SYDNEY, Oct. 27, 2025 -- Kazia Therapeutics Limited ("Kazia" or the "Company") today announced its intention to request and hold a follow-up Type C meeting with the U.S. Food & Drug Administration (FDA) to discuss overall survival (OS) findings in newly diagnosed glioblastoma (GBM) patients treated with paxalisib and to seek agency feedback on a potential regulatory pathway aligned with the FDA Oncology Center of Excellence's Project FrontRunner initiative.
"GBM remains one of the most lethal cancers with limited therapeutic options. In line with the FDA Oncology Center of Excellence's Project FrontRunner initiative, we intend to engage the Agency to discuss whether the overall survival data generated in newly diagnosed GBM patients treated with paxalisib may be adequate to support a conditional approval pathway," said Dr. John Friend, M.D., Chief Executive Officer of Kazia Therapeutics. "Consistent with this framework, Kazia will propose initiation of the post-approval, randomized Phase 3 confirmatory study prior to submission of the NDA, ensuring that our regulatory strategy fully reflects the FDA's renewed emphasis on overall survival as the most meaningful endpoint for patients and clinicians."
In its recently issued draft guidance, the FDA stated that overall survival is the "gold standard" endpoint in oncology and "should be prioritized as the primary endpoint when feasible," particularly in diseases with a short natural history where survival can be reliably assessed. Kazia believes GBM is precisely such a setting and intends to present survival analyses, supporting clinical safety, and planned confirmatory trial design for FDA discussion.
Project FrontRunner is an FDA Oncology Center of Excellence initiative encouraging sponsors to consider when it may be appropriate to seek approval of cancer drugs for advanced or metastatic disease in an earlier clinical setting, rather than the traditional approach of developing therapies only for patients who have exhausted available treatment options.
As announced in July 2024, in the prespecified secondary analysis in newly diagnosed (up-front) unmethylated GBM patients, median OS was 15.54 months in the paxalisib arm (n = 54) versus 11.89 months for concurrent standard of care (SOC) (n = 46). Kazia intends to reference Project FrontRunner principles in its Type C briefing package, including an OS-driven confirmatory study plan in newly diagnosed GBM.
"We are moving decisively to bring paxalisib forward in GBM using the endpoints that matter most to patients and physicians," added Dr. Friend. "Our objective is to work collaboratively with the FDA under the guiding principles of Project FrontRunner to pursue a conditional approval in the front-line treatment setting of glioblastoma. In parallel, Kazia will initiate the post-approval, randomized Phase 3 study prior to filing the NDA, ensuring that our development plan fully aligns with the Agency's modernized, patient-focused framework."
Kazia also notes that leading oncology companies have begun publicly referencing Project FrontRunner in successful FDA actions, underscoring the initiative's growing relevance for sponsors developing first-line or earlier-setting therapies.
For investor and media, please contact Alex Star, Managing Director LifeSci Advisors LLC, Astarr@lifesciadvisors.com, +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 central nervous system lymphoma, with several of these trials having reported encouraging interim data. Paxalisib was granted Orphan Drug Designation for glioblastoma by the U.S. Food and Drug Administration (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 tumor 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 tumors 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 tumor 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 contains forward-looking statements, which can generally be identified as such by the use of words such as "may," "will," "plan," "intend," "estimate," "future," "forward," "potential," "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: Kazia's intention to request and hold a Type C meeting with the FDA to discuss OS findings in GBM patients treated with paxalisib and to seek agency feedback on a potential regulatory pathway, the plan to propose initiation of the post-approval, randomized Phase 3 confirmatory study prior to submission of the NDA, the intention to present survival analyses, supporting clinical safety and planned confirmatory trial design for FDA discussion, Kazia's intention to reference Project FrontRunner principles in its Type C briefing package, the objective to work collaboratively with the FDA under the guiding principles of Project FrontRunner, the plan to pursue a conditional approval in the front-line treatment setting of GBM, the plan to initiate the post-approval, randomized Phase 3 study prior to filing the NDA, the goal of ensuring that Kazia's development plan and regulatory strategy fully reflects and aligns with the FDA's framework and emphasis, 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, timing for any regulatory submissions or discussions with regulatory agencies and the potential market opportunity for paxalisib. 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 U.S. government shutdown, 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.
