Pharmaxis presents new cystic fibrosis trial data

Pharmaxis (ASX:PXS) has presented new analysis from data generated by its phase III trials of Bronchitol in cystic fibrosis (CF), concentrating on the long-term benefits of the treatment.

The analysis was provided in a poster presentation at the European Cystic Fibrosis Society conference in Portugal.

It shows that adult patients responding to Bronchitol - defined as the treatment causing any improvement in lung function - experienced on average 34% fewer exacerbations in symptoms over the 26-week period of the studies.

Separate analysis shows that patients not currently receiving recombinant human deoxyribonuclease (rhDNase) - a popular CF treatment - demonstrated an improvement in lung function and a 50% reduction in hospitalised exacerbations with Bronchitol compared to standard-of-care alone.

Pharmaxis CEO Gary Phillips said the “subgroup of patients not taking rhDNase represent a group with a high medical need as many have previously tried rhDNAse and are seeking new treatments.”

He said Bronchitol patients in the subgroup were also discharged from hospital earlier when they had exacerbations, and also required less time interacting with physicians.

Bronchitol is a formulation of mannitol, delivered to the lungs as a dry powder through an inhaler.

The product is approved in Australia for cystic fibrosis patients aged six years or older, and in the EU in patients over 18. But the US FDA in March rejected Pharmaxis’ New Drug Application and recommended that a new clinical trial be conducted.

The company is also trialling Bronchitol in bronchiectasis, but a recent phase III trial failed to meet its primary end point.

Pharmaxis shares were trading 6.9% higher at $0.155 as of around 2 pm on Tuesday.