Kazia Therapeutics presents ‘positive data’ on paxalisib in childhood brain cancers

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“Positive data” is being presented regarding the activity of Kazia's lead drug paxalisib in two forms of childhood brain cancers.

The company is presenting the data via two presentations this week at the 20th International Symposium on Pediatric Neuro-Oncology (ISPNO) in Germany.

Johns Hopkins University’s Dr Jeffrey Rubens spoke about the activity of paxalisib in combination with a second drug in animal models of atypical teratoid/rhabdoid tumours.

The second presentation is from Associate Professor Matt Dun at the Hunter Medical Research Institute within Australia’s University of Newcastle.

Dr Dun’s presentation covers work carried out in collaboration with 10 international laboratories and leading paediatric centres.

This work relates to the combination of paxalisib with ONC201, which is a Chimerix manufactured investigational therapy for treating diffuse intrinsic pontine glioma (DIPG).

‘Dramatic reduction’ of tumour

According to Kazia, diffuse midline gliomas (DMGs) are a group of childhood brain cancers that include DIPG, which is categorised as highly aggressive and hard to treat.

There are no United States Food and Drug Administration approved drug treatments for DMGs, which account for 15% of all brain tumours in children.

Additionally, from diagnosis, life expectancy is between nine and 11 months.

While ONC201 has demonstrated efficacy in DIPG, Kazia noted patients become resistant to the drug over time.

Under compassionate access, two patients who received a combination of ONC201 and paxalisib demonstrated “dramatic reductions in tumour volume and complete resolution of disease symptoms – extending overall survival”.

In two animal models of DIPG, paxalisib and ONC201 “synergistically extended survival” from 73 to 100 days and from 36 to 43 days.

This research prompted the clinical trial of the drug combination to begin in November last year.

The Pacific Pediatric Neuro-Oncology Consortium (PNOC) has sponsored the phase II trial, which is ongoing.

Initial data from this study is anticipated in the first half of 2023.

Read more here.