Knoa Pharma and GCAR Announce First Newly Diagnosed Glioblastoma Patient Randomized in Phase 2/3 GBM AGILE Evaluation of Tinostamustine and First Recurrent Patient Dosed

Tinostamustine is being studied in newly diagnosed glioblastoma patients who often face limited treatment options

STAMFORD, Conn., June 24, 2026 /PRNewswire/ -- Knoa Pharma LLC ("Knoa Pharma"), a public health-focused pharmaceutical company, and the Global Coalition for Adaptive Research ("GCAR") announced 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

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.

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 Knoa Pharma LLC
Knoa Pharma makes important medicines safely and responsibly, while innovating to benefit patients and the public health. The company is dedicated to safely distributing medicines that are critical to patient care, enhancing its robust generics portfolio, addressing unmet medical needs, and supporting initiatives to address the opioid crisis. Our public health minded mission enables us to expand access to lifesaving overdose reversal medicines and affordable treatments for opioid use disorder - at no profit. Owned by a not-for-profit foundation, everything we do is centered on improving public health, saving lives, supporting recovery, and bringing forward medicines that make a meaningful difference. Learn more at www.knoapharma.com.

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.

References

1. Glioblastoma (GBM). https://my.clevelandclinic.org/health/diseases/17032-glioblastoma. Accessed May 27, 2026.
2. Glioblastoma Brain Tumor Information. (n.d.). Glioblastoma Foundation. https://glioblastomafoundation.org/patients/glioblastoma-brain-tumor-information. Accessed May 27, 2026.
3. Liu, D., Yang, T., Ma, W., Wang, Y. (2022). Clinical strategies to manage adult glioblastoma patients without MGMT hypermethylation. Journal of Cancer, 13(1), 354-363. https://doi.org/10.7150/jca.63595. Accessed May 27, 2026.

SOURCE Knoa Pharma