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On November 4, 2021, Chimerix announced that ONC201, an investigational therapy with a dual mechanism of action, has achieved positive top-line results in clinical trials for the treatment of recurrent gliomas
ONC201 targets DRD2 and ClpP at the same time, blocking DRD2 can inhibit the RAS signaling pathway, and activating ClpP can promote the degradation of proteins in the mitochondria of cells that are related to the survival of cancer cells
▲The mechanism of ONC201 (picture source: reference [2])
The 50 patients whose efficacy can be evaluated in this trial have diffuse midline gliomas, which carry the H3 K27M mutation.
In terms of safety, the complete safety data collection and analysis of the cohort are in progress, and one serious adverse event was attributed to ONC201
"For these patients who do not have effective systemic treatment options, these data are exciting