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

LARKSPUR, Calif. and STAMFORD, Conn., April 8, 2026 /PRNewswire/ -- The Global Coalition for Adaptive Research (GCAR) and Purdue Pharma L.P. (Purdue) today announced the activation of investigational tinostamustine in GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment – NCT03970447), a pioneering, international adaptive platform trial designed to accelerate the identification of effective treatments for glioblastoma (GBM). Tinostamustine will be evaluated for the treatment of adult patients with newly diagnosed GBM across GBM AGILE. It will also be studied in a cohort of patients with recurrent GBM at select GBM AGILE sites.

GBM is the most common and aggressive form of primary brain cancer. Treatment options remain limited and patient outcomes have seen minimal improvement over the past several decades.

GBM AGILE is a seamless phase 2/3 study conducted under a master protocol enabling multiple therapies or combinations of therapies from different pharmaceutical companies to be evaluated simultaneously against a shared 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 U.S. FDA and other health authorities. Since its launch in 2019, GBM AGILE has evaluated multiple investigational therapies and has screened over 2,600 patients at approximately 60 trial locations in six countries.

Dr. John de Groot, Neuro-Oncology Division Chief, Department of Neurosurgery, University of California, San Francisco and Dr. Shiao-Pei Weathers, Brain Tumor Section Chief, Department of Neuro-Oncology, University of Texas MD Anderson Cancer Center, are serving as the Principal Investigators for tinostamustine'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 remains one of the most aggressive and difficult-to-treat cancers we encounter in clinical practice," said Dr. John de Groot. "There is a pressing need to explore novel mechanisms of action in well-designed studies. GBM AGILE's adaptive platform design allows us to rigorously evaluate promising therapies like tinostamustine while generating high-quality data efficiently. The addition of tinostamustine strengthens our ability to investigate new approaches that may improve outcomes for patients with GBM."

"Despite advances in oncology, outcomes for patients with glioblastoma remain poor," said Dr. Shiao-Pei Weathers. "Participating in a global study like GBM AGILE gives patients access to innovative investigational therapies that would otherwise not be available outside of a clinical trial. The inclusion of tinostamustine offers hope and reflects the ongoing commitment to expanding meaningful treatment opportunities for this devastating disease."

Tinostamustine is a first-in-class, new chemical entity that combines two potentially synergistic mechanisms of action: bifunctional DNA alkylating activity, which triggers apoptosis, and pan histone deacetylase inhibition (or HDAC inhibition), which in addition to improving alkylating agent access to DNA, has been shown to disrupt oncogenic signaling pathways and enhance immune recognition of tumor cells. This dual mechanism may be particularly relevant in aggressive and treatment-resistant cancers like GBM, where both genomic instability and epigenetic dysregulation drive disease progression. Tinostamustine has the potential to be a first-line treatment and is being investigated in patients with newly diagnosed GBM as an adjuvant therapy following standard treatment with surgery, chemotherapy and radiation, as well as in a limited cohort for patients in whom the disease has recurred following initial treatment.

"We are pleased to initiate the evaluation of tinostamustine in GBM AGILE, an innovative adaptive trial designed to efficiently determine whether promising therapies like tinostamustine can provide meaningful benefit to patients with glioblastoma," said Dr. Julie Ducharme, Vice President and Chief Scientific Officer, Purdue. "Encouraging findings from prior clinical studies support continued investigation, and we look forward to advancing the development of tinostamustine for this devastating disease, where significant unmet need remains."

"Activation of the first trial sites represents the culmination of years of hard work and dedication, scientific rigor, and partnership," said Dr. Craig Landau, President and CEO, Purdue. "It underscores our determination to apply sustained scientific and clinical investment to help deliver new treatments patients urgently need."

"At GCAR, our mission is to rethink how therapies are developed for aggressive cancers like glioblastoma," said Dr. Meredith Buxton, CEO and President, GCAR. "Through master protocols and adaptive platform trials, we aim to streamline evaluation and accelerate decision-making. Our collaboration to evaluate tinostamustine represents an important step toward rapidly advancing new treatments in GBM AGILE and bringing new hope to patients."

This press release discusses investigational uses of an agent in development and is not intended to convey conclusions about efficacy or safety. There is no guarantee that tinostamustine will successfully complete development or gain FDA approval.

About Global Coalition for Adaptive Research (GCAR)
Global Coalition for Adaptive Research (GCAR) is a 501(c)(3) non-profit corporation pioneering collaborative, innovative trials to speed the discovery and development of cures. GCAR unites physicians, clinical researchers, advocacy and philanthropic organizations, biotech/pharma companies, health authorities, and other key stakeholders in healthcare to expedite research. 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.

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.

Purdue's subsidiary Imbrium will engage with investors and potential partners at the Neuroscience Innovation Partnering & Licensing Summit (April 28–29, Boston) where the pipeline, including tinostamustine, will be highlighted.

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SOURCE Global Coalition for Adaptive Research