Kazia Therapeutics Supports Australian MRFF-Funded Project Developing AI-Driven Sequential Therapy Strategy for DIPG/DMG

SYDNEY, Oct. 1, 2025 /PRNewswire/ -- Kazia Therapeutics Limited (NASDAQ: KZIA), a clinical-stage biotechnology company developing innovative therapies for brain cancers, today announced its participation in a fully funded Australian Medical Research Future Fund (MRFF) project titled "Sequential & Temporal Therapeutic Agility for the Treatment of Diffuse Midline Glioma (DMG)".

Under this three-year initiative, a consortium of leading researchers will establish DMG-ADAPTS, an advanced AI-enabled clinical decision-making platform designed to optimize sequencing and timing of targeted therapies for diffuse midline glioma (DMG), including diffuse intrinsic pontine glioma (DIPG). Kazia will provide its investigational drug paxalisib, a brain-penetrant, dual PI3K/mTOR inhibitor, to support the study.

Project Overview

DMG/DIPG represents one of the most devastating pediatric cancers, accounting for up to 25% of childhood brain cancer deaths, with median survival of less than one year. While early clinical studies of targeted and immune therapies have demonstrated biological activity, durable responses remain limited due to rapid resistance driven by epigenomic plasticity.

The MRFF-funded project aims to address this challenge by:

• Developing DMG-ADAPTS, a cloud-based AI platform integrating multiomics, tumor, CSF, and blood profiling to guide individualized, adaptive therapy decisions in real time.
• Testing a therapeutic toolkit of twelve brain-penetrant small molecules (including paxalisib) and immunotherapies across preclinical patient-derived models.
• Establishing non-invasive biomarkers to anticipate resistance and inform timely therapeutic transitions.

Lead investigator, University of Newcastle Professor of Paediatric Haematology and Oncology Matt Dun, of HMRI's Precision Medicine Research Program, said "Our extensive work, alongside that of the international community, has identified key genetic and adaptive vulnerabilities in DMG. Therapeutics such as paxalisib represent an important addition to the treatment toolkit, ensuring patients have every chance at long-term survival. We are delighted to partner with Kazia to define where paxalisib best fits within our therapeutic strategy".

Kazia's Role

Kazia will contribute paxalisib, already under evaluation in multiple clinical studies for both adult and pediatric brain cancers. Notably, positive signals have been reported in the PNOC 022 study, a multi-arm pediatric brain cancer trial where paxalisib has shown encouraging activity.

"Our participation in this MRFF project underscores Kazia's continued commitment to finding innovative solutions for children facing DIPG and DMG," said Dr. John Friend, Chief Executive Officer of Kazia Therapeutics. "By integrating cutting-edge AI with precision therapies such as paxalisib, this initiative has the potential to revolutionize how these aggressive tumors are managed. We are proud to provide drug supply support in this cost-conscious collaboration that leverages global research excellence."

For investor and media, please contact Alex Star, Managing Director LifeSci Advisors LLC,  [email protected], +1-201-786-8795.

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 cancer. Licensed from Genentech in late 2016, paxalisib is or has been the subject of ten clinical trials in this disease. A completed Phase 2/3 study in glioblastoma (GBM-Agile) was reported in 2024 and discussions are ongoing for designing and executing a pivotal registrational study in pursuit of a standard approval. Other clinical trials involving paxalisib are ongoing in advanced breast cancer, brain metastases, diffuse midline gliomas, and primary CNS lymphoma, with several of these trials having reported encouraging interim data. Paxalisib was granted Orphan Drug Designation for glioblastoma by the FDA in February 2018, and Fast Track Designation (FTD) for glioblastoma by the FDA in August 2020. Paxalisib was also granted FTD in July 2023 for the treatment of solid tumour brain metastases harboring PI3K pathway mutations in combination with radiation therapy. In addition, 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. Kazia is also developing EVT801, a small molecule inhibitor of VEGFR3, which was licensed from Evotec SE in April 2021. Preclinical data has shown EVT801 to be active against a broad range of tumour types and has provided evidence of synergy with immuno-oncology agents. A Phase I study has been completed and preliminary data was presented at 15th Biennial Ovarian Cancer Research Symposium in September 2024. For more information, please visit www.kaziatherapeutics.com or follow us on X @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, Kazia's strategy and plans with respect to its paxalisib program, the potential benefits of paxalisib as an investigational PI3K/mTOR inhibitor, timing for any regulatory submissions or discussions with regulatory agencies, the potential market opportunity for paxalisib and Kazia's intent and efforts to regain and/or maintain compliance with the applicable Nasdaq continued listing requirements and standards. 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, including the risk that interim or early data may not be consistent with final data, risks related to regulatory approvals, risks related to the impact of global economic conditions, and risks related to Kazia's ability to regain and/or maintain compliance with the applicable Nasdaq continued listing requirements and standards. These and other risks and uncertainties are described more fully in Kazia's Annual Report, filed on form 20-F with the SEC, and in subsequent filings with the United States Securities and Exchange Commission. 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

SOURCE Kazia Therapeutics Limited