Cenobamate

Summary

Cenobamate (CHEMBL3989949) is an approved small molecule (ATC N03AX25) targeting GABRA4 and GABRA6; indicated across 5 conditions including epilepsy and visual epilepsy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: N03AX25
• Targets: 2 (GABRA4, GABRA6)
• Indications: 5 conditions
• Clinical trials: 19
• Chemistry: 267.67 Da · C10H10ClN5O2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3989949
Name	Cenobamate
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	11962412
ATC	N03AX25
Molecular formula	C10H10ClN5O2
Molecular weight	267.67
InChIKey	GFHAXPJGXSQLPT-VIFPVBQESA-N

SMILES: C1=CC=C(C(=C1)[C@H](CN2N=CN=N2)OC(=O)N)Cl

IUPAC name: [(1R)-1-(2-chlorophenyl)-2-(tetrazol-2-yl)ethyl] carbamate

Also known as: Cenobamate, Cenobamato, Ontozry, Xcopri, YKP-3089, YKP3089, CENOBAMATE

Patent coverage: 144 distinct patent families (347 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 292 (84%) 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
GABRA4	GABAA receptor α4 subunit	Positive	4.38	0%	P48169
GABRA6	GABAA receptor α6 subunit	Positive	4.24	0.1%	Q16445

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

Aggregated over 2 target gene(s): GABRA4, GABRA6.

Top Reactome pathways

1 total, by targets touching each:

Pathway	Targets	Genes
GABA receptor activation	2	GABRA4, GABRA6

Dominant GO biological processes

GO term	Targets
gamma-aminobutyric acid signaling pathway	2
synaptic transmission, GABAergic	2
chloride transmembrane transport	2
inhibitory synapse assembly	2
monoatomic ion transport	2
chloride transport	2
monoatomic ion transmembrane transport	2
regulation of postsynaptic membrane potential	2
central nervous system development	1
signal transduction	1

Indications & clinical

Indications

2 approved indications. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
epilepsy	4	MONDO:0005027	EFO:0000474
visual epilepsy	4	MONDO:0001386	HP:0001250

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
focal epilepsy	3	MONDO:0005384	EFO:0004263
liver disorder	1	MONDO:0005154	EFO:0001421

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

Clinical trials

Total trials: 19.

Phase distribution

Phase	Trials
PHASE3	6
PHASE1	5
PHASE2	4
Not specified	2
PHASE4	1
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06453213	PHASE4	ACTIVE_NOT_RECRUITING	Open-label Study of Cenobamate Monotherapy in Adult Subjects With Newly Diagnosed or Recurrent Partial-Onset Epilepsy
NCT03961568	PHASE3	ENROLLING_BY_INVITATION	Cenobamate Open-Label Extension Study for YKP3089C025
NCT05067634	PHASE3	ACTIVE_NOT_RECRUITING	Safety and Efficacy Study of Cenobamate in Pediatric Subjects 2-17 Years of Age With Partial-onset (Focal) Seizures
NCT06579573	PHASE3	RECRUITING	ONO-2017 Study Japanese Patients With Primary Generalized Tonic Clonic Seizures.
NCT02535091	PHASE3	COMPLETED	Safety and Pharmacokinetic Study of YKP3089 as Adjunctive Therapy in Subjects With Partial Onset Seizures
NCT03678753	PHASE3	COMPLETED	Randomized, Double-Blind Study to Evaluate Efficacy and Safety of Cenobamate Adjunctive Therapy in PGTC Seizures
NCT04557085	PHASE3	UNKNOWN	Randomized, Double-blind Study to Evaluate Efficacy and Safety of Cenobamate Adjunctive Therapy in POS
NCT06590896	PHASE2	RECRUITING	Safety of Cenobamate in Japanese Subjects With Partial Onset Seizures
NCT00616148	PHASE2	COMPLETED	Efficacy of YKP3089 in Patients With Photosensitive Epilepsy
NCT01397968	PHASE2	COMPLETED	Efficacy and Safety of YKP3089 in Subjects With Treatment Resistant Partial Onset Seizures
NCT01866111	PHASE2	COMPLETED	A Double-Blind, Randomized, Placebo-Controlled, Phase 2 Trial of YKP3089 as Adjunctive Therapy in Subjects With Partial Onset Seizures
NCT03234699	PHASE1	COMPLETED	Assess the Influence of Cenobamate on the PK of Cytochrome P450 (CYP) Probe Drugs As a Means of Predicting Drug-drug Interactions
NCT03509285	PHASE1	COMPLETED	A Study of the Abuse Liability Potential of Cenobamate in Recreational Drug Users
NCT04791553	PHASE1	COMPLETED	Safety Study of Cenobamate in Subjects With Normal Hepatic Function and Subjects With Severe Hepatic Impairment
NCT04903314	PHASE1	COMPLETED	Dose-Escalation Study of Cenobamate (YKP3089) in Pediatric Subjects With Partial-Onset Seizures
NCT05572255	PHASE1	COMPLETED	A Randomized, Open-label, Three-way Crossover Study to Evaluate the Relative Bioavailability of 200 Mg Cenobamate Administered Orally
NCT06352723	EARLY_PHASE1	NOT_YET_RECRUITING	Cenobamate in the Intensive Care Unit
NCT06716801	Not specified	RECRUITING	Real-world Clinical Response to Cenobamate Early add-on in France, Germany and Spain
NCT04513860	Not specified	APPROVED_FOR_MARKETING	Cenobamate Expanded Access Program (EAP)

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

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.

26 molecules share ≥1 primary target. Top 26 by shared-target count:

Molecule	Source	Status	Shared targets
APALUTAMIDE	ChEMBL + PubChem	Phase 4 (approved)	GABRA4, GABRA6
CLONAZEPAM	ChEMBL + PubChem	Phase 4 (approved)	GABRA4, GABRA6
DIAZEPAM	ChEMBL + PubChem	Phase 4 (approved)	GABRA4, GABRA6
ENZALUTAMIDE	ChEMBL + PubChem	Phase 4 (approved)	GABRA4, GABRA6
FLUMAZENIL	ChEMBL + PubChem	Phase 4 (approved)	GABRA4, GABRA6
BREXANOLONE	ChEMBL	Phase 4 (approved)	GABRA4, GABRA6
GANAXOLONE	ChEMBL	Phase 4 (approved)	GABRA4, GABRA6
LIOTHYRONINE	ChEMBL	Phase 4 (approved)	GABRA4, GABRA6
PROPOFOL	ChEMBL	Phase 4 (approved)	GABRA4, GABRA6
ABECARNIL	ChEMBL	Phase 2	GABRA4, GABRA6
BAICALEIN	ChEMBL	Phase 2	GABRA4, GABRA6
DELORAZEPAM	ChEMBL	Phase 2	GABRA4, GABRA6
FLAVONE	ChEMBL	Phase 2	GABRA4, GABRA6
PROGABIDE	ChEMBL	Phase 2	GABRA4, GABRA6
.gamma.-aminobutyric acid	PubChem	Approved	GABRA4, GABRA6
alfaxalone	PubChem	Approved	GABRA4, GABRA6
Caffeine	PubChem	Approved	GABRA4, GABRA6
Ciprofloxacin	PubChem	Approved	GABRA4, GABRA6
Fipronil	PubChem	Approved	GABRA4, GABRA6
Phenytoin	PubChem	Approved	GABRA4, GABRA6
valproate	PubChem	Approved	GABRA4, GABRA6
ESZOPICLONE	ChEMBL	Phase 4 (approved)	GABRA4
LINDANE	ChEMBL	Phase 4 (approved)	GABRA6
ZURANOLONE	ChEMBL	Phase 4 (approved)	GABRA4
BRETAZENIL	ChEMBL	Phase 2	GABRA6
PANADIPLON	ChEMBL	Phase 2	GABRA6