Trilostane

Summary

Trilostane (CHEMBL1200907) is an approved small-molecule antineoplastic agent (ATC H02CA01) targeting HSD3B2; indicated across 3 conditions including adrenal gland disorder and prostate adenocarcinoma.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: H02CA01
• Targets: 1 (HSD3B2)
• Indications: 3 conditions
• Clinical trials: 2
• Chemistry: 329.4 Da · C20H27NO3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1200907
Name	Trilostane
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	656583
ChEBI	CHEBI:32260
ATC	H02CA01
Molecular formula	C20H27NO3
Molecular weight	329.4
InChIKey	KVJXBPDAXMEYOA-CXANFOAXSA-N

SMILES: C[C@]12CC[C@H]3[C@H]([C@@H]1CC[C@@H]2O)CC[C@]45[C@@]3(CC(=C([C@H]4O5)O)C#N)C

IUPAC name: (1S,2R,6R,8S,11S,12S,15S,16S)-5,15-dihydroxy-2,16-dimethyl-7-oxapentacyclo[9.7.0.02,8.06,8.012,16]octadec-4-ene-4-carbonitrile

ChEBI definition: An epoxy steroid that is 3,17β-dihydroxy-5α-androst-2-ene-2-carbonitrile in which the oxygen of the epoxy group is joined to the 4α and 5 α positions.

Pharmacological roles (ChEBI): antineoplastic agent, abortifacient, EC 1.1.1.210 [3β(or 20α)-hydroxysteroid dehydrogenase] inhibitor.

Also known as: Desopan, Modrastane, Modrenal, NSC-758904, Trilocur, Trilorale, Trilostane, Trilostano, WIN 24,540, WIN-24540, TRILOSTANE, SID144206007

Patent coverage: 7,141 distinct patent families (30,906 SureChEMBL compound mentions), from 1 matched compound structure(s). 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
HSD3B2	hydroxy-delta-5-steroid dehydrogenase, 3 beta- and steroid delta-isomerase 2	Inhibition	8.4	0.2%	P26439

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

Aggregated over 1 target gene(s): HSD3B2.

Top Reactome pathways

7 total, by targets touching each:

Pathway	Targets	Genes
Metabolism	1	HSD3B2
Androgen biosynthesis	1	HSD3B2
Mineralocorticoid biosynthesis	1	HSD3B2
Glucocorticoid biosynthesis	1	HSD3B2
Metabolism of steroid hormones	1	HSD3B2
Metabolism of lipids	1	HSD3B2
Metabolism of steroids	1	HSD3B2

Dominant GO biological processes

GO term	Targets
steroid biosynthetic process	1
androgen biosynthetic process	1
C21-steroid hormone metabolic process	1
lipid metabolic process	1

Indications & clinical

Indications

3 indications (1 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
adrenal gland disorder	4	MONDO:0005495	EFO:0005539
prostate adenocarcinoma	2	MONDO:0005082	EFO:0000673

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

Clinical trials

Total trials: 2.

Phase distribution

Phase	Trials
PHASE2	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00181597	PHASE2	COMPLETED	Trilostane for Androgen-Independent Prostate Cancer
NCT01615211	PHASE2	TERMINATED	Randomized Study of Letrozole and Trilostane for Medical Abortion

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).