Pharmaxis Ltd. has concluded an interim analysis of data from 6 patients who have completed 6 months' treatment with PXS-5505 in its open label phase 2 clinical trial in patients with the bone marrow cancer myelofibrosis. The phase 2 trial known as MF-101 was cleared by the FDA under the Investigational New Drug (IND) scheme and aims to demonstrate that PXS-5505, the lead asset in Pharmaxis' drug discovery pipeline, is safe and effective as a monotherapy in myelofibrosis patients who are intolerant, unresponsive or ineligible for treatment with approved JAK inhibitor drugs. These patients have very limited treatment options and a life expectancy of approximately 1 year.

A total of 15 patients have been enrolled in the cohort expansion phase of the study with 6 patients having completed 24 weeks of treatment. Four patients have dropped out of the study due to a lack of clinical response. PXS-5505 has been well tolerated with no serious treatment related adverse events reported.

2/6 patients had clinically important improvement in symptoms. 5/6 patients had either stable or improved bone marrow fibrosis scores of 1 grade. 5/6 have stable or improved platelet and/or haemoglobin scores. No reductions were seen in spleen volume.

A total of 18 clinical trial sites are now actively recruiting in Australia, South Korea, Taiwan and the US with two more sites due to open in Q4. Recruitment is expected to complete by the end of 2022 with top line results available in Third Quarter 2023. While Pharmaxis' primary focus is the development of PXS-5505 for myelofibrosis, the drug also has potential in several other cancers including liver and pancreatic cancer where it aims to break down the fibrotic tissue in tumours and enhance the chemotherapy treatment.

An investigator led phase 1c study in newly diagnosed hepatocellular cancer patients, where PXS-5505 will be used in addition to immunotherapy standard of care, is due to commence recruitment at Rochester University New York in Fourth Quarter 2022.