Ann: Pharmaxis Announces Positive Results of Phas, page-2

re: Ann: Pharmaxis Announces Positive Results... Pharmaceutical company Pharmaxis (ASX:PXS, Nasdaq:PXSL) is pleased to announce positive
results of its recently completed international Phase III trial of BronchitolTM in people with cystic
fibrosis. The study represents one of the largest clinical trials conducted in 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 1 second (FEV1) when administered 400
mg 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 6 and was sustained
through to week 26.
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,
dornase alfa (PulmozymeTM). This endpoint was also successfully achieved. For patients being
treated with concurrent dornase alfa, FEV1 improved after 6 months by 5.2% from baseline
(p=0.002 versus placebo).
Over the 6 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).
Consistent loss of lung function, is the leading cause of death for people with cystic fibrosis and
this deterioration now averages 1-2% per year.
Dr Alan Robertson, Pharmaxis Chief Executive Officer 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.”
For the 324 subjects randomized, the treatment groups were balanced with respect to key
demographic and background characteristics: the average age was approximately 23 years old,
the mean lung function on entry to the trial was 62% of the predicted normal FEV1, and 55% of
the population were using dornase alfa. The ages ranged from 6 years to 56 years and the lung
function ranges were from 26% to 94% of the predicted FEV1.
In the trial subjects, Bronchitol was well-tolerated overall and had a favourable safety profile.
At screening, only 7% of recruited subjects were unable to tolerate Bronchitol and were
therefore not entered into the study. There was no difference in adverse events or serious
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adverse events between the treatment groups. The most common adverse event was cough,
which was mild to moderate in most cases and similar between the treatment arms.
The trial was conducted in 40 centres in the United Kingdom, Ireland, Australia and New
Zealand. It was a double blind, placebo controlled study designed in consultation with the
European Medicines Agency (EMEA) with the objective of seeking a marketing authorisation for
Bronchitol for treating cystic fibrosis in Europe and elsewhere. Pharmaxis will now move to file
a marketing application later this year.
The first scientific presentation of the results will be made at the June European Cystic Fibrosis
Society meeting in France. In addition a more detailed account of the results of the trial is
planned to be presented at the North American Cystic Fibrosis conference in Minneapolis in
October.
Bronchitol has received Orphan Drug Designation and development fast track status from the
U.S. Food and Drug Administration and Orphan Drug Designation from the European Medicines
Agency.
Bronchitol is designed to hydrate the airway surface of the lungs, and promote normal lung
mucus clearance. No new products have been approved anywhere in the world for cystic
fibrosis for more than 10 years.
Dr Alan Robertson said: ”We recognise that many people have been involved in the
development of Bronchitol and their assistance has been invaluable. The trial was complex and
challenging and this landmark result is a tribute to their efforts.”