Pharmaxis Ltd (ASX:PXS, OTC:PMXSF) has initiated the first of five trial sites in Taiwan as the next stage of phase 2 clinical testing of its drug PXS-5505 in patients with the bone marrow cancer myelofibrosis.
Four more sites in the US will come online in the coming months, joining a cohort of 11 active sites across Australia and South Korea.
The phase 2 trial – dubbed MF-101 – was permitted by the US Federal Drug Administration (FDA) under the Investigational New Drug (IND) scheme.
The trial is designed to demonstrate that PXS-5505 is safe and effective as a monotherapy in myelofibrosis patients who are intolerant, unresponsive, or ineligible for treatment with approved JAK inhibitor drugs.
“Good tolerability profile being maintained”
“This planned initiation of new trial sites in Taiwan will bring the total number of active sites to 16,” Pharmaxis CEO Gary Phillips said.
“The study is making pleasing progress as it builds to a recruitment target of 24 patients. This latest expansion of sites is part of a focused effort by the company to bring patients into the trial and deliver results by the year-end.
“We know from the earlier dose-escalation study that PXS-5505 will fully block the LOX enzymes that play a fundamental part in the bone marrow fibrosis that characterises myelofibrosis, giving hope that the drug can modify the course of the disease.
“The good tolerability profile, which is a key differentiator from current standard of care, was first demonstrated in healthy volunteers and then backed up by myelofibrosis patients on 1 month of therapy in the dose-escalation study.
“I am pleased to report that we now have experience with patients who have had three or more months of therapy on PXS-5505 and that the good tolerability profile is being maintained.”
An effective pan-LOX inhibitor for myelofibrosis would open a market that is estimated at US$1 billion per year.
PXS-5505 also has potential in several other cancers, including liver and pancreatic cancer, where it is targeted at breaking down the fibrotic tissue in the tumour and enhancing the effects of chemotherapy treatment.
About Pharmaxis
Pharmaxis is an Australian pharmaceutical research company developing drugs for inflammatory and fibrotic diseases, with a focus on myelofibrosis.
Pharmaxis’ lead drug candidate (PXS-5505) is for the treatment of the bone marrow cancer myelofibrosis, which affects 1 in 500,000 people.
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.
Pharmaxis has also developed two products from its proprietary spray drying technology that are manufactured and exported from its Sydney facility; Bronchitol® for cystic fibrosis, and Aridol® for the assessment of asthma. Both products are approved in major markets worldwide and sold through a network of commercial partners.