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Kazia Therapeutics (NASDAQ: KZIA) has announced new data presentations from its lead program, paxalisib, at the 21st International Symposium on Pediatric Neuro-Oncology (ISPNO 2024) in Philadelphia from June 29 to July 2, 2024. The data includes results from the Phase 2 PNOC DMG-ACT study evaluating paxalisib in combination with ONC201, revealing median overall survival rates of 13.2 months (Cohort 1), 15.8 months (Cohort 2), and 8.8 months (Cohort 3). Additional presentations will cover preclinical data on paxalisib combined with an HDAC inhibitor and gemcitabine for treating atypical teratoid/rhabdoid tumors. Concurrently, Kazia's research has been published in the European Journal of Cancer, emphasizing the need for mutation-specific, CNS-penetrant inhibitors for pediatric Diffuse Midline Glioma (DMG). These developments highlight potential advancements in treating pediatric brain cancers.

  • Presentation of new data on paxalisib at ISPNO 2024 could enhance market visibility and credibility.
  • Phase 2 study reveals promising median overall survival rates: 13.2 months (Cohort 1), 15.8 months (Cohort 2), and 8.8 months (Cohort 3).
  • Publication in European Journal of Cancer may boost recognition and validation of Kazia's research efforts.
  • Preclinical data shows potential synergy of paxalisib with HDAC inhibitors and gemcitabine, indicating new treatment avenues.
  • None.

SYDNEY, June 27, 2024 /PRNewswire/ -- Kazia Therapeutics Limited (NASDAQ: KZIA), a biotechnology company specialising in oncology, is pleased to announce the presentation of new data from its lead program, paxalisib, at the upcoming 21st International Symposium on Pediatric Neuro-Oncology (ISPNO 2024) June 29 – July 2, 2024, in Philadelphia, PA. Kazia concurrently announces  publication of an article in European Journal of Cancer highlighting the need for evaluating mutation-specific, CNS penetrant, inhibitors to treat pediatric patients with Diffuse Midline Glioma (DMG).  

There will be three paxalisib-related presentations in total at ISPNO, including data from the Phase 2 PNOC DMG-ACT (DMG-Adaptive Combination Trial, PNOC022) study evaluating the efficacy and safety of paxalisib in combination with ONC201. As a follow up to data presented last year at the Society for NeuroOncology, 28th Annual Meeting, lead researchers will discuss survival, pharmacokinetics, and tumor biomarkers from 132 diffuse midline glioma (DMG) patients enrolled in the Phase 2 study.  Highlights of the abstract include median overall survival of 13.2 months in Cohort 1 (newly diagnosed, enrolled pre-radiation n=33), 15.8 months in Cohort 2 (newly diagnosed, enrolled post-radiation n=69) and 8.8 months in Cohort 3 (relapsed patients, enrolled after progression n=30).

The second presentation is based on novel preclinical data utilizing the addition of a novel HDAC inhibitor to the backbone therapy of paxalisib in DMG models. The third presentation will highlight preclinical data results of the combination therapy of paxalisib and gemcitabine for patients with relapsed/recurrent atypical teratoid/rhabdoid tumors AT/RT by Johns Hopkins University researchers. Based on these findings, the Pacific Pediatric Neuro-Oncology Consortium is planning to include this combination therapy in its next AT/RT international clinical trial.

Summary of Abstracts (
Clinical Trials; July 2, 2024; 8:15am
TRLS-14:  PNOC022 report: a combination therapy trial using an adaptive platform design for patients with diffuse midline glioma at initial diagnosis, post-radiation therapy, or progression
Cassie Kline, Andrea Franson, Anuradha Banerjee, Alyssa T Reddy, et al

Poster Session I; June 30, 2024; 5pm
ATRT-15 Combining the PI3K inhibitor paxalisib with nucleoside analog gemcitabine to improve survival of atypical teratoid/rhabdoid tumors
Tyler Findlay, Kristen Malebranche, Anupa Geethadevi, Charles Eberhart, Jeffrey Rubens, Eric Raabe

DIPG-21 Preclinical assessment of a multimodal treatment approach with Givinostat, Paxalisib, and radiotherapy for Diffuse Midline Glioma (DMG)
Aimée du Chatinier, Michaël H Meel, Piotr Waranecki, Dennis S Metselaar, Esther

The European Journal of Cancer publication titled Paediatric Strategy Forum for Medicinal Product Development of PI3-K, mTOR, AKT and GSK3β Inhibitors in Children and Adolescents with Cancer is the output from a two-day forum in April 2023 at Dana Farber Cancer Institute.  Consisting of patient advocates, regulators, researchers and pediatric clinicians, the publication concludes "Evaluation of mutation-specific, CNS-penetrant PI3-K inhibitors in children with DMG should be prioritised and innovative regulatory approaches are needed in view of the rarity of the population." The paper can be accessed at the following website:

About Kazia Therapeutics Limited
Kazia Therapeutics Limited (NASDAQ: KZIA) is an oncology-focused drug development company, based in Sydney, Australia. Our lead program is paxalisib, an investigational brain-penetrant inhibitor of the PI3K / Akt / mTOR pathway, which is being developed to treat multiple forms of brain cancer. Licensed from Genentech in late 2016, paxalisib is or has been the subject of ten clinical trials in this disease. A completed Phase 2 study in glioblastoma reported early signals of clinical activity in 2021, and a pivotal study in glioblastoma, GBM AGILE, has completed enrollment, with final data expected imminently. Other clinical trials are ongoing in brain metastases, diffuse midline gliomas, and primary CNS lymphoma, with several of these having reported encouraging interim data. Paxalisib was granted Rare Pediatric Disease Designation and Orphan Drug Designation by the FDA for diffuse intrinsic pontine glioma in August 2020, and for atypical teratoid / rhabdoid tumours in June 2022 and July 2022, respectively.

For more information, please visit or follow us on Twitter @KaziaTx.

Forward-Looking Statements
This announcement may contain forward-looking statements, which can generally be identified as such by the use of words such as "may," "will," "estimate," "future," "forward," "anticipate," or other similar words. Any statement describing Kazia's future plans, strategies, intentions, expectations, objectives, goals or prospects, and other statements that are not historical facts, are also forward-looking statements, including, but not limited to, statements regarding: the timing for results and data related to Kazia's clinical and preclinical trials and investigator-initiated trials of Kazia's product candidates, and Kazia's strategy and plans with respect to its programs, including paxalisib and EVT801. Such statements are based on Kazia's current expectations and projections about future events and future trends affecting its business and are subject to certain risks and uncertainties that could cause actual results to differ materially from those anticipated in the forward-looking statements, including risks and uncertainties: associated with clinical and preclinical trials and product development, related to regulatory approvals, and related to the impact of global economic conditions. These and other risks and uncertainties are described more fully in Kazia's Annual Report, filed on form 20-F with the United States Securities and Exchange Commission (SEC), and in subsequent filings with the SEC. Kazia undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise, except as required under applicable law. You should not place undue reliance on these forward-looking statements, which apply only as of the date of this announcement.

This announcement was authorized for release by Dr John Friend, CEO.

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What new data will Kazia Therapeutics present at ISPNO 2024?

Kazia Therapeutics will present data from the Phase 2 PNOC DMG-ACT study, including survival rates and preclinical data on paxalisib with HDAC inhibitors and gemcitabine.

What are the survival rates reported in Kazia's Phase 2 PNOC DMG-ACT study?

The Phase 2 study reported median overall survival rates of 13.2 months (Cohort 1), 15.8 months (Cohort 2), and 8.8 months (Cohort 3).

What is the significance of Kazia's publication in the European Journal of Cancer?

The publication highlights the need for mutation-specific, CNS-penetrant inhibitors for treating pediatric Diffuse Midline Glioma (DMG), validating Kazia's research efforts.

How might the new preclinical data impact Kazia's treatment strategies?

Preclinical data showing potential synergy of paxalisib with HDAC inhibitors and gemcitabine may open new avenues for treating atypical teratoid/rhabdoid tumors.

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