Pharmaxis announced that the first pivotal phase-3 clinical trial of Bronchitol for the treatment of cystic fibrosis has completed enrolment. The first efficacy data from the trial is expected to be available during the first half of 2009. The trial commenced its dosing phase in April 2007, reached its initial recruitment target of 250 subjects in June 2008 and closed with 325 subjects enrolled. Recruitment was extended to cater for a lower numbers of patients than anticipated entering the study on rhDNASE.
The trial is a double blind, comparator controlled, randomised study comparing 400 mg of Bronchitol twice a day to control. The trial includes a 26-week efficacy and safety component, followed by a 26-week open-label Bronchitol safety extension. The primary efficacy end-point is change from baseline in FEV1 (forced expiratory volume in one second) after 26 weeks.
Pharmaxis chief executive officer Alan Robertson said, "It is rewarding to reach this milestone in bringing Bronchitol to the marketplace. The study has enrolled well, and we look forward to the results helping to shape cystic fibrosis clinical practice in the future."
Pharmaxis has received Orphan Drug Designation and fast track status from the US Food and Drug Administration and Orphan Drug Designation from the European Medicines Agency for Bronchitol in cystic fibrosis. Bronchitol is designed to hydrate the airway surface of the lungs, improve lung hygiene and promote normal lung clearance. Approximately 75,000 people in the major pharmaceutical markets are affected with cystic fibrosis and no products have been approved to improve lung hydration.
Pharmaxis Ltd is developing Bronchitol for the management of chronic obstructive lung diseases including bronchiectasis, cystic fibrosis and chronic bronchitis. Bronchitol is a proprietary formulation of mannitol administered as a dry powder in a convenient hand-held inhaler.
Pharmaxis is a specialist pharmaceutical company involved in the research, development and commercialization of therapeutic products for chronic and acute respiratory diseases.