National Brain Tumor Society to Provide Funding for ONC201 Trial in EGFR-Independent High-Grade Gliomas

Philadelphia, PA (January 27, 2020) – Oncoceutics announced that the National Brain Tumor Society (NBTS) has committed to providing more than $200,000 in funding, with the potential for multiple years, to support a Phase II clinical trial of ONC201, the first imipridone dopamine receptor D2 antagonist (DRD2), in a molecular subset of high grade gliomas.

The future clinical trial will evaluate the efficacy of single agent ONC201 in patients who have a recurrent form of high-grade glioma that exhibits low expression of epidermal growth factor receptor (EGFR), which is associated with elevated DRD2 expression and ONC201 sensitivity. The trial will be led by Clark C. Chen, MD, PhD, Lyle French Chair in Neurosurgery and Head of the Department of Neurosurgery, at the University of Minnesota Medical School who is co-PI of the trial along with Joshua Allen, PhD, Senior Vice President of Research and Development at Oncoceutics. Each year, approximately 5,000 patients are affected by this subtype of gliomas in the United States for which there are no effective therapies following first-line chemoradiation.

The project builds on previous results demonstrating the safety and efficacy of ONC201 in patients with high grade gliomas, in particular those with H3 K27M-mutant gliomas. Preclinical studies, led by Dr. Chen, using tumor tissue associated with The Cancer Genome Atlas (TCGA) and patient-derived xenograft (PDX) models of glioblastoma (GBM) have identified an additional subset of high-grade gliomas that exhibit low EGFR expression and, conversely, elevated DRD2 expression. These glioma cells are highly sensitive to ONC201 treatment both in vitro and in vivo. Preliminary data from clinical trial patients with recurrent GBM that harbor these characteristics suggest that treatment with ONC201 may translate into improved survival endpoints relative to patients who do not express these biomarkers.

“NBTS is committed to rapidly and directly improving treatments for brain tumor patients,” said Kirk Tanner, PhD, Chief Scientific Officer, National Brain Tumor Society. “This clinical trial aims to extend the clinical utility of ONC201 to high-grade glioma patients beyond those that harbor the H3 K27M mutation where it has shown compelling single agent efficacy.”

"It is gratifying to see the work done over the past seven years in my laboratory translate into a pivotal clinical trial. I am optimistic that the insights gained from this trial will contribute to improving the lives of patients afflicted with high-grade gliomas," said Clark C. Chen, MD, PhD, Lyle French Chair in Neurosurgery and Head of the Department of Neurosurgery, at the University of Minnesota Medical School.

“The NBTS’s critical support for this project represents an important milestone to further inform patient selection for clinical trials with ONC201 based on biomarkers that expand its current development scope. I am delighted that Dr. Chen’s collaboration with our team has resulted in the convincing rationale to evaluate a new biomarker approach that will hopefully result in similarly positive outcomes that we are seeing in patients with H3 K27M-mutant high grade gliomas,” said Wolfgang Oster, MD, PhD, CEO and Chairman of Oncoceutics.

About Oncoceutics

Oncoceutics, Inc. is a clinical-stage drug discovery and development company with a novel class of compounds, called imipridones, that selectively target G protein-coupled receptors for oncology. The first lead compound to emerge from this program is ONC201, an orally active small molecule DRD2 antagonist. The company is supported by grants from NCI, FDA, The Musella Foundation and a series of private and public partnerships. Visit for more information.

About NBTS

National Brain Tumor Society invests in, mobilizes, and unites the brain tumor community to discover a cure, deliver effective treatments, and advocate for patients and care partners. We are the largest patient advocacy non-profit solely dedicated to the brain tumor community and a leading thought leader in the neuro-oncology field. Headquartered in Newton, Massachusetts, our organization raises funds to invest in accelerating brain tumor treatments, prepare the community to navigate their unique brain tumor experience, and convene stakeholders while changing public policy to improve the lives and survival of brain tumor patients. Visit us at


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