Pharmaxis Ltd (ASX:PXS) has completed the recruitment for its Bronchitol clinical trial for the U.S. market, the largest cystic fibrosis market in the world.
Bronchitol is a respiratory product currently approved to treat cystic fibrosis and being sold in Australia and Europe.
This current multicentre clinical trial aims to secure approval for Bronchitol to treat adult patients in the United States.
Pharmaxis has partnered its work on Bronchitol in the U.S. with global pharmaceutical company Chiesi Farmaceutici SpA. 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 US$26 million.
Subject to a positive trial outcome, the company will submit a response to the FDA and a decision on approval can be expected in 2018.
Gary Phillips, CEO, commented:
“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 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 FEV.”
Trial details
The Phase 3 trial is being conducted in accordance with the requirements of the FDA. It will last 26 weeks and Bronchitol will be administered twice daily in patients aged 18 and over.
The final enrolment is expected to reach 420 adult patients.
Background
Pharmaxis is a pharmaceutical research company with a portfolio including two respiratory products, Bronchitol and Aridol, approved and sold in various world markets.
It also has a research pipeline focused on areas of high unmet clinical need in inflammatory and fibrotic diseases.
The company’s product pipeline is founded on its expertise in the chemistry of amine oxidase inhibitors.
This expertise has attracted interest from leading pharmaceutical companies looking to make acquisitions or partner in this rapidly expanding area of medical need.
In May 2015, Boehringer Ingelheim acquired the Pharmaxis phase 1 investigational drug PXS-4728A, to develop it for the treatment of the cardiometabolic liver-related condition NASH.
This involved an upfront payment of €27.5 million and a total potential deal value of over A$750 million. Expected timing for the commencement of a phase 2 clinical trial is the first quarter of 2017. The commencement of a phase 2 trial triggers the payment of a development milestone to Pharmaxis of approximately A$25 million.
Analysis
This completion of recruitment represents another milestone for Pharmaxis as it attempts to enter the U.S. - the largest cystic fibrosis market in the world.
This progression gives Pharmaxis greater certainty around when the study will report and, subject to that report being positive, when the FDA will consider the drug application.
The company had $41.5 million in cash at the end of March with an upcoming $25 million milestone payment due in Q1 2017.
Pharmaxis maintains coverage from an independent investment research house with a recommendation of Speculative Buy Class B. The company recently completed a successful round of meetings at the annual BIO International Convention in San Francisco, where it received strong interest and feedback for its drug development pipeline.
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