Kazia Therapeutics Limited ("Kazia" or the "Company")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In its recently issued draft guidance, the FDA stated that overall survival is the "gold standard" endpoint in oncology and "should be prioritized as the primary endpoint when feasible,"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.