Sydney-based Pharmaxis (ASX:PXS) has completed recruitment of its international clinical trial evaluating Bronchitol (mannitol) in adults with cystic fibrosis.
The results of the trial (CF303) are expected to be reported in the second quarter of 2017.
The Phase 3 trial is being conducted in accordance with the requirements of the US FDA to gain approval for Bronchitol to treat cystic fibrosis (CF) in the US in adult patients. The company said it will submit a response to the FDA and a decision on approval can be expected in 2018, subject to a positive trial outcome.
The trial is a 26 week randomised, double-blind parallel group investigation of Bronchitol administered twice daily in patients aged 18 and over with cystic fibrosis to assess improvements in lung function, pulmonary exacerbations and safety. Subject to final randomisation of patients screened by the trial sites, the final enrolment is expected to reach 420 adult CF patients.
Pharmaxis has partnered its work on Bronchitol for the US with global pharmaceutical company Chiesi Farmaceutici SpA (Chiesi).
The company said Chiesi is responsible for funding up to US$22 million of the cost of the trial, the total cost of which is expected to be approximately US$26 million. Chiesi is also responsible for completing the Bronchitol New Drug Application with the FDA.
"Subject to approval in the United States, Bronchitol will be sold as part of Chiesi’s cystic fibrosis portfolio. Milestones totaling up to US$25 million are payable including US$10 million on the launch of Bronchitol," said the company.
Pharmaxis will manufacture Bronchitol on commercial terms for Chiesi with Pharmaxis retaining a high teens percent share of sales revenue as its margin.
Gary Phillips, Pharmaxis Chief Executive Officer, said: “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. The US is the largest CF market and we now have greater certainty around when the study will report and, subject to that report being positive, when the FDA will complete its consideration of our new drug application.
“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 (307 in total) showed a significant improvement in FEV1. I’d like to thank the CF community for its support and participation. We look forward to working with Chiesi to make this new treatment option available for CF patients in the United States.”
The CF303 trial is being managed by INC, a global contract research organisation with significant experience running international trials in the cystic fibrosis community.
Bronchitol is a precision spray‐dried form of mannitol, delivered to the lungs by a specially designed, portable inhaler. The product is approved for marketing for the treatment of cystic fibrosis patients aged over six years in Australia and for patients aged 18 years and over throughout the European Union and in Israel.