Valbenazine

Summary

Valbenazine (CHEMBL2364639) is an approved small molecule (ATC N07XX13) targeting SLC18A2; indicated across 5 conditions including movement disorder and huntington disease.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: N07XX13
• Targets: 1 (SLC18A2)
• Indications: 5 conditions
• Clinical trials: 31
• Chemistry: 418.6 Da · C24H38N2O4

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2364639
Name	Valbenazine
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	24795069
ATC	N07XX13
Molecular formula	C24H38N2O4
Molecular weight	418.6
InChIKey	GEJDGVNQKABXKG-CFKGEZKQSA-N

SMILES: CC(C)C[C@@H]1CN2CCC3=CC(=C(C=C3[C@H]2C[C@H]1OC(=O)[C@H](C(C)C)N)OC)OC

IUPAC name: [(2R,3R,11bR)-9,10-dimethoxy-3-(2-methylpropyl)-2,3,4,6,7,11b-hexahydro-1H-benzo[a]quinolizin-2-yl] (2S)-2-amino-3-methylbutanoate

Also known as: Mt-5199, NBI-98854, Valbenazina, Valbenazine, VALBENAZINE, valbenazine

Parent form; salt/anhydrous children: CHEMBL3707248

Patent coverage: 186 distinct patent families (495 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 317 (64%) 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
SLC18A2	Vesicular monoamine transporter 2	Inhibition	6.73	0%	Q05940

Broader ChEMBL bioactivity targets: 6 (assay-derived). Sample: Thromboxane A2 receptor, 5-hydroxytryptamine receptor 1A, Prostaglandin G/H synthase 1, Mu-type opioid receptor, Voltage-gated inwardly rectifying potassium channel KCNH2, Synaptic vesicular amine transporter.

Bioactivity

ChEMBL activities: 4 potent at pChembl ≥ 5 of 7 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
Q01827	6.34	AC50	460	nM	CHEMBL_ACT_25197399
HTR1A	5.85	AC50	1410	nM	CHEMBL_ACT_25165442
KCNH2	5.77	IC50	1700	nM	CHEMBL_ACT_23243518
PTGS1	5.26	AC50	5440	nM	CHEMBL_ACT_25205642

Target pathways

Aggregated over 1 target gene(s): SLC18A2.

Top Reactome pathways

4 total, by targets touching each:

Pathway	Targets	Genes
Serotonin Neurotransmitter Release Cycle	1	SLC18A2
Norepinephrine Neurotransmitter Release Cycle	1	SLC18A2
Dopamine Neurotransmitter Release Cycle	1	SLC18A2
SLC-mediated transport of neurotransmitters	1	SLC18A2

Dominant GO biological processes

GO term	Targets
response to amphetamine	1
serotonin secretion by mast cell	1
histamine secretion by mast cell	1
neurotransmitter transport	1
chemical synaptic transmission	1
locomotory behavior	1
response to toxic substance	1
post-embryonic development	1
aminergic neurotransmitter loading into synaptic vesicle	1
obsolete monoamine transport	1
obsolete dopamine transport	1
serotonin uptake	1
histamine uptake	1
neurotransmitter loading into synaptic vesicle	1
somato-dendritic dopamine secretion	1

Indications & clinical

Indications

5 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
movement disorder	3	MONDO:0005395	EFO:0004280
Huntington disease	3	MONDO:0007739	MONDO:0007739
Tourette syndrome	2	MONDO:0007661	EFO:0004895
trichotillomania	2	MONDO:0013189	HP:0012167

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

Clinical trials

Total trials: 31.

Phase distribution

Phase	Trials
PHASE2	14
PHASE3	9
PHASE4	5
PHASE1	2
PHASE2/PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07105111	PHASE4	RECRUITING	A Study to Evaluate the Effectiveness of Valbenazine in Adult Participants With Tardive Dyskinesia (TD) Who Remain Symptomatic While Receiving or After Stopping a Vesicular Monoamine Transporter 2 (VMAT2) Inhibitor
NCT03698331	PHASE4	COMPLETED	The Potential for Clinical Dependence and Withdrawal Symptoms Associated With Valbenazine
NCT03891862	PHASE4	COMPLETED	Persistence of Effect and Safety of Valbenazine for the Treatment of Tardive Dyskinesia
NCT05859698	PHASE4	COMPLETED	Study of the Effectiveness of Valbenazine on Patient- and Clinician-Reported Outcomes in Participants With Tardive Dyskinesia
NCT06107829	PHASE4	WITHDRAWN	Valbenazine Treatment of Tardive Dyskinesia in Adults With Intellectual/Developmental Disabilities
NCT04400331	PHASE3	ACTIVE_NOT_RECRUITING	Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated With Huntington Disease
NCT06312189	PHASE3	ENROLLING_BY_INVITATION	Long-term Study to Evaluate Safety and Tolerability of Valbenazine in Participants With Chorea Associated With Huntington Disease in Canada
NCT02274558	PHASE3	COMPLETED	A Phase 3 Study of NBI-98854 for the Treatment of Tardive Dyskinesia
NCT02405091	PHASE3	COMPLETED	Safety and Tolerability Study of NBI-98854 for the Treatment of Tardive Dyskinesia
NCT02736955	PHASE3	COMPLETED	Rollover Study for Continuing Valbenazine (NBI-98854) Administration for the Treatment of Tardive Dyskinesia
NCT03176771	PHASE2/PHASE3	COMPLETED	Efficacy and Safety of MT-5199 in Subjects With Tardive Dyskinesia
NCT04102579	PHASE3	COMPLETED	Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Chorea Associated With Huntington Disease
NCT05110157	PHASE3	COMPLETED	Journey Study: Evaluate the Efficacy, Safety, and Tolerability of Valbenazine as Adjunctive Treatment for Schizophrenia
NCT05206513	PHASE3	COMPLETED	Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia Due to Cerebral Palsy
NCT05654870	PHASE3	TERMINATED	Study to Evaluate the Efficacy, Safety, and Tolerability of Valbenazine as an Adjunctive Treatment for Schizophrenia
NCT05207085	PHASE2	RECRUITING	Efficacy of Valbenazine for the Treatment of Trichotillomania in Adults
NCT06771323	PHASE2	RECRUITING	Safety and Effectiveness of Valbenazine as Adjunct Therapy to Botulinum Toxin Injections in Cervical Dystonia
NCT07111988	PHASE2	NOT_YET_RECRUITING	Valbenazine in Obsessive Compulsive Disorder
NCT01267188	PHASE2	COMPLETED	Efficacy and Safety of NBI-98854 in Subjects With Tardive Dyskinesia
NCT01393600	PHASE2	COMPLETED	NBI-98854 for the Treatment of Tardive Dyskinesia in Subjects With Schizophrenia or Schizoaffective Disorder
NCT01688037	PHASE2	COMPLETED	NBI-98854 for the Treatment of Tardive Dyskinesia in Subjects With Schizophrenia or Schizoaffective Disorder (KINECT Study)
NCT01733121	PHASE2	COMPLETED	NBI-98854 Dose Titration Study for the Treatment of Tardive Dyskinesia
NCT02581865	PHASE2	COMPLETED	Safety and Efficacy Study of NBI-98854 in Adults With Tourette Syndrome
NCT02679079	PHASE2	COMPLETED	Safety and Efficacy Study of NBI-98854 in Children and Adolescents With Tourette Syndrome
NCT02879578	PHASE2	COMPLETED	Safety and Tolerability Study of NBI-98854 for the Treatment of Subjects With Tourette Syndrome
NCT03325010	PHASE2	COMPLETED	Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome
NCT03444038	PHASE2	COMPLETED	Open-Label Safety and Tolerability Study of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome
NCT03530293	PHASE2	TERMINATED	Safety and Efficacy of NBI-98854 in Pediatric Subjects With Tourette Syndrome
NCT03732534	PHASE2	TERMINATED	Rollover Study for Continuing NBI-98854 Administration in Pediatric Subjects With Tourette Syndrome
NCT02256475	PHASE1	COMPLETED	Safety, Pharmacokinetics, and Pharmacodynamics of NBI-98854 in Children and Adolescents With Tourette Syndrome
NCT05053321	PHASE1	WITHDRAWN	Reduction of Demoralization in Patients With Tardive Dyskinesia After Treatment With Valbenazine

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.

6 molecules share ≥1 primary target. Top 6 by shared-target count:

Molecule	Source	Status	Shared targets
RESERPINE	ChEMBL + PubChem	Phase 4 (approved)	SLC18A2
TETRABENAZINE	ChEMBL + PubChem	Phase 4 (approved)	SLC18A2
KETANSERIN	ChEMBL	Phase 4 (approved)	SLC18A2
SEROTONIN	ChEMBL + PubChem	Phase 3 (approved)	SLC18A2
FLORBENAZINE	ChEMBL	Phase 2	SLC18A2
LOBELINE	ChEMBL	Phase 2	SLC18A2