Kazia Therapeutics Limited announced plans to hold a follow-up Type C meeting with the U.S. Food and Drug Administration (FDA) to discuss new overall survival (OS) findings from patients with newly diagnosed glioblastoma (GBM) treated with its investigational therapy, paxalisib. The company intends to seek FDA guidance on a potential conditional approval pathway under the Oncology Center of Excellence’s Project FrontRunner initiative.
“GBM remains one of the most lethal cancers with limited therapeutic options,” said Dr. John Friend, M.D., Chief Executive Officer of Kazia Therapeutics. “We plan to engage the FDA to determine whether the overall survival data from newly diagnosed GBM patients treated with paxalisib could support a conditional approval pathway. In keeping with FDA guidance, we also intend to initiate a post-approval, randomized Phase 3 confirmatory study before submitting the NDA.”
The FDA’s recent draft guidance reaffirmed overall survival as the ‘gold standard’ endpoint in oncology, emphasizing its importance in diseases with short natural histories where survival outcomes can be clearly measured. Kazia believes glioblastoma meets these criteria and plans to present survival data, safety findings, and a confirmatory trial design at the upcoming meeting.
In a previously reported analysis, median overall survival was 15.54 months for newly diagnosed unmethylated GBM patients treated with paxalisib (n=54) compared to 11.89 months for those receiving the standard of care (n=46). The company’s proposed regulatory strategy will incorporate Project FrontRunner principles, which encourage the evaluation of promising therapies earlier in the treatment pathway.
“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 to pursue conditional approval for front-line GBM treatment while launching a confirmatory Phase 3 trial in parallel.”
Kazia noted that several leading oncology developers have successfully engaged with the FDA under Project FrontRunner, underscoring its growing role in shaping modern oncology drug development.