Balovaptan

Summary

Balovaptan (CHEMBL4297183) is a phase-3 clinical-stage small molecule targeting AVPR1A, AVPR2, and OXTR; indicated across 3 conditions including autism spectrum disorder and post-traumatic stress disorder.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 4 (AVPR1A, AVPR2, OXTR…)
• Indications: 3 conditions
• Clinical trials: 15
• Chemistry: 409.9 Da · C22H24ClN5O

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297183
Name	Balovaptan
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	46200932
Molecular formula	C22H24ClN5O
Molecular weight	409.9
InChIKey	GMPZPHGHNDMRKL-UHFFFAOYSA-N

SMILES: CN1CC2=C(C=CC(=C2)Cl)N3C(=NN=C3C4CCC(CC4)OC5=CC=CC=N5)C1

IUPAC name: 8-chloro-5-methyl-1-(4-pyridin-2-yloxycyclohexyl)-4,6-dihydro-[1,2,4]triazolo[4,3-a][1,4]benzodiazepine

Also known as: Balovaptan, RG-7314, Rg7314, RG7314, RO-5285119, RO5285119, BALOVAPTAN

Patent coverage: 29 distinct patent families (84 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 61 (73%) 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
AVPR1A	V1A receptor	Antagonist	8.59	4.6%	P37288
AVPR2	V2 receptor	Antagonist	5	0%	P30518
OXTR	OT receptor	Antagonist	5.15	0.1%	P30559
HTR2B	5-HT2B receptor	Antagonist	11.57	0.4%	P41595

Broader ChEMBL bioactivity targets: 3 (assay-derived). Sample: Vasopressin V2 receptor, Vasopressin V1a receptor, Voltage-gated inwardly rectifying potassium channel KCNH2.

Bioactivity

ChEMBL activities: 10 potent at pChembl ≥ 5 of 11 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
AVPR1A	9.15	Ki	0.7	nM	CHEMBL_ACT_22399242
AVPR1A	9.15	Ki	0.7	nM	CHEMBL_ACT_22882069
AVPR1A	9.15	Ki	0.7	nM	CHEMBL_ACT_24397600
AVPR1A	9.15	Ki	0.7	nM	CHEMBL_ACT_25943774
AVPR1A	8.52	IC50	3	nM	CHEMBL_ACT_22399274
AVPR1A	8.52	IC50	3	nM	CHEMBL_ACT_22882108
AVPR1A	8.52	IC50	3	nM	CHEMBL_ACT_24397620
AVPR1A	8.52	IC50	3	nM	CHEMBL_ACT_25943844
AVPR2	5.92	Ki	1200	nM	CHEMBL_ACT_24397628
AVPR2	5.8	Ki	1600	nM	CHEMBL_ACT_25944174

Target pathways

Aggregated over 4 target gene(s): AVPR1A, AVPR2, OXTR, HTR2B.

Top Reactome pathways

15 total, by targets touching each:

Pathway	Targets	Genes
Vasopressin-like receptors	3	AVPR1A, AVPR2, OXTR
G alpha (q) signalling events	3	AVPR1A, HTR2B, OXTR
Signal Transduction	1	HTR2B
Signaling by GPCR	1	HTR2B
Class A/1 (Rhodopsin-like receptors)	1	HTR2B
Amine ligand-binding receptors	1	HTR2B
GPCR downstream signalling	1	HTR2B
Serotonin receptors	1	HTR2B
G alpha (s) signalling events	1	AVPR2
Vasopressin regulates renal water homeostasis via Aquaporins	1	AVPR2
GPCR ligand binding	1	HTR2B
Defective AVP does not bind AVPR1A,B and causes neurohypophyseal diabetes insipidus (NDI)	1	AVPR1A
Cargo recognition for clathrin-mediated endocytosis	1	AVPR2
Clathrin-mediated endocytosis	1	AVPR2
Defective AVP does not bind AVPR2 and causes neurohypophyseal diabetes insipidus (NDI)	1	AVPR2

Dominant GO biological processes

GO term	Targets
G protein-coupled receptor signaling pathway	4
signal transduction	4
regulation of systemic arterial blood pressure by vasopressin	3
positive regulation of cell population proliferation	3
cellular response to hormone stimulus	3
positive regulation of vasoconstriction	3
positive regulation of blood pressure	3
positive regulation of systemic arterial blood pressure	2
calcium-mediated signaling	2
telencephalon development	2
maternal aggressive behavior	1
generation of precursor metabolites and energy	1
positive regulation of cytosolic calcium ion concentration	1
negative regulation of female receptivity	1
grooming behavior	1

Indications & clinical

Indications

2 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
autism spectrum disorder	3	MONDO:0005258	EFO:0003756
post-traumatic stress disorder	2	MONDO:0005146	EFO:0001358

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

Clinical trials

Total trials: 15.

Phase distribution

Phase	Trials
PHASE1	10
PHASE2	4
PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03504917	PHASE3	TERMINATED	A Study of Balovaptan in Adults With Autism Spectrum Disorder With a 2-Year Open-Label Extension
NCT01793441	PHASE2	COMPLETED	A Study of RG7314 to Investigate Efficacy and Safety in Individuals With Autism Spectrum Disorders (ASD)
NCT02901431	PHASE2	TERMINATED	A Study to Investigate the Efficacy and Safety of Balovaptan (RO5285119) in Participants With Autism Spectrum Disorder (ASD)
NCT05399550	PHASE2	WITHDRAWN	Study to Evaluate The Safety and Efficacy of Balovaptan in Participants With Acute Ischemic Stroke at a High Risk of Developing Malignant Brain Edema
NCT05401565	PHASE2	COMPLETED	Study To Evaluate The Efficacy And Safety Of Balovaptan In Adults With Post-Traumatic Stress Disorder (PTSD)
NCT01418963	PHASE1	COMPLETED	A Study of RO5285119 in Healthy Volunteers
NCT01708616	PHASE1	COMPLETED	A Study of the Combination of Multiple Doses of RO5285119 With a Single Dose of Risperidone in Healthy Volunteers
NCT01967979	PHASE1	COMPLETED	A Study of the Effect of Multiple Doses of Itraconazole and Fluoxetine on the Pharmacokinetics of a Single Dose of RO5285119 in Healthy Subjects
NCT02179866	PHASE1	COMPLETED	A Single-center Study to Investigate How Quickly and to What Extent a Radioactive Dose of RO5285119 is Absorbed, Metabolized and Eliminated From the Body of Healthy Male Volunteers
NCT03579719	PHASE1	COMPLETED	A Study to Investigate the Effect of Itraconazole on the PK of Multiple Doses of Balovaptan in Healthy Volunteers
NCT03586726	PHASE1	COMPLETED	A Study to Investigate the Effect of Rifampicin on the PK of Multiple Doses of Balovaptin In Healthy Volunteers
NCT03764449	PHASE1	COMPLETED	A Study Investigating the Absolute Oral Bioavailability of Balovaptan After Single and Multiple Daily Oral Doses of Balovaptan in Healthy Volunteers
NCT03912350	PHASE1	WITHDRAWN	The Effect of Hepatic Impairment on the Pharmacokinetics of Balovaptan
NCT04049578	PHASE1	TERMINATED	A Study to Investigate the Pharmacokinetics, Safety, and Tolerability of Balovaptan in Children With Autism Spectrum Disorder
NCT04156646	PHASE1	COMPLETED	A Study to Investigate the Effect of Esomeprazole and the Effect of Food on the Pharmacokinetics of Balovaptan in Healthy Volunteers

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.

482 molecules share ≥1 primary target. Top 100 by shared-target count:

Molecule	Source	Status	Shared targets
Belzutifan	PubChem	Approved	AVPR1A, AVPR2, HTR2B, OXTR
Almotriptan	ChEMBL + PubChem	Phase 4 (approved)	AVPR1A, AVPR2, HTR2B
Clozapine	ChEMBL + PubChem	Phase 4 (approved)	AVPR1A, AVPR2, HTR2B
Crizotinib	ChEMBL + PubChem	Phase 4 (approved)	AVPR1A, AVPR2, HTR2B
Desloratadine	ChEMBL + PubChem	Phase 4 (approved)	AVPR1A, AVPR2, HTR2B
Dihydroergotamine	ChEMBL + PubChem	Phase 4 (approved)	AVPR1A, AVPR2, HTR2B
Olanzapine	ChEMBL + PubChem	Phase 4 (approved)	AVPR1A, AVPR2, HTR2B
OXYTOCIN	ChEMBL + PubChem	Phase 4 (approved)	AVPR1A, AVPR2, OXTR
PIMOZIDE	ChEMBL + PubChem	Phase 4 (approved)	AVPR1A, AVPR2, HTR2B
Pramipexole	ChEMBL + PubChem	Phase 4 (approved)	AVPR1A, AVPR2, HTR2B
Tamsulosin	ChEMBL + PubChem	Phase 4 (approved)	AVPR1A, AVPR2, HTR2B
TEGASEROD	ChEMBL + PubChem	Phase 4 (approved)	AVPR1A, AVPR2, HTR2B
ATOSIBAN	ChEMBL	Phase 4 (approved)	AVPR1A, AVPR2, OXTR
CARBETOCIN	ChEMBL	Phase 4 (approved)	AVPR1A, AVPR2, OXTR
CHLORHEXIDINE	ChEMBL	Phase 4 (approved)	AVPR1A, AVPR2, HTR2B
DESMOPRESSIN	ChEMBL	Phase 4 (approved)	AVPR1A, AVPR2, OXTR
FLUSPIRILENE	ChEMBL	Phase 4 (approved)	AVPR1A, AVPR2, HTR2B
MOZAVAPTAN	ChEMBL	Phase 4 (approved)	AVPR1A, AVPR2, OXTR
NITAZOXANIDE	ChEMBL	Phase 4 (approved)	AVPR1A, AVPR2, HTR2B
PYRVINIUM	ChEMBL	Phase 4 (approved)	AVPR1A, AVPR2, HTR2B
VASOPRESSIN	ChEMBL	Phase 4 (approved)	AVPR1A, AVPR2, OXTR
LIXIVAPTAN	ChEMBL	Phase 3	AVPR1A, AVPR2, OXTR
OTILONIUM BROMIDE	ChEMBL	Phase 3	AVPR1A, AVPR2, HTR2B
BENZETHONIUM	ChEMBL	Phase 2	AVPR1A, AVPR2, HTR2B
DOMIPHEN	ChEMBL	Phase 2	AVPR1A, AVPR2, HTR2B
NELIVAPTAN	ChEMBL	Phase 2	AVPR1A, AVPR2, OXTR
ORNIPRESSIN	ChEMBL	Phase 2	AVPR1A, AVPR2, OXTR
PECAVAPTAN	ChEMBL	Phase 2	AVPR1A, AVPR2, OXTR
SELEPRESSIN	ChEMBL	Phase 2	AVPR1A, AVPR2, OXTR
Aclidinium Bromide	PubChem	Approved	AVPR1A, AVPR2, HTR2B
Afatinib	PubChem	Approved	AVPR1A, AVPR2, HTR2B
Alogliptin	PubChem	Approved	AVPR1A, AVPR2, HTR2B
Apixaban	PubChem	Approved	AVPR1A, AVPR2, HTR2B
Bosentan	PubChem	Approved	AVPR1A, AVPR2, HTR2B
Fidaxomicin	PubChem	Approved	AVPR1A, AVPR2, HTR2B
Fulvestrant	PubChem	Approved	AVPR1A, AVPR2, HTR2B
Glycopyrrolate	PubChem	Approved	AVPR1A, AVPR2, HTR2B
Linagliptin	PubChem	Approved	AVPR1A, AVPR2, HTR2B
Propoxyphene	PubChem	Approved	AVPR1A, AVPR2, HTR2B
Pyrazinamide	PubChem	Approved	AVPR1A, AVPR2, HTR2B
saxagliptin	PubChem	Approved	AVPR1A, AVPR2, HTR2B
Tadalafil	PubChem	Approved	AVPR1A, AVPR2, HTR2B
Tafamidis	PubChem	Approved	AVPR1A, AVPR2, HTR2B
Tiotropium Bromide Monohydrate	PubChem	Approved	AVPR1A, AVPR2, HTR2B
Amiloride	ChEMBL + PubChem	Phase 4 (approved)	AVPR2, HTR2B
Apremilast	ChEMBL + PubChem	Phase 4 (approved)	AVPR1A, HTR2B
CINACALCET	ChEMBL + PubChem	Phase 4 (approved)	AVPR2, HTR2B
Escitalopram	ChEMBL + PubChem	Phase 4 (approved)	AVPR2, HTR2B
Olodaterol	ChEMBL + PubChem	Phase 4 (approved)	AVPR1A, HTR2B
RIFAMPIN	ChEMBL + PubChem	Phase 4 (approved)	AVPR1A, AVPR2
AMIODARONE	ChEMBL	Phase 4 (approved)	AVPR2, HTR2B
AMOXAPINE	ChEMBL	Phase 4 (approved)	AVPR2, HTR2B
ARIPIPRAZOLE	ChEMBL	Phase 4 (approved)	AVPR1A, HTR2B
BALSALAZIDE	ChEMBL	Phase 4 (approved)	AVPR1A, AVPR2
BOSUTINIB	ChEMBL	Phase 4 (approved)	AVPR1A, AVPR2
CLOTRIMAZOLE	ChEMBL	Phase 4 (approved)	AVPR2, HTR2B
CONIVAPTAN	ChEMBL	Phase 4 (approved)	AVPR1A, AVPR2
DIETHYLSTILBESTROL	ChEMBL	Phase 4 (approved)	AVPR1A, HTR2B
IDEBENONE	ChEMBL	Phase 4 (approved)	AVPR2, HTR2B
IPRINDOLE	ChEMBL	Phase 4 (approved)	AVPR2, HTR2B
LASOFOXIFENE	ChEMBL	Phase 4 (approved)	AVPR2, HTR2B
MEFLOQUINE	ChEMBL	Phase 4 (approved)	AVPR2, HTR2B
NEBIVOLOL	ChEMBL	Phase 4 (approved)	AVPR2, HTR2B
PONATINIB	ChEMBL	Phase 4 (approved)	AVPR1A, HTR2B
RIFAXIMIN	ChEMBL	Phase 4 (approved)	AVPR1A, AVPR2
RIMONABANT	ChEMBL	Phase 4 (approved)	AVPR1A, HTR2B
RITONAVIR	ChEMBL	Phase 4 (approved)	AVPR1A, HTR2B
SAQUINAVIR	ChEMBL	Phase 4 (approved)	AVPR1A, HTR2B
SERTINDOLE	ChEMBL	Phase 4 (approved)	AVPR2, HTR2B
SERTRALINE	ChEMBL	Phase 4 (approved)	AVPR2, HTR2B
SUNITINIB	ChEMBL	Phase 4 (approved)	AVPR2, HTR2B
TERFENADINE	ChEMBL	Phase 4 (approved)	AVPR2, HTR2B
THIOTHIXENE	ChEMBL	Phase 4 (approved)	AVPR2, HTR2B
TOLVAPTAN	ChEMBL	Phase 4 (approved)	AVPR1A, AVPR2
TRIFLUOPERAZINE	ChEMBL	Phase 4 (approved)	AVPR2, HTR2B
NOLASIBAN	ChEMBL	Phase 3	AVPR1A, OXTR
QUERCETIN	ChEMBL	Phase 3	AVPR2, HTR2B
RETOSIBAN	ChEMBL	Phase 3	AVPR2, OXTR
SEMAXANIB	ChEMBL	Phase 3	AVPR1A, OXTR
EPELSIBAN	ChEMBL	Phase 2	AVPR2, OXTR
RELCOVAPTAN	ChEMBL	Phase 2	AVPR1A, AVPR2
ZOTEPINE	ChEMBL	Phase 2	AVPR1A, HTR2B
Abiraterone	PubChem	Approved	AVPR1A, AVPR2
acetylcysteine	PubChem	Approved	AVPR1A, AVPR2
Acyclovir	PubChem	Approved	AVPR1A, AVPR2
Allopurinol	PubChem	Approved	AVPR1A, AVPR2
aminolevulinic acid	PubChem	Approved	AVPR1A, AVPR2
Anagrelide	PubChem	Approved	AVPR1A, AVPR2
Aprepitant	PubChem	Approved	AVPR1A, AVPR2
Binimetinib	PubChem	Approved	AVPR1A, HTR2B
chenodiol	PubChem	Approved	AVPR1A, HTR2B
Clofarabine	PubChem	Approved	AVPR1A, AVPR2
Dacarbazine	PubChem	Approved	AVPR1A, AVPR2
Desoxycorticosterone Pivalate	PubChem	Approved	AVPR1A, AVPR2
Didanosine	PubChem	Approved	AVPR1A, AVPR2
Erythromycin	PubChem	Approved	AVPR1A, AVPR2
Ethambutol	PubChem	Approved	AVPR1A, AVPR2
Ganciclovir	PubChem	Approved	AVPR1A, AVPR2
Gefitinib	PubChem	Approved	AVPR1A, AVPR2
Imipenem	PubChem	Approved	AVPR1A, HTR2B