Therapy Detail

Therapy Name H3B-8800
Therapy Description

H3B-8800 binds to the SF3B complex and modulates splicing events, preferentially kills splicesome mutant tumor cells (Blood 2016 128: 966)

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Drug Name Trade Name Synonyms Drug Classes Drug Description
H3B-8800 H3B-8800 binds to the SF3B complex and modulates splicing events, preferentially kills splicesome mutant tumor cells (Blood 2016 128: 966)
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
SF3B1 K700E chronic myeloid leukemia sensitive H3B-8800 Preclinical - Cell line xenograft Actionable In a preclinical study, H3B-8800 inhibited tumor growth in cell line xenograft models of chronic myeloid leukemia harboring SF3B1 K700E while had no effect in SF3B1 wild-type controls (Blood 2016 128:966). detail...
Unknown unknown chronic myelomonocytic leukemia not applicable H3B-8800 Preclinical - Pdx Actionable In a preclinical study, H3B-8800 treatment reduced tumor burden in patient-derived xenograft (PDX) models of chronic myelomonocytic leukemia harboring spliceosome mutations but not spliceosome wild-type models (Blood 2016 128:966). detail...
U2AF1 S34F lung cancer sensitive H3B-8800 Preclinical - Cell line xenograft Actionable In a preclinical study, H3B-8800 preferentially inhibited growth of lung cancer cells harboring U2AF1 S34F compared to U2AF1 wild-type cells in culture and in cell line xenograft models (Cancer Res 2017;77(13 Suppl):Abstract nr 1185). detail...
KMT2A-MLLT3 SRSF2 P95H acute myeloid leukemia sensitive H3B-8800 Preclinical Actionable In a preclinical study, H3B-8800 inhibited tumor growth in transplant animal models of mouse acute myeloid leukemia cells harboring SRSF2 P95H and KMT2A-MLLT3 (MLL-AF9) while had no effect in SRSF2 wild-type controls (Blood 2016 128:966). detail...
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...
SF3B1 R1074H Advanced Solid Tumor resistant H3B-8800 Preclinical - Cell culture Actionable In a preclinical study, transformed cells overexpressing SF3B1 R1074H were resistant to H3B-8800 in culture (Blood 2016 128:966). detail...
Clinical Trial Phase Therapies Title Recruitment Status
NCT02841540 Phase I H3B-8800 Phase 1 Trial to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Splicing Modulator H3B-8800 for Subjects With Myelodysplastic Syndromes, Acute Myeloid Leukemia, and Chronic Myelomonocytic Leukemia Recruiting