Kazia Therapeutics Ltd (ASX:KZA, NASDAQ:KZIA) has formed a partnership with the Australian and New Zealand Children’s Haematology/Oncology Group (ANZCHOG) for a Phase 2 clinical study examining paxalisib in children with advanced solid tumours, including brain tumours.
This is the first Australian-led clinical trial of paxalisib.
Specific genetic mutations
The study, OPTIMISE, will combine paxalisib with chemotherapy for children with specific genetic mutations in their tumours. It will harness expertise and insights gained from the Zero Childhood Cancer Program, which aims to match childhood cancer patients with targeted therapies suited to the unique characteristics of their tumour.
The Zero Childhood Cancer Program, led out of the Children’s Cancer Institute and the Kids Cancer Centre at Sydney Children’s Hospital, has already enrolled more than 900 children with high-risk malignancies.
Patients with P13K pathway alterations may now be eligible to receive paxalisib.
High-risk malignancies
OPTIMISE will explore paxalisib in combination with existing chemotherapy agents for the treatment of children with high-risk malignancies, including (but not confined to) brain tumours. It will first seek to establish the optimal dosing for children in combination with chemotherapy and will then determine the efficacy and safety in biomarker-defined populations.
As many as 18 children are anticipated to be enrolled into an initial dose escalation cohort and up to 100 patients in a dose expansion cohort.
The study is funded by the Australian Government, through a Medical Research Future Fund (MRFF) grant, with Kazia’s contribution consisting of drug supply and in-kind support.
“We are pleased to see this very exciting new trial move forward with paxalisib as a matched targeted therapy in a biomarker-selected paediatric population,” said Kazia chief executive officer Dr James Garner.
“Our commitment to childhood cancer, especially childhood brain cancer, is already substantial. The OPTIMISE study is very complementary to the ongoing PNOC022 study in DIPG and in addition, represents a promising new opportunity to explore paxalisib in a broader range of patients.
“This new project speaks to the substantial ongoing interest in the drug among leading clinicians and has the potential to yield important new insights into the use of paxalisib in some new areas with very substantial unmet clinical need.”
About the Zero Childhood Cancer Program
The Zero Childhood Cancer Precision Medicine Program (ZERO) is an international effort to identify targeted therapies for childhood malignancies.
One of the key insights of recent decades in cancer treatment has been the understanding that tumours are typically driven by specific genetic mutations.
Instead of taking a ‘one size fits all’ approach to their treatment, ZERO aims to provide tailored individual treatment regimens for children diagnosed with cancer.
ZERO is led by the Children’s Cancer Institute and the Kid’s Cancer Centre at Sydney Children’s Hospital, Randwick in Sydney, NSW. The Institute is a leading Australian Medical Research Institute dedicated to paediatric cancer.
The principal investigators of the paxalisib arm of the OPTIMISE study are Dr Marion Mateos and Professor David Ziegler, senior clinicians at the Kids Cancer Centre with extensive track records in the field.
Next steps
OPTIMISE is expected to commence enrolment in the second half of 2023.
The study will initially launch in Australia but may expand in due course to other countries.