Pharmaxis' Bronchitol inhaler has encouraging Phase 3 trial results for cystic fibrosis patients
Australian listed pharmaceutical company Pharmaxis (ASX:PXS, Nasdaq:PXSL) has received encourgaging results for its recently completed international Phase III trial of the company's Bronchitol portable inhaler in patients with cystic fibrosis.
Bronchitol has received Orphan Drug Designation and fast track status for cystic fibrosis from the U.S. Food and Drug Administration and Orphan Drug Designation from the European Medicines Agency.
The trial was a multi-centre, randomised, double blind, placebo controlled, 26 week study, with an optional further 6 month open label uncontrolled period. It was conducted in 40 centres in the United Kingdom, Ireland, Australia and New Zealand.
The primary endpoint of the trial was to assess whether Bronchitol improves lung function as measured by a change in FEV when administered twice per day for six months.
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, rhDNase. Additional endpoints included changes in the Forced Vital Capacity of the lung, pulmonary exacerbations and antibiotic use.
Clinical Results showed there was a clinically meaningful change from baseline (119mL) and placebo (93mL) at week 26 with Bronchitol for FEV1 (p<0.001).>
For the subgroup of patients on concomitant rhDNase there was also a significant improvement in FEV1 from baseline (88mL) and from placebo (109mL) at week 26 with Bronchitol (p=0.001). Again, there was an immediate and sustained improvement in FEV1 over the 26 week period of the study (p=0.003).
While the study was not powered to show a reduction in the secondary endpoint of exacerbation, the rate of a protocol defined pulmonary exacerbation (PDPE) per subject for the 26 weeks was lower for Bronchitol versus control: overall reduction in rate of 25% (p=0.2).
In September 2008 Pharmaxis submitted a marketing application in Australia for Bronchitol as a treatment for bronchiectasis. This follows a long-term Phase 3 safety study of more than 360 subjects finding that Bronchitol is safe, with no serious adverse events attributable to the medicine. This supports results from an earlier stage of the trial showing that Bronchitol delivered a highly significant improvement in users' quality of life.
The medicine is expected to be approved for bronchiectasis first in Australia, and later in the U.S. and Europe following the completion of a further Phase 3 trial. Bronchitol will be the first targeted medication for sufferers in over 20 years.
Small cap fund manager Acorn Capital is one of the largest shareholders in Pharmaxis owning 7.8% of the company.