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Boehringer Ingelheim Program on Track


In May 2015 Boehringer acquired the Pharmaxis investigational drug PXS-4728A to develop initially as a treatment for non-alcoholic steatohepatitis (NASH). Boehringer has confirmed the planned timing for the commencement of a phase 2 clinical trial as the second quarter of 2017. The commencement of a phase 2 trial in NASH triggers the payment of a development milestone to Pharmaxis of approximately A$25 million.

In May 2017, Boehringer confirmed it would pursue a second indication for PXS-4728 in a separate phase 2 study, also commencing in 2017. Pharmaxis will receive
a milestone payment of €10m at the commencement of the trial in the second indication.

Find out more about the sale to Boehringer

Find out more about NASH

Latest news on NASH

A silent liver disease epidemic As nonalcoholic steatohepatitis, or NASH, stealthily becomes a leading cause of liver transplants, drug companies are racing to develop treatments. Lisa M. Jarvis, Chemical & Engineering News, October 3, 2016

Big Pharma, Biotech Attack NASH Liver Illness From All SidesSeptember 21, 2016




Pharmaxis Completes Treatment Phase in Pivotol Bronchitol Cystic Fibrosis Clinical Trial for US Market

On 23 February Pharmaxis announced that the last of 423 patients has concluded treatment in its international clinical trial evaluating Bronchitol®(mannitol) for cystic fibrosis (CF). The topline results of the trial are expected to be reported in the second quarter of 2017.

The Phase 3 trial known as DPM-CF-303has been conducted in accordance with the requirements of the US Food and Drug Administration (FDA) to gain marketing approval for Bronchitol to treat adult CF 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 is a 26 week randomised, double-blind parallel group investigation of Bronchitol administered twice daily in cystic fibrosis patients aged 18 and over to assess improvements in lung function, pulmonary exacerbations and safety.  The trial recruited a total of 423 patients across 126 sites in 21 countries in North and South America, Western and Eastern Europe.

The trial 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.  The CF303 trial is being managed by INC, a global contract research organisation with significant experience running international trials in the cystic fibrosis community.

Read the media release



Research Collaborations

Pharmaxis Announces
Research Collaboration with Woolcock Institute on Novel Cystic Fibrosis Therapy

In November 2016 Pharmaxis announced a research collaboration with Sydney’s prestigious Woolcock Institute of Medical Research to develop a novel inhalation therapy for the treatment of cystic fibrosis (CF).

The National Health and Medical Research Council (NHMRC) has awarded a research grant of $421,545 for development and testing of the Orbital® Inhaler with a dry powder formulation of the antibiotic tobramycin. The Orbital inhaler is a Pharmaxis invention which has been designed to deliver high-doses of dry powder drugs to the lungs in a more effective and convenient manner than existing technology.

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Stopping Scars Before They Form

2016 08 24 ACS

Pharmaxis is collaborating on a potential new scar treatment for burns, keloids and Dupuytren contracture. Early stage research from a group including The University of Western Australia, Fiona Wood Foundation, Royal Perth Hospital Burns Unit and Pharmaxis has been presented at the prestigious American Chemical Society meeting in Philadelphia.

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