Table 1

Hh pathway inhibitors and clinical trials in hematologic malignancies

NameActionProducerActive hematologic clinical trialTrial number*
Cyclopamine SMO antagonist Generic Nonclinical NA 
LDE225 SMO antagonist Novartis Phase 1: CML, in combination with nilotinib NCT1456676 
LEQ506 SMO antagonist Novartis Solid tumors only (phase 1 and 2) NA 
GDC-0449 SMO antagonist Genentech Phase 1b: myeloma in first remission or first relapse after ASCT NCT01330173 
BMS-833923 SMO antagonist BMS Phase 1: CML, in combination with dasatinib NCT 01218477 
   Phase 1b: myeloma, in combination with lenalidomide with dexamethasone or bortezomib with dexamethasone NCT00884546 
IPI926 SMO antagonist Infinity Phase 2: myelofibrosis NCT01371617 
PF-04449913 SMO antagonist Pfizer Phase 1: myeloid malignancies, including CML, in combination with dasatinib or bosutinib NCT00953758 
GANT61 Direct GLI inhibition Generic Nonclinical NA 
NameActionProducerActive hematologic clinical trialTrial number*
Cyclopamine SMO antagonist Generic Nonclinical NA 
LDE225 SMO antagonist Novartis Phase 1: CML, in combination with nilotinib NCT1456676 
LEQ506 SMO antagonist Novartis Solid tumors only (phase 1 and 2) NA 
GDC-0449 SMO antagonist Genentech Phase 1b: myeloma in first remission or first relapse after ASCT NCT01330173 
BMS-833923 SMO antagonist BMS Phase 1: CML, in combination with dasatinib NCT 01218477 
   Phase 1b: myeloma, in combination with lenalidomide with dexamethasone or bortezomib with dexamethasone NCT00884546 
IPI926 SMO antagonist Infinity Phase 2: myelofibrosis NCT01371617 
PF-04449913 SMO antagonist Pfizer Phase 1: myeloid malignancies, including CML, in combination with dasatinib or bosutinib NCT00953758 
GANT61 Direct GLI inhibition Generic Nonclinical NA 

NA indicates not applicable.

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