On June 24, 2026 Knoa Pharma LLC ("Knoa Pharma"), a public health-focused pharmaceutical company, and the Global Coalition for Adaptive Research ("GCAR") reported that the first patient was randomized to the tinostamustine arm on GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment – NCT03970447) in early April. In addition, the first patient in recurrent setting received the first dose of tinostamustine. Glioblastoma (GBM) is an aggressive brain cancer that is challenging to treat and currently has no cure.1
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Tinostamustine is an investigational, first-in-class chemical entity that combines two potentially synergistic mechanisms of action, bifunctional alkylating activity and pan histone deacetylase inhibition (or HDAC inhibition). 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.
Prior clinical research has evaluated tinostamustine in patients with MGMT promoter-unmethylated glioblastoma (uMGMT GBM). Patients with uMGMT GBM are associated with poor prognosis and limited treatment options. Nearly 15,000 people in the U.S. are diagnosed with GBM each year,2 and 60% of those patients have uMGMT GBM.3
The trial, known as 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.
"GBM AGILE was designed to accelerate the development of treatment options, which is why we’re honored to work with GCAR to determine whether tinostamustine could provide meaningful benefit to patients," said Dr. Julie Ducharme, Vice President and Chief Scientific Officer, Knoa Pharma. "Encouraging findings from prior clinical studies support continued investigation."
"Glioblastoma patient outcomes have seen minimal improvement over the past several decades," said Dr. Meredith Buxton, CEO and President, GCAR. "The first patients randomized and dosed with tinostamustine marks an important milestone as we work to advance new treatment options and bring new hope to patients. By leveraging an adaptive platform design, we can assess promising treatments more rapidly than traditional clinical trials and make smarter, data-driven decisions sooner. GBM AGILE’s ability to evaluate therapies in newly diagnosed patients while simultaneously identifying signals in recurrent disease provides a powerful opportunity to accelerate the development of potential new options for patients with glioblastoma."
In an earlier Phase 1 trial of tinostamustine in patients with uMGMT GBM, results showed tinostamustine to be tolerable at doses of 80 to 100 mg/m², with manageable side effects. While the Phase 1 study was not designed to demonstrate efficacy, exploratory analyses of progression-free and overall survival outcomes showed encouraging signals of clinical activity.
(Press release, Global Coalition for Adaptive Research, JUN 24, 2026, View Source [SID1234668939])