The study is set to open a new arm with Kazia’s investigational new drug, paxalisib, and will now move into an operational phase with recruitment of patients to the paxalisib arm expected to begin in the first quarter of 2021.
“Ground-breaking clinical trial”
Chief executive officer James Garner said: “We have spent the last nine months or so working closely with the GCAR team to plan paxalisib’s entry into GBM AGILE and we are very gratified to now be moving into the operational phase of the study.
“GBM AGILE is truly a ground-breaking clinical trial, driven by some of the world’s leading experts in the field, and we are proud to be a part of it.
“We expect GBM AGILE to provide definitive clinical evidence for the approval of paxalisib by regulatory agencies in key markets.
“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.”
Costs and timeline
Kazia will initially pay a fee of US$5 million to GCAR in consideration for paxalisib joining GBM AGILE.
Additional payments will be due throughout the duration of the study, dependent on the attainment of key milestones.
The full financial terms of the agreement between Kazia and GCAR are considered commercially confidential.
Additionally, the total cost of the study will depend on the number of patients ultimately recruited and other operational variables.
Kazia and GCAR expect that necessary regulatory filings and submissions to institutional review boards will be actioned during the current quarter.
The first patient in to the paxalisib arm is anticipated to occur early in 2021.
GCAR chief executive officer Dr Meredith Buxton said: “We are pleased to welcome paxalisib into GBM AGILE.
“Our mission is to help drive the development of new therapies for glioblastoma, by creating an efficient model for testing and confirming new potentially beneficial treatments for patients with GBM.
“We look forward to continuing to work closely with the Kazia team to bring paxalisib into the study and support its evaluation.”
Dr Ingo Mellinghoff and Dr Eudocia Q Lee will serve as principal investigators for the paxalisib arm while Dr Timothy Cloughesy is principal investigator for the overall study.
Dr Mellinghoff is the chair of the Department of Neurology at Memorial Sloan Kettering Cancer Center in New York, NY.
He is a highly experienced neuro-oncologist with an extensive track record of published research in brain tumours and is a professor at the Gerstner Sloan Kettering Graduate School of Biomedical Sciences and the Graduate School of Medical Sciences at Weill Cornell University.
Dr Lee is a neuro-oncologist at Dana-Farber Cancer Institute in Boston, MA, Director of Clinical Research at the Center for Neuro-Oncology at Dana-Farber, and an Assistant Professor of neurology at Harvard Medical School.
She is a widely published clinical researcher, with a primary research interest in tumours of the brain and spinal cord, and their neurologic complications.
Dr Lee said: “GBM AGILE has been designed to provide a definitive assessment of the efficacy of new drugs for glioblastoma.
“Paxalisib has already been evaluated in two clinical trials in this disease, and GBM AGILE will now greatly enrich our understanding of how best to use it for the benefit of patients.”
GBM AGILE is an adaptive study where the number of patients recruited, and their allocation within the study, will be continuously adjusted in the light of emerging results.
It is expected that between 50 and 200 patients will receive paxalisib, depending on the safety and efficacy of the drug.
Data from these patients will be compared against data from an estimated several hundred patients in a shared control arm, allowing for considerable operational efficiency.
The primary endpoint of GBM AGILE is overall survival (OS), which is considered the gold standard endpoint for the assessment of new cancer therapies.