Vamorolone

Summary

Vamorolone (CHEMBL2348780) is an approved small-molecule anti-inflammatory drug (ATC H02AB18) targeting NR3C1; indicated across 3 conditions including duchenne muscular dystrophy and ulcerative colitis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: H02AB18
• Targets: 1 (NR3C1)
• Indications: 3 conditions
• Clinical trials: 12
• Chemistry: 356.5 Da · C22H28O4

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2348780
Name	Vamorolone
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	3035000
ChEBI	CHEBI:228304
ATC	H02AB18
Molecular formula	C22H28O4
Molecular weight	356.5
InChIKey	ZYTXTXAMMDTYDQ-DGEXFFLYSA-N

SMILES: C[C@@H]1C[C@H]2[C@@H]3CCC4=CC(=O)C=C[C@@]4(C3=CC[C@@]2([C@]1(C(=O)CO)O)C)C

IUPAC name: (8S,10S,13S,14S,16R,17R)-17-hydroxy-17-(2-hydroxyacetyl)-10,13,16-trimethyl-7,8,12,14,15,16-hexahydro-6H-cyclopenta[a]phenanthren-3-one

ChEBI definition: A 3-oxo-Δ1,Δ4-steroid that is pregna-1,4,9(11)-trien-17-ol substituted by oxo, oxo, and hydroxy groups at positions 3, 20, and 21, respectively. It is approved by the FDA for the treatment of patients with Duchenne muscular dystrophy aged 2 years and older.

Pharmacological roles (ChEBI): anti-inflammatory drug, immunosuppressive agent, glucocorticoid receptor agonist.

Also known as: Agamree, Vamorolona, Vamorolone, Vamorolone acetate, VBP-15, Vbp-15 free alcohol, VAMOROLONE

Patent coverage: 69 distinct patent families (151 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
NR3C1	Glucocorticoid receptor	Binding		0.9%	P04150

Broader ChEMBL bioactivity targets: 2 (assay-derived). Sample: Nuclear factor NF-kappa-B complex, Voltage-gated inwardly rectifying potassium channel KCNH2.

Bioactivity

ChEMBL activities: 1 potent at pChembl ≥ 5 of 2 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
NFKB1	7.18	IC50	65.9	nM	CHEMBL_ACT_25878325

Target pathways

Aggregated over 1 target gene(s): NR3C1.

Top Reactome pathways

8 total, by targets touching each:

Pathway	Targets	Genes
HSP90 chaperone cycle for steroid hormone receptors (SHR) in the presence of ligand	1	NR3C1
Nuclear Receptor transcription pathway	1	NR3C1
SUMOylation of intracellular receptors	1	NR3C1
PTK6 Expression	1	NR3C1
Regulation of RUNX2 expression and activity	1	NR3C1
FOXO-mediated transcription of oxidative stress, metabolic and neuronal genes	1	NR3C1
Potential therapeutics for SARS	1	NR3C1
Regulation of NPAS4 gene transcription	1	NR3C1

Dominant GO biological processes

GO term	Targets
negative regulation of transcription by RNA polymerase II	1
regulation of gluconeogenesis	1
chromatin organization	1
regulation of DNA-templated transcription	1
regulation of transcription by RNA polymerase II	1
apoptotic process	1
chromosome segregation	1
signal transduction	1
glucocorticoid metabolic process	1
response to wounding	1
gene expression	1
microglia differentiation	1
adrenal gland development	1
nuclear receptor-mediated steroid hormone signaling pathway	1
regulation of glucocorticoid biosynthetic process	1

Indications & clinical

Indications

3 indications (2 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
Duchenne muscular dystrophy	4	MONDO:0010679	MONDO:0010311
ulcerative colitis	1	MONDO:0005101	EFO:0000729

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

Clinical trials

Total trials: 12.

Phase distribution

Phase	Trials
PHASE2	6
PHASE1	2
Not specified	2
PHASE4	1
PHASE1/PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06713135	PHASE4	ACTIVE_NOT_RECRUITING	A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy
NCT02760264	PHASE2	COMPLETED	A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
NCT02760277	PHASE2	COMPLETED	An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
NCT03038399	PHASE2	COMPLETED	Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
NCT03439670	PHASE2	COMPLETED	A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
NCT04348890	PHASE1/PHASE2	WITHDRAWN	Proof of Concept Trial of Vamorolone in Pediatric Ulcerative Colitis
NCT05166109	PHASE2	COMPLETED	A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD)
NCT05185622	PHASE2	COMPLETED	A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)
NCT06649409	PHASE1	COMPLETED	Evaluation of Vamorolone Mineralocorticoid Receptor Antagonism in Healthy Subjects
NCT06689527	PHASE1	COMPLETED	Evaluation of Vamorolone CYP3A4 Induction on Midazolam (a Sensitive CYP 3A4 Substrate) Pharmacokinetics
NCT03863119	Not specified	AVAILABLE	Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
NCT06564974	Not specified	RECRUITING	Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT

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.

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

Molecule	Source	Status	Shared targets
FULVESTRANT	ChEMBL + PubChem	Phase 4 (approved)	NR3C1
PODOFILOX	ChEMBL + PubChem	Phase 4 (approved)	NR3C1
REGORAFENIB	ChEMBL + PubChem	Phase 4 (approved)	NR3C1
ACENOCOUMAROL	ChEMBL	Phase 4 (approved)	NR3C1
ALCLOMETASONE DIPROPIONATE	ChEMBL	Phase 4 (approved)	NR3C1
AMCINONIDE	ChEMBL	Phase 4 (approved)	NR3C1
AMIODARONE	ChEMBL	Phase 4 (approved)	NR3C1
APREPITANT	ChEMBL	Phase 4 (approved)	NR3C1
AURANOFIN	ChEMBL	Phase 4 (approved)	NR3C1
BAZEDOXIFENE	ChEMBL	Phase 4 (approved)	NR3C1
BECLOMETHASONE DIPROPIONATE	ChEMBL	Phase 4 (approved)	NR3C1
BENZBROMARONE	ChEMBL	Phase 4 (approved)	NR3C1
BETAMETHASONE	ChEMBL	Phase 4 (approved)	NR3C1
BETAMETHASONE DIPROPIONATE	ChEMBL	Phase 4 (approved)	NR3C1
BETAMETHASONE PHOSPHORIC ACID	ChEMBL	Phase 4 (approved)	NR3C1
BETAMETHASONE VALERATE	ChEMBL	Phase 4 (approved)	NR3C1
BUDESONIDE	ChEMBL	Phase 4 (approved)	NR3C1
BUTOCONAZOLE	ChEMBL	Phase 4 (approved)	NR3C1
CANDESARTAN CILEXETIL	ChEMBL	Phase 4 (approved)	NR3C1
CANRENONE	ChEMBL	Phase 4 (approved)	NR3C1
CHLORMADINONE	ChEMBL	Phase 4 (approved)	NR3C1
CICLESONIDE	ChEMBL	Phase 4 (approved)	NR3C1
CLOBETASOL PROPIONATE	ChEMBL	Phase 4 (approved)	NR3C1
CLOFAZIMINE	ChEMBL	Phase 4 (approved)	NR3C1
CLOTRIMAZOLE	ChEMBL	Phase 4 (approved)	NR3C1
CORTISONE	ChEMBL	Phase 4 (approved)	NR3C1
DANAZOL	ChEMBL	Phase 4 (approved)	NR3C1
DAUNORUBICIN	ChEMBL	Phase 4 (approved)	NR3C1
DEFLAZACORT	ChEMBL	Phase 4 (approved)	NR3C1
DESOGESTREL	ChEMBL	Phase 4 (approved)	NR3C1
DESONIDE	ChEMBL	Phase 4 (approved)	NR3C1
DESOXIMETASONE	ChEMBL	Phase 4 (approved)	NR3C1
DESOXYCORTICOSTERONE PIVALATE	ChEMBL	Phase 4 (approved)	NR3C1
DEXAMETHASONE	ChEMBL	Phase 4 (approved)	NR3C1
DEXTROTHYROXINE	ChEMBL	Phase 4 (approved)	NR3C1
DIENESTROL	ChEMBL	Phase 4 (approved)	NR3C1
DIETHYLSTILBESTROL	ChEMBL	Phase 4 (approved)	NR3C1
DIFLORASONE DIACETATE	ChEMBL	Phase 4 (approved)	NR3C1
DOXORUBICIN	ChEMBL	Phase 4 (approved)	NR3C1
DROSPIRENONE	ChEMBL	Phase 4 (approved)	NR3C1
ECONAZOLE	ChEMBL	Phase 4 (approved)	NR3C1
EFAVIRENZ	ChEMBL	Phase 4 (approved)	NR3C1
ENZALUTAMIDE	ChEMBL	Phase 4 (approved)	NR3C1
EPLERENONE	ChEMBL	Phase 4 (approved)	NR3C1
ESTRADIOL	ChEMBL	Phase 4 (approved)	NR3C1
ESTRIOL	ChEMBL	Phase 4 (approved)	NR3C1
ETHINYL ESTRADIOL	ChEMBL	Phase 4 (approved)	NR3C1
EXEMESTANE	ChEMBL	Phase 4 (approved)	NR3C1
FELODIPINE	ChEMBL	Phase 4 (approved)	NR3C1
FLUDROCORTISONE	ChEMBL	Phase 4 (approved)	NR3C1
FLUNISOLIDE	ChEMBL	Phase 4 (approved)	NR3C1
FLUOCINOLONE ACETONIDE	ChEMBL	Phase 4 (approved)	NR3C1
FLUOCINONIDE	ChEMBL	Phase 4 (approved)	NR3C1
FLUOROMETHOLONE	ChEMBL	Phase 4 (approved)	NR3C1
FLUOXYMESTERONE	ChEMBL	Phase 4 (approved)	NR3C1
FLURANDRENOLIDE	ChEMBL	Phase 4 (approved)	NR3C1
FLUTICASONE FUROATE	ChEMBL	Phase 4 (approved)	NR3C1
FLUTICASONE PROPIONATE	ChEMBL	Phase 4 (approved)	NR3C1
GLUCAGON	ChEMBL	Phase 4 (approved)	NR3C1
HALCINONIDE	ChEMBL	Phase 4 (approved)	NR3C1