The U.S. Food and Drug Administration has awarded an orphan grant to ONC201, a selective antagonist of DRD2, in a multiple myeloma clinical trial.1

ONC201 has demonstrated excellent anticancer activity in various preclinical models of refractory multiple myeloma, and it has shown manageable toxicity and early signals of efficacy in several cancer types, including non-Hodgkin lymphoma.

The orally bioavailable inhibitor of Akt and ERK has also exhibited a therapeutic pharmacokinetic profile and induction of pharmacodynamic markers.

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ONC201 activates the integrated stress response (ISR) using unique triggers, leading to the upregulation of a host of proteins that induce apoptosis and inhibit cancer growth. Multiple myeloma cells are especially sensitive to ISR activation, which is also a key mechanism of action of proteasome inhibitors like bortezomib.

“While significant progress has been made in the field of multiple myeloma and the treatments have meaningfully improved, patients with resistant disease need additional options,” said Bart Barlogie, MD, Director of Research of the Myeloma Program of the Tisch Cancer Institute of the Icahn School of Medicine at Mount Sinai in New York. “It is gratifying to see introductions of new classes of compounds, such as ONC201 that represents the first clinical stage drug of the imipridone family that offer disruptive approaches in how to tackle this challenging disease.”

RELATED: In Search of the Right Treatment Combinations for Multiple Myeloma

The grant awarded totals $1.7 million to support the development of ONC201 in patients with relapsed/refractory multiple myeloma in a phase 2 program.


  1. Oncoceutics awarded FDA orphan grant for ONC201 multiple myeloma trial. Oncoceutics website. Updated August 16, 2016. Accessed August 17, 2016.