Investigational Drug Tinostamustine to be Included in GBM AGILE Clinical Trial for Glioblastoma

By: Purdue Pharma L.P. via Business Wire

September 17, 2025 at 09:00 AM EDT

Purdue Pharma L.P. (“Purdue”) has entered into an agreement to have its pipeline medication tinostamustine included 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).

Tinostamustine is a first-in-class, new chemical entity that combines two potentially synergistic mechanisms of action, bifunctional alkylating activity and pan histone deacetylase inhibition (or HDAC inhibition). Tinostamustine is under investigation in patients with glioblastoma, an aggressive brain cancer that is very challenging to treat and for which there is no cure.¹ Most patients do not survive more than 15 months with current treatment approaches.¹ Tinostamustine has the potential to be a first-line treatment. Nearly 14,000 people in the U.S. are diagnosed with glioblastoma 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.

GBM AGILE is a pioneering, international adaptive platform trial designed to accelerate the identification of effective treatments for glioblastoma. It 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. It 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.

“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, Purdue. “GBM AGILE accelerates the clinical trial timeline to speed medicines to patients, which aligns with our mission to address serious, unmet medical needs.”

“We are pleased to be evaluating tinostamustine in GBM AGILE,” said Dr. Meredith Buxton, Chief Executive Officer/President, GCAR. “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.”

The study is anticipated to begin following IND submission and FDA approval of the protocol with activation in Canada, Europe, and Australia to follow.

*Tinostamustine is an investigational agent. This announcement discusses its use in clinical development and is not intended to convey conclusions about efficacy or safety. There is no guarantee that tinostamustine will successfully complete development or receive regulatory approval.

Reference

Glioblastoma (GBM). https://my.clevelandclinic.org/health/diseases/17032-glioblastoma. Accessed May 23, 2025.

About Purdue Pharma L.P.

Purdue Pharma and its subsidiaries develop, manufacture and market medications to meet the evolving needs of healthcare professionals, patients, and caregivers. Purdue and its subsidiaries focus on balancing innovative science with clinically effective, compassionate care. The Company’s goals are to serve patients who rely on its medicines, pursue public health initiatives intended to help abate the opioid crisis, advance its pipeline of branded and generic medications, and introduce medicines that will help save and improve lives.

About Imbrium Therapeutics L.P.

Imbrium is a clinical-stage biopharmaceutical company dedicated to advancing medical science through the development of important new pharmacologic and biologic therapeutics. We are pursuing oncology chemotherapeutics, treatments for disorders of the central nervous system, and non-opioid approaches to the management of pain. As a subsidiary of Purdue Pharma L.P., Imbrium strives to develop and bring to market new medicines that serve the unmet needs of patients, physicians and health systems worldwide. We have built a robust and diversified pipeline of investigational drug candidates, and we actively collaborate with industry and academic partners to identify and advance future impactful medicines. For more information, please visit www.imbriumthera.com.