Pharmaxis progresses clinical trial in bone marrow cancer

Pharmaxis Ltd (ASX:PXS) (FRA:UUD) has completed dosing the first of three stages in its phase 1c clinical trial (MF‐101) on bone marrow cancer myelofibrosis – a disorder in which normal bone marrow tissue is gradually replaced with a fibrous scar‐like material. The study’s safety monitoring committee has given the green light to progress to the second dose level after reviewing factors including the safety and pharmacokinetic properties of the company’s PXS‐5505 treatment for myelofibrosis patients. Participating Australian and South Korean hospitals will shortly commence dosing of the second dose level for a treatment period of 28 days. The dose escalation phase of the study will inform the selection of the optimum dose of the drug to be used in the six‐month dose expansion phase (24 patients) to evaluate safety and efficacy. “Demonstrated good tolerability” Pharmaxis CEO Gary Phillips said: PXS‐5505 has demonstrated good tolerability and very consistent pharmacokinetic properties in the first dose escalation stage of MF‐101.   “This is very much in line with the results from our phase 1 healthy volunteer study reported last year.  “Further data from this first dose will be available in coming weeks but based on the pharmacokinetic profile I anticipate that we will see significant inhibition of all lysyl oxidase enzymes even at this lowest level of dose.  “We look forward to seeing the data from the next two dose cohorts in 2H 2021 and delivering safety and efficacy data from the 6‐month dose expansion study by the end of next year.” Sites in other countries including the USA are currently being approached in anticipation of the dose expansion phase commencing recruitment later this year. PXS‐5505 potential In pre‐clinical models of myelofibrosis PXS‐5505 reversed the bone marrow fibrosis that drives morbidity and mortality in myelofibrosis and reduced many of the abnormalities associated with this disease. It has already received Investigational New Drug (IND)  approval and Orphan Drug Designation for the treatment of myelofibrosis from the FDA.  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.