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Table 1 CSF1/CSF1R inhibitors as monotherapy in current clinical development

From: Colony-stimulating factor 1 receptor (CSF1R) inhibitors in cancer therapy

Class Target Compound Clinical Phase Sponsor Indication ClinicalTrials.gov identifier Status/Results Reference
Small molecules CSF1R (and cKIT, Flt3) Pexidartinib (PLX3397, PLX108-01) 2 The Christie NHS Foundation Trust KIT-mutated advanced acral and mucosal melanoma NCT02071940 Ongoing -
1/2 Plexxikon Unresectable or metastatic KIT-mutated melanoma NCT02975700 Ongoing -
2 Plexxikon Advanced castration-resistant prostate cancer with bone metastasis and high circulating tumor cell counts NCT01499043 Not yet reported -
2 Plexxikon Recurrent GBM NCT01349036 ORR: 0%
CBR: 7/38 (18%)
[14, 26]
1/2 NCI Refractory leukemias and refractory solid tumors, including neurofibromatosis type 1-associated plexiform neurofibromas NCT02390752 Ongoing -
2 Plexxikon Relapsed or refractory cHL NCT01217229 ORR: 1/20 (5%) [15]
1/2 Plexxikon Relapsed or refractory FLT3-ITD-positive acute myeloid leukemia NCT01349049 Ongoing -
1 Plexxikon Advanced, incurable, solid tumors in which the target kinases are linked to disease pathophysiology NCT01004861 Ongoing -
CSF1R (and Trk) PLX7486 1 Plexxikon Solid tumors NCT01804530 Ongoing -
CSF1R ARRY-382 1 Array BioPharma Solid tumors NCT01316822 ORR: 0%
CBR: 4/26 (15%)
[17]
CSF1R JNJ-40346527 1/2 Johnson& Johnson cHL NCT01572519 ORR: 1/21 (5%)
CBR: 11/21 (52%)
[16]
CSF1R BLZ945 1/2 Novartis Solid tumors NCT02829723 Ongoing -
Monoclonal antibodies CSF1R Emactuzumab (RG7155) 1 Roche Solid tumors NCT01494688 PMR: 5/44 (11%)
ORR: 0%
CBR: 6/40 (24%)
[18]
CSF1R AMG820 1 Amgen Solid tumors NCT01444404 ORR: 1/25 (4%)
CBR: 6/25 (24%)
[19]
CSF1R IMC-CS4 (LY3022855) 1 Eli Lilly Solid tumors NCT01346358 Ongoing -
1 Eli Lilly Breast and prostate cancer NCT02265536 Ongoing -
CSF1 MCS110 1/2 Novartis Prostate cancer NCT00757757 Terminated -
  1. CBR clinical benefit rate, cHL classical Hodgkin lymphoma, CSF1 colony-stimulating factor 1, CSF1R colony-stimulating factor 1 receptor, GBM glioblastoma, NCI National Cancer Institute, NHS National Health Service, ORR objective response rate, PMR partial metabolic response