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National Brain Tumor Society Backs Oncoceutics' Phase II Trial for Subset of High-Grade Gliomas

NEW YORK – Drugmaker Oncoceutics said Monday that the National Brain Tumor Society will provide $200,000 to support an upcoming Phase II trial of ONC201 in a molecularly defined subset of glioma patients.

ONC201 is the first imipridone dopamine receptor D2 antagonist (DRD2) that treats high-grade glioma by selectively targeting G protein-coupled receptors. The Phase II trial will evaluate the efficacy of ONC201 in patients who have a recurrent form of high-grade glioma with low epidermal growth factor receptor expression, which is associated with elevated DRD2 expression and ONC201 sensitivity, according to the drugmaker.

Previously, ONC201 has shown encouraging activity in adults with high-grade gliomas harboring the H3 K27M mutation and has obtained fast-track designation from the FDA. 

In preclinical studies using tumor tissue from The Cancer Genome Atlas and patient-derived xenograft models of glioblastoma, researchers identified an additional subset of high-grade gliomas that exhibit low EGFR expression and elevated DRD2 expression. These glioma cells are sensitive to ONC201-mediated DRD2 inhibition both in vitro and in vivo, which halts tumor growth.

According to Oncoceutics, preliminary data from the clinical trial patients with recurrent glioblastomas with low EGFR expression and high DRD2 expression suggest that when treated with ONC201, these biomarkers may result in improved survival endpoints.

"Funding support for this upcoming trial of ONC201 stems from our interest in understanding the full potential of this compelling investigational drug, which has shown early signs of efficacy in both adult and pediatric high-grade glioma patients whose tumors harbor H3K27M mutation, to an additional subpopulation who can be identified through biomarker analysis," National Brain Tumor Society Chief Scientific Officer Kirk Tanner said in a statement. "In doing so, we hope to extend the number of patients who could conceivably benefit from ONC201."

Oncoceutics will also continue to be evaluate ONC201 in multiple, parallel clinical trials of adult and pediatric high-grade glioma patients whose tumors harbor H3K27M mutation, including diffuse intrinsic pontine glioma patients.