Kazia Therapeutics has commenced a Phase II clinical trial of its investigational new drug, paxalisib, along with metformin and a ketogenic diet to treat recently diagnosed and recurrent glioblastoma, a type of primary brain cancer.
The trial was launched at Weill Cornell Medicine in the US, with the first subject initiated and a second participant in the screening stage.
Licenced by Kazia from Genentech in 2016, paxalisib is a brain-penetrant inhibitor of the PI3K / Akt / mTOR pathway.
The open-label, two-arm trial will analyse the efficacy of paxalisib in combination with a ketogenic diet and metformin in patients with glioblastoma.
Cohort 1 of the trial will have subjects with recently detected glioblastoma with unmethylated MGMT promotor status.
Cohort 2 will enrol individuals with recurrent glioblastoma, irrespective of the methylation status of their MGMT promotor and have progressed following standard-of-care treatment.
Subjects in both cohorts will be given paxalisib plus metformin and a ketogenic diet.
Each arm will have two stages with nearly 16 subjects enrolled in the initial stage to identify initial signs of activity.
Nearly 15 subjects will be enrolled in the second confirmatory stage on confirming the activity.
The proportion of subjects who are alive and progression-free at six months will be the primary endpoint of the trial.
Apart from safety and efficacy, the trial will evaluate various metabolic, pharmacodynamic and new radiographic imaging biomarkers that could aid research and clinical practice in the future.
The company intends to conclude subject recruitment in the second half of CY2023.
Kazia Therapeutics CEO Dr James Garner said: “This important milestone kicks off an exciting year for Kazia, with at least six potential data read-outs anticipated over the course of 2022.
“The GBM AGILE study continues to progress well, and additional studies, such as this one, have the potential to substantially extend and enhance the use of paxalisib in clinical practice.”
The commencement of the latest trial for glioblastoma treatment takes the number of trials of paxalisib underway in brain cancer to a total of eight.
In December last year, the company reported positive data from a Phase II trial of paxalisib as a first-line therapy to treat glioblastoma.