Clinical stage drug development company Pharmaxis Ltd (ASX: PXS) has initiated the first of five new trial sites in Taiwan as it progresses the phase 2 clinical trial of its drug PXS‐ 5505 in patients with the bone marrow cancer myelofibrosis.
These latest centres join 11 active sites across Australia and South Korea with a further four centres in the US expected to come on stream in coming months.
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.”
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.
An effective pan‐LOX inhibitor for myelofibrosis would open a market that is conservatively estimated at US$1 billion per annum.
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 breakdown the fibrotic tissue in the tumour and enhance the effect of chemotherapy treatment.