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IO-202 (anti-LILRB4) receives FDA orphan drug designation for the treatment of R/R AML

By Sumayya Khan

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Nov 12, 2020


On November 10, 2020, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to IO-202 for the treatment of relapsed/refractory (R/R) acute myeloid leukemia (AML). IO-202 is a first-in-class monoclonal antibody that blocks the signaling of the immune inhibitory receptor LILRB4, thus activating T-cell cytotoxicity against leukemia cells.1

IO-202 is currently being investigated in a multicenter, open-label, phase I dose escalation and expansion study (NCT04372433), which was previously described on the AML Hub when reporting on the FDA clearance of IO-202 for investigation as a new drug in AML and chronic myelomonocytic leukemia. The novel mechanism of action of IO-202 and this ongoing trial are due to be presented as a trial-in-progress poster at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, in December.1

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