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FF-10501-01

Development stage: Phase 2
Lead developer: Fujifilm Holdings
Modality: Small Molecules
Administration: Oral

Overview

FF-10501-01 is an orally bioavailable, small molecule inhibitor of inosine monophosphate dehydrogenase (IMPDH), developed as an antineoplastic agent. By competitively inhibiting IMPDH, it blocks the conversion of inosine monophosphate to xanthosine monophosphate, thereby suppressing de novo guanine nucleotide synthesis. This leads to anti-proliferative and pro-apoptotic effects in hematologic malignancies such as acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), including cases resistant to hypomethylating agents. The drug has shown clinical activity in early-phase trials but development was limited by adverse events such as mucositis[1][2][3][4][5].

Other names: IMPDH inhibitor FF-10501

Targets

IMPDH (Inosine-5'-monophosphate dehydrogenase 1)

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