Pharmaxis breathing easier after positive Phase II results

Pharmaxis (ASX:PXS) investors will be breathing easy after the company announced a positive result from its Phase II clinical trial of Bronchitol, its lead compound for treating the chronic lung disorder bronchiectasis.

Describing the results of the trial as "fantastic", CEO Dr Alan Robertson said his company has taken a step closer to the marketplace.

It has begun planning for a Phase III trial - potentially an AUD$15 million exercise that Robertson says Pharmaxis may fund from its own resources, without help from Big Pharma.

The double-blind, placebo-controlled crossover trial, conducted at hospitals in Sydney, Melbourne and Auckland, recruited 60 Australian and New Zealand patients aged between 18 and 75, who suffer from bronchiectasis.

Bronchiectasis is a form of chronic, obstructive lung disease in which an increase in the viscosity of the protective mucus layer on the surface of the lung, impairing the patient's ability to remove inhaled debris and microbes by coughing.

The thick mucus impedes the beating of the tiny, hair-like cilia on the cells lining the lungs, which in healthy individuals generates currents that sweep foreign material from the lung surface.

According to the company, doctors observed a significant improvement in indices of patients' quality of life, and sleep quality, after only two weeks of treatment with Bronchitol.

Patients on Bronchitol who were unable to clear their congested lungs at the beginning of the trial had a highly significant improvement in their symptoms, compared with patients on a placebo.

Bronchitol is a patented, inhalable dry powder that is administered via a hand-held puffer. Robertson said the drug, developed by researchers at Sydney's Royal Prince Alfred Hospital, is an osmotic agent, that restores the normal viscosity of the lung's protective fluid layer by drawing water from the interstitial spaces between epithelial cells.

The drug also appears to work by reducing the hydrogen bonding that thickens mucus, and may also have a direct effect on the activity of cilia - although this effect may simply reflect the reduced viscosity of the mucus layer.

The most encouraging aspect of the trial, Robertson said, was that Bronchitol appeared to work well, where many other drugs, including Roche's Pulmozine for cystic fibrosis, had been unsuccessful in treating bronchiectasis.

"Bronchiectasis is an interesting disease, with an estimated 600,000 patients around the world," Robertson said. "China, Japan and South Korea would be the major markets for a successful new drug.

He said Pharmaxis was now working with international experts on obstructive lung disease to develop protocols for a Phase III trial, which could begin early in 2005.

"Our intention is to go it alone, but how we finance it is another matter," he said. "We had $25 million in cash at the end of the last financial year, and we estimate that $15 million would be sufficient to fund the trial.

"But we have other funding commitments, so we're working through the options."