Kazia Therapeutics Announces Phase II/III Clinical Trial Results for Paxalisib in Glioblastoma

Core Insights - Kazia Therapeutics Limited announced results from the GBM-AGILE study, highlighting the efficacy of paxalisib in treating glioblastoma, a severe brain cancer with significant unmet medical needs [1][9]. Study Overview - The GBM-AGILE trial is an adaptive phase II/III global study sponsored by the Global Coalition for Adaptive Research, aimed at evaluating the response of glioblastoma patients to new investigational agents using innovative Bayesian design [2]. - The trial included 313 patients, with newly diagnosed unmethylated (NDU) and recurrent glioblastoma patients randomized to receive either paxalisib or standard of care [3]. Efficacy Results - In the primary analysis, the median overall survival (OS) for paxalisib-treated NDU patients was 14.77 months compared to 13.84 months for the cumulative standard of care [10]. - A prespecified secondary analysis showed a median OS of 15.54 months for paxalisib-treated NDU patients versus 11.89 months for concurrent standard of care, indicating a 3.8-month improvement, approximately a 33% increase in survival [10][19]. - No efficacy signal was detected in the recurrent disease population, with median OS of 9.69 months for standard of care versus 8.05 months for paxalisib [5]. Safety Profile - Paxalisib was well tolerated in the GBM-AGILE trial, with no new safety signals identified in the patient population [11]. Future Plans - Based on the data from the GBM-AGILE study, Kazia plans to request a meeting with the FDA to discuss potential pathways for accelerated approval of paxalisib [12]. - Full data, including secondary endpoints from the paxalisib arm, is expected to be presented at a scientific meeting later in 2024 [18]. Company Background - Kazia Therapeutics is focused on developing paxalisib, an investigational drug targeting the PI3K/Akt/mTOR pathway for various brain cancers, and has conducted multiple clinical trials since its licensing from Genentech in 2016 [6]. - The company is also developing EVT801, a small-molecule inhibitor of VEGFR3, with ongoing Phase I studies [7].