Promising early study results released today by clinical stage drug development company Pharmaxis (ASX: PXS), should be a reason for investors to stir.  

Pharmaxis’ latest clinical trial (MF-101) focuses on bone marrow cancer myelofibrosis and potential treatment with their hero drug, PXS-5505.

Having already proven the drug’s tolerability and efficacy in terms of absorption, distribution, metabolism and excretion, Pharmaxis has today released more details around the first dose level results. 

PXS-5505 works to inhibit LOX and LOXL2, two enzymes key to the process of bone marrow fibrosis in myelofibrosis. Traditional chemotherapeutic drugs target a different enzyme and signalling pathway however, despite treatment being available, myelofibrosis still portends a poor prognosis. Hence, Pharmaxis is targeting the LOX and LOXL2 enzymes in the hopes that they can revolutionise the way we treat myelofibrosis and other fibrotic diseases in the clinic.

With FDA approval under the Investigational New Drug scheme, the current phase 1c/2a clinical trial has yielded promising results so far.

CEO Gary Phillips said: “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 these models PXS-5505 caused disease modifying effects with improvements in blood cell count, diminished spleen size and reduced bone marrow fibrosis. Comparing these results with those achieved in the phase 1 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 6-month dose expansion study.” 

Recruitment for the second dose cohort is complete and dosing has commenced across study sites in both Australia and South Korea. Patients will have 28 days of treatment at this new dosage before safety, enzyme inhibition levels and pharmacokinetic properties of the drug are assessed. Following this, the third and final cohort of patients will commence dosing. 

This dose escalation phase of the study is designed to inform the optimal dosage of PXS-5505 which will be carried through to a dose expansion study. Pharmaxis anticipates recruitment for the dose expansion to begin later this year. 

PXS-5505 may well be the answer for patients who are unable to receive or respond to traditional myelofibrosis therapies and open up Pharmaxis to a US $1 billion per annum market opportunity. 

The drug is also being investigated for its potential as a treatment for other conditions and cancers such as liver and pancreatic cancer to enhance the chemotherapeutic effects of traditional cancer drugs. 

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