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Pharmaxis (ASX:PXS) gets first results from bone marrow cancer trial

Health Care
ASX:PXS
29 June 2021 14:10 (AEST)
Pharmaxis (ASX:PXS) - CEO, Gary Phillips (left)

Source: Pharmaxis

Pharmaxis (PXS) has received initial data from its phase 1c clinical trial that’s studying a potential new treatment for myelofibrosis.

Myelofibrosis is a bone marrow cancer which causes scar tissue to build up, leading to the loss of production of red and white blood cells and platelets.

Positively, the first dose level of the company’s lead drug, PXS‐5505, has shown strong inhibition of two target enzymes, LOX and LOXL2, in patients.

The trial aims to demonstrate the PXS-5505 drug as a monotherapy in myelofibrosis patients who are intolerant, unresponsive or ineligible for treatment.

Company CEO, Gary Phillips, was pleased with the results.

“We are delighted to see that along with excellent tolerability we are achieving levels of LOX and LOXL2 inhibition in myelofibrosis patients that are already exceeding those levels seen in the preclinical models of myelofibrosis.”

In the preclinical models, PXS‐5505 showed improvements in blood cell count, diminished spleen size and reduced bone marrow fibrosis.

“Comparing these results with those achieved in the phase one study with healthy volunteers, we are now very confident that we will achieve a level of inhibition of the lysyl oxidase enzyme family that allows us to fully test the clinical relevance of these targets in myelofibrosis patients in the upcoming six‐month dose expansion study,” Mr Phillips said.

Pharmaxis has already recruited the second dose cohort and dosing of all patients across the Australia and South Korea sites has begun.

After 28 days on this second dose, the safety and pharmacokinetics will be assessed before starting myelofibrosis patients on the final and highest dose. The dose escalation phase will determine what the optimal dose of PXS-5505 is for the six-month expansion study which will include. 24 patients.

Sites in other countries, including the US, are currently being engaged ahead of the dose expansion phase commencing recruitment later this year.

Company shares were up 10.7 per cent to trade at 8.3 cents at 1:44 pm AEST.

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