CSF1/CSF1R inhibitors as monotherapy in current clinical development

ClassTargetCompoundClinical PhaseSponsorIndicationClinicalTrials.gov identifierStatus/ResultsReference
Small moleculesCSF1R (and cKIT, Flt3)Pexidartinib (PLX3397, PLX108-01)2The Christie NHS Foundation TrustKIT-mutated advanced acral and mucosal melanomaNCT02071940Ongoing-
1/2PlexxikonUnresectable or metastatic KIT-mutated melanomaNCT02975700Ongoing-
2PlexxikonAdvanced castration-resistant prostate cancer with bone metastasis and high circulating tumor cell countsNCT01499043Not yet reported-
2PlexxikonRecurrent GBMNCT01349036ORR: 0%CBR: 7/38 (18%)[14, 26]
1/2NCIRefractory leukemias and refractory solid tumors, including neurofibromatosis type 1-associated plexiform neurofibromasNCT02390752Ongoing-
2PlexxikonRelapsed or refractory cHLNCT01217229ORR: 1/20 (5%)[15]
1/2PlexxikonRelapsed or refractory FLT3-ITD-positive acute myeloid leukemiaNCT01349049Ongoing-
1PlexxikonAdvanced, incurable, solid tumors in which the target kinases are linked to disease pathophysiologyNCT01004861Ongoing-
CSF1R (and Trk)PLX74861PlexxikonSolid tumorsNCT01804530Ongoing-
CSF1RARRY-3821Array BioPharmaSolid tumorsNCT01316822ORR: 0%CBR: 4/26 (15%)[17]
CSF1RJNJ-403465271/2Johnson& JohnsoncHLNCT01572519ORR: 1/21 (5%)CBR: 11/21 (52%)[16]
CSF1RBLZ9451/2NovartisSolid tumorsNCT02829723Ongoing-
Monoclonal antibodiesCSF1REmactuzumab (RG7155)1RocheSolid tumorsNCT01494688PMR: 5/44 (11%)ORR: 0%CBR: 6/40 (24%)[18]
CSF1RAMG8201AmgenSolid tumorsNCT01444404ORR: 1/25 (4%)CBR: 6/25 (24%)[19]
CSF1RIMC-CS4 (LY3022855)1Eli LillySolid tumorsNCT01346358Ongoing-
1Eli LillyBreast and prostate cancerNCT02265536Ongoing-
CSF1MCS1101/2NovartisProstate cancerNCT00757757Terminated-

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