Retinol

Summary

Retinol (CHEMBL986) is an approved small molecule (ATC D10AD02) targeting NR2C2; indicated across 15 conditions including acne and eye disorder.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: D10AD02 (+3 more)
• Targets: 1 (NR2C2)
• Indications: 15 conditions
• Clinical trials: 82
• Chemistry: 286.5 Da · C20H30O

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL986
Name	Retinol
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	445354
ChEBI	CHEBI:17336
ATC	D10AD02, S01XA02, A11CA01, R01AX02
Molecular formula	C20H30O
Molecular weight	286.5
InChIKey	FPIPGXGPPPQFEQ-OVSJKPMPSA-N

SMILES: CC1=C(C(CCC1)(C)C)/C=C/C(=C/C=C/C(=C/CO)/C)/C

IUPAC name: (2E,4E,6E,8E)-3,7-dimethyl-9-(2,6,6-trimethylcyclohexen-1-yl)nona-2,4,6,8-tetraen-1-ol

ChEBI definition: A retinol in which all four exocyclic double bonds have E- (trans-) geometry.

Other ChEBI roles (chemical / environmental): human metabolite, mouse metabolite, plant metabolite.

Also known as: Arovit, Nepalm, NSC-122759, Retinol, Retinol (vit a), Ro-a-vit, Vitamin a, Vitamin a 1, Vitamin A, retinol, SID17389530, SID26747393

Parent form; salt/anhydrous children: CHEMBL5314606

Patent coverage: 128,473 distinct patent families (277,603 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
NR2C2	Testicular receptor 4	Full agonist	4.71	1.1%	P49116

Broader ChEMBL bioactivity targets: 16 (assay-derived). Sample: Beta-lactoglobulin, Microtubule-associated protein tau, Ubiquitin carboxyl-terminal hydrolase 2, Nuclear receptor ROR-gamma, 4’-phosphopantetheinyl transferase ffp, Thyrotropin receptor, Menin/Histone-lysine N-methyltransferase MLL, Amyloid-beta precursor protein, Polyunsaturated fatty acid lipoxygenase ALOX15, Retinol-binding protein 4.

Bioactivity

ChEMBL activities: 9 potent at pChembl ≥ 5 of 24 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
RBP4	8.08	Ki	8.3	nM	CHEMBL_ACT_25901529
MAPK1	7.5	Potency	31.6	nM	CHEMBL_ACT_4542009
P02754	7.44	Kd	36	nM	CHEMBL_ACT_2989045
APP	6.75	IC50	180	nM	CHEMBL_ACT_22754685
RBP4	6.74	Kd	182	nM	CHEMBL_ACT_6276119
SNCA	6.72	IC50	190	nM	CHEMBL_ACT_22754655
NR1I2	5.32	AC50	4800	nM	CHEMBL_ACT_25188374
ALDH1A1	5.22	Potency	5967	nM	CHEMBL_ACT_4155187
USP2	5	Potency	10000	nM	CHEMBL_ACT_4719813

Target pathways

Aggregated over 1 target gene(s): NR2C2.

Top Reactome pathways

2 total, by targets touching each:

Pathway	Targets	Genes
Nuclear Receptor transcription pathway	1	NR2C2
Interaction of NuRD complexes with transcription factors	1	NR2C2

Dominant GO biological processes

GO term	Targets
negative regulation of transcription by RNA polymerase II	1
regulation of DNA-templated transcription	1
spermatogenesis	1
nervous system development	1
cell differentiation	1
tumor necrosis factor-mediated signaling pathway	1
positive regulation of embryonic development	1
positive regulation of canonical NF-kappaB signal transduction	1
positive regulation of transcription by RNA polymerase II	1
positive regulation of JNK cascade	1
intracellular receptor signaling pathway	1

Indications & clinical

Indications

15 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
acne	4	MONDO:0011438	EFO:0003894
eye disorder	4	MONDO:0005328	EFO:0003966
malaria	3	MONDO:0005136	EFO:0001068
vitamin A deficiency	3	MONDO:0007016	EFO:1001237
diarrheal disease	3	MONDO:0001673	MONDO:0001673
parasitic infectious disease	3	MONDO:0005135	EFO:0001067
tuberculosis	3	MONDO:0018076	MONDO:0018076
retinitis pigmentosa	3	MONDO:0019200	MONDO:0019200
heart failure	2	MONDO:0005252	EFO:0003144
purpura	2	MONDO:0002610	HP:0000979
severe acute respiratory syndrome	2	MONDO:0005091	MONDO:0100096
skin neoplasm	0	MONDO:0002531	MONDO:0002898

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

Clinical trials

Total trials: 82.

Phase distribution

Phase	Trials
Not specified	33
PHASE4	17
PHASE3	15
PHASE2	8
EARLY_PHASE1	3
PHASE2/PHASE3	2
PHASE1/PHASE2	2
PHASE1	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00168584	PHASE4	COMPLETED	Different Doses of Vitamin A and Childhood Morbidity and Mortality
NCT00168597	PHASE4	COMPLETED	Vitamin A With BCG Vaccine
NCT00168610	PHASE4	UNKNOWN	Vitamin A Supplementation With Bacille Calmette Guerin (BCG) Vaccine
NCT00168623	PHASE4	UNKNOWN	Vitamin A Supplementation With Routine Childhood Vaccines and Mortality and Morbidity
NCT00168636	PHASE4	COMPLETED	Different Doses of Vitamin A Supplementation and Male and Female Morbidity and Mortality
NCT00222547	PHASE4	COMPLETED	Deworming and Enhanced Vitamin A Every 6 Month in Rural Indian Children Aged 1-6
NCT00228254	PHASE4	COMPLETED	Vitamin A and Zinc: Prevention of Pneumonia (VAZPOP) Study
NCT00514891	PHASE4	TERMINATED	Impact of the WHO Recommended Vitamin A Supplementation at Immunisation Contacts
NCT00712647	PHASE4	COMPLETED	Carotene and Retinol Efficacy Trial
NCT00963222	PHASE4	UNKNOWN	The Study of the Effects of Vitamin A on Immune System in Patients With Atherosclerosis
NCT01225289	PHASE4	COMPLETED	Impact of Vitamin A Supplementation on Immune System in Multiple Sclerosis Patients
NCT01405352	PHASE4	UNKNOWN	The Effect of Vitamin A Supplementation on Cytokine Profile in Obesity
NCT01407211	PHASE4	UNKNOWN	Impact of Vitamin A on Gene Expression, in Multiple Sclerosis Patient
NCT01414972	PHASE4	UNKNOWN	The Effect of Vitamin A on Atherosclerosis
NCT01417273	PHASE4	UNKNOWN	Impact of Vitamin A on Multiple Sclerosis (MS)
NCT02906566	PHASE4	COMPLETED	Retinol on Human Skin Aging in East Asian Descent
NCT03152474	PHASE4	COMPLETED	Adrenal Cortical Function and Vitamin A Deficiency in Sepsis
NCT00000114	PHASE3	COMPLETED	Randomized Trial of Vitamin A and Vitamin E Supplementation for Retinitis Pigmentosa
NCT00000116	PHASE3	COMPLETED	Randomized Trial of DHA for Retinitis Pigmentosa Patients Receiving Vitamin A
NCT00114868	PHASE3	COMPLETED	Vitamin A Supplementation in Newborns Study
NCT00133406	PHASE3	UNKNOWN	Long-term Impact and Intervention for Diarrhea in Brazil
NCT00170404	PHASE3	COMPLETED	TB Nutrition, Immunology and Epidemiology
NCT00197743	PHASE3	COMPLETED	Trial of Vitamins in HIV Progression and Transmission
NCT00211341	PHASE3	COMPLETED	Trial of the Impact of Vitamin A on Maternal Mortality
NCT00707785	PHASE3	COMPLETED	Effect of Vitamin A in the Treatment of Neonatal Sepsis and Necrotizing Enterocolitis
NCT00742937	PHASE2/PHASE3	COMPLETED	Impact of Maternal Supplementation With Dual Megadose of Vitamin A
NCT01152931	PHASE3	COMPLETED	Antioxidant Micronutrients in Malaria
NCT01265589	PHASE3	UNKNOWN	Intratracheal Vitamin A Administration With Surfactant for Newborn Respiratory Distress Syndrome
NCT01782001	PHASE3	COMPLETED	Evaluation of Vitamin A and Zinc Supplementation on Malarial Morbidity
NCT02043223	PHASE2/PHASE3	COMPLETED	Stopping Postpartum Vitamin A Supplementation: Missing Concealed Benefit
NCT02319499	PHASE3	COMPLETED	Zinc, Iron, Vitamin A and Psychosocial Care for Child Growth and Development
NCT02699099	PHASE3	COMPLETED	Immunogenicity and Safety Study of GSK Biologicals’ Candidate Malaria Vaccine Given at 6, 7.5 and 9 Months of Age in Co-administration With Measles, Rubella and Yellow Fever (YF) Vaccines Followed by a Booster of the Malaria Vaccine.
NCT02878265	PHASE3	COMPLETED	The Effect of Vitamin A With and Without Zinc and Multivitamin Supplementation on Malaria Morbidity in Ghanaian Children
NCT07577180	PHASE3	COMPLETED	Oral Vitamin A Supplementation for Prevention of Bronchopulmonary Dysplasia in Preterm Infants
NCT05720559	PHASE2	NOT_YET_RECRUITING	Early Blocking Strategy for Metachronous Liver Metastasis of Colorectal Cancer Based on Pre-hepatic CTC Detection
NCT06450925	PHASE2	RECRUITING	Vitamin A Supplementation in Allogeneic Stem Cell Transplantation.
NCT06508099	PHASE2	RECRUITING	Vitamin A and D Supplementation in Allogeneic HCT
NCT00053612	PHASE2	COMPLETED	Vitamin A to Reduce HIV in Vaginal Secretions and Prevent Viral Transmission
NCT00133419	PHASE2	COMPLETED	Micronutrients and Enteric Infections in African Children
NCT01203488	PHASE1/PHASE2	COMPLETED	Vitamin A Supplementation for Extremely-Low-Birth-Weight Infants

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.

3 molecules share ≥1 primary target. Top 3 by shared-target count:

Molecule	Source	Status	Shared targets
CEP-11981	ChEMBL	Phase 2	NR2C2
Sunitinib	PubChem	Approved	NR2C2
tretinoin	PubChem	Approved	NR2C2