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Molecular Profile SF3B1 K700E
Therapy H3B-8800
Indication/Tumor Type acute myeloid leukemia
Response Type sensitive

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
SF3B1 K700E acute myeloid leukemia sensitive H3B-8800 Preclinical - Pdx Actionable In a preclinical study, H3B-8800 inhibited aberrant splicing and tumor growth in a cell line xenograft and a patient-derived xenograft (PDX) model of acute myeloid leukemia harboring SF3B1 K700E (PMID: 29457796). 29457796
SF3B1 K700E acute myeloid leukemia sensitive H3B-8800 Preclinical - Cell culture Actionable In a preclinical study, H3B-8800 preferentially inhibited growth of mouse acute myeloid leukemia cells harboring SF3B1 K700E compared to SF3B1 wild-type cells in culture (Blood 2016 128:966). detail...
PubMed Id Reference Title Details
H3B-8800, an Orally Bioavailable Modulator of the SF3b Complex, Shows Efficacy in Spliceosome-Mutant Myeloid Malignancies Full reference...
(29457796) H3B-8800, an orally available small-molecule splicing modulator, induces lethality in spliceosome-mutant cancers. Full reference...