Bayer’s Regorafenib is the First Drug in Innovative Global Glioblastoma Platform Trial

  • GBM AGILE is backed by more than 150 collaborators around the world
  • GBM treatment options and patient outcomes have remained largely unchanged over several decades and there is no known cure


The Global Coalition for Adaptive Research (GCAR) and Bayer announced on 11/14/18 that Bayer’s regorafenib will be the first drug to enter “GBM AGILE” (Glioblastoma Adaptive Global Innovative Learning Environment). GBM AGILE is a revolutionary patient-centered adaptive platform trial that will evaluate multiple therapies for patients with newly diagnosed and recurrent glioblastoma (GBM) – the deadliest form of brain cancer.

“The signals seen with regorafenib in earlier studies warrant further exploration in glioblastoma, a rare cancer where patients are desperately needing new treatment options. We are excited to be the first partner in GBM AGILE, the potentially largest trial ever with an innovative setup that has the possibility to accelerate the evaluation of potentially new effective treatments for patients with glioblastoma, and ultimately could lead to new treatment options for patients,” said Dr. Svetlana Kobina, Head of Medical Affairs Oncology at Bayer. “Bayer supports the active clinical research of regorafenib in a range of different tumor types to explore the potential of the compound to help even more patients in need.”


GBM treatment options and patient outcomes have remained largely unchanged over several decades and there is no known cure. 95% of patients succumb within five years of diagnosis, with more than half dying within the first 15 months after diagnosis.

GBM AGILE is an international, innovative platform trial designed to more rapidly identify effective therapies for patients with glioblastoma through response adaptive randomization and a seamless phase 2/3 design. The trial will be conducted under a master protocol, allowing multiple therapies or combinations of therapies from different pharmaceutical partners to be evaluated simultaneously. Experimental treatments will be added to or dropped from the trial over time. The trial design and infrastructure constitutes a more efficient approach to testing new therapies for GBM, thus bringing new potentially beneficial treatments to patients sooner.

GBM AGILE is billed as the biggest global collaboration in the history of brain cancer research, backed by more than 150 collaborators around the world. Traditionally, pharmaceutical companies test one drug at a time and patients can be on trials for up to two years before they know whether the treatment is working or not. GBM AGILE employs an adaptive clinical trial design, This allows researchers to test treatments tailored to patients’ genomic profiles using their individual biological and genomic information. Results are learned and assessed as the trial progresses. This allows clinicians to continuously add data and apply those learnings to the next patient who enters the trial.

“GBM AGILE is a testament to the many contributors from the international GBM research and advocacy communities who collaboratively ‘crowdsourced’ the knowledge needed to create an adaptive trial that will more efficiently test many therapies, and also serve as a learning system to help oncologists choose the best treatment for every GBM patient. It’s great to see these innovations and the commitments of the GBM AGILE Knowledge Network deployed to support patients,” said Dr. Anna Barker.