The Phase 3 trial recruited 423 patients across 126 sites in 21 countries in North and South America, Western and Eastern Europe.
The topline results of the trial are expected to be reported in the second quarter of this year.
The last of 423 has concluded treatment as part of the 26 week investigation of Bronchitol administered twice daily in cystic fibrosis patients aged 18 and over to assess improvements in lung function, pulmonary exacerbations and safety.
Pharmaxis has partnered its work on Bronchitol for the U.S. with global pharmaceutical company Chiesi Farmaceutici SpA, who has funded US$22 million of the expected US$26 million trial cost.
Gary Phillips, CEO, commented: “Having the last of more than 400 participants at 126 sites in 21 countries complete their last clinic visit is a very significant milestone for the Bronchitol business unit.”
The Phase 3 trial known as DPM‐CF‐303 has been conducted in accordance with the requirements of the U.S Food and Drug Administration (FDA) to gain marketing approval for Bronchitol in the U.S.
Subject to a positive trial outcome, a response will be submitted to the FDA and a decision on approval can be expected in the second half of 2018.
The trial is a 26 week randomised, double‐blind parallel group investigation of Bronchitol administered twice daily in cystic fibrosis patients.
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 Russia and for patients aged 18 years and over throughout the European Union and in Israel.
Chiesi has funded US$22 million of the expected total US$26 million cost of the trial and is also responsible for completing the Bronchitol New Drug Application with the FDA.
Subject to approval in the U.S, Bronchitol will be sold as part of Chiesi’s cystic fibrosis portfolio.
Milestones totalling up to US$25 million are payable including US$10 million on the launch of Bronchitol.
Pharmaxis will manufacture Bronchitol in Australia on commercial terms for Chiesi with Pharmaxis retaining a high teens percentage share of sales revenue as its margin.
The completion of the treatment phase is another step forward to realising the commercial value of Bronchitol.
The U.S. is the largest market for cystic fibrosis and the pending trial results will potentially make Bronchitol available in clinics across the U.S.
Pharmaxis entered 2017 well-funded with a cash balance of A$29 million.
The company continues to build into a biotech powerhouse focused on fibrosis and inflammation with a number of opportunities and milestones expected in the current calendar year.
Story by ProactiveInvestors