Purdue Pharma L.P. has entered into an agreement to include its pipeline medication tinostamustine in the Phase 2/3 GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment) adaptive clinical trial for glioblastoma patients, led by the Global Coalition for Adaptive Research (GCAR).
Novel Dual-Mechanism Approach
Tinostamustine represents a first-in-class, new chemical entity that combines two potentially synergistic mechanisms of action: bifunctional alkylating activity and pan histone deacetylase inhibition (HDAC inhibition). The investigational drug is being developed to address glioblastoma, an aggressive brain cancer that is very challenging to treat and for which there is no cure. Most patients do not survive more than 15 months with current treatment approaches.
The drug has the potential to serve as a first-line treatment for the nearly 14,000 people diagnosed with glioblastoma in the U.S. each year. Tinostamustine will be investigated in patients with newly diagnosed glioblastoma as an adjuvant therapy following standard treatment with surgery, chemotherapy and radiation, as well as in patients with recurrent disease.
Innovative Trial Design
GBM AGILE is a pioneering, international adaptive platform trial designed to accelerate the identification of effective treatments for glioblastoma. The trial is led by GCAR, a non-profit corporation, and is supported by a global network of oncologists, neuro-oncologists, clinicians, researchers, biostatisticians, and patient advocates.
Unlike traditional trials, GBM AGILE offers a seamless phase 2/3 design that supports a well-defined path for FDA registration. The platform allows for the simultaneous assessment of multiple investigational therapies against a common control arm, significantly reducing development time by adapting to emerging data and prioritizing promising new treatments.
Industry Perspectives
"We are pleased to partner with GCAR in GBM AGILE to further study tinostamustine, which has shown promise in early trials," said Dr. Julie Ducharme, Vice President and Chief Scientific Officer at Purdue. "GBM AGILE accelerates the clinical trial timeline to speed medicines to patients, which aligns with our mission to address serious, unmet medical needs."
Dr. Meredith Buxton, Chief Executive Officer/President of GCAR, emphasized the clinical urgency: "Glioblastoma is the deadliest brain cancer, and our mission is to find promising new treatments to improve overall survival as compared to standard treatments. We are now hard at work collaborating on the Investigational New Drug Application (IND) and Clinical Trial Application (CTA) submissions necessary to include tinostamustine in our innovative and patient-centric adaptive trial."
Regulatory Timeline
The study is anticipated to begin following IND submission and FDA approval of the protocol, with activation in Canada, Europe, and Australia to follow. The companies are currently collaborating on the necessary regulatory submissions to advance the trial initiation.
