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Kazia Therapeutics welcomes presentation of positive paxalisib data at international symposium

Last updated: 19:29 13 Jun 2022 EDT, First published: 19:20 13 Jun 2022 EDT

Kazia Therapeutics Ltd -

Kazia Therapeutics Ltd (ASX:KZA, NASDAQ:KZIA) welcomes the presentation of positive data regarding the activity of paxalisib in two forms of childhood brain cancer with poor prognosis and limited treatment options.

The data is the subject of two poster presentations at the 20th International Symposium on Paediatric Neuro-Oncology (ISPNO), held in Hamburg, Germany this week.

The first poster was presented by Dr Jeffrey Rubens at Johns Hopkins University and described the activity of paxalisib in combination with a second drug in animal models of atypical teratoid/rhabdoid tumours (AT/RT).

This builds on work previously presented at the American Association of Cancer Research (AACR) Annual Meeting in April 2022.

The second poster presents work led by Associate Professor Matt Dun at the Hunter Medical Research Institute at the University of Newcastle, Australia, and involving collaboration by 10 international laboratories and leading paediatric cancer centres.

Two clinical studies reported

The research principally relates to the combination of paxalisib with ONC201, an investigational therapy manufactured by Chimerix Inc in the treatment of diffuse intrinsic pontine glioma (DIPG).

While the primary focus of the ISPNO presentation concerns animal models of the disease, two clinical case studies were also reported. Importantly, these data underpin the ongoing Phase 2 clinical trial testing the safety and efficacy of paxalisib and ONC201 in DIPG.

Targeting aggressive childhood brain cancers

Diffuse midline gliomas (DMGs) are a group of childhood brain cancers including DIPG, which represent approximately 15% of brain tumours in children. Life expectancy from diagnosis is estimated to be 9 to 11 months and there are no FDA-approved drug treatments.

ONC201, a dopamine receptor D2 (DRD2) antagonist, shows single-agent efficacy in DIPG, but patients invariably become resistant to therapy over time. A key resistance mechanism involves activation of the PI3K pathway, which is the target of paxalisib.

Two patients who received the combination of ONC201 and paxalisib under compassionate access demonstrated “dramatic reductions in tumour volume and complete resolution of disease symptoms, extending overall survival”.

Using two aggressive autopsy animal models of DIPG the combination of paxalisib and ONC201 synergistically extended survival from 73 to 100 days (37%) and from 36 to 43 days (19%), respectively.

Study is ongoing

On the basis of this research, a clinical trial of the combination commenced recruitment in November 2021 under the sponsorship of the Pacific Pediatric Neuro-Oncology Consortium (PNOC). The study is ongoing, with initial data anticipated in the first half of next year.

“Brain tumours are the second most common cancer of children, but the leading cause of death, with the prognosis for aggressive forms of the disease such as DIPG remaining woeful,” said Dr Matt Dun, lead investigator of the DIPG research.

“It is vital that we find new treatments. This research collaboration has united many of the leading international centres in this field and has deployed cutting-edge research tools to identify an extremely promising combination of therapies.

"My colleagues and I hope to see these laboratory findings validated in the ongoing PNOC clinical trial, which may establish ONC201 and paxalisib as the cornerstones of treatment for children and their families affected by this disease.”

Emphatic demonstration

Kazia CEO Dr James Garner added, “Dr Dun’s painstaking research over several years has yielded enormous insight into potential treatments for DIPG. We are excited to see such an emphatic demonstration of the potential for ONC201 and paxalisib to provide benefit in this disease.

"We are firmly committed to taking paxalisib forward in childhood brain cancer, and very much hope that the work of world-leading scientists such as Dr Dun will help to bring hope to all those touched by diseases such as DIPG.”

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