GDC-0084, a small molecule inhibitor of the PI3K/AKT/mTOR pathway, is being developed to treat glioblastoma, the most common and aggressive form of primary brain cancer in adults.
It is expected that data from GBM AGILE will be used to seek marketing approval for GDC-0084 from FDA and other regulatory agencies.
Important treatment option for brain cancer
GBM AGILE global principal investigator Dr Timothy Cloughesy said: “We see an urgent need for new therapies in glioblastoma, and GBM AGILE has been designed to provide an opportunity for industry to test new therapeutic agents in a cutting-edge, registration-level study, at considerably lower cost and in a faster time than would typically be possible for a company-driven study.
“GDC-0084 has the potential to become an important treatment option for brain cancer, and this study is the best way to definitively determine its efficacy in this challenging disease.”
GBM AGILE is sponsored and administered by the Global Coalition for Adaptive Research (GCAR), a non-profit organization which includes many of the world’s leading scientists and clinicians in the field of brain cancer.
The study commenced recruitment of its first investigational arm in June 2019.
Primary endpoint is overall survival
GBM AGILE is designed as a ‘master protocol’ study, into which different drug candidates can be placed for testing against a common control arm.
It is an ‘adaptive study’, utilizing Bayesian statistical techniques to dynamically adjust the number of patients in a given arm according to emerging signals of activity.
This minimises redundant patient recruitment, saving cost and time.
The primary endpoint is overall survival (OS), which is considered the ‘gold standard’ for the approval of new cancer therapies by FDA and other regulatory agencies.
Participating drugs are first examined in a stage 1 (phase II) component, which then progresses seamlessly into a stage 2 (phase III) component once pre-defined efficacy hurdles are met.
Kazia CEO Dr James Garner said: “GBM AGILE offers three enormous advantages to Kazia.
“First, the highly innovative adaptive design allows us to test GDC-0084 in the fastest and most cost-effective way possible.
“Second, the considerable technical, scientific, and operational capability in GCAR gives us access to resources that we could never hope to draw upon otherwise.
“Third, the quality of the study, and the caliber of the participating sites, means that GDC-0084 will have the best possible opportunity to demonstrate its potential.
“No company our size could run a study like this single-handedly, so we have adopted GBM AGILE as our primary path-to-market strategy for GDC-0084.”