Kazia Therapeutics’ paxalisib shows activity against rare childhood brain cancer in pre-clinical research

Kazia Therapeutics ASX KZA paxalisib childhood brain cancer paediatric tumour
John Hopkins University scientists describe paxalisib as a “backbone therapy” in treating atypical teratoid/rhabdoid tumours, which are rare brain cancers in children.

Kazia Therapeutics (ASX: KZA) has revealed pre-clinical data that its drug paxalisib demonstrates activity in two forms of childhood brain cancer where there is a very high unmet medical need.

The company presented the pre-clinical data at the Annual Meeting of the American Association for Cancer Research (AACR) in New Orleans.

According to Kazia, this is the first-time data has been presented on exploring paxalisib in this form of brain cancer known as atypical teratoid/rhabdoid tumours.

These are rare and fast-growing tumours that begin in the brain and spinal cord. The tumours usually present in children aged three and younger.

Scientists working at the Johns Hopkins University in Baltimore described paxalisib as a “backbone therapy” in atypical teratoid/rhabdoid tumours in their presentation abstracts.

The scientists were working in the laboratory of Assistant Professor Jeffrey Rubens.

Aggressive childhood brain cancer

A third abstract from a different team at Johns Hopkins showed “strong synergy” between paxalisib and other cancer therapies. This was demonstrated in a model of diffuse intrinsic pontine glioma (DIPG).

DIPG is another childhood brain tumour that is highly aggressive and hard to treat.

Kazia noted that paxalisib has previously shown activity in DIPG as both a sole therapy as well in in combination with other cancer therapies.

Chief executive officer Dr James Garner said the data was “very promising”.

“Paxalisib is already the subject of an ongoing phase II clinical trail in DIPG and diffuse midline gliomas and this new data suggests potential wider applications of the drug in childhood brain cancers.”


Paxalisib is Kazia’s lead drug, which has been developed as a brain-penetrant inhibitor of the PI2K/Akt/mTOR pathway.

The drug has been designed to treat glioblastoma, which is the most aggressive form of primary brain cancer in adults.

At present, Kazia has eight additional studies evaluating paxalisib on various forms of brain cancer.

Paxalisib has US FDA orphan drug designation and fast track designation for treating glioblastoma. It also has rare paediatric disease designation and orphan drug designation for DIPG.


While at the AACR conference in New Orleans, Kazia also presented on its novel drug EVT801, which has the potential to treat a variety of cancers.

The drug is an inhibitor of VEGFR3, which is receptor involved in the formation of new lymphatic vessels around a growing tumour.

Pre-clinical data demonstrates EVT801 inhibits the growth and spread of new tumours, while also working synergistically with other immuno-oncology therapies.

Dr Garner said EVT801 is an “exciting new drug candidate with enormous potential”.

Kazia is recruiting patients at two sites in France for a phase I clinical trial of EVT801.

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