Pharmaxis Announces Positive Results of Phase 3 Cystic Fibrosis Trial
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
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.
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
designed in consultation with the European Medicines Agency (EMEA) with the
objective of seeking a marketing authorisation for Bronchitol for treating
cystic fibrosis in
marketing application later this year.
The first scientific presentation of the results will be made at the June
European Cystic Fibrosis Society meeting in
detailed account of the results of the trial is planned to be presented at the
North American Cystic Fibrosis conference in
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.
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
Tuesday 5th May at
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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
information about Pharmaxis, go to http://www.pharmaxis.com.au or contact
Investor Relations on phone +61 2 9454 7200.
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.
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: firstname.lastname@example.org RELEASED THROUGH: Australia: Felicity Moffatt Tel: +61-418-677-701 Email: email@example.com United States: Brandon Lewis Trout Group Tel: +1-646-378-2915 Email: firstname.lastname@example.org
SOURCE Pharmaxis Ltd