Kazia Therapeutics reports positive phase II/III results for paxalisib in glioblastoma, showing promising survival outcomes in newly diagnosed patients and plans FDA discussions for potential accelerated approval.

Australia: Kazia Therapeutics Limited (NASDAQ: KZIA), a company specializing in oncology drug development, has announced the results of the GBM-AGILE phase II/III study evaluating paxalisib compared to standard of care (SOC) for glioblastoma patients. Glioblastoma, a severe form of brain cancer, lacks effective treatment options, underscoring the significance of these findings.

GBM-AGILE, sponsored by the Global Coalition for Adaptive Research (GCAR), includes leading researchers from institutions like Memorial Sloan Kettering Cancer Center and Dana-Farber Cancer Institute. This adaptive trial employs Bayesian principles to analyze primary endpoints, particularly Overall Survival (OS), comparing investigational agents with concurrent SOC patients enrolled since the study's inception in July 2019.

Paxalisib, the third drug evaluated in the study, targeted newly diagnosed unmethylated MGMT promoter status glioblastoma patients and those with recurrent disease. Initial results from the study's Stage 1 showed promising OS outcomes: newly diagnosed patients treated with paxalisib exhibited a median OS of 15.54 months, compared to 11.89 months with SOC. Similar trends were observed in sensitivity analyses, reinforcing earlier phase II findings.

Importantly, paxalisib demonstrated tolerability with no new safety concerns identified. However, efficacy in recurrent disease patients did not meet statistical significance compared to SOC. Despite this, Kazia plans further analyses to explore potential signals warranting continued investigation.

Building on these findings, Kazia intends to request a meeting with the FDA to discuss potential accelerated approval pathways for paxalisib, which has received orphan drug and fast track designations for glioblastoma treatment in unmethylated MGMT promoter status patients.

These results mark a pivotal step forward in glioblastoma research, offering hope for improved treatment strategies in a challenging clinical landscape.

Kazia CEO, Dr 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.”

According to TechSci Research, Kazia Therapeutics' positive results for paxalisib in glioblastoma could significantly impact the healthcare market. The potential for accelerated FDA approval may enhance treatment options for patients with unmethylated MGMT promoter status, addressing a critical unmet need in glioblastoma therapy. This development may attract investor interest in Kazia Therapeutics and other companies involved in oncology drug development, bolstering the healthcare market's growth trajectory. In the coming years, successful FDA discussions and potential market entry of paxalisib could lead to increased competition and innovation in glioblastoma treatment, potentially benefiting patients worldwide.