Mifepristone

Summary

Mifepristone (CHEMBL1276308) is an approved small-molecule contraceptive drug (ATC G03XB01) targeting NR1I2, NR3C1, and PGR; indicated across 38 conditions including cushing syndrome and uterine corpus leiomyoma.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: G03XB01 (+1 more)
• Targets: 4 (NR1I2, NR3C1, PGR…)
• Indications: 38 conditions
• Clinical trials: 150
• Chemistry: 429.6 Da · C29H35NO2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1276308
Name	Mifepristone
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	55245
ChEBI	CHEBI:50692
ATC	G03XB01, G03XB51
Molecular formula	C29H35NO2
Molecular weight	429.6
InChIKey	VKHAHZOOUSRJNA-GCNJZUOMSA-N

SMILES: CC#C[C@@]1(CC[C@@H]2[C@@]1(C[C@@H](C3=C4CCC(=O)C=C4CC[C@@H]23)C5=CC=C(C=C5)N(C)C)C)O

IUPAC name: (8S,11R,13S,14S,17S)-11-[4-(dimethylamino)phenyl]-17-hydroxy-13-methyl-17-prop-1-ynyl-1,2,6,7,8,11,12,14,15,16-decahydrocyclopenta[a]phenanthren-3-one

Pharmacological roles (ChEBI): abortifacient, contraceptive drug, synthetic oral contraceptive, hormone antagonist.

Also known as: Korlym, Mifegyne, Mifeprex, Mifepristona, Mifepristone, NSC-759862, RU 38486, RU 486, RU-38486, RU-486, RU486, mifepristone

Patent coverage: 8,252 distinct patent families (30,535 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
NR1I2	Pregnane X receptor	Agonist	5	0.3%	O75469
NR3C1	Glucocorticoid receptor	Antagonist	9.4	0.9%	P04150
PGR	Progesterone receptor	Mixed	8.96	0.1%	P06401
AR	Androgen receptor	Antagonist	9.19		P10275

Broader ChEMBL bioactivity targets: 45 (assay-derived). Sample: Tyrosyl-DNA phosphodiesterase 1, Nuclear receptor ROR-gamma, Survival motor neuron protein, Prelamin-A/C, RecQ-like DNA helicase BLM, Inositol monophosphatase 1, Ferritin light chain, Geminin, Endonuclease 4, Peripheral myelin protein 22.

Bioactivity

ChEMBL activities: 214 potent at pChembl ≥ 5 of 251 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
PGR	10.68	IC50	0.02	nM	CHEMBL_ACT_2391144
PGR	10.6	IC50	0.03	nM	CHEMBL_ACT_184565
PGR	10.55	IC50	0.03	nM	CHEMBL_ACT_184563
PGR	10.35	IC50	0.04	nM	CHEMBL_ACT_2391063
PGR	10.3	IC50	0.05	nM	CHEMBL_ACT_3125135
PGR	10.27	IC50	0.05	nM	CHEMBL_ACT_2046181
PGR	10.14	IC50	0.07	nM	CHEMBL_ACT_18407048
NR3C1	10.05	Ki	0.09	nM	CHEMBL_ACT_18235952
PGR	10	IC50	0.1	nM	CHEMBL_ACT_1170993
NR3C1	10	Ki	0.1	nM	CHEMBL_ACT_1463070
PGR	9.89	IC50	0.13	nM	CHEMBL_ACT_1002415
PGR	9.74	IC50	0.18	nM	CHEMBL_ACT_493315
PGR	9.74	IC50	0.18	nM	CHEMBL_ACT_540698
PGR	9.7	IC50	0.2	nM	CHEMBL_ACT_2119997
PGR	9.7	IC50	0.2	nM	CHEMBL_ACT_2369837
PGR	9.7	IC50	0.2	nM	CHEMBL_ACT_2463066
PGR	9.7	IC50	0.2	nM	CHEMBL_ACT_3383449
PGR	9.7	IC50	0.2	nM	CHEMBL_ACT_402714
PGR	9.7	IC50	0.2	nM	CHEMBL_ACT_824254
NR3C1	9.62	Ki	0.24	nM	CHEMBL_ACT_275737
NR3C1	9.62	Ki	0.24	nM	CHEMBL_ACT_3252319
PGR	9.6	Ki	0.25	nM	CHEMBL_ACT_1481479
PGR	9.6	IC50	0.25	nM	CHEMBL_ACT_1742825
NR3C1	9.53	IC50	0.3	nM	CHEMBL_ACT_15684859
PGR	9.52	IC50	0.3	nM	CHEMBL_ACT_1002414
PGR	9.52	IC50	0.3	nM	CHEMBL_ACT_1002418
NR3C1	9.52	Ki	0.3	nM	CHEMBL_ACT_15684193
PGR	9.52	IC50	0.3	nM	CHEMBL_ACT_18407140
PGR	9.52	IC50	0.3	nM	CHEMBL_ACT_338277
PGR	9.52	IC50	0.3	nM	CHEMBL_ACT_641296

Target pathways

Aggregated over 4 target gene(s): NR1I2, NR3C1, PGR, AR.

Top Reactome pathways

32 total, by targets touching each:

Pathway	Targets	Genes
Nuclear Receptor transcription pathway	4	AR, NR1I2, NR3C1, PGR
SUMOylation of intracellular receptors	4	AR, NR1I2, NR3C1, PGR
HSP90 chaperone cycle for steroid hormone receptors (SHR) in the presence of ligand	3	AR, NR3C1, PGR
Nuclear signaling by ERBB4	1	PGR
Signal Transduction	1	AR
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
Metabolism of proteins	1	AR
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
PTK6 Expression	1	NR3C1
Transcriptional regulation by RUNX2	1	AR
Regulation of RUNX2 expression and activity	1	NR3C1
RUNX2 regulates osteoblast differentiation	1	AR
RUNX2 regulates bone development	1	AR
Cellular responses to stimuli	1	AR
Estrogen-dependent gene expression	1	PGR
FOXO-mediated transcription of oxidative stress, metabolic and neuronal genes	1	NR3C1
Potential therapeutics for SARS	1	NR3C1
Signaling by Rho GTPases, Miro GTPases and RHOBTB3	1	AR
Regulation of NPAS4 gene transcription	1	NR3C1

Dominant GO biological processes

GO term	Targets
regulation of DNA-templated transcription	4
signal transduction	4
positive regulation of transcription by RNA polymerase II	4
negative regulation of transcription by RNA polymerase II	3
gene expression	3
positive regulation of gene expression	3
positive regulation of DNA-templated transcription	3
regulation of transcription by RNA polymerase II	3
nuclear receptor-mediated steroid hormone signaling pathway	3
intracellular receptor signaling pathway	2
negative regulation of DNA-templated transcription	2
cellular response to steroid hormone stimulus	2
positive regulation of miRNA transcription	2
response to ketone	2
cell-cell signaling	2

Indications & clinical

Indications

38 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
Cushing syndrome	4	MONDO:0018912	EFO:0003099
uterine corpus leiomyoma	3	MONDO:0007886	EFO:0000731
ACTH-dependent Cushing syndrome	3	MONDO:0020528	EFO:1001110
leiomyoma	3	MONDO:0001572	MONDO:0001572
major depressive disorder	3	MONDO:0002009	MONDO:0002009
depressive disorder	3	MONDO:0002050	MONDO:0002050
breast neoplasm	3	MONDO:0021100	MONDO:0007254
meningioma	3	MONDO:0016642	MONDO:0016642
mental disorder	3	MONDO:0005084	EFO:0000677
cocaine dependence	2	MONDO:0005186	EFO:0002610
hepatitis C virus infection	2	MONDO:0005231	EFO:0003047
post-traumatic stress disorder	2	MONDO:0005146	EFO:0001358
HIV infectious disease	2	MONDO:0005109	EFO:0000764
endometriosis	2	MONDO:0005133	EFO:0001065
non-small cell lung carcinoma	2	MONDO:0005233	EFO:0003060
prostate adenocarcinoma	2	MONDO:0005082	EFO:0000673
borderline personality disorder	2	MONDO:0001156	HP:0012076
adenomyosis	2	MONDO:0010888	EFO:1001757
central serous chorioretinopathy	2	MONDO:0018616	EFO:0009784
alcohol abuse	2	MONDO:0002046	MONDO:0002046
peritoneal neoplasm	2	MONDO:0006901	MONDO:0002087
fallopian tube neoplasm	2	MONDO:0021092	MONDO:0002158
ovarian cancer	2	MONDO:0008170	MONDO:0008170
endometrium neoplasm	2	MONDO:0021251	MONDO:0011962
Alzheimer disease	2	MONDO:0004975	MONDO:0004975
bipolar disorder	2	MONDO:0004985	MONDO:0004985
anxiety	1	MONDO:0011918	EFO:0005230
male breast carcinoma	1	MONDO:0005628	EFO:0006861
diabetes mellitus	1	MONDO:0005015	EFO:0000400
persian gulf syndrome	1	MONDO:0005907	EFO:0007430
ductal breast carcinoma in situ	0	MONDO:0005023	EFO:0000432
nicotine dependence	0	MONDO:0008575	EFO:0003768

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

Clinical trials

Total trials: 150.

Phase distribution

Phase	Trials
PHASE4	34
Not specified	31
PHASE2	27
PHASE3	26
PHASE1	13
PHASE1/PHASE2	8
PHASE2/PHASE3	7
EARLY_PHASE1	4

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05341817	PHASE4	RECRUITING	The Use of Letrozole or Mifepristone for Pretreatment of Medical Termination of Pregnancy
NCT00177372	PHASE4	COMPLETED	Mifepristone and Misoprostol for Fetal Demise
NCT00269568	PHASE4	COMPLETED	Mifepristone at Same Time Multicenter Study
NCT00410345	PHASE4	COMPLETED	Cervical Rippening With Antiprogesterone in Midtrimester Abortions
NCT00691067	PHASE4	COMPLETED	A Controlled Trial of Mifepristone in Gulf War Veterans With Chronic Multisymptom Illness
NCT00784797	PHASE4	COMPLETED	Misopristol Versus Pitocin for Second Trimester Abortion
NCT00997347	PHASE4	COMPLETED	The Extended Gestational Age Medical Abortion Study
NCT01490697	PHASE4	COMPLETED	Developing Memory Reconsolidation Blockers as Novel Posttraumatic Stress Disorder (PTSD) Treatments
NCT01631682	PHASE4	COMPLETED	Pilot Study of Pharmaceutical and Behavioral Interventions to Treat Anxiety Disorders
NCT01768299	PHASE4	COMPLETED	Medical Abortion With Mifepristone + Misoprostol (13 - 22 Weeks)
NCT01798017	PHASE4	COMPLETED	Introducing Mifepristone-Misoprostol for Menstrual Regulation in Public Sector Facilities in Bangladesh
NCT01856985	PHASE4	COMPLETED	Acceptability of an Out-patient Regimen of Medical Abortion With Mifepristone and 800 Mcg Misoprostol Administered at 78-84 Days Gestation
NCT01920022	PHASE4	COMPLETED	Quickstart of Nexplanon® at Medical Abortion
NCT01990560	PHASE4	COMPLETED	Glucocorticoid Receptor Blockade With Mifepristone in Patients With Mild Adrenal Hypercortisolism
NCT02342002	PHASE4	TERMINATED	Mifepristone and Misoprostol Versus Misoprostol Alone for Missed Abortion: A Randomized-controlled Trial
NCT02412618	PHASE4	COMPLETED	Same-Day Mifepristone-Misoprostol Compared to Misoprostol Only for Surgical Abortion Cervical Preparation
NCT02620904	PHASE4	TERMINATED	Mifepristone Induction for Fetal Demise
NCT02704481	PHASE4	TERMINATED	Non-surgical Alternatives to Treatment of Failed Medical Abortion
NCT02708446	PHASE4	UNKNOWN	A Comparison of Sublingual and Buccal Misoprostol Regimens After Mifepristone for Mid-trimester Abortion
NCT02720991	PHASE4	COMPLETED	A Pilot of an Outpatient Regimen of Medical Abortion With Mifepristone and Sublingual Misoprostol in the 11 and 12 Weeks
NCT02745093	PHASE4	UNKNOWN	Medical Abortion at Gestational Age of 8 to ≤9 Weeks Versus >9 to ≤12 Weeks
NCT02981030	PHASE4	COMPLETED	Acceptability and Feasibility of a Simplified Medical Abortion Service Delivery in Western Ukraine
NCT03044093	PHASE4	UNKNOWN	Mifepristone and Misoprostol Compared With Misoprostol Alone for Second Trimester Abortion
NCT03210324	PHASE4	TERMINATED	A Study on the Mifepristone Tablets in the Treatment of Symptomatic Uterine Fibroids With Safety and Efficacy
NCT03212352	PHASE4	TERMINATED	Comparing Two Medical Treatments for Early Pregnancy Failure.
NCT03320057	PHASE4	COMPLETED	Medication Abortion Via Pharmacy Dispensing
NCT03346629	PHASE4	COMPLETED	Outpatient Service for Mid-trimester Termination of Pregnancy
NCT03913104	PHASE4	COMPLETED	Mail Order Mifepristone Study
NCT04063904	PHASE4	TERMINATED	Pilot Study of an Ambulatory Medical Abortion Service at 13-18 Weeks of Gestation in Colombia
NCT04458558	PHASE4	UNKNOWN	Improving Access to Abortion in the Republic of Georgia
NCT05046041	PHASE4	COMPLETED	Assessing Outpatient ‘Day Procedure’ for Second-trimester Medical Abortion at Two Public Sector Hospitals in Nepal
NCT05119439	PHASE4	TERMINATED	Mifepristone and Two Doses of Misoprostol for Abortion at 11&12 Weeks
NCT05124314	PHASE4	UNKNOWN	Comparison of Two Different Drug Regimens for Medical Treatment of Early Pregnancy Loss
NCT05322252	PHASE4	COMPLETED	Simultaneous Mifepristone and Misoprostol Versus Misoprostol Alone for Induction of Labor of Nonviable Second Trimester Pregnancy: a Pilot Randomized Controlled Trial
NCT06394999	PHASE3	RECRUITING	Efficacy, Safety, and Acceptability of Mifepristone 50 mg Once-weekly as a Contraceptive
NCT06492889	PHASE2/PHASE3	NOT_YET_RECRUITING	Assessing the Efficacy and Acceptability of Two Missed Period Pills Regimens
NCT07506512	PHASE3	NOT_YET_RECRUITING	Optimal Time Interval Between Mifepristone and Misoprostol Administration for Early Pregnancy Loss
NCT00128479	PHASE3	COMPLETED	A United States Study of the Safety and Tolerability of Corlux for Psychotic Symptoms in Psychotic Major Depression
NCT00128505	PHASE3	COMPLETED	An International Extension Study of Corlux for Recurrent Psychotic Symptoms in Psychotic Major Depression
NCT00130676	PHASE3	COMPLETED	A United States Study of Corlux for Psychotic Symptoms in Psychotic Major Depression

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

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.

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

Molecule	Source	Status	Shared targets
REGORAFENIB	ChEMBL + PubChem	Phase 4 (approved)	AR, NR1I2, NR3C1, PGR
DESOGESTREL	ChEMBL	Phase 4 (approved)	AR, NR1I2, NR3C1, PGR
DESOXIMETASONE	ChEMBL	Phase 4 (approved)	AR, NR1I2, NR3C1, PGR
DIETHYLSTILBESTROL	ChEMBL	Phase 4 (approved)	AR, NR1I2, NR3C1, PGR
ENZALUTAMIDE	ChEMBL	Phase 4 (approved)	AR, NR1I2, NR3C1, PGR
ETHINYL ESTRADIOL	ChEMBL	Phase 4 (approved)	AR, NR1I2, NR3C1, PGR
FLUTICASONE PROPIONATE	ChEMBL	Phase 4 (approved)	AR, NR1I2, NR3C1, PGR
METHYLPREDNISOLONE	ChEMBL	Phase 4 (approved)	AR, NR1I2, NR3C1, PGR
PROGESTERONE	ChEMBL	Phase 4 (approved)	AR, NR1I2, NR3C1, PGR
TESTOSTERONE PROPIONATE	ChEMBL	Phase 4 (approved)	AR, NR1I2, NR3C1, PGR
TROGLITAZONE	ChEMBL	Phase 4 (approved)	AR, NR1I2, NR3C1, PGR
Pyrazinamide	PubChem	Approved	AR, NR1I2, NR3C1, PGR
BOSENTAN	ChEMBL + PubChem	Phase 4 (approved)	NR1I2, NR3C1, PGR
DESOXYCORTICOSTERONE PIVALATE	ChEMBL + PubChem	Phase 4 (approved)	NR1I2, NR3C1, PGR
Fidaxomicin	ChEMBL + PubChem	Phase 4 (approved)	NR1I2, NR3C1, PGR
FULVESTRANT	ChEMBL + PubChem	Phase 4 (approved)	NR1I2, NR3C1, PGR
MEGESTROL	ChEMBL + PubChem	Phase 4 (approved)	AR, NR3C1, PGR
PODOFILOX	ChEMBL + PubChem	Phase 4 (approved)	AR, NR3C1, PGR
TADALAFIL	ChEMBL + PubChem	Phase 4 (approved)	NR1I2, NR3C1, PGR
BECLOMETHASONE DIPROPIONATE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
BENZBROMARONE	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
BETAMETHASONE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
BETAMETHASONE DIPROPIONATE	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
BITHIONOL	ChEMBL	Phase 4 (approved)	AR, NR1I2, PGR
BUDESONIDE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
BUTOCONAZOLE	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
CANDESARTAN CILEXETIL	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
CHLORMADINONE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
CICLESONIDE	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
CLOBETASOL PROPIONATE	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
DANAZOL	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
DAUNORUBICIN	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
DEXAMETHASONE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
DIFLORASONE DIACETATE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
DROSPIRENONE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
ECONAZOLE	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
EFAVIRENZ	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
EPLERENONE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
ESTRADIOL	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
ESTRIOL	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
FELODIPINE	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
FLUOCINOLONE ACETONIDE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
FLUOCINONIDE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
FLUOROMETHOLONE	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
FLUOXYMESTERONE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
FLURANDRENOLIDE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
FLUTICASONE FUROATE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
HALCINONIDE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
HYDROCORTISONE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
HYDROXYPROGESTERONE CAPROATE	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
ISOCONAZOLE	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
LOTEPREDNOL ETABONATE	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
MASOPROCOL	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
MEDROXYPROGESTERONE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
MICONAZOLE	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
MITOTANE	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
MOMETASONE FUROATE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
NISOLDIPINE	ChEMBL	Phase 4 (approved)	NR1I2, NR3C1, PGR
NOMEGESTROL	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR
NORETHINDRONE	ChEMBL	Phase 4 (approved)	AR, NR3C1, PGR