Pharmaxis reaches milestone in cystic fibrosis trial

Pharmaxis has announced completion of the first component of its Phase III international trial assessing the effectiveness of Bronchitol in people with cystic fibrosis.

The final subject has now completed their final clinical visit and the trial has run to time and budget. The first subject entered the trial in April 2007.

The trial was conducted in 40 centres in four different countries and involved 325 randomized subjects with cystic fibrosis. The trial was a double blind, placebo controlled study designed with the assistance of the European regulatory agency (EMEA) and with the objective of seeking a marketing approval for Bronchitol for treating cystic fibrosis in Europe and elsewhere.

The results of the trial are expected to be available later this month after data review and statistical analysis of the various endpoints have been completed. The first objective of the trial is to demonstrate an improvement in lung function. Consistent loss of lung function is the leading cause of death of people with cystic fibrosis.

Dr Alan Robertson, Pharmaxis Chief Executive Officer said: "We are very pleased to announce this major milestone for Pharmaxis and look forward to the results of the study with great interest. It is hoped Bronchitol will change the therapeutic landscape for people living with cystic fibrosis and provide a new therapeutic regimen that helps to prolong life."

The second component of the trial will examine the safety of Bronchitol in people with cystic fibrosis.

Pharmaxis has received Orphan Drug Designation and fast track status from the U.S. 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, and promote normal lung mucus clearance in order to improve lung function, break the cycle of repeated respiratory infections and blockages, and improve quality of life for patients with CF. Approximately 75,000 people in the major pharmaceutical markets are affected with cystic fibrosis and no products have been approved to improve lung hydration.

A second Phase III clinical trial is actively recruiting in centres across the USA, Canada, Argentina, Germany, Belgium and France. This second trial has been designed with the assistance of the FDA through its Special Protocol Assessment Scheme and is the second of two trials required by the FDA before Bronchitol is considered for marketing in the USA.