On February 3, 2021 Henry Ford Cancer Institute reported that it is the first site in the world to activate two new treatments for glioblastoma (GBM), the deadliest form of brain cancer, as part of a patient-centered adaptive platform trial known as GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment) (Press release, Kintara Therapeutics, FEB 3, 2021, View Source [SID1234577608]). Led by Global Coalition for Adaptive Research (GCAR), GBM AGILE tests multiple therapies for patients with newly-diagnosed and recurrent GBM.
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Henry Ford Cancer Institute was first-in-the-world to enroll a patient in GBM AGILE when enrollment began in 2019.
"We are excited for this major step forward in the GBM AGILE trial, and especially for the hope it provides those battling glioblastoma brain cancer," said Tom Mikkelsen, M.D., principal investigator for GBM AGILE at Henry Ford Cancer Institute, and medical director of the Precision Medicine Program and Clinical Trials Office at Henry Ford Health System. "Through global collaboration, GBM AGILE is making it possible for some of the world’s foremost experts in glioblastoma research and treatment to collaborate and advance the pace at which scientific and clinical breakthroughs can be achieved."
After opening at Henry Ford Cancer Institute, the two new interventions – VAL-083 from Kintara Therapeutics, Inc. and paxalisib from Kazia Therapeutics Limited– will subsequently open at more than 35 trial sites across the United States, with additional global sites in Canada, Europe and China to follow. VAL-083 is being evaluated in all three glioblastoma patient subtypes: newly-diagnosed methylated MGMT; newly-diagnosed unmethylated MGMT; and recurrent. Paxalisib is being evaluated in newly-diagnosed unmethylated and recurrent glioblastoma.
VAL-083 is a "first-in-class" small molecule that has been studied in more than 40 Phase I and Phase II clinical trials in multiple indications sponsored by the National Cancer Institute. VAL-083 is independent of the MGMT resistance mechanism and has been granted Orphan Drug Designation for glioblastoma by the FDA and for Glioma by the European Medicines Agency. In addition, the FDA granted Fast Track Designation for VAL-083 in recurrent glioblastoma.
Paxalisib is a small molecule inhibitor of the PI3K / AKT / mTOR pathway. The PI3K pathway appears to be disordered in more than 85% of cases of glioblastoma, making this pathway a high-potential target for new glioblastoma therapies. Paxalisib is a potent inhibitor of the PI3K pathway, and has been shown to have an anti-tumor effect in animal models of glioblastoma. Paxalisib was granted Orphan Drug Designation for glioblastoma by the FDA in February 2018, and Fast Track Designation for glioblastoma by the FDA in August 2020.
GBM AGILE is an international, innovative platform trial designed to more rapidly identify and confirm effective therapies for patients with glioblastoma through response adaptive randomization and a seamless phase II/III design. The trial, conceived by over 130 key opinion leaders, is conducted under a master protocol, allowing multiple therapies or combinations of therapies from different pharmaceutical partners to be evaluated simultaneously. With its innovative design and efficient operational infrastructure, data from GBM AGILE can be used as the foundation for a new drug application and biologics license application submissions and registrations to the FDA and other health authorities.