Ulipristal

Summary

Ulipristal (CHEMBL2103846) is an approved small molecule (ATC G03XB02) targeting PGR; indicated across 2 conditions including benign muscle neoplasm and leiomyoma.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: G03XB02 (+1 more)
• Targets: 1 (PGR)
• Indications: 2 conditions
• Clinical trials: 12
• Chemistry: 433.6 Da · C28H35NO3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2103846
Name	Ulipristal
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	13559281
ATC	G03XB02, G03AD02
Molecular formula	C28H35NO3
Molecular weight	433.6
InChIKey	HKDLNTKNLJPAIY-WKWWZUSTSA-N

SMILES: CC(=O)[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,17R)-17-acetyl-11-[4-(dimethylamino)phenyl]-17-hydroxy-13-methyl-1,2,6,7,8,11,12,14,15,16-decahydrocyclopenta[a]phenanthren-3-one

Also known as: Uliprisnil, Ulipristal, Ulipristal acetate ulipristal, ULIPRISTAL

Patent coverage: 157 distinct patent families (283 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
PGR	Progesterone receptor	Partial agonist	7	0.1%	P06401

Bioactivity

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

Target pathways

Aggregated over 1 target gene(s): PGR.

Top Reactome pathways

7 total, by targets touching each:

Pathway	Targets	Genes
Nuclear signaling by ERBB4	1	PGR
HSP90 chaperone cycle for steroid hormone receptors (SHR) in the presence of ligand	1	PGR
Nuclear Receptor transcription pathway	1	PGR
SUMOylation of intracellular receptors	1	PGR
Estrogen-dependent gene expression	1	PGR
Developmental Lineage of Mammary Gland Luminal Epithelial Cells	1	PGR
Developmental Lineage of Mammary Gland Alveolar Cells	1	PGR

Dominant GO biological processes

GO term	Targets
ovulation from ovarian follicle	1
glandular epithelial cell maturation	1
regulation of DNA-templated transcription	1
regulation of transcription by RNA polymerase II	1
signal transduction	1
cell-cell signaling	1
positive regulation of gene expression	1
negative regulation of gene expression	1
nuclear receptor-mediated steroid hormone signaling pathway	1
paracrine signaling	1
positive regulation of transcription by RNA polymerase II	1
lung alveolus development	1
regulation of epithelial cell proliferation	1
progesterone receptor signaling pathway	1
maintenance of protein location in nucleus	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
benign muscle neoplasm	3	MONDO:0003061	MONDO:0003061
leiomyoma	2	MONDO:0001572	MONDO:0001572

Clinical trials

Total trials: 12.

Phase distribution

Phase	Trials
PHASE4	5
PHASE3	3
PHASE2	2
PHASE1	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT01569113	PHASE4	COMPLETED	Assessment of Effects on Ovarian Activity of a Combined Oral Contraceptive Pill When Preceded by the Intake of ellaOne® or Placebo.
NCT02213081	PHASE4	COMPLETED	Ulipristal for Endometriosis-related Pelvic Pain
NCT02288130	PHASE4	UNKNOWN	Ulipristal vs. GnRHa Prior to Laparoscopic Myomectomy
NCT04132349	PHASE4	TERMINATED	Ulipristal Acetate in Symptomatic Uterine Fibroid
NCT04832906	PHASE4	COMPLETED	UA Versus UAE in Treatment of Fibroids
NCT01642472	PHASE3	COMPLETED	PGL4001 Efficacy Assessment in Reduction of Symptoms Due to Uterine Leiomyomata
NCT02147158	PHASE3	COMPLETED	A Study of the Efficacy and Safety of Ulipristal Acetate Intermittent Treatment for Abnormal Uterine Bleeding Associated With Leiomyomas
NCT02147197	PHASE3	COMPLETED	A Study of the Efficacy and Safety of a 3-month Treatment Course of Ulipristal Acetate for the Treatment of Abnormal Uterine Bleeding Associated With Leiomyomas
NCT00290251	PHASE2	COMPLETED	Treatment of Uterine Fibroids With the Selective Progesterone Receptor Modulator CDB-2914
NCT02465814	PHASE2	COMPLETED	Assess Safety and Efficacy of Vilaprisan in Patients With Uterine Fibroids
NCT03230539	PHASE1	UNKNOWN	A proof-of Concept, Randomized 3-month Study to Evaluate the Effects of Three Contraceptive Intrauterine Systems Delivering Copper and a Daily Dose of 5, 20 or 40 μg of Ulipristal Acetate (UPA)
NCT03342859	PHASE1	TERMINATED	Mode of Action Study of Vilaprisan and Ulipristal Acetate in Patients With Uterine Fibroids for Whom Surgery is Planned

Clinical evidence (CIViC)

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

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.

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

Molecule	Source	Status	Shared targets
FIDAXOMICIN	ChEMBL + PubChem	Phase 4 (approved)	PGR
FULVESTRANT	ChEMBL + PubChem	Phase 4 (approved)	PGR
GENTIAN VIOLET	ChEMBL + PubChem	Phase 4 (approved)	PGR
ABIRATERONE	ChEMBL	Phase 4 (approved)	PGR
ACETYLCHOLINE	ChEMBL	Phase 4 (approved)	PGR
ADAPALENE	ChEMBL	Phase 4 (approved)	PGR
ALCLOMETASONE DIPROPIONATE	ChEMBL	Phase 4 (approved)	PGR
ALECTINIB	ChEMBL	Phase 4 (approved)	PGR
AMCINONIDE	ChEMBL	Phase 4 (approved)	PGR
APOMORPHINE	ChEMBL	Phase 4 (approved)	PGR
ARFORMOTEROL	ChEMBL	Phase 4 (approved)	PGR
ARIPIPRAZOLE	ChEMBL	Phase 4 (approved)	PGR
AZATHIOPRINE	ChEMBL	Phase 4 (approved)	PGR
BECLOMETHASONE DIPROPIONATE	ChEMBL	Phase 4 (approved)	PGR
BENPERIDOL	ChEMBL	Phase 4 (approved)	PGR
BENZBROMARONE	ChEMBL	Phase 4 (approved)	PGR
BENZIODARONE	ChEMBL	Phase 4 (approved)	PGR
BETAMETHASONE	ChEMBL	Phase 4 (approved)	PGR
BETAMETHASONE DIPROPIONATE	ChEMBL	Phase 4 (approved)	PGR
BETAMETHASONE VALERATE	ChEMBL	Phase 4 (approved)	PGR
BEXAROTENE	ChEMBL	Phase 4 (approved)	PGR
BICALUTAMIDE	ChEMBL	Phase 4 (approved)	PGR
BIFONAZOLE	ChEMBL	Phase 4 (approved)	PGR
BITHIONOL	ChEMBL	Phase 4 (approved)	PGR
BOSUTINIB	ChEMBL	Phase 4 (approved)	PGR
BUDESONIDE	ChEMBL	Phase 4 (approved)	PGR
BUTOCONAZOLE	ChEMBL	Phase 4 (approved)	PGR
CABOZANTINIB	ChEMBL	Phase 4 (approved)	PGR
CALCIPOTRIENE	ChEMBL	Phase 4 (approved)	PGR
CALCITRIOL	ChEMBL	Phase 4 (approved)	PGR
CANDESARTAN CILEXETIL	ChEMBL	Phase 4 (approved)	PGR
CANRENONE	ChEMBL	Phase 4 (approved)	PGR
CARBARIL	ChEMBL	Phase 4 (approved)	PGR
CASPOFUNGIN	ChEMBL	Phase 4 (approved)	PGR
CEFAMANDOLE	ChEMBL	Phase 4 (approved)	PGR
CEFMENOXIME	ChEMBL	Phase 4 (approved)	PGR
CEFTAZIDIME	ChEMBL	Phase 4 (approved)	PGR
CEFTIZOXIME	ChEMBL	Phase 4 (approved)	PGR
CEFTRIAXONE	ChEMBL	Phase 4 (approved)	PGR
CEFUROXIME	ChEMBL	Phase 4 (approved)	PGR
CEPHRADINE	ChEMBL	Phase 4 (approved)	PGR
CHLORHEXIDINE	ChEMBL	Phase 4 (approved)	PGR
CHLORMADINONE	ChEMBL	Phase 4 (approved)	PGR
CHOLECALCIFEROL	ChEMBL	Phase 4 (approved)	PGR
CICLESONIDE	ChEMBL	Phase 4 (approved)	PGR
CLOBETASOL PROPIONATE	ChEMBL	Phase 4 (approved)	PGR
CLOCORTOLONE PIVALATE	ChEMBL	Phase 4 (approved)	PGR
CLOMIPHENE	ChEMBL	Phase 4 (approved)	PGR
CYCLOFENIL	ChEMBL	Phase 4 (approved)	PGR
DANAZOL	ChEMBL	Phase 4 (approved)	PGR
DAUNORUBICIN	ChEMBL	Phase 4 (approved)	PGR
DEFLAZACORT	ChEMBL	Phase 4 (approved)	PGR
DEQUALINIUM	ChEMBL	Phase 4 (approved)	PGR
DESOGESTREL	ChEMBL	Phase 4 (approved)	PGR
DESONIDE	ChEMBL	Phase 4 (approved)	PGR
DESOXIMETASONE	ChEMBL	Phase 4 (approved)	PGR
DESOXYCORTICOSTERONE PIVALATE	ChEMBL	Phase 4 (approved)	PGR
DEXAMETHASONE	ChEMBL	Phase 4 (approved)	PGR
DIETHYLPROPION	ChEMBL	Phase 4 (approved)	PGR
DIETHYLSTILBESTROL	ChEMBL	Phase 4 (approved)	PGR