Tackling a cancer treatment market estimated to be worth USD $1 billion per annum, drug development company Pharmaxis (ASX: PXS) has entered the final dosing of their Phase 1c trials in the study of a potential new treatment for bone marrow cancer, myelofibrosis.
The third and final stage of dosing follows successful first and second rounds, where doses of Pharmaxis’ lead drug candidate PXS-5505 were gradually increased for trial recruits. Each delivered good tolerability confirming the safety and pharmacokinetics of the drug.
With this third and final round of dosing, response data will be collected over the following 28 days. Pharmaxis will then derive the optimal dose of PXS-5505 for their Phase 2 clinical trials which have already been cleared by the US Food and Drug Administration to proceed.
“We’re working to apply PXS-5505 to cancer treatment by inhibiting the LOX and LOXL2 enzymes which play a significant role. The results from this second of three dose cohorts in our myelofibrosis clinical trial show a reassuring dose related increase in blood drug levels and good tolerability,” said Pharmaxis CEO, Gary Phillips.
“We anticipate that we will also see dose related increases in levels of LOX and LOXL2 inhibition when that data becomes available later this month. We are on track to commence dosing in the 6-month dose expansion study later this year and deliver results by the end of next year.”
A rare form of currently incurable bone marrow cancer, myelofibrosis affects 15 in every 1 million Americans. Current treatments however, are insufficient. Pharmaxis has received Orphan Drug status from the US FDA and is conducting the development of PXS-5505 under the Investigational New Drug (IND) scheme.
With a novel approach to treatment, PXS-5505 has potential to be game changing for an industry that generates more than USD $1 billion in limited treatments annually.
Upon completion of this final stage dosing, Pharmaxis will progress to the Phase 2a trial where 24 patients in Australia, South Korea, the United States and Taiwan will undergo a 6-month trial of PXS-5505 as a monotherapy in myelofibrosis patients.
PXS-5505 remains the core focus for Pharmaxis as their most advanced drug candidate as a treatment for myelofibrosis. However, successful clinical data also has potential to expedite current research by international cancer centres into its use in treating other diseases including liver and pancreatic cancer.
Pharmaxis CEO Gary Phillips recently hosted a special investor briefing which can be viewed below.