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Kazia Therapeutics' Paxalisib to be Evaluated in Glioblastoma Study

NEW YORK – Sydney, Australia-based Kazia Therapeutics announced on Friday that a new arm within the Glioblastoma Adaptive Global Innovative Learning Environment (GBM AGILE) study will explore the activity of its investigational drug paxalisib.

The paxalisib arm will recruit newly diagnosed glioblastoma patients with unmethylated MGMT promotor status, as well as patients with recurrent disease. The drug firm is also studying newly diagnosed glioblastoma patients with unmethylated MGMT promotor status in an ongoing Phase II paxalisib trial. The primary endpoint of the trial is overall survival.

Between 50 and 200 patients are expected to receive paxalisib within GBM AGILE. The safety and efficacy of the drug in these patients will be compared against the outcomes of several hundred patients in a shared control arm. Kazia expects to begin enrolling patients in early 2021 and continue recruitment for up to three years.

"We expect GBM AGILE to provide definitive clinical evidence for the approval of paxalisib by regulatory agencies in key markets," Kazia CEO James Garner said in a statement. "This is a faster, more cost effective, and higher quality study than any company of our size could mount independently, and we are confident that it will provide the best possible opportunity for paxalisib to demonstrate its potential in this very challenging disease."

GBM AGILE, an international adaptive study sponsored by nonprofit the Global Coalition for Adaptive Research (GCAR), is focused on finding new treatments for glioblastoma. Kazia will pay an initial fee of $5 million to GCAR to explore its drug in the study.

Paxalisib, a PI3K/AKT/mTOR pathway inhibitor that Kazia licensed from Genentech, is the second drug protocol added to GBM AGILE since the study began in 2019. The trial's first drug protocol was for regorafenib (Bayer's Stivarga). The study is ongoing at 30 sites in the US and Canada, and there are plans to expand into Europe and China next year.

Ingo Mellinghoff from Memorial Sloan Kettering and Eudocia Lee from the Dana-Farber Cancer Institute will be principal investigators for the paxalisib arm.

The US Food and Drug Administration has granted paxalisib fast track and orphan disease designation for glioblastoma.