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The FDA awarded an orphan grant to evaluate ONC201, a selective antagonist of DRD2 that belongs to the superfamily of G protein-coupled receptors, in a multiple myeloma clinical trial. The award totals $1.7 million in funding to support a phase II program with ONC201 in relapsed/refractory multiple myeloma.

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ONC201 has shown anti-cancer activity in several challenging preclinical models of refractory multiple myeloma. In clinical trials, ONC201 has exhibited exceptional safety, a therapeutic pharmacokinetic profile, induction of pharmacodynamic markers, and early efficacy signals in a number of different cancer types, including non-Hodgkin Lymphoma.


As previously reported in companion research articles published in Science Signaling, ONC201 uses unique triggers to activate the integrated stress response (ISR), which upregulates a host of proteins that induce tumor cell death and inhibit the synthesis of proteins that are key for cancer growth. Multiple myeloma cells are particularly sensitive to ISR activation, which is also a key mechanism of action of proteasome inhibitors that are approved for the treatment of multiple myeloma.