On December 11, 2019 Kazia Therapeutics Limited (ASX: KZA;NASDAQ: KZIA), an Australian oncology-focused biotechnology company, is reported that it’s lead program, GDC-0084, has been selected to join GBM AGILE, an international, academic-led, multi-drug adaptive phase II / III study in glioblastoma (Press release, Kazia Therapeutics, DEC 11, 2019, View Source;iii-study-in-glioblastoma-300972810.html [SID1234552281]). It is expected that data from GBM AGILE will be used to seek marketing approval for GDC-0084 from FDA and other regulatory agencies.
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Key Points
GBM AGILE (NCT03970447) is an adaptive ‘master protocol’ study, in which different drug candidates can be tested for potential use in glioblastoma
Study is designated phase II / III and data from it is considered acceptable for product registration purposes by US FDA
Kazia has entered into a preliminary agreement to commence planning and set-up activities for inclusion of GDC-0084 in GBM AGILE, with a view to commencing recruitment in Q2 / Q3 CY2020, subject to a definitive agreement
Kazia plans to participate in GBM AGILE in place of a company-run registration study, and GBM AGILE will serve as the path-to-market for GDC-0084
Study is expected to recruit up to 200 patients into the GDC-0084 arm
Dr Timothy Cloughesy, GBM AGILE Global Principal Investigator, commented, "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 (www.gcaresearch.org).
The study commenced recruitment of its first investigational arm in June 2019. 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 minimizes 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.
Dr James Garner, Kazia CEO, commented, "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."
Dr Garner was speaking from the inaugural International Glioblastoma Drug Development Summit in Boston, MA, where Kazia is an invited speaker. The Summit has convened many of the leading researchers, clinicians, and industry participants in the field of glioblastoma to discuss new approaches to the development of novel therapies.
GBM AGILE has the potential to test new drug candidates in several different patient subgroups. In addition to the newly-diagnosed unmethylated group, which Kazia has already identified as the primary target population, the intent is to also test GDC-0084 in recurrent patients. The company may consider future use in newly-diagnosed methylated patients in consultation with clinicians as further data becomes available.
Kazia and GCAR have entered into a preliminary agreement to begin set-up work for inclusion of GDC-0084 in GBM AGILE, and it is expected that this work will take approximately four to six months. The proceeds of Kazia’s recent institutional financing round will be used to support these activities. Patient recruitment is expected to begin in Q2 / Q3 CY2020, and will be contingent upon execution of a definitive agreement between the parties.
Dr Meredith Buxton, COO of GCAR, added, "The future of drug development requires new approaches, particularly in challenging diseases such as glioblastoma. GBM AGILE is scientifically rigorous, highly efficient, and statistically innovative, and has been designed to provide the best possible platform to generate new treatment options for patients. We look forward to working with the Kazia team to bring GDC-0084 into the study as swiftly as possible."