Kazia Provides Secondary OS Data for FDA Glioblastoma Drug Case

Kazia Therapeutics recently disclosed further data from the GBM-AGILE study, aiming for FDA accelerated approval for its glioblastoma treatment, paxalisib. GBM-AGILE, a large, global clinical trial, evaluates various glioblastoma treatments against standard therapies. The study investigates paxalisib combined with temozolomide for newly diagnosed patients and lomustine for recurrent cases, with overall survival (OS) as the primary outcome.

In recurrent disease settings, paxalisib showed no efficacy. The median OS for treated patients was 8.05 months, nearly two months shorter than the 9.69-month median OS for those treated with lomustine. Kazia is conducting further analysis to explore potential insights from these data.

Paxalisib demonstrated more promise in newly diagnosed glioblastoma patients, achieving a median OS of 14.77 months, slightly higher than the 13.84-month median OS for those treated with temozolomide. The study also examined a smaller subset of control patients enrolled concurrently with paxalisib's inclusion. In this group, the median OS for paxalisib increased to 15.54 months, while it decreased to 11.89 months for comparators.

John Friend, CEO of Kazia, highlighted the significance of the 3.8-month improvement in overall survival for newly diagnosed unmethylated glioblastoma patients compared to the concurrent standard-of-care group. He emphasized the importance of these findings and noted the company's plans to discuss potential accelerated approval pathways with the FDA.

Following the announcement, Kazia's stock surged 142%, reflecting market optimism. Paxalisib, initially developed by Genentech, inhibits PI3K, a crucial protein in cell growth and division, which is often dysregulated in various cancers. Unlike other PI3K inhibitors, paxalisib can traverse the blood-brain barrier, making it a promising candidate for central nervous system cancers.

In a previous update in August 2022, Kazia reported that paxalisib had not met the criteria for advancing to the second phase of the GBM-AGILE study. At that time, the biotech company was still blinded to the study’s results and could not fully interpret the reasons behind the setback.