, /PRNewswire/ -- Kazia Therapeutics Limited (NASDAQ: ), an oncology-focused drug development company, is pleased to announce results from GBM-AGILE, a phase II/III study that included an evaluation of paxalisib versus standard of care (SOC) for patients with glioblastoma (NCT03522298), a life-threatening brain cancer, where there is an urgent unmet need for new therapeutics. GBM AGILE is an adaptive phase II/III global trial sponsored by the Global Coalition for Adaptive Research (GCAR), a nonprofit organization comprised of some of the world's foremost clinical, translational, and basic science researchers, from institutions such as Memorial Sloan Kettering Cancer Center and Dana-Farber Cancer Institute. The trial is designed to efficiently screen for and characterize the response of glioblastoma (GBM) patients to novel investigative agents.
Utilizing a complex innovative design, Bayesian principles are applied to the primary endpoint (Overall Survival) comparison of the investigational agents to patients receiving Standard of Care (SOC) enrolled from the study start (also referred to as cumulative control population). In general, secondary analyses and endpoints are assessed based on established statistical models in comparison to the control patients enrolled at the same time as the investigational agent (concurrent control population). Paxalisib is the third drug candidate to complete its evaluation in the study and was evaluated in newly diagnosed glioblastoma patients .