Oral pan-LOX Inhibitor (PXS-5505)

Oral pan-LOX Inhibitor - myelofibrosis and pancreatic cancer

Pharmaxis has developed an oral drug inhibiting all lysyl oxidase family members. The compound has shown significant reductions in fibrosis in in-vivo models of kidney fibrosis, lung fibrosis, myelofibrosis and pancreatic cancer. This all-encompassing LOX inhibitor is well positioned for the treatment of severe fibrosis as well as cancer with prominent stroma (connective tissue) or fibrotic metastatic niches. Pharmaxis plans to initially develop the compound for myelofibrosis.

Pharmaxis is also collaborating with Garvan Institute of Medical Research to investigate the therapeutic potential of LOX inhibition in pancreatic cancer.

A Phase 1 clinical trial in healthy volunteers commenced in February 2019 and reported in April 2020. The Phase 1 trial demonstrated a well-tolerated drug that effectively inhibits all enzymes in the lysyl oxidase family that are involved in fibrosis. Long term toxicity studies were completed clearing the way for 6-month phase 2 studies in several cancers with the opportunity to demonstrate disease modifying efficacy.

In mid 2020 the US Food and Drug Administration granted orphan-drug designation for the pan-LOX inhibitor PXS-5505 for the treatment of myelofibrosis. In August the FDA completed a safety review of the company’s Investigational New Drug (IND) application PXS-5505 and gave Pharmaxis permission to proceed with a phase 1c/2 clinical trial for the treatment of myelofibrosis in adults. 

The study will incorporate a one-month dose escalation phase followed by 6 months’ treatment in an open label study of patients with intermediate or high-risk myelofibrosis, including primary myelofibrosis, post-polycythemia vera myelofibrosis and post-essential thrombocythemia myelofibrosis patients.  

Access Pharmaxis Publications and Posters on LOX inhibitors

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Fibrosis