Clinical stage drug development company Pharmaxis (ASX:PXS) has commenced dosing in the phase 2 clinical trial studying its investigative PXS-5505 in patients with the bone marrow cancer myelofibrosis.
CEO Gary Phillips said, “The first patients entering the phase 2 study were participants in the just completed phase 1c dose escalation study. We are pleased that these patients want to continue on PXS-5505 allowing us a rapid start to the next stage of the clinical trial.
"Having shown that the drug has a good tolerability profile at a dose which can fully inhibit all the family of lysyl oxidase enzymes that are implicated in myelofibrosis, we are confident of being able to assess the impact of this mechanism of action in the ongoing study. The trial infrastructure and funding is in place and we are on track to complete the study by the end of 2022.”
The company said the phase 2 trial MF-101 cleared by the FDA under the Investigational New Drug (IND) scheme aims to demonstrate that PXS-5505, the lead asset in Pharmaxis’ drug discovery pipeline, is safe and well-tolerated as a monotherapy in myelofibrosis patients who are intolerant, unresponsive or ineligible for treatment with approved JAK inhibitor drugs.
The trial has additional secondary endpoints to explore the impact of inhibiting lysyl oxidase enzymes on a number of disease parameters such as bone marrow fibrosis, cytopenia and spleen volume.
Trial sites are open to recruiting myelofibrosis patients into the six-month phase 2 study in Australia and South Korea with preparations well advanced to open more sites in Taiwan and the US.
Pharmaxis said that while myelofibrosis is the primary focus for the development of PXS-5505, the drug also has potential in several other cancers including liver and pancreatic cancer where it aims to break down the fibrotic tissue in the tumour and enhance the effect of chemotherapy treatment.