have complex daily schedules dominated by difficult treatment regimens.