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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