Kazia Therapeutics Limited (NASDAQ: KZIA), an oncology-focused drug development company based in Australia, on Tuesday provided a regulatory update on paxalisib for the treatment of glioblastoma (GBM) following its Type C clinical meeting with the US Food and Drug Administration (FDA).

In July 2024, the company reported topline results from the GBM-AGILE study in which newly diagnosed unmethylated (NDU) patients with glioblastoma treated with paxalisib showed a clinically meaningful improvement in a prespecified secondary analysis for overall survival (OS) compared to standard of care. Based on these results and the data from all completed paxalisib clinical studies, Kazia requested a meeting with the FDA to discuss potential clinical and regulatory paths.

Following discussions with the FDA and feedback from Kazia's recent Type C meeting, the FDA's current position is that data on OS would generally not be appropriate for accelerated approval, but could be considered to support a traditional/standard approval. The Agency further stated that the secondary endpoint OS data from the GBM-AGILE study are supportive and informative for designing and executing a pivotal registrational study in pursuit of a standard approval.

Kazia noted that the company aligned with the FDA on key aspects of the design of a proposed registrational/pivotal phase 3 study, including patient population, primary endpoint, and the comparator arm to be used.

"We appreciate the extensive and thoughtful feedback from the FDA, which provides us with added clarity with respect to paxalisib's potential registration pathway for the treatment of patients with NDU glioblastoma," commented Dr. John Friend, Kazia's CEO. "We believe data from the GBM-AGILE trial, including the prespecified secondary endpoint, which demonstrated a 3.8-month OS improvement, provides evidence supporting a clinically meaningful efficacy signal that merits further testing paxalisib in this patient population in a larger, pivotal study."