Bexarotene

Summary

Bexarotene (CHEMBL1023) is an approved small-molecule antineoplastic agent (ATC L01XF03) targeting PPARG, RXRA, and RXRB; indicated across 26 conditions including primary cutaneous t-cell non-hodgkin lymphoma and neoplasm.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: L01XF03
• Targets: 4 (PPARG, RXRA, RXRB…)
• Indications: 26 conditions
• Clinical trials: 38
• Chemistry: 348.5 Da · C24H28O2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1023
Name	Bexarotene
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	82146
ChEBI	CHEBI:50859
ATC	L01XF03
Molecular formula	C24H28O2
Molecular weight	348.5
InChIKey	NAVMQTYZDKMPEU-UHFFFAOYSA-N

SMILES: CC1=CC2=C(C=C1C(=C)C3=CC=C(C=C3)C(=O)O)C(CCC2(C)C)(C)C

IUPAC name: 4-[1-(3,5,5,8,8-pentamethyl-6,7-dihydronaphthalen-2-yl)ethenyl]benzoic acid

Pharmacological roles (ChEBI): antineoplastic agent.

Also known as: Bexarotene, Bexaroteno, LG-100069, LG100069, LGD-1069, LGD1069, NSC-747528, Targretin, Targretine, bexarotene, SID50125754, BEXAROTENE

Parent form; salt/anhydrous children: CHEMBL5290401

Patent coverage: 9,832 distinct patent families (40,951 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 40,930 (100%) 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
PPARG	Peroxisome proliferator-activated receptor-γ	Antagonist	5.52	2.6%	P37231
RXRA	Retinoid X receptor-α	Agonist	7.4	5.1%	P19793
RXRB	Retinoid X receptor-β	Agonist	7.7	0.2%	P28702
RXRG	Retinoid X receptor-γ	Agonist	7.54	0.2%	P48443

Broader ChEMBL bioactivity targets: 36 (assay-derived). Sample: Nuclear receptor ROR-gamma, ATP-dependent DNA helicase Q1, RecQ-like DNA helicase BLM, Alpha-2A adrenergic receptor, Retinoic acid receptor RXR-beta, Retinoic acid receptor gamma, Retinoic acid receptor RXR-gamma, Retinoic acid receptor beta, Retinoic acid receptor alpha, D(1A) dopamine receptor.

Bioactivity

ChEMBL activities: 108 potent at pChembl ≥ 5 of 121 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
RXRA	8.57	EC50	2.7	nM	CHEMBL_ACT_5207547
RXRA	8.3	EC50	5.01	nM	CHEMBL_ACT_24986588
RXRB	8.23	Ki	5.9	nM	CHEMBL_ACT_659013
RARA	8.22	EC50	6	nM	CHEMBL_ACT_1108538
RXRG	8.08	Ki	8.3	nM	CHEMBL_ACT_659014
RXRA	8	EC50	10	nM	CHEMBL_ACT_16511009
RXRA	7.85	Kd	14	nM	CHEMBL_ACT_1072803
RXRA	7.85	Ki	14	nM	CHEMBL_ACT_1093034
RXRA	7.8	Ki	16	nM	CHEMBL_ACT_659012
RXRG	7.7	EC50	20	nM	CHEMBL_ACT_1108546
P28705	7.7	EC50	20	nM	CHEMBL_ACT_1283418
RXRA	7.7	EC50	20	nM	CHEMBL_ACT_15249469
RXRA	7.7	EC50	20	nM	CHEMBL_ACT_19452242
RXRG	7.7	EC50	20	nM	CHEMBL_ACT_26116386
P28705	7.7	EC50	20	nM	CHEMBL_ACT_325359
RXRA	7.7	EC50	19.8	nM	CHEMBL_ACT_3423814
RXRA	7.7	EC50	20	nM	CHEMBL_ACT_5294285
P28704	7.68	Kd	21	nM	CHEMBL_ACT_1072804
RXRB	7.68	Ki	21	nM	CHEMBL_ACT_1093035
RXRB	7.68	Ki	21	nM	CHEMBL_ACT_1108542
P28704	7.68	Ki	21	nM	CHEMBL_ACT_1283429
RXRA	7.68	Ki	21	nM	CHEMBL_ACT_2926769
P28704	7.68	Kd	21	nM	CHEMBL_ACT_325361
RXRA	7.66	EC50	22	nM	CHEMBL_ACT_19307697
RXRA	7.64	EC50	23	nM	CHEMBL_ACT_29190610
P28704	7.62	EC50	24	nM	CHEMBL_ACT_1072798
RXRB	7.62	EC50	24	nM	CHEMBL_ACT_1093023
RXRB	7.62	EC50	24	nM	CHEMBL_ACT_18168826
RXRB	7.62	EC50	24	nM	CHEMBL_ACT_18727311
RXRB	7.62	EC50	24	nM	CHEMBL_ACT_19304543

Target pathways

Aggregated over 4 target gene(s): PPARG, RXRA, RXRB, RXRG.

Top Reactome pathways

68 total, by targets touching each:

Pathway	Targets	Genes
Nuclear Receptor transcription pathway	4	PPARG, RXRA, RXRB, RXRG
Signal Transduction	3	RXRA, RXRB, RXRG
PPARA activates gene expression	3	PPARG, RXRA, RXRB
Generic Transcription Pathway	3	RXRA, RXRB, RXRG
Signaling by Retinoic Acid	3	RXRA, RXRB, RXRG
RNA Polymerase II Transcription	3	RXRA, RXRB, RXRG
Gene expression (Transcription)	3	RXRA, RXRB, RXRG
Signaling by Nuclear Receptors	3	RXRA, RXRB, RXRG
Metabolism	2	RXRA, RXRB
Transcriptional regulation of white adipocyte differentiation	2	PPARG, RXRA
Regulation of lipid metabolism by PPARalpha	2	RXRA, RXRB
SUMOylation of intracellular receptors	2	PPARG, RXRA
Metabolism of lipids	2	RXRA, RXRB
NR1H2 and NR1H3-mediated signaling	2	RXRA, RXRB
NR1H2 & NR1H3 regulate gene expression linked to lipogenesis	2	RXRA, RXRB
NR1H3 & NR1H2 regulate gene expression linked to cholesterol transport and efflux	2	RXRA, RXRB
NR1H2 & NR1H3 regulate gene expression to limit cholesterol uptake	2	RXRA, RXRB
NR1H2 & NR1H3 regulate gene expression linked to triglyceride lipolysis in adipose	2	RXRA, RXRB
NR1H2 & NR1H3 regulate gene expression to control bile acid homeostasis	2	RXRA, RXRB
NR1H2 & NR1H3 regulate gene expression linked to gluconeogenesis	2	RXRA, RXRB
MLL4 and MLL3 complexes regulate expression of PPARG target genes in adipogenesis and hepatic steatosis	2	PPARG, RXRA
Transcriptional regulation of brown and beige adipocyte differentiation by EBF2	2	PPARG, RXRA
Developmental Biology	1	RXRA
R-HSA-1368082	1	RXRA
BMAL1:CLOCK,NPAS2 activates circadian expression	1	RXRA
Mitochondrial biogenesis	1	RXRA
Recycling of bile acids and salts	1	RXRA
Regulation of cholesterol biosynthesis by SREBP (SREBF)	1	RXRA
Organelle biogenesis and maintenance	1	RXRA
Synthesis of bile acids and bile salts	1	RXRA

Dominant GO biological processes

GO term	Targets
cell differentiation	4
positive regulation of transcription by RNA polymerase II	4
retinoic acid receptor signaling pathway	4
regulation of DNA-templated transcription	4
hormone-mediated signaling pathway	3
mRNA transcription by RNA polymerase II	3
positive regulation of DNA-templated transcription	3
nervous system development	3
nuclear receptor-mediated steroid hormone signaling pathway	3
negative regulation of transcription by RNA polymerase II	2
positive regulation of cholesterol efflux	2
monocyte differentiation	2
peroxisome proliferator activated receptor signaling pathway	2
positive regulation of lipid metabolic process	2
cell maturation	2

Indications & clinical

Indications

26 indications (3 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
primary cutaneous T-cell non-Hodgkin lymphoma	4	MONDO:0000607	EFO:0002913
neoplasm	4	MONDO:0005070	EFO:0000616
T-cell non-Hodgkin lymphoma	4	MONDO:0015760	MONDO:0015760
non-small cell lung carcinoma	3	MONDO:0005233	EFO:0003060
lymphoma	3	MONDO:0005062	EFO:0000574
anaplastic large cell lymphoma	3	MONDO:0020325	EFO:0003032
schizoaffective disorder	3	MONDO:0005487	EFO:0005411
mycosis fungoides	3	MONDO:0009691	EFO:1001051
acute myeloid leukemia	2	MONDO:0018874	EFO:0000222
alopecia areata	2	MONDO:0005340	EFO:0004192
psoriasis	2	MONDO:0005083	EFO:0000676
parapsoriasis	2	MONDO:0006592	EFO:1000747
Sezary syndrome	2	MONDO:0017844	EFO:1000785
myelodysplastic syndrome	2	MONDO:0018881	EFO:0000198
leukemia	2	MONDO:0005059	EFO:0000565
breast neoplasm	2	MONDO:0021100	MONDO:0007254
lung neoplasm	2	MONDO:0021117	MONDO:0008903
myelodysplastic/myeloproliferative disease	2	MONDO:0020077	MONDO:0020077
Alzheimer disease	2	MONDO:0004975	MONDO:0004975
plasma cell myeloma	1	MONDO:0009693	EFO:0001378
ACTH-dependent Cushing syndrome	1	MONDO:0020528	EFO:1001110
ductal breast carcinoma in situ	1	MONDO:0005023	EFO:0000432
triple-negative breast carcinoma	1	MONDO:0005494	EFO:0005537

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

Clinical trials

Total trials: 38.

Phase distribution

Phase	Trials
PHASE2	14
PHASE1	11
PHASE3	6
PHASE1/PHASE2	4
PHASE4	1
EARLY_PHASE1	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT01007448	PHASE4	COMPLETED	Study Evaluating Two Dose Levels of Targretin Capsules in Participants With Refractory Cutaneous T-Cell Lymphoma (CTCL)
NCT00050973	PHASE3	COMPLETED	Evaluation of Efficacy, Safety and Tolerability of Targretin Capsules in Patients With Advanced or Metastatic Non-small Cell Lung Cancer
NCT00056056	PHASE3	TERMINATED	Ultraviolet Light Therapy Using Methoxsalen With or Without Bexarotene in Treating Patients With Mycosis Fungoides
NCT00141947	PHASE3	UNKNOWN	Bexarotene Treatment in Schizophrenia
NCT00534898	PHASE3	WITHDRAWN	Bexarotene and Fenretinide as Addition to Antipsychotic Treatment in Schizophrenia Patients
NCT00535574	PHASE3	COMPLETED	Bexarotene Augmentation of Antipsychotic Treatment for Chronic Schizophrenia
NCT01578499	PHASE3	COMPLETED	A Phase 3 Trial of Brentuximab Vedotin(SGN-35) Versus Physician’s Choice (Methotrexate or Bexarotene) in Participants With CD30-Positive Cutaneous T-Cell Lymphoma (ALCANZA Study)
NCT00002212	PHASE2	COMPLETED	A Study of Targretin Capsules in Patients With AIDS-Related Kaposi’s Sarcoma
NCT00003752	PHASE2	COMPLETED	Bexarotene in Treating Patients With Metastatic Breast Cancer
NCT00030589	PHASE2	UNKNOWN	Chemotherapy and Photodynamic Therapy in Treating Patients With Cutaneous T-Cell Lymphoma
NCT00030849	PHASE2	COMPLETED	Bexarotene and Interferon Alfa in Treating Patients With Cutaneous T-Cell Lymphoma
NCT00125359	PHASE2	COMPLETED	Study of Tarceva and Targretin Oral Capsules in Patients With Advanced Lung Cancer
NCT00151008	PHASE2	COMPLETED	Bexarotene With Narrow-Band UVB for Psoriasis
NCT00153842	PHASE1/PHASE2	TERMINATED	A Study of Paclitaxel and Carboplatin in Combination With Bexarotene Oral Capsules in Patients With Advanced Lung Cancer
NCT00178841	PHASE2	COMPLETED	Combination Drug Study of Bexarotene and Rosiglitazone to Treat CTCL
NCT00238628	PHASE1/PHASE2	COMPLETED	A Phase I/II Study of Bexarotene in Combination With ZD1839 (IRESSA®) in the Treatment of Non-Small Cell Lung Cancer
NCT00306969	PHASE1/PHASE2	COMPLETED	Study of Oral Bexarotene in Combination With Photopheresis for Treatment of Cutaneous T-Cell Lymphoma
NCT00322296	PHASE2	TERMINATED	Phase II Trial to Evaluate the Efficacy of Topical Bexarotene Gel in Patients With Parapsoriasis
NCT00411632	PHASE2	COMPLETED	BATTLE Program: Tarceva and Targretin in Patients With Advanced Non-Small Cell Lung Cancer (NSCLC)
NCT00425477	PHASE2	COMPLETED	Bexarotene and GM-CSF in Treating Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia
NCT00514293	PHASE2	UNKNOWN	Bexarotene, Tretinoin, and Combination Chemotherapy in Treating Patients With Stage III or Stage IV Non-Small Cell Lung Cancer
NCT00615784	PHASE2	TERMINATED	Phase II Study of Bexarotene in Patients With Acute Myeloid Leukemia
NCT00660231	PHASE2	COMPLETED	Gemcitabine and Bexarotene in Treating Patients With Progressive or Refractory Stage IB, Stage II, Stage III, or Stage IV Cutaneous T-cell Non-Hodgkin Lymphoma
NCT00845351	PHASE1/PHASE2	UNKNOWN	Preoperative Bexarotene Treatment for Cushing’s Disease
NCT01782742	PHASE2	COMPLETED	Bexarotene Amyloid Treatment for Alzheimer’s Disease
NCT05296304	PHASE1	RECRUITING	A Study of Bexarotene Combined With Radiotherapy in People With Mycosis Fungoides
NCT00055991	PHASE1	COMPLETED	Bexarotene in Preventing Breast Cancer in Women at Genetic Risk
NCT00127101	PHASE1	TERMINATED	An Investigational Study of a Histone Deacetylase (HDAC) Inhibitor Plus Targretin in Cutaneous T-Cell Lymphoma Patients (0683-016)(TERMINATED)
NCT00316030	PHASE1	COMPLETED	Study of Bexarotene in Patients With Acute Myeloid Leukemia
NCT01001143	PHASE1	COMPLETED	Intravenous (IV) Decitabine and Oral Bexarotene for Acute Myelogenous Leukemia (AML)
NCT01116622	PHASE1	COMPLETED	Erlotinib (Tarceva) and Bexarotene (Targretin) Oral Capsules in Advanced Cancers of the Aerodigestive Tract
NCT01134341	PHASE1	COMPLETED	Pralatrexate and Bexarotene in Patients With Relapsed or Refractory Cutaneous T-cell Lymphoma
NCT01504490	PHASE1	TERMINATED	Phase I Study of CS-7017 and Bexarotene
NCT02061878	PHASE1	COMPLETED	A Study to Evaluate the Effect of Bexarotene on Beta-Amyloid and Apolipoprotein E Metabolism in Healthy Subjects
NCT03323658	PHASE1	COMPLETED	Bexarotene in Preventing Breast Cancer in Patients at High Risk for Breast Cancer
NCT04664829	PHASE1	COMPLETED	The Role of Bexarotene in Inducing Susceptibility to Chemotherapy in Metastatic TNBC
NCT00718770	EARLY_PHASE1	COMPLETED	A Pilot Clinical Trial for Poorly Differentiated Thyroid Cancer - Correlation to Retinoid and Peroxisome-proliferator-activated Receptor (PPARy) Expression
NCT05106192	Not specified	WITHDRAWN	Triamcinolone Acetonide Injections in Primary Cutaneous Lymphoma Plaques With a Novel Needle-free Drug-delivery System.

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.

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

Molecule	Source	Status	Shared targets
IRX-4204	ChEMBL	Phase 2	PPARG, RXRA, RXRB, RXRG
PIRINIXIC ACID	ChEMBL	Phase 2	PPARG, RXRA, RXRB, RXRG
ALITRETINOIN	ChEMBL + PubChem	Phase 4 (approved)	RXRA, RXRB, RXRG
CALCITRIOL	ChEMBL + PubChem	Phase 4 (approved)	RXRA, RXRB, RXRG
OXAPROZIN	ChEMBL + PubChem	Phase 4 (approved)	RXRA, RXRB, RXRG
TRETINOIN	ChEMBL + PubChem	Phase 4 (approved)	RXRA, RXRB, RXRG
SULINDAC	ChEMBL + PubChem	Phase 4 (approved)	PPARG, RXRA
LEVOTHYROXINE	ChEMBL	Phase 4 (approved)	PPARG, RXRA
PIOGLITAZONE	ChEMBL	Phase 4 (approved)	PPARG, RXRA
ROSIGLITAZONE	ChEMBL	Phase 4 (approved)	PPARG, RXRA
DOCONEXENT	ChEMBL	Phase 3	PPARG, RXRA
ICOSAPENT	ChEMBL	Phase 3	PPARG, RXRA
TIRATRICOL	ChEMBL	Phase 3	PPARG, RXRA
FULVESTRANT	ChEMBL + PubChem	Phase 4 (approved)	PPARG
BENZBROMARONE	ChEMBL	Phase 4 (approved)	PPARG
CANDESARTAN CILEXETIL	ChEMBL	Phase 4 (approved)	PPARG
CANNABIDIOL	ChEMBL	Phase 4 (approved)	PPARG
CARVEDILOL	ChEMBL	Phase 4 (approved)	PPARG
CEFAMANDOLE	ChEMBL	Phase 4 (approved)	PPARG
CEFOTAXIME	ChEMBL	Phase 4 (approved)	PPARG
CEFOXITIN	ChEMBL	Phase 4 (approved)	PPARG
CEFTAZIDIME	ChEMBL	Phase 4 (approved)	PPARG
CEFTRIAXONE	ChEMBL	Phase 4 (approved)	PPARG
CLOBETASOL PROPIONATE	ChEMBL	Phase 4 (approved)	PPARG
DOMPERIDONE	ChEMBL	Phase 4 (approved)	RXRA
EFAVIRENZ	ChEMBL	Phase 4 (approved)	PPARG
ELAFIBRANOR	ChEMBL	Phase 4 (approved)	PPARG
FENOFIBRATE	ChEMBL	Phase 4 (approved)	PPARG
FENOFIBRIC ACID	ChEMBL	Phase 4 (approved)	PPARG
FLUVASTATIN	ChEMBL	Phase 4 (approved)	RXRA
GEMFIBROZIL	ChEMBL	Phase 4 (approved)	PPARG
GLYBURIDE	ChEMBL	Phase 4 (approved)	PPARG
INDOMETHACIN	ChEMBL	Phase 4 (approved)	PPARG
LASOFOXIFENE	ChEMBL	Phase 4 (approved)	PPARG
LIOTHYRONINE	ChEMBL	Phase 4 (approved)	PPARG
LUMIRACOXIB	ChEMBL	Phase 4 (approved)	PPARG
MASOPROCOL	ChEMBL	Phase 4 (approved)	PPARG
MECLOFENAMIC ACID	ChEMBL	Phase 4 (approved)	RXRA
METHYLENE BLUE	ChEMBL	Phase 4 (approved)	PPARG
MONTELUKAST	ChEMBL	Phase 4 (approved)	PPARG
NINTEDANIB	ChEMBL	Phase 4 (approved)	PPARG
PEMAFIBRATE	ChEMBL	Phase 4 (approved)	PPARG
PITAVASTATIN	ChEMBL	Phase 4 (approved)	RXRA
RIMONABANT	ChEMBL	Phase 4 (approved)	PPARG
TELMISARTAN	ChEMBL	Phase 4 (approved)	PPARG
TERIFLUNOMIDE	ChEMBL	Phase 4 (approved)	PPARG
TIPRANAVIR	ChEMBL	Phase 4 (approved)	PPARG
TROGLITAZONE	ChEMBL	Phase 4 (approved)	PPARG
ZAFIRLUKAST	ChEMBL	Phase 4 (approved)	PPARG
ALEGLITAZAR	ChEMBL	Phase 3	PPARG
BALAGLITAZONE	ChEMBL	Phase 3	PPARG
BEZAFIBRATE	ChEMBL	Phase 3	PPARG
CANDESARTAN	ChEMBL	Phase 3	PPARG
GAMOLENIC ACID	ChEMBL	Phase 3	PPARG
IMIGLITAZAR	ChEMBL	Phase 3	PPARG
LANIFIBRANOR	ChEMBL	Phase 3	PPARG
LERIGLITAZONE	ChEMBL	Phase 3	PPARG
LOBEGLITAZONE	ChEMBL	Phase 3	PPARG
MURAGLITAZAR	ChEMBL	Phase 3	PPARG
NAMODENOSON	ChEMBL	Phase 3	PPARG