Bayer's regorafenib kicks off brain cancer platform trial

Bayer has become the first pharma company to take part in a new platform trial designed to find new treatments for brain cancer.

GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment) is an international trial testing several therapies for patients with newly diagnosed and recurrent glioblastoma.

Using a master protocol, several therapies or combinations of therapies from pharma companies can be tested simultaneously, with potential treatments added or dropped from the phase 2/3 trial over time.

The idea is to adopt a more efficient approach to testing new therapies for glioblastoma, and Bayer is starting proceedings by opening enrolment for US patients in an arm evaluating its cancer drug regorafenib.

Sponsored by the non-profit charitable organisation the Global Coalition for Adaptive Research (GCAR), by the end of the year GBM AGILE will open in over 40 academic medical centres and community-based institutions across the US.

There are plans to expand across Europe, China, Canada, and Australia through 2020.

GCAR aims to expand and replicate what is learned using this model for GBM to benefit patients with other rare and deadly diseases.

A similar approach has already been used in breast cancer, where the I-SPY trials have used a platform design to test several different breast cancer drugs against the same control group.

This represents a more efficient and ethical approach that reduces the number of patients required, and limits exposure to the potentially less effective standard therapy.

Glioblastoma treatment options are limited and patient outcomes have remained largely unchanged over several decades and 95% of patients die within five years of diagnosis, with more than half dying within the first 15 months after diagnosis.

Regorafenib showed promise compared to standard of care in the randomised multi-institutional investigator-sponsored Phase 2 trial REGOMA, published in The Lancet Oncology in December, 2018.

Regorafenib is already approved under the brand name Stivarga in more than 90 countries, including the US, countries of the European Union, China and Japan for metastatic colorectal cancer, metastatic gastrointestinal stromal tumors and hepatocellular carcinoma.

The drug is an oral multi-kinase inhibitor that potently blocks multiple protein kinases involved in tumor angiogenesis (VEGFR1, -2, -3, TIE2), oncogenesis (KIT, RET, RAF-1, BRAF), metastasis (VEGFR3, PDGFR, FGFR) and tumour immunity (CSF1R).