Clinical stage pharmaceutical company Pharmaxis (PXS: ASX) is full steam ahead with their Phase 2 clinical trial for flagship drug, PXS-5505. The Company has launched new clinical trial sites in Taiwan in a planned expansion to boost patient recruitment.

Designed for use in the treatment of myelofibrosis, a rare blood cancer with a poor prognosis, and limited treatment options, PXS-5505 works by inhibiting a key pathway in the formation of fibrotic scar tissue which is a process central to the disease progression of myelofibrosis.

The current standard of care for myelofibrosis patients are JAK inhibitors. Since their market entry in 2011 they have dominated, however, JAK inhibitors only address symptomatology and do not affect the course of the disease. Extensive preclinical and Phase 1 trial work has shown that PXS-5505 has the potential to be disease modifying which could be a real game changer in the myelofibrosis treatment market.

The Company is initiating five new trial sites in Taiwan which will join the 11 already active sites in Australia and South Korea. A further 4 centres in the US will begin dosing patients in the coming months.

PXS-5505 passed Phase 1 clinical trials with flying colours, demonstrating good tolerability and safety. Another point towards the drug came when it was cleared by the FDA under the Investigational New Drug scheme, giving Pharmaxis increased support along the approval process.

The progression of the trial puts Pharmaxis another step closer towards getting PXS-5505 approved as a safe and effective monotherapy for myelofibrosis patients where JAK inhibitors are ineffective.

Results from the study are expected by the end of the year thanks to the site expansion. CEO of Pharmaxis, Gary Phillips said: “This planned initiation of new trial sites in Taiwan will bring the total number of active sites to 16. The study is making pleasing progress as it builds to a recruitment target of 24 patients.”

“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 3 or more months of therapy on PXS-5505 and that the good tolerability profile is being maintained.”

The myelofibrosis treatment market is conservatively estimated at USD $1 billion per annum, but other trials involving PXS-5505 open the Company to wider markets.

In addition to progression in myelofibrosis, PXS-5505 is also planned to go into Phase 1c/2 trials for liver cancer following successful preclinical work by researchers at the University of Rochester, New York. The demonstrated ability to break down solid tumours and enhance chemotherapeutic effects puts PXS-5505 in a unique position to address more cancers, and help more people.

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