Pharmaxis boosted by cystic fibrosis study

Pharmaxis' (ASX:PXS) drug Bronchitol has produced positive results from a phase II trial in cystic fibrosis patients, described by CEO Alan Robertson as "a landmark study and transforming day for Pharmaxis and the cystic fibrosis community".

"I am very happy with our results for Bronchitol, which position it as an important treatment for cystic fibrosis," Robertson said. "They are as good as I could have hoped for -- to have done something to improve lung function is fantastic."

The trial achieved its primary end point of improvement in lung function. At the end of the two-week treatment periods, patients receiving Bronchitol had statistically significantly improved lung function compared to two weeks of placebo treatment, Pharmaxis said.

The study was a double-blind, placebo controlled, randomised comparison of 420mg of Bronchitol to placebo in 49 patients with cystic fibrosis at eight centres across Australian and New Zealand. Bronchitol or placebo was administered twice a day for 14 days.

Secondary endpoints of the study include quality of life measures. Pharmaxis said significantly better respiratory symptoms were achieved among patients taking Bronchitol compared to those taking a placebo. Additionally, the trial found Bronchitol had no deleterious effect on the microbiology of the sputum, and no serious adverse side-effects.

"Now we have to proven its capability all we need is interest from the cystic fibrosis community and the cystic fibrosis foundation," Robertson said. "The path forward is phase III trials and then to put it on the market -- we hope to have it on the market by 2008 at the latest. Bronchitol has the chance to change the lives of people with cystic fibrosis and it positions us very well as a company."

The US Food and Drug Administration has expanded an 'orphan drug' designation to Bronchitol, for the additional indication of facilitating mucus clearance in patients with cystic fibrosis. Pharmaxis was initially granted orphan drug status for Bronchitol as a treatment for patients with bronchiectasis in February.

Orphan drug status is granted to treatment aimed at serious or life-threatening diseases. Cystic fibrosis patients have a defective gene which causes the body to produce an abnormally thick, sticky mucus that clogs the lungs and leads to life-threatening lung infections. "There have been no therapeutic advances to help clear congested lungs for patients with cystic fibrosis in the past 10 years," Robertson said.

Under orphan drug status, Pharmaxis has a seven-year period of market exclusivity. Other incentives include assistance from the FDA with clinical trial design and an exemption from FDA user fees.