| Hazard Information | Back Directory | [Uses]
H3B-8800 is a potent and orally active SF3B splicing modulator. H3B-8800 direct interaction with the SF3b complex and shows anti-cancer activity. H3B-8800 has the potential for the research of acute myeloid leukemia (AML) with SF3B1 mutant[1]. | [in vivo]
H3B-8800 (2, 4 mg/kg; p.o.; daily) shows anti tumor activity in AML derived xenografts (PDXs) with a mutation in SF3B1[1]. | Animal Model: | 6-8 weeks, female NSG or CB17-SCID mice (K562 cells with SF3B1WT or SF3B1K700E)[1] | | Dosage: | 2, 4 mg/kg | | Administration: | P.o.; daily | | Result: | Resulted in antileukemic efficacy and splicing modulation in mice bearing AML patient-derived xenografts (PDXs) with a mutation in SF3B1 but had little effect in mice bearing SF3B1WT AML PDXs, significantly reduced leukemic burden relative in SF3B1K700E PDX. |
| [References]
[1] Seiler M, et al. H3B-8800, an orally available small-molecule splicing modulator, induces lethality in spliceosome-mutant cancers. Nat Med. 2018 May;24(4):497-504. DOI:10.1038/nm.4493 |
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