Kazia Therapeutics reports results from GBM-AGILE phase II/III study

Kazia Therapeutics announced results from GBM-AGILE, a phase II/III study that included an evaluation of paxalisib versus standard of care, or SOC, for patients with glioblastoma, or NCT03522298, a life-threatening brain cancer, where there is an urgent unmet need for new therapeutics. Kazia CEO, John Friend stated, “We are excited to have shown a 3.8 month improvement in overall survival, an approximate 33% improvement, for newly diagnosed unmethylated patients with GBM compared to the concurrent standard of care arm. Having comparable Overall Survival data across two independent studies is a compelling outcome in this difficult to treat glioblastoma population. We look forward to discussing possible approaches for an accelerated approval pathway for paxalisib with the FDA.” A total of 313 newly diagnosed unmethylated patients and recurrent patients being treated at top US cancer hospitals were randomized in Stage 1 to either a paxalisib treatment arm or the SOC concurrent control arm from January 2021 to May 2022. The cumulative control arm was enrolled from July 2019 to May 2022. For the primary analysis the median Overall Survival was 14.77 months for paxalisib-treated NDU patients versus 13.84 months for cumulative SOC NDU patients.Full data including secondary endpoints from the paxalisib arm of the GBM AGILE study is expected to be presented at a scientific meeting later this year.