Global Coalition for Adaptive Research Announces Initiation of AZD1390 in GBM AGILE Trial

AstraZeneca's AZD1390 Now Enrolling in GBM AGILE, a Registrational-Intent Phase 2/3 Adaptive Platform Trial for Patients with Glioblastoma

AZD1390 Arm Will Recruit Patients with Newly Diagnosed Glioblastoma Across Multiple International Trial Sites

LARKSPUR, Calif., May 13, 2025 /PRNewswire/ -- The Global Coalition for Adaptive Research (GCAR) today announced the activation of AstraZeneca's AZD1390 in GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment – NCT03970447), the world's first global adaptive platform trial for glioblastoma. The AZD1390 arm will be evaluated for the treatment of adult patients with newly diagnosed glioblastoma.

Glioblastoma is the most aggressive and common form of primary brain cancer. Treatment options and patient outcomes have seen little progress over the past several decades.

AstraZeneca's AZD1390 is entering the GBM AGILE trial as the 7^th investigational arm. Since launching in 2019, the GBM AGILE trial has evaluated multiple therapies and has screened over 2,300 patients at trial locations in six countries.

GBM AGILE, a seamless phase 2/3 trial, is conducted under a master protocol, allowing multiple therapies or combinations of therapies from different pharmaceutical companies to be evaluated simultaneously against a common control arm. With its innovative design and efficient operational infrastructure, data from GBM AGILE can potentially be used as the foundation for a new drug application (NDA) and registrations to the US FDA and other health authorities.

Professor Anthony Chalmers, Chair of Clinical Oncology at the University of Glasgow and Dr. Patrick Wen, Director, Center for Neuro-Oncology at Dana-Farber Cancer Institute, and Professor of Neurology at Harvard Medical School, are serving as arm Principal Investigators for AZD1390's evaluation in GBM AGILE. Dr. Timothy Cloughesy, Director, Neuro-Oncology Program and Distinguished Professor of Neurology at the University of California, Los Angeles, is the Global Principal Investigator for the overall study.

"Glioblastoma is an invariably fatal cancer with few effective treatment options. There is an urgent need for better treatment options," said Professor Anthony Chalmers. "We are excited to evaluate AZD1390 in GBM AGILE based on encouraging data from previous studies and the extensive evidence that AZD1390 can increase the effectiveness of radiation therapy. This investigational drug has clear potential to improve outcomes for glioblastoma patients."

"GBM AGILE is designed to rapidly identify and confirm effective therapies for patients with glioblastoma through response adaptive randomization," said Dr. Patrick Wen. "With the addition of AZD1390 to GBM AGILE, a global clinical trial, we are able to offer GBM patients access to the latest and most promising investigational therapies."

AZD1390 is a brain penetrant ataxia telangiectasia mutant (ATM) kinase inhibitor that blocks ATM-dependent signaling and repair of DNA double strand breaks (DSBs) in the genome. Preclinically, AZD1390 exhibits activity in combination with agents such as radiation therapy that induce DSBs. AZD1390 has also been shown to achieve clinically relevant concentrations in resected glioblastoma tissue and suppress the ability of tumor cells to repair the DNA damage that is induced by radiation. Furthermore, data from a Phase I trial of AZD1390 with radiotherapy showed a manageable toxicity profile and preliminary efficacy for patients with recurrent glioblastoma and was shared in an oral presentation at the American Association for Cancer Research (AACR) Annual Meeting 2024.

"At GCAR, we're dedicated to accelerating the development of critically needed therapies for patients facing rare and deadly diseases like glioblastoma," said Dr. Meredith Buxton, CEO and President of GCAR. "We believe master protocols and adaptive platform trials are a powerful way to expedite that progress. We're excited to collaborate with AstraZeneca and look forward to rapidly advancing the development of AZD1390 in GBM AGILE to help address the urgent need for more effective glioblastoma therapies." 

About Global Coalition for Adaptive Research (GCAR)
Global Coalition for Adaptive Research (GCAR) is a 501(c)(3) non-profit corporation uniting physicians, clinical researchers, advocacy and philanthropic organizations, biotech/pharma companies, health authorities, and other key stakeholders in healthcare to expedite the discovery and development of treatments for patients with rare and deadly diseases. As the sponsor of innovative trials, including master protocols and adaptive platform trials, GCAR is dedicated to the advancement of science by modernizing clinical trials that support more efficient, less costly drug development. To learn more about GCAR and its initiatives, visit gcaresearch.org and follow us on social media @GCAResearch.

SOURCE Global Coalition for Adaptive Research