On March 7, 2023 Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA), an oncology-focused drug development company, reported that it has entered into a collaboration with the Australian and New Zealand Children’s Haematology / Oncology Group (ANZCHOG) for a phase II clinical study examining paxalisib in children with advanced solid tumours, including brain tumours (Press release, Kazia Therapeutics, MAR 7, 2023, View Source [SID1234628376]).
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The study, named OPTIMISE, will combine paxalisib with chemotherapy for children with specific genetic mutations in their tumours. The study 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.
Key Points
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New collaboration is the first Australian-led clinical trial of paxalisib.
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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 PI3K pathway alterations may now be eligible to receive paxalisib.
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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.
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Up to 18 children are anticipated to be enrolled into an initial dose escalation cohort, and up to 100 patients in a dose expansion cohort.
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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 Dr James Garner, Chief Executive Officer of Kazia. "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."
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 2H CY2023. The study will initially launch in Australia but may expand in due course to other countries.
An international phase II study of paxalisib in combination with ONC-201 for the treatment of DIPG and DMGs, sponsored by the Pacific Pediatric Neuro-Oncology Consortium (PNOC), commenced recruitment in November 2021. Initial data is anticipated in 1H CY2023.