Pharmaxis Announces Positive Results of Phase 3 Cystic Fibrosis Trial
04 May 2009 - 6:02PM
PR Newswire (US)
SYDNEY, Australia, May 4 /PRNewswire-Asia-FirstCall/ --
Pharmaceutical company Pharmaxis (ASX:PXS)(NASDAQ:PXSL) is pleased
to announce positive results of its recently completed
international Phase III trial of Bronchitol(TM) 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
(Pulmozyme(TM)). 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 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." Dr Alan Robertson
will host a teleconference to discuss the results on Tuesday 5th
May at 8.00am (Sydney), (Monday 4th May at 6.00pm U.S. East Coast,
3.00pm U.S. West Coast). Telephone access (toll free) details are
below: -- Australia: 1800 131 617 -- USA/Canada: 866 746 2596 --
UK: 0800 376 8339 -- NZ: 0800 446 958 -- Switzerland: 0800 001 230
Online (global access):
http://services.choruscall.com/links/pharmaxis090505.html About
Pharmaxis Pharmaxis (ACN 082 811 630) is a specialist
pharmaceutical company involved in the research, development and
commercialization of therapeutic products for chronic respiratory
and immune disorders. Its development pipeline of products includes
Aridol for the diagnosis of asthma, Bronchitol for cystic fibrosis,
bronchiectasis and chronic obstructive pulmonary disease (COPD),
PXS25 for the treatment of lung fibrosis and PXS4159 for asthma.
Founded in 1998, Pharmaxis is listed on the Australian Securities
Exchange (symbol PXS), and on NASDAQ Global Market (symbol PXSL).
The company is headquartered in Sydney at its TGA-approved
manufacturing facilities. For more information about Pharmaxis, go
to http://www.pharmaxis.com.au/ or contact Investor Relations on
phone +61 2 9454 7200. About Bronchitol Pharmaxis Ltd is developing
Bronchitol for the management of chronic obstructive lung diseases
including cystic fibrosis, and bronchiectasis. Bronchitol is a
proprietary dry-powder mannitol, precision formulated for delivery
to the lungs through an easy-to-use, pocket-size, portable inhaler.
Once inhaled its action on mucus helps restore normal lung
clearance mechanisms. Clinical studies have shown Bronchitol to be
effective and well tolerated in stimulating mucus hydration and
clearance in people with chronic obstructive lung diseases. In
particular, Bronchitol has been shown to increase mucus clearance
from the lungs and significantly improve quality of life for people
with bronchiectasis. Additional studies have also shown Bronchitol
to improve lung function in people affected by cystic fibrosis.
About Cystic Fibrosis In a healthy person, there is a constant flow
of mucus over the surfaces of the air passages in the lungs,
removing debris and bacteria. In CF, an inherited disease, a
defective gene disrupts ion transport across the epithelial
membrane within cells. In the lungs, this leads to a depletion of
the airway surface liquid that normally bathes the cilia, and a
resultant reduction in mucociliary clearance. The result is thick,
sticky mucus that clogs the lungs, severely restricting the natural
airway-clearing process. It also increases the potential for
bacteria to become trapped and for inflammation, thus creating an
unhealthy lung environment that leads to life- threatening lung
infections. Forward-Looking Statements The statements contained in
this media release that are not purely historical are
forward-looking statements within the meaning of Section 21E of the
Securities Exchange Act of 1934, as amended. Forward-looking
statements in this media release include statements regarding our
expectations, beliefs, hopes, goals, intentions, initiatives or
strategies, including statements regarding the potential for Aridol
and/or Bronchitol. All forward-looking statements included in this
media release are based upon information available to us as of the
date hereof, and we assume no obligation to update any such
forward-looking statement as a result of new information, future
events or otherwise. We can not guarantee that any product
candidate will receive FDA or other regulatory approval or that we
will seek any such approval. Factors that could cause or contribute
to such differences include, but are not limited to, factors
discussed in the "Risk Factors and Other Uncertainties" section of
our Form 20-F lodged with the U.S. Securities and Exchange
Commission. CONTACT: Alan Robertson Chief Executive Officer Tel:
+61-2-9454-7200 Email: RELEASED THROUGH: Australia: Felicity
Moffatt Tel: +61-418-677-701 Email: United States: Brandon Lewis
Trout Group Tel: +1-646-378-2915 Email: DATASOURCE: Pharmaxis Ltd
CONTACT: Alan Robertson, +61-2-9454-7200, ; Felicity Moffatt,
+61-418-677-701, , both of Pharmaxis; Brandon Lewis of Trout Group
for Pharmaxis, +1-646-378-2915, Web site:
http://www.pharmaxis.com.au/
Copyright
Pharmaxis Limited - Sponsored Adr (Australia) (MM) (NASDAQ:PXSL)
Historical Stock Chart
From Apr 2024 to May 2024
Pharmaxis Limited - Sponsored Adr (Australia) (MM) (NASDAQ:PXSL)
Historical Stock Chart
From May 2023 to May 2024