Kazia Therapeutics adds recruitment sites to paxalisib arm of global glioblastoma study

Oncology-focused drug development company Kazia Therapeutics (ASX: KZA) has confirmed the multi-drug platform study GBM AGILE has opened recruitment to its paxalisib arm in Europe.

GBM AGILE is a global, academic-led study designed to identify promising new therapies for glioblastoma, which is the most common and most aggressive form of primary brain cancer in adults.

The study is sponsored by the Global Coalition for Adaptive Research (GCAR) and involves three drug candidates— Bayer’s regorafenib, Kazia’s paxalisib, and Kintara Therapeutics’ VAL-083.

Biohaven Pharmaceuticals troriluzole drug and Vigeo Therapeutics’ VG1021 will enter the study later this year.

Study sites

Kazia announced the University Hospital of Zurich will join over 40 study sites in the US and Canada which are currently recruiting patients to the paxalisib arm.

It represents the first time that paxalisib has been the subject of clinical trial activity in Switzerland.

Additional sites are expected to open in Switzerland and in several other European countries in the near future.

The university’s department of neurology director Dr Michael Weller said expansion of the trial into Europe was a positive development.

“There is a profound need for new treatment options in glioblastoma, and GBM AGILE has been designed to evaluate new therapies in the most efficient way possible,” he said.

“We look forward to making a significant contribution to the study, and to seeing results in due course.”

Brain-penetrant inhibitor

Paxalisib is a brain-penetrant inhibitor of the PI3K / Akt / mTOR pathway and is being developed to treat glioblastoma.

Licenced from Genentech in late 2016, the drug commenced recruitment to GBM AGILE in January last year.

Paxalisib was granted orphan drug designation for glioblastoma by the US Food and Drug Administration in early 2018, and fast track designation in mid-2020.

It was also granted rare paediatric disease designation and orphan designation for diffuse intrinsic pontine glioma (another highly-aggressive tumour) by the US FDA in 2020.

Paxalisib recruitment

The paxalisib arm of GBM AGILE will recruit newly-diagnosed patients with the unmethylated MGMT promotor (a genetic marker which denotes near-total resistance to existing FDA-approved standard of care drug temozolomide) and recurrent patients who have progressed despite treatment with temozolomide.

Patients recruited to the paxalisib arm will be compared against a shared control group.

The duration of enrolment is estimated to be between 30 months and 36 months, and final data is expected by the end of next year.

The primary endpoint of GBM AGILE is overall survival, which is also considered the gold standard for the evaluation of new cancer therapies and is the preferred approval endpoint for regulators including the US FDA.

Kazia expects GBM AGILE to serve as the pivotal study for registration in key markets including in the European Union.