Acipimox

Summary

Acipimox (CHEMBL345714) is an approved small molecule (ATC C10AD06) targeting HCAR2; indicated across 7 conditions including cardiovascular disorder and type 1 diabetes mellitus.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: C10AD06
• Targets: 1 (HCAR2)
• Indications: 7 conditions
• Clinical trials: 20
• Chemistry: 154.12 Da · C6H6N2O3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL345714
Name	Acipimox
Type	Small molecule
Max phase	4
FDA approved	no
PubChem CID	5310993
ATC	C10AD06
Molecular formula	C6H6N2O3
Molecular weight	154.12
InChIKey	DJQOOSBJCLSSEY-UHFFFAOYSA-N

SMILES: CC1=CN=C(C=[N+]1[O-])C(=O)O

IUPAC name: 5-methyl-4-oxidopyrazin-4-ium-2-carboxylic acid

Also known as: Acipimox, NSC-759818, Olbetam, acipimox, SID50110108, SID144205421, SID144206051, SID144206358, ACIPIMOX

Patent coverage: 2,026 distinct patent families (7,101 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 7,078 (100%) 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
HCAR2	HCA2 receptor	Full agonist	5.6	0.1%	Q8TDS4

Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Hydroxycarboxylic acid receptor 2.

Bioactivity

ChEMBL activities: 2 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
HCAR2	5.41	AC50	3900	nM	CHEMBL_ACT_25153492
HCAR2	5.28	EC50	5300	nM	CHEMBL_ACT_1824988

Target pathways

Aggregated over 1 target gene(s): HCAR2.

Top Reactome pathways

3 total, by targets touching each:

Pathway	Targets	Genes
Hydroxycarboxylic acid-binding receptors	1	HCAR2
Class A/1 (Rhodopsin-like receptors)	1	HCAR2
G alpha (i) signalling events	1	HCAR2

Dominant GO biological processes

GO term	Targets
neutrophil apoptotic process	1
G protein-coupled receptor signaling pathway	1
positive regulation of neutrophil apoptotic process	1
negative regulation of lipid catabolic process	1
positive regulation of adiponectin secretion	1
apoptotic process	1
signal transduction	1

Indications & clinical

Indications

7 indications (1 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
cardiovascular disorder	4	MONDO:0004995	EFO:0000319
type 1 diabetes mellitus	3	MONDO:0005147	MONDO:0005147
metabolic syndrome X	2	MONDO:0011565	EFO:0000195
heart failure	2	MONDO:0005252	EFO:0003144
bulimia nervosa	2	MONDO:0005452	EFO:0005204
type 2 diabetes mellitus	2	MONDO:0005148	MONDO:0005148
eating disorder	2	MONDO:0005451	EFO:0005203

Clinical trials

Total trials: 20.

Phase distribution

Phase	Trials
Not specified	12
PHASE2	3
PHASE2/PHASE3	2
PHASE3	1
PHASE1/PHASE2	1
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00759291	PHASE2/PHASE3	COMPLETED	The Impact of Free Fatty Acid Reduction on Vascular Function in the Metabolic Syndrome
NCT01816165	PHASE3	COMPLETED	Effects of Acipimox on Insulin Action, Vascular Function, and Muscle Function in Type 1 Diabetes
NCT01980524	PHASE2/PHASE3	COMPLETED	The Impact of Free Fatty Acid (FFA-) Suppression on Myocardial Lipids and Function in Patients With Type 2 Diabetes
NCT03338387	PHASE2	ENROLLING_BY_INVITATION	Co-Feedback Action of Growth Hormone, PP and PYY on Ghrelin in Bulimia
NCT00153179	PHASE1/PHASE2	COMPLETED	Free Fatty Acids and Vascular Function in Subjects With Diabetes
NCT00449423	PHASE2	TERMINATED	Metabolic Modulation as Treatment in Acute Heart Failure
NCT01488409	PHASE2	COMPLETED	Effects of Acipimox on Mitochondrial Function in Obesity
NCT02256722	PHASE1	COMPLETED	In Vivo Specificity of KUC 7483 CL Co-administered With Bisoprolol, Propranolol, and Acipimox in Healthy Male Subjects
NCT00246402	Not specified	COMPLETED	Acipimox to Improve Hyperlipidemia and Insulin Sensitivity Associated With HIV
NCT00549614	Not specified	COMPLETED	Effect of 4 Weeks Treatment With Acipimox in Patients With Chronic Heart Failure
NCT00943059	Not specified	COMPLETED	Cross-over Study on Effect of Lipid Lowering by Acipimox on Cardiac and Skeletal Muscle Mitochondrial Function
NCT01209416	Not specified	COMPLETED	The Effect of Pharmacological Antilipolysis on the Metabolic Effects of Ghrelin
NCT01260376	Not specified	COMPLETED	Effect of Pharmacological Anti-lipolysis on FFA and VLDL-TG Metabolism Before and During Exercise
NCT01299831	Not specified	COMPLETED	The Relationship Between Body Composition and Growth Hormone, SIRT Signaling, Protein Turnover and Insulin Sensitivity
NCT01580813	Not specified	COMPLETED	Evaluating the Effects of a Study Medication on Exercise Function in Type 2 Diabetes
NCT02782208	Not specified	COMPLETED	Lipolytic Effects of GH in Hypopituitary Patients in Vivo
NCT02792621	Not specified	COMPLETED	Increasing Mitochondrial Function on Skeletal Muscle Performance in Older Men
NCT02796950	Not specified	COMPLETED	The Effect of Acipimox on GLP (Glucagon-like Peptide)-1 Secretion
NCT03325491	Not specified	TERMINATED	Assessing the Effects of Increased Mitochondrial Function Exercise Training on Muscle Performance
NCT03809793	Not specified	COMPLETED	Can the Health Benefits of a Walking-based Exercise Programme be Enhanced by Co-ingestion of a Lipid-lowering Drug?

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.

45 molecules share ≥1 primary target. Top 45 by shared-target count:

Molecule	Source	Status	Shared targets
DIMETHYL	ChEMBL	Phase 4 (approved)	HCAR2
MONOMETHYL	ChEMBL	Phase 4 (approved)	HCAR2
NIACIN	ChEMBL	Phase 4 (approved)	HCAR2
5-FLUORONICOTINIC ACID	ChEMBL	Phase 3	HCAR2
ACIFRAN	ChEMBL	Phase 2	HCAR2
SCH-900271	ChEMBL	Phase 2	HCAR2
Aclidinium Bromide	PubChem	Approved	HCAR2
Allopurinol	PubChem	Approved	HCAR2
Alprazolam	PubChem	Approved	HCAR2
Amitriptyline	PubChem	Approved	HCAR2
Beclomethasone	PubChem	Approved	HCAR2
Beclomethasone Dipropionate	PubChem	Approved	HCAR2
Belzutifan	PubChem	Approved	HCAR2
Carisoprodol	PubChem	Approved	HCAR2
Celecoxib	PubChem	Approved	HCAR2
cephalexin	PubChem	Approved	HCAR2
Cetirizine	PubChem	Approved	HCAR2
Cinnamic Acid	PubChem	Approved	HCAR2
Citalopram	PubChem	Approved	HCAR2
Clindamycin	PubChem	Approved	HCAR2
Clonazepam	PubChem	Approved	HCAR2
Clonidine	PubChem	Approved	HCAR2
Desloratadine	PubChem	Approved	HCAR2
Diazepam	PubChem	Approved	HCAR2
Diltiazem	PubChem	Approved	HCAR2
Famciclovir	PubChem	Approved	HCAR2
Fenofibrate	PubChem	Approved	HCAR2
Fluconazole	PubChem	Approved	HCAR2
Fluticasone Propionate	PubChem	Approved	HCAR2
Gemfibrozil	PubChem	Approved	HCAR2
Guaifenesin	PubChem	Approved	HCAR2
Hydroxyzine	PubChem	Approved	HCAR2
Isosorbide Mononitrate	PubChem	Approved	HCAR2
Lamotrigine	PubChem	Approved	HCAR2
Lorazepam	PubChem	Approved	HCAR2
Meclizine	PubChem	Approved	HCAR2
Metformin	PubChem	Approved	HCAR2
Metoclopramide	PubChem	Approved	HCAR2
Nifedipine	PubChem	Approved	HCAR2
Pantoprazole	PubChem	Approved	HCAR2
Pimozide	PubChem	Approved	HCAR2
Propoxyphene	PubChem	Approved	HCAR2
Valacyclovir	PubChem	Approved	HCAR2
Verapamil	PubChem	Approved	HCAR2
Zidovudine	PubChem	Approved	HCAR2