Pharmaxis Ltd is an Australian pharmaceutical research company with expertise developing drugs for inflammatory and fibrotic diseases and focussed on myelofibrosis. The company has a highly productive drug discovery engine, drug candidates in clinical trials and two respiratory products approved in global markets generating ongoing revenue.

Established in 1998 and listed on the Australian Securities Exchange in 2003 (symbol PXS) the Company’s head office, manufacturing and research laboratories are located in Sydney, Australia.

Pharmaxis’ lead drug candidate (PXS-5505) is for the treatment of the bone marrow cancer myelofibrosis, which affects 1 in 500,000 people. In myelofibrosis patients, normal bone marrow tissue is gradually replaced with a fibrous scar-like material, destroying the normal bone marrow environment and preventing the production of adequate numbers of red and white bloods cells and platelets. The US Food and Drug Administration (FDA) has granted PXS-5505 orphan drug designation for the treatment of myelofibrosis and has approved an Investigational New Drug application so that clinical trials in myelofibrosis patients can commence.

PXS‐5505 is also being investigated as a potential treatment for other cancers such as liver and pancreatic and brain cancer.

PXS-5505 was developed from the Company’s product pipeline which is founded on its expertise in the chemistry of amine oxidase inhibitors. Drug candidates being developed from the pipeline are targeting diseases including:

  • fibrotic diseases such as kidney fibrosis, NASH, pulmonary fibrosis and cardiac fibrosis
  • inflammatory diseases such as Duchenne Muscular Dystrophy (DMD)

Another Pharmaxis drug discovery, PXS‐6302 has  entered  a  world  first  clinical  trial  aiming  to  stop  scars  that are formed after surgery or trauma, particularly following burn injuries. The research is being conducted by a team led by burns specialist Prof Fiona Wood.

Pharmaxis has successfully executed large scale clinical trials resulting in the approval and marketing of two products which are manufactured and exported from the company's high tech production facility in Sydney.

  • Bronchitol® (mannitol), an inhaled dry powder for the treatment of cystic fibrosis is approved and marketed in Europe, Russia and Australia and following approval in October 2020, was commercially launched in the United States in Q1 2021. Pharmaxis partners with a number of specialist companies who have responsibility for ongoing commercialisation of Bronchitol in their local jurisdictions. For more information visit the Bronchitol website.
  • Aridol® a lung function test to help diagnose asthma is approved and sold in the United States, Europe, Australia, Canada and Asia. For more information visit the Aridol website. 

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