Pharmaxis announced that it has completed recruiting adult patients with cystic fibrosis (CF) for its large and potentially pivotal Phase 3 clinical trial evaluating Bronchitol (mannitol) as an inhaled treatment. The company expects to report results in the second quarter of 2017.
Based on those results, the company will apply for U.S. Food and Drug Administration (FDA) approval of Bronchitol as a CF treatment.
The trial (NCT02134353) is a 26-week, randomized and double-blind parallel group study assessing Bronchitol, administered twice daily, in more than 400 CF patients ages 18 and older. Its primary endpoints include safety, and improvements in lung function and pulmonary exacerbations as measured by change from baseline in FEV1 (forced expiration) over weeks six, 14 and 26.
“We are pleased to have attained this significant milestone in such a large undertaking, securing more than 400 study participants at 126 sites in 21 countries,” said Gary Phillips, Pharmaxis chief executive officer, in a recent press release. “The clinical study protocol closely follows the design of the two large scale clinical trials already undertaken by Pharmaxis (CF 301 and CF 302) in which a post-hoc analysis of the subgroups of adult patients … showed a significant improvement in FEV1. I’d like to thank the CF community for its support and participation.”
Bronchitol, a dry powder mannitol (an osmotic diuretic) inhaled twice daily using a small handheld device, works by rehydrating the airways and promoting productive cough. Previous clinical trials have shown that Bronchitol helps to increase mucus clearance, improving the lung function and the quality of life in people with CF.
It is approved to treat CF patients over age 6 in Australia, and those 18 years and older throughout the European Union and in Israel.
Pharmaxis is collaborating with Chiesi Farmaceutici SpA to advance Bronchitol in the U.S. market. Chiesi which is responsible for financing up to $22 million of the trial’s costs, expected to total around $26 million.