Azimilide

Summary

Azimilide (CHEMBL123558) is a phase-3 clinical-stage small molecule targeting KCNQ1; indicated across 1 condition including congestive heart failure.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 1 (KCNQ1)
• Indications: 1 condition
• Clinical trials: 1

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL123558
Name	Azimilide
Type	Small molecule
Max phase	3

Also known as: Azimilida, Azimilide, azimilide, AZIMILIDE, Azimilide (Dihydrochloride)

Parent form; salt/anhydrous children: CHEMBL537847, CHEMBL3140370

Patent coverage: 553 distinct patent families (2,246 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
KCNQ1	Kv7.1		5.3	0.2%	P51787

Broader ChEMBL bioactivity targets: 3 (assay-derived). Sample: Voltage-gated inwardly rectifying potassium channel KCNH2, Potassium voltage-gated channel subfamily KQT member 1, Voltage-gated inwardly rectifying potassium channel KCNH2.

Bioactivity

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

Target	pChembl	Type	Value	Unit	Activity ID
O08703	6.75	IC50	180	nM	CHEMBL_ACT_2293432
O70344	6.62	IC50	240	nM	CHEMBL_ACT_2293433
KCNH2	6.4	IC50	400	nM	CHEMBL_ACT_1449628
KCNH2	6.25	IC50	562.3	nM	CHEMBL_ACT_2645517
KCNH2	6.25	IC50	560	nM	CHEMBL_ACT_345126
KCNH2	6.25	IC50	562.3	nM	CHEMBL_ACT_5218933
KCNH2	5.85	IC50	1413	nM	CHEMBL_ACT_1047051
KCNH2	5.85	IC50	1413	nM	CHEMBL_ACT_1523685
KCNH2	5.85	IC50	1413	nM	CHEMBL_ACT_2358325

Target pathways

Aggregated over 1 target gene(s): KCNQ1.

Top Reactome pathways

7 total, by targets touching each:

Pathway	Targets	Genes
Neuronal System	1	KCNQ1
Potassium Channels	1	KCNQ1
Voltage gated Potassium channels	1	KCNQ1
Muscle contraction	1	KCNQ1
Phase 3 - rapid repolarisation	1	KCNQ1
Cardiac conduction	1	KCNQ1
Phase 2 - plateau phase	1	KCNQ1

Dominant GO biological processes

GO term	Targets
action potential	1
gastrin-induced gastric acid secretion	1
glucose metabolic process	1
heart development	1
sensory perception of sound	1
rhythmic behavior	1
regulation of heart contraction	1
regulation of blood pressure	1
positive regulation of heart rate	1
iodide transport	1
erythrocyte differentiation	1
intracellular chloride ion homeostasis	1
response to insulin	1
social behavior	1
corticosterone secretion	1

Indications & clinical

Indications

1 indication (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
congestive heart failure	2	MONDO:0005009	EFO:0000373

Clinical trials

Total trials: 1.

Phase distribution

Phase	Trials
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00752596	PHASE2	COMPLETED	Compassionate Use Study of 125 mg Per Day of Azimilide Dihydrochloride in One Patient

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.

35 molecules share ≥1 primary target. Top 35 by shared-target count:

Molecule	Source	Status	Shared targets
ALVIMOPAN	ChEMBL + PubChem	Phase 4 (approved)	KCNQ1
AMBRISENTAN	ChEMBL + PubChem	Phase 4 (approved)	KCNQ1
DARIFENACIN	ChEMBL + PubChem	Phase 4 (approved)	KCNQ1
DARUNAVIR	ChEMBL + PubChem	Phase 4 (approved)	KCNQ1
DULOXETINE	ChEMBL + PubChem	Phase 4 (approved)	KCNQ1
EVEROLIMUS	ChEMBL + PubChem	Phase 4 (approved)	KCNQ1
NEBIVOLOL	ChEMBL + PubChem	Phase 4 (approved)	KCNQ1
NELFINAVIR	ChEMBL + PubChem	Phase 4 (approved)	KCNQ1
PALONOSETRON	ChEMBL + PubChem	Phase 4 (approved)	KCNQ1
RALTEGRAVIR	ChEMBL + PubChem	Phase 4 (approved)	KCNQ1
SOLIFENACIN	ChEMBL + PubChem	Phase 4 (approved)	KCNQ1
SUNITINIB	ChEMBL + PubChem	Phase 4 (approved)	KCNQ1
TOLTERODINE	ChEMBL + PubChem	Phase 4 (approved)	KCNQ1
MARAVIROC	ChEMBL	Phase 4 (approved)	KCNQ1
VOLINANSERIN	ChEMBL	Phase 3	KCNQ1
Alfuzosin	PubChem	Approved	KCNQ1
Belzutifan	PubChem	Approved	KCNQ1
Deferasirox	PubChem	Approved	KCNQ1
Desvenlafaxine	PubChem	Approved	KCNQ1
Dofetilide	PubChem	Approved	KCNQ1
Etravirine	PubChem	Approved	KCNQ1
Lacosamide	PubChem	Approved	KCNQ1
Lamotrigine	PubChem	Approved	KCNQ1
Lapatinib	PubChem	Approved	KCNQ1
Loperamide	PubChem	Approved	KCNQ1
Mefenamic Acid	PubChem	Approved	KCNQ1
Moxifloxacin	PubChem	Approved	KCNQ1
Nilotinib	PubChem	Approved	KCNQ1
Paliperidone	PubChem	Approved	KCNQ1
Sildenafil	PubChem	Approved	KCNQ1
Silodosin	PubChem	Approved	KCNQ1
Sitagliptin	PubChem	Approved	KCNQ1
Tadalafil	PubChem	Approved	KCNQ1
Vardenafil	PubChem	Approved	KCNQ1
Zuranolone	PubChem	Approved	KCNQ1