Galeterone

Summary

Galeterone (CHEMBL2105738) is a phase-3 clinical-stage small molecule targeting CYP17A1 and AR; indicated across 2 conditions including malignant pancreatic neoplasm; with CIViC clinical evidence for 1 variant-indication association (e.g. AR AR-V7 in prostate cancer).

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 2 (CYP17A1, AR)
• Indications: 2 conditions
• Clinical trials: 5
• Precision-oncology evidence (CIViC): 1 variant–indication association
• Chemistry: 388.5 Da · C26H32N2O

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2105738
Name	Galeterone
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	11188409
Molecular formula	C26H32N2O
Molecular weight	388.5
InChIKey	PAFKTGFSEFKSQG-PAASFTFBSA-N

SMILES: C[C@]12CC[C@@H](CC1=CC[C@@H]3[C@@H]2CC[C@]4([C@H]3CC=C4N5C=NC6=CC=CC=C65)C)O

IUPAC name: (3S,8R,9S,10R,13S,14S)-17-(benzimidazol-1-yl)-10,13-dimethyl-2,3,4,7,8,9,11,12,14,15-decahydro-1H-cyclopenta[a]phenanthren-3-ol

Also known as: Galeterona, Galeterone, TOK 001, TOK-001, VN 124, VN-124, VN/124, GALETERONE

Patent coverage: 380 distinct patent families (971 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 805 (83%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
CYP17A1	CYP17A1	Inhibition	6.52	0.1%	P05093
AR	Androgen receptor	Antagonist	6.42		P10275

Broader ChEMBL bioactivity targets: 3 (assay-derived). Sample: Androgen receptor, Steroid 21-hydroxylase, Steroid 17-alpha-hydroxylase/17,20 lyase.

Bioactivity

ChEMBL activities: 17 potent at pChembl ≥ 5 of 19 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
CYP17A1	7.92	IC50	12.16	nM	CHEMBL_ACT_25020219
CYP17A1	7.8	IC50	16	nM	CHEMBL_ACT_24747416
CYP17A1	7.8	IC50	16	nM	CHEMBL_ACT_25514412
CYP17A1	7.55	IC50	28.1	nM	CHEMBL_ACT_18505294
CYP21A2	7.11	IC50	77.2	nM	CHEMBL_ACT_18505283
CYP17A1	6.85	IC50	140	nM	CHEMBL_ACT_19474739
CYP21A2	6.61	IC50	248	nM	CHEMBL_ACT_18710951
CYP21A2	6.61	IC50	248	nM	CHEMBL_ACT_27120059
CYP17A1	6.55	IC50	282	nM	CHEMBL_ACT_18710925
CYP17A1	6.55	IC50	282	nM	CHEMBL_ACT_27120017
CYP17A1	6.52	IC50	300	nM	CHEMBL_ACT_15216601
AR	6.39	EC50	405	nM	CHEMBL_ACT_15216603
AR	6.17	EC50	670	nM	CHEMBL_ACT_13344975
CYP17A1	6.12	IC50	752	nM	CHEMBL_ACT_13344922
AR	6.07	EC50	845	nM	CHEMBL_ACT_15216602
AR	5.96	IC50	1108	nM	CHEMBL_ACT_24747427
CYP17A1	5.45	IC50	3548	nM	CHEMBL_ACT_25020218

Target pathways

Aggregated over 2 target gene(s): CYP17A1, AR.

Top Reactome pathways

26 total, by targets touching each:

Pathway	Targets	Genes
Signal Transduction	1	AR
Androgen biosynthesis	1	CYP17A1
Glucocorticoid biosynthesis	1	CYP17A1
Signaling by Rho GTPases	1	AR
RHO GTPase Effectors	1	AR
Generic Transcription Pathway	1	AR
Cellular responses to stress	1	AR
SUMOylation	1	AR
SUMO E3 ligases SUMOylate target proteins	1	AR
HSP90 chaperone cycle for steroid hormone receptors (SHR) in the presence of ligand	1	AR
Nuclear Receptor transcription pathway	1	AR
Metabolism of proteins	1	AR
SUMOylation of intracellular receptors	1	AR
Defective CYP17A1 causes AH5	1	CYP17A1
RHO GTPases activate PKNs	1	AR
Activated PKN1 stimulates transcription of AR (androgen receptor) regulated genes KLK2 and KLK3	1	AR
Deubiquitination	1	AR
Ub-specific processing proteases	1	AR
Post-translational protein modification	1	AR
RNA Polymerase II Transcription	1	AR
Gene expression (Transcription)	1	AR
Transcriptional regulation by RUNX2	1	AR
RUNX2 regulates osteoblast differentiation	1	AR
RUNX2 regulates bone development	1	AR
Cellular responses to stimuli	1	AR
Signaling by Rho GTPases, Miro GTPases and RHOBTB3	1	AR

Dominant GO biological processes

GO term	Targets
steroid biosynthetic process	1
androgen biosynthetic process	1
glucocorticoid biosynthetic process	1
sex differentiation	1
steroid metabolic process	1
cortisol biosynthetic process	1
hormone biosynthetic process	1
progesterone metabolic process	1
lipid metabolic process	1
hormone metabolic process	1
olefinic compound metabolic process	1
negative regulation of transcription by RNA polymerase II	1
MAPK cascade	1
in utero embryonic development	1
regulation of systemic arterial blood pressure	1

Indications & clinical

Indications

2 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
malignant pancreatic neoplasm	2	MONDO:0009831	EFO:1000359

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 5.

Phase distribution

Phase	Trials
PHASE2	2
PHASE1	2
PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02438007	PHASE3	TERMINATED	A Study of Galeterone Compared to Enzalutamide In Men Expressing Androgen Receptor Splice Variant-7 mRNA (AR-V7) Metastatic CRPC
NCT04098081	PHASE2	ACTIVE_NOT_RECRUITING	1911GCCC: Galeterone With Gemcitabine for Patients With Metastatic Pancreatic Adenocarcinoma
NCT01709734	PHASE2	TERMINATED	A 2 Part, Phase 2 Trial of Galeterone in the Treatment of Castration Resistant Prostate Cancer
NCT00959959	PHASE1	COMPLETED	ARMOR1: Study of TOK-001 to Treat Castration Resistant Prostate Cancer
NCT02729376	PHASE1	COMPLETED	Single-Dose Study to Assess the Absorption, Metabolism, Excretion, and Mass Balance of Radiolabeled Galeterone

Clinical evidence (CIViC)

Variant × indication × effect (1 predictive associations from 1 curated evidence items):

Variant	Indication	Effect	Therapy	Level	CIViC
AR AR-V7	Prostate Cancer	Sensitivity/Response	Galeterone + Enzalutamide	CIViC B	EID9549

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

144 molecules share ≥1 primary target. Top 60 by shared-target count:

Molecule	Source	Status	Shared targets
ABIRATERONE	ChEMBL + PubChem	Phase 4 (approved)	AR, CYP17A1
Bicalutamide	ChEMBL + PubChem	Phase 4 (approved)	AR, CYP17A1
Enzalutamide	ChEMBL + PubChem	Phase 4 (approved)	AR, CYP17A1
Flutamide	ChEMBL + PubChem	Phase 4 (approved)	AR, CYP17A1
TESTOSTERONE PROPIONATE	ChEMBL	Phase 4 (approved)	AR, CYP17A1
CLOTRIMAZOLE	ChEMBL + PubChem	Phase 4 (approved)	CYP17A1
ECONAZOLE	ChEMBL + PubChem	Phase 4 (approved)	CYP17A1
MEGESTROL	ChEMBL + PubChem	Phase 4 (approved)	AR
MICONAZOLE	ChEMBL + PubChem	Phase 4 (approved)	CYP17A1
OSILODROSTAT	ChEMBL + PubChem	Phase 4 (approved)	CYP17A1
AMINOGLUTETHIMIDE	ChEMBL	Phase 4 (approved)	CYP17A1
APALUTAMIDE	ChEMBL	Phase 4 (approved)	AR
ARIPIPRAZOLE	ChEMBL	Phase 4 (approved)	AR
BECLOMETHASONE DIPROPIONATE	ChEMBL	Phase 4 (approved)	AR
BETAMETHASONE	ChEMBL	Phase 4 (approved)	AR
BIFONAZOLE	ChEMBL	Phase 4 (approved)	CYP17A1
BITHIONOL	ChEMBL	Phase 4 (approved)	AR
BROMHEXINE	ChEMBL	Phase 4 (approved)	AR
BUDESONIDE	ChEMBL	Phase 4 (approved)	AR
CHLORMADINONE	ChEMBL	Phase 4 (approved)	AR
CLARITHROMYCIN	ChEMBL	Phase 4 (approved)	AR
CLASCOTERONE	ChEMBL	Phase 4 (approved)	AR
CLOCORTOLONE PIVALATE	ChEMBL	Phase 4 (approved)	AR
CLOMIPHENE	ChEMBL	Phase 4 (approved)	AR
CORTISONE	ChEMBL	Phase 4 (approved)	AR
CYCLOFENIL	ChEMBL	Phase 4 (approved)	AR
DAROLUTAMIDE	ChEMBL	Phase 4 (approved)	AR
DESOGESTREL	ChEMBL	Phase 4 (approved)	AR
DESOXIMETASONE	ChEMBL	Phase 4 (approved)	AR
DEXAMETHASONE	ChEMBL	Phase 4 (approved)	AR
DIETHYLSTILBESTROL	ChEMBL	Phase 4 (approved)	AR
DIFLORASONE DIACETATE	ChEMBL	Phase 4 (approved)	AR
DORZOLAMIDE	ChEMBL	Phase 4 (approved)	AR
DROSPIRENONE	ChEMBL	Phase 4 (approved)	AR
DYDROGESTERONE	ChEMBL	Phase 4 (approved)	AR
EPLERENONE	ChEMBL	Phase 4 (approved)	AR
ESTRADIOL	ChEMBL	Phase 4 (approved)	AR
ESTRADIOL CYPIONATE	ChEMBL	Phase 4 (approved)	AR
ESTRADIOL VALERATE	ChEMBL	Phase 4 (approved)	AR
ESTRIOL	ChEMBL	Phase 4 (approved)	AR
ESTRONE	ChEMBL	Phase 4 (approved)	AR
ETHINYL ESTRADIOL	ChEMBL	Phase 4 (approved)	AR
ETHYNODIOL DIACETATE	ChEMBL	Phase 4 (approved)	AR
ETONOGESTREL	ChEMBL	Phase 4 (approved)	AR
FLUMETHASONE PIVALATE	ChEMBL	Phase 4 (approved)	AR
FLUOCINOLONE ACETONIDE	ChEMBL	Phase 4 (approved)	AR
FLUOCINONIDE	ChEMBL	Phase 4 (approved)	AR
FLUOXYMESTERONE	ChEMBL	Phase 4 (approved)	AR
FLURANDRENOLIDE	ChEMBL	Phase 4 (approved)	AR
FLUTICASONE FUROATE	ChEMBL	Phase 4 (approved)	AR
FLUTICASONE PROPIONATE	ChEMBL	Phase 4 (approved)	AR
HALCINONIDE	ChEMBL	Phase 4 (approved)	AR
HALOBETASOL PROPIONATE	ChEMBL	Phase 4 (approved)	AR
HEXACHLOROPHENE	ChEMBL	Phase 4 (approved)	AR
HEXESTROL	ChEMBL	Phase 4 (approved)	AR
HYDROCORTISONE	ChEMBL	Phase 4 (approved)	AR
INDOMETHACIN	ChEMBL	Phase 4 (approved)	AR
ISOCONAZOLE	ChEMBL	Phase 4 (approved)	CYP17A1
KETOCONAZOLE	ChEMBL	Phase 4 (approved)	CYP17A1
LEVONORGESTREL	ChEMBL	Phase 4 (approved)	AR