Pharmaxis Ltd. (PXSL)

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Bronchitol Cystic Fibrosis Dose Trial Results Positive
Monday August 11, 8:10 pm ET
Bronchitol Demonstrates Dose-Related Improvements in Lung Function of Cystic Fibrosis Patients

SYDNEY, Australia, Aug. 11 /Xinhua-PRNewswire-FirstCall/ -- Speciality pharmaceutical company Pharmaxis (ASX: PXS - News; Nasdaq: PXSL - News) today announced results from its Phase II trial, DPM-CF-202, in subjects with cystic fibrosis.

The trial achieved its primary end point of demonstrating a dose dependent improvement in lung function as measured by FVC (forced vital capacity) and FEV1 (the amount of air that can be forcibly exhaled in 1 second). At the end of the two-week Bronchitol treatment periods, changes in lung function were as follows:

-- 400 mg treatment group: FEV1 increased by 8.6%
(139 mls, p=0.0006 vs 40 mg)
-- 240 mg treatment group: FEV1 increased by 4.6% (87 mls)
-- 120 mg treatment group: FEV1 increased by 3.7% (42 mls)
-- 40 mg treatment group: FEV1 decreased by -1.6% (-33 mls)

FVC changed by +7.9% on 400 mg (p=0.0004 vs 40 mg), +3.9% on 240 mg, +1.5% on 120 mg and -0.6% on 40 mg.

Pharmaxis Chief Executive Officer Alan Robertson said, "The excellent result from this trial reaffirms that the 400 mg Bronchitol dose being used in the Phase 3 trials is optimal for its clinical effectiveness. We look forward to the results from the ongoing Phase 3 studies and to bringing Bronchitol to the market as rapidly as possible."

The study was an open, randomised comparison of 400mg, 240 mg, 120 mg and 40 mg of Bronchitol in 48 patients with cystic fibrosis at 12 centres across Canada and Argentina. Bronchitol was administered twice a day for 14 days in a crossover design.

Secondary endpoints of the study included other spirometry and quality of life measures. These measures also showed a positive effect for 400 mg Bronchitol on MMEF (maximum mid expiratory flow) and the respiratory domain of the cystic fibrosis quality of life questionnaire (CFQR).

Additionally, no serious adverse events emerged during the 400 mg treatment period and the adverse event profile was similar across all doses.

People affected by cystic fibrosis typically experience a decline in lung function of 1-2% per year during their life, as measured by FEV1.

Pharmaxis has received Orphan Drug Designation and fast track status from the Food and Drug Administration (FDA) for Bronchitol in cystic fibrosis.

Bronchitol is designed to hydrate the airway surface, improve lung hygiene and promote normal lung clearance. Additional data from this trial will be presented 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 due to report preliminary data early in 2009.

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 .

About the Trial

The following information is provided in accord with the ASX and AusBiotech Code of Best Practice for Reporting by Biotechnology, Medical Device and other Life Sciences Companies.

Name of Trial DPM-CF-202 (a Phase II study with Bronchitol)
Blinding Status Open
Design Crossover, Dose response
Treatment Route Inhalation
Frequency Twice daily
Dose levels 400mg, 240 mg, 120 mg, 40 mg for 2 weeks,
1-2 weeks washout between doses
No of subjects PP population 38
Subject Selection diagnosis of cystic fibrosis (sweat test or
Criteria genotype), of either gender, aged 7 years or
more, baseline FEV1 of between 40%
and 80% of the predicted normal value or a
decline in FEV1 of 20% or more in the last 12
months for those >80% predicted
Study population Median age: 16 yrs, mean FEV1: 63% predicted
Trial Location Canada and Argentina
Commercial partners None

Primary end points:
Change in FEV1 8.6% increase (139 mls) on 400 mg Bronchitol.
-1.6% on 40 mg (p=0.0006). 4.6% on 240 mg,
3.7% on 120 mg
Change in FVC 7.9% increase in FVC on 400 mg vs -0.6% on 40 mg
(p=0.0004). 3.9% on 240 mg, 1.5% on 120 mg

Secondary end points:
Other lung function 11.9% increase in MMEF on 400 mg (vs -0.3% on 40
measures; mg, change did not reach significance)
CF Questionnaire Improved 6.3 points on 400 mg vs -0.2 on 40 mg
Safety/adverse events No serious adverse events on 400 mg Bronchitol

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Pharmaxis Concludes Special Protocol Assessment with FDA for Bronchitol Phase 3 Trial
Thursday June 19, 7:05 pm ET

SYDNEY, Australia, June 19 /Xinhua-PRNewswire-FirstCall/ -- Pharmaxis (ASX: PXS - News; Nasdaq: PXSL - News) today announced that the company and the U.S. Food and Drug Administration (FDA) have reached agreement on the phase 3 registration trial of Bronchitol for bronchiectasis via the Special Protocol Assessment process.

The SPA process allows for FDA evaluation of a clinical trial protocol intended to form the primary basis of an efficacy claim in support of a New Drug Application, and provides an agreement that the study design, including trial size, clinical endpoints and/or data analyses are acceptable to the FDA.

Pharmaxis previously agreed on the trial design with the European regulatory agency (EMEA). This trial will therefore form the basis of a marketing application in both the U.S. and Europe.

The phase 3 trial has been designed in collaboration with internationally renowned experts in the field of bronchiectasis and will be a randomized, placebo controlled, double-blind investigation of Bronchitol twice daily in approximately 350 adults with bronchiectasis. Participants will be treated for 52 weeks and the primary endpoints are reduction in frequency of exacerbations and improvement in quality of life. Secondary endpoints include time to first exacerbation and duration of exacerbation. Additional secondary endpoints are antibiotic use, sputum volume, exercise tolerance and lung function measurements. The trial includes health economic analysis and will be conducted in centers across Europe and the U.S.

This trial is the second Phase III study to be undertaken for Bronchitol in bronchiectasis and follows the completion of a successful shorter trial reported last year.

Pharmaxis CEO, Alan Robertson, said the company was pleased to have concluded its discussions with the FDA and the EMEA.

''We believe this phase 3 trial design will allow us to thoroughly demonstrate the clinical benefits of Bronchitol in a patient population for which mucus build up and clearance is a daily problem. Our bronchiectasis program follows closely behind our work in cystic fibrosis where a phase 3 clinical trial is expected to soon close recruitment.''

Pharmaxis is developing Bronchitol as a treatment to improve mucus clearance in the lungs of patients with cystic fibrosis, bronchiectasis and other acute and chronic pulmonary conditions. The U.S. FDA has granted Bronchitol fast track status and it is designated as an orphan drug in the U.S.

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Pharmaxis' Aridol Authorised for Sale in Germany
Sunday May 18, 10:13 pm ET

SYDNEY, Australia, May 18 /Xinhua-PRnewswire-Firstcall/ -- Specialist Pharmaceutical Company Pharmaxis Ltd (ASX: PXS - News; Nasdaq: PXSL - News) today announced that it has received national approval to market Aridol in Germany.

Aridol is indicated for measuring airway hyperresponsiveness and has been approved in 14 European countries under the mutual recognition procedure (MRP). The necessary national approvals that follow the MRP have now been received for Denmark, Germany, Ireland, The Netherlands, Portugal, Sweden, and the United Kingdom.

In Germany a total of 660,000 lung function tests are conducted annually, of which approximately 90% are conducted by office-based physicians and the remainder in the major hospitals. To enter the market, Pharmaxis will first negotiate with insurance companies that cover the office-based physician market before launching with a local distributor.

A simple-to-use airways inflammation test, Aridol is a dry powder administered to patients' lungs via a small hand-held inhaler. Doctors can use the results of this test to identify airway hyperresponsiveness -- a hallmark of asthma. Medications can be adjusted according to the severity of the disease. (see overleaf for more details on Aridol)

"We are pleased that Aridol is becoming globally recognised as a useful test for identifying airway hyperresponsiveness," said Pharmaxis CEO Dr Alan Robertson. "With this latest approval, Aridol is on the way to becoming the worldwide standard for detecting sensitive airways in people with conditions such as asthma.

"The Aridol test provides objective information on airway hyperresponsiveness and assists in the diagnosis and assessment of severity of asthma and how much medication should be used."

As well as being included as one of the tests recommended by the International Olympic Committee -- Medical Commission Independent Panel and the World Anti-Doping Agency, Aridol is also included in the GINA Report of Global Strategy for Asthma Management and Prevention, the US Asthma Management Guidelines, the British Guideline on the Management of Asthma and the Australian Asthma Management Handbook. To find out more about Pharmaxis go to http://www.pharmaxis.com.au .

About Aridol

Aridol is the first and only approved Europe-wide lung function test and the world's first approved indirect challenge test for asthma. The Aridol lung function test, developed by Australian researchers and Pharmaxis, helps doctors more accurately determine the severity of a patient's airways inflammation -- a hallmark of asthma -- and allow prescription of the right amount of medication. The simple 15-25 minute test uses powdered mannitol, which the patient inhales in increasing doses. In asthmatic patients, this causes the airways to narrow and contract, which is detected by measuring the amount of air a person can exhale in one second. The smaller the dose required to cause contraction, the more severe the patient's asthma. People without airway inflammation do not respond to an Aridol challenge test. Asthma affects 52 million people worldwide, many of whom may be receiving inappropriate medication because of the absence of an objective test -- until now. Clinical trial results suggest that 25% of asthmatic patients are being treated with sub-optimal dosages of asthma medication, and up to 17% could reduce their medication without adverse effects.