ONC201 is a highly selective antagonist of dopamine receptor D2 (DRD2) and ClpP agonist that is able to penetrate the blood-brain-barrier effectively. ONC201 engages proven anti-cancer pathways that lead to apoptosis in cancer cells. ONC201 is currently in a pivotal trial for H3 K27M-mutant gliomas and is in Phase II trials in a variety of oncology indications, such as neuroendocrine tumors, endometrial cancer, and acute myeloid leukemia. Based on its lead indication, FDA granted to ONC201: Fast Track Designation for the treatment of adult recurrent H3 K27M-mutant high-grade glioma, Orphan Drug Designation for the indication of glioblastoma and H3 K27M-mutant glioma, and Rare Pediatric Disease Designation for treatment of H3 K27M-mutant glioma.
Based on clinical results indicating that ONC201 may be effective against gliomas with the H3 K27M mutation, Oncoceutics has launched Phase II clinical trials targeting H3 K27M-mutant gliomas.