Pharmaxis Ltd (PXS.AX) Cystic Fibrosis Trial In Children Returns Positive Data
/4/2008
SYDNEY, Australia, April 3, 2008 -- Pharmaxis Ltd announced today that a Phase II clinical trial in children with cystic fibrosis demonstrated excellent lung function improvement following three months treatment with Bronchitol that matched that achieved by the current marketed product rhDNase(1). The improvement in lung function after three months on Bronchitol reflects that seen in a previous study following two weeks of treatment.
The study was an independent investigator initiated study conducted in the United Kingdom in 20 children with a mean age of 13 years. Those children enrolled in the trial completed three months treatment with each of three different therapies - Bronchitol alone, both Bronchitol and rhDNase together and rhDNase alone. The trial measured changes in lung function, airway inflammation, infections, and quality of life.
At the end of the treatment period, lung function as determined by measuring FEV1 (the amount of air that can be forcibly exhaled in 1 second) improved by:
-- 7% while the subjects were on Bronchitol -- 7% while subjects were on rhDNase and -- 2% while subjects were on both agents together.
The study had insufficient numbers to reach a definitive statistical conclusion.
People affected by cystic fibrosis typically experience a decline in lung function of 1-2% every year of their life, as measured by FEV1.
Pharmaxis Chief Executive Officer Alan Robertson said: 'While not on the regulatory approval path, this is the first time we have had an opportunity to measure the performance of Bronchitol following 3 months continuous treatment and it bodes well for the pivotal 6 month Phase 3 trial that is currently in progress. Although the two agents worked less well when taken together, the patient numbers are low and a previous study has shown a benefit to using both therapies together.'
Bronchitol is designed to hydrate the airway surface, improve lung hygiene and promote normal lung clearance. rhDNase is designed to improve mucus viscosity. Additional data from this trial will be presented by the independent investigator at a forthcoming scientific congress. A European, Pharmaxis sponsored, regulatory Phase III clinical trial, designed to lead to a marketing application for Bronchitol in adults and children with cystic fibrosis, is currently enrolling subjects.
Approximately 75,000 people in the major pharmaceutical markets are affected with cystic fibrosis and no products have been approved to improve lung hydration.
To find out more about Pharmaxis, go to http://www.pharmaxis.com.au.
1: rhDNase is marketed as Pulmozyme, which is a trade mark of Genentech Inc.
SOURCE: Pharmaxis Ltd, Sydney, Australia CONTACT: Alan Robertson - Chief Executive Officer Ph: +61 2 9454 7200 or email [email protected]