Clinical Trials

Date: 2017-02-23

Type of information: Completion of patient enrollment

phase: 3

Announcement: completion of the trial

Company: Pharmaxis (Australia)

Product: Bronchitol® (mannitol)

Action mechanism: Bronchitol® is a precision spray-dried form of mannitol, delivered to the lungs by a specially designed, portable inhaler. Pharmaxis has partnered its work on Bronchitol for the United States with global pharmaceutical company Chiesi Farmaceutici . Chiesi has funded US$22 million of the expected total US$26 million cost of the trial. 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. Pharmaxis will manufacture Bronchitol in Australia on commercial terms for Chiesi with Pharmaxis retaining a high teens percent share of sales revenue as its margin.

Disease: cystic fibrosis

Therapeutic area: Rare diseases - Genetic diseases

Country: Argentina, Australia, Belgium, Canada, Czech Republic, Hungary, Israel, Italy, Mexico, New Zealand, Poland, Romania, Russian Federation, Slovakia, South Africa, Spain, Ukraine, France, USA

Trial details: The CF303 trial aims to provide prospective evidence of the safety and efficacy of mannitol 400 mg b.i.d. in subjects aged 18 years and above. The study 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 enrollment is expected to reach 420 adult CF patients. Two other large scale clinical trials have been already undertaken by Pharmaxis (CF 301 and CF 302) . (NCT02134353)

Latest news:

• • On February 23, 2017, Pharmaxis announced that the last of 423 patients has concluded treatment in its international clinical trial evaluating Bronchitol® (mannitol) for cystic fibrosis. The topline results of the trial are expected to be reported in the second quarter of this year. The Phase 3 trial known as DPM?CF?303 has been conducted in accordance with the requirements of the FDA to gain marketing approval for Bronchitol to treat adult cystic fibrosis patients in the United States. 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 recruited a total of 423 patients across 126 sites in 21 countries in North and South America, Western and Eastern Europe.  The design was guided by the FDA and follows 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 statistically significant improvement in FEV1.
• • On July 15, 2016, Pharmaxis  announced it has completed recruitment ofits 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 FDA to gain approval for Bronchitol to treat cystic fibrosis in the United States in adult patients. Subject to a positive trial outcome, Pharmaxis will submit a response to the FDA and a decision on approval can be expected in 2018. Pharmaxis has partnered its work on Bronchitol for the United States with Chiesi Farmaceutici. Chiesi is responsible for funding up to $22 million of the cost of the trial, the total cost of which is expected to be approximately $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 $25 million are payable including $10 million on the launch of Bronchitol. Pharmaxis will manufacture Bronchitol on commercial terms for Chiesi with Pharmaxis retaining a high teens percent share of sales revenue as its margin.

Is general: Yes