Staff Writer

Australian biotech firm Pharmaxis Ltd. reported positive results from a Phase III study of Bronchitol in cystic fibrosis patients, one of the largest clinical trials conducted in that disease.

Patients treated with Bronchitol had a statistically significant improvement in lung function from baseline of 6.6 percent compared to placebo. Lung function improved at week six and was sustained through to week 26.

Bronchitol, which is designed to reduce the amount of mucus buildup in the lungs, also improved lung function in study patients already being treated with the most commonly used cystic fibrosis treatment, dornase alfa (sold as Pulmozyme by Genentech USA Inc.).

During the six-month treatment period, there was significant lung function improvement for both those patients being treated with Bronchitol and dornase alfa, and those being treated with Bronchitol alone compared to placebo.

Sydney, Australia-based Pharmaxis was planning to discuss the study results during a teleconference set for late Monday.

The first scientific presentation of the results will be made at a June meeting of the European Cystic Fibrosis Society in France. More detailed results are expected to be presented at the North American Cystic Fibrosis conference in Minneapolis in October.

No new products have been approved anywhere in the world for cystic fibrosis for more than 10 years, according to Pharmaxis. Genentech's Pulmozyme was first approved by the FDA in December 1993.

Bronchitol has received orphan drug and development fast-track status from the FDA, as well as orphan status from the European Medicines Agency. Consistent loss of lung function is the leading cause of death for people with cystic fibrosis and that deterioration averages 1 percent to 2 percent per year.

Bronchitol is an inhalable dry power formulation of the naturally occurring sugar alcohol mannitol. It works to hydrate the airway surface of the lungs, and promote normal lung mucus clearance.

Pharmaxis CEO Alan Robertson said in a statement: "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."

He added, "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."

Bronchitol also is being tested in bronchiectasis, an obstructive lung disease, and Pharmaxis recently reached an agreement with the FDA on the design of a Phase III trial in that indication. The pivotal study is expected to enroll 350 patients randomized to receive either Bronchitol or placebo for 52 weeks, with the primary endpoints defined as a reduction in the frequency of exacerbations and improvement in quality of life.

Pharmaxis' first commercial product, the Aridol lung function test, is designed to help doctors diagnose and manage asthma by identifying airway hyperresponsiveness - a hallmark of asthma. Aridol is approved for sale in Australia, major European countries and Korea. The company also is seeking U.S. approval of Aridol.

Beyond Aridol and Bronchitol, Pharmaxis has an earlier-stage pipeline, including PXS25, aimed at interstitial lung diseases such as pulmonary fibrosis, and PXS4159, a preclinical program for asthma.