ONC201 is the first of a new group of cancer therapies called imipridones that have a novel mechanism of action. Unlike other oncology drugs, ONC201 and other imipridones selectively target G protein-coupled receptors (GPCRs) that are dysregulated in cancers.

ONC201 is an orally active dopamine receptor D2 (DRD2) antagonist that is well-tolerated and has been shown in Phase I and II clinical trials to be active in specific advanced cancers.

ONC201 has shown efficacy in gliomas with the H3 K27M mutation. About 10% of adults with glioma have the H3 K27M mutation. About 50-60% of children with high-grade glioma (and 80-90% of children with Diffuse Intrinsic Pontine Gliomas, or DIPG) have the H3 K27M mutation.

Phase I/II clinical trials of ONC201 in glioma patients with the H3 K27M mutation are underway at several locations in the U.S.

Patients should be tested for the H3 K27M mutation and, if they meet the eligibility criteria, should be enrolled in these clinical trials.

The test is commercially available and easily accessible either through immunohistochemistry (IHC) staining or gene sequencing.

If your patient does not have the H3 K27M mutation or the tumor has not been biopsied, check the eligibility for this ONC201 clinical trial: NCT02525692.


ONC201 is being evaluated for efficacy in several cancer types and in trials across the United States.

These trials include treatment for multiple myeloma, leukemia, lymphoma, neuroendocrine tumors (e.g. DSRCT, adrenal tumors), endometrial cancer and other solid tumors.


Find a clinical trial for your patient: patientwing.com/oncoceutics

Contact us: info@oncoceutics.com

More about ONC201’s mechanism of action: oncoceutics.com/therapeutic-approach

More about our publications: oncoceutics.com/publications

More about ONC201 safety (Phase I experience): First-in-human Clinical Trial of Oral ONC201 in Patients with Refractory Solid Tumors

More about ONC201 in gliomas: A phase 2 study of the first imipridone ONC201, a selective DRD2 antagonist for oncology, in recurrent glioblastoma