Bempedoic Acid

drug
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Also known as Acide bempedoiqueAcido bempedoicoBempedoic acid component of nexlizetESP-55016ETC-1002ETC1002NexletolNilemdo

Summary

Bempedoic Acid (CHEMBL3545313) is an approved small-molecule antilipemic drug (ATC C10AX15) targeting ACLY; indicated across 9 conditions including cardiovascular disorder and familial hypercholesterolemia.

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • ATC class: C10AX15
  • Targets: 1 (ACLY)
  • Indications: 9 conditions
  • Clinical trials: 36
  • Chemistry: 344.5 Da · C19H36O5

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL3545313
NameBempedoic Acid
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID10472693
ChEBICHEBI:149601
ATCC10AX15
Molecular formulaC19H36O5
Molecular weight344.5
InChIKeyHYHMLYSLQUKXKP-UHFFFAOYSA-N

SMILES: CC(C)(CCCCCC(CCCCCC(C)(C)C(=O)O)O)C(=O)O

IUPAC name: 8-hydroxy-2,2,14,14-tetramethylpentadecanedioic acid

ChEBI definition: An α,ω-dicarboxylic acid that is pentadecanedioic acid which is substituted by methyl groups groups at positions 2 and 14, and by a hydroxy group at position 8. It is a drug used for the treatment of high LDL cholesterol, which is sometimes referred to as ‘bad cholesterol’.

Pharmacological roles (ChEBI): antilipemic drug, EC 2.3.3.8 (ATP citrate synthase) inhibitor, prodrug.

Also known as: Acide bempedoique, Acido bempedoico, Bempedoic acid, Bempedoic acid component of nexlizet, ESP-55016, ETC-1002, ETC1002, Nexletol, Nilemdo, BEMPEDOIC ACID, bempedoic acid

Parent form; salt/anhydrous children: CHEMBL5397694, CHEMBL5410861, CHEMBL5412638, CHEMBL5418282

Patent coverage: 306 distinct patent families (787 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).

GeneTargetActionpAffinityCancer dependencyUniProt
ACLYATP citrate lyaseInhibition5.747.4%P53396

Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Acetyl-CoA carboxylase.

Bioactivity

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

Target pathways

Aggregated over 1 target gene(s): ACLY.

Top Reactome pathways

9 total, by targets touching each:

PathwayTargetsGenes
Metabolism1ACLY
Integration of energy metabolism1ACLY
ChREBP activates metabolic gene expression1ACLY
Innate Immune System1ACLY
Immune System1ACLY
Metabolism of lipids1ACLY
Neutrophil degranulation1ACLY
Fatty acyl-CoA biosynthesis1ACLY
Fatty acid metabolism1ACLY

Dominant GO biological processes

GO termTargets
acetyl-CoA biosynthetic process1
citrate metabolic process1
oxaloacetate metabolic process1
fatty acid biosynthetic process1
cholesterol biosynthetic process1
lipid biosynthetic process1
coenzyme A metabolic process1
negative regulation of ferroptosis1
lipid metabolic process1

Indications & clinical

Indications

9 indications (6 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).

IndicationTrial phaseMONDOEFO
cardiovascular disorder4MONDO:0004995EFO:0000319
familial hypercholesterolemia4MONDO:0005439EFO:0004911
coronary artery disorder4MONDO:0005010EFO:0001645
atherosclerosis4MONDO:0005311EFO:0003914
hyperlipidemia3MONDO:0021187MONDO:0021187
type 2 diabetes mellitus2MONDO:0005148MONDO:0005148

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

Clinical trials

Total trials: 36.

Phase distribution

PhaseTrials
PHASE213
PHASE39
PHASE16
Not specified6
PHASE42

Top trials by phase / activity

NCTPhaseStatusTitle
NCT07255820PHASE4NOT_YET_RECRUITINGDual vs Triple Lipid-Lowering Therapy in Type 2 Diabetes Mellitus Patients With Elevated LDL Cholesterol
NCT05263778PHASE4UNKNOWNCholesterol Lowering Via Bempedoic Acid/Ezetimibe, an ACL-Inhibiting Regimen in Acute Coronary Syndrome Study
NCT07474649PHASE3NOT_YET_RECRUITINGA Study of Bempedoic Acid/Ezetimibe/High-intensity Statin in Patients Without Cardiovascular Events
NCT07614958PHASE3RECRUITINGPhase 3 Clinical Study Evaluating Efficacy, Safety, and Tolerability of Obicetrapib Vs Bempedoic Acid to Treat Dyslipidemia in Adults With Primary Non-familial Hypercholesterolemia or Mixed Dyslipidemia at High to Very High Cardiovascular Risk, During a Treatment Period of Approximately 84 Days
NCT02666664PHASE3COMPLETEDEvaluation of Long-Term Safety and Tolerability of ETC-1002 in High-Risk Patients With Hyperlipidemia and High CV Risk (CLEAR Harmony)
NCT02988115PHASE3COMPLETEDEvaluation of the Efficacy and Safety of Bempedoic Acid (ETC-1002) in Patients With Hyperlipidemia and Statin Intolerant
NCT02991118PHASE3COMPLETEDEvaluation of Long-Term Efficacy of Bempedoic Acid (ETC-1002) in Patients With Hyperlipidemia at High Cardiovascular Risk
NCT03001076PHASE3COMPLETEDEvaluation of the Efficacy and Safety of Bempedoic Acid (ETC-1002) as Add-on to Ezetimibe Therapy in Patients With Elevated LDL-C (CLEAR Tranquility)
NCT03067441PHASE3COMPLETEDAssessment of the Long-Term Safety and Efficacy of Bempedoic Acid (CLEAR Harmony OLE)
NCT03337308PHASE3COMPLETEDA Study Evaluating the Safety and Efficacy of Bempedoic Acid Plus Ezetimibe Fixed-Dose Combination Compared to Bempedoic Acid, Ezetimibe, and Placebo in Patients Treated With Maximally Tolerated Statin Therapy
NCT06450366PHASE3COMPLETEDA Study to Evaluate the Efficacy and Safety of Enlicitide (MK-0616, Oral PCSK9 Inhibitor) Compared With Ezetimibe or Bempedoic Acid or Ezetimibe and Bempedoic Acid in Adults With Hypercholesterolemia (MK-0616-018/CORALreef AddOn)
NCT05488431PHASE2RECRUITINGCholesterol and Inflammation Lowering Via Bempedoic Acid, an ACL-inhibiting Regimen in HIV Trial (CLEAR HIV Trial)
NCT01262638PHASE2COMPLETEDA Study to Assess the Efficacy and Safety of ETC-1002 in Subjects With Elevated Blood Cholesterol and Either Normal or Elevated Triglycerides
NCT01607294PHASE2COMPLETEDA Single Center Study to Evaluate the Efficacy and Safety of ETC 1002 in Subjects With Type 2 Diabetes
NCT01751984PHASE2COMPLETEDA Study of the Efficacy and Safety of ETC-1002 in Participants With Statin Intolerance
NCT01779453PHASE2COMPLETEDA Study of the Safety, Pharmacokinetic Drug Interaction and Efficacy of ETC-1002 and Atorvastatin in Subjects With Hypercholesterolemia
NCT01941836PHASE2COMPLETEDEvaluation of ETC-1002, Ezetimibe, and the Combination in Hypercholesterolemic Patients
NCT02072161PHASE2COMPLETEDEvaluation of ETC-1002 vs Placebo in Patients Receiving Ongoing Statin Therapy
NCT02178098PHASE2COMPLETEDEvaluation of ETC-1002 in Participants With Hypercholesterolemia and Hypertension
NCT02659397PHASE2COMPLETEDA Study Of Pharmacokinetics, Pharmacodynamics And Safety Of Adding ETC-1002 To Atorvastatin 80 mg
NCT03051100PHASE2COMPLETEDEvaluation of the Efficacy and Safety of Bempedoic Acid (ETC-1002) 180mg, Ezetimibe 10mg, and Atorvastatin 20 mg Triplet Therapy in Patients With Elevated LDL-C
NCT03193047PHASE2COMPLETEDEvaluation of the Efficacy and Safety of Bempedoic Acid (ETC-1002) 180mg When Added to PCSK9 Inhibitor Therapy
NCT03531905PHASE2COMPLETEDBempedoic Acid + Ezetimibe Fixed-Dose Combination (FDC) Study in Patients With Type 2 Diabetes and Elevated LDL-C
NCT05694260PHASE2COMPLETEDA Clinical Study in Children With Heterozygous Familial Hypercholesterolemia (HeFH) Aged 6 to 17 Treated Once Daily With Bempedoic Acid Oral Dosing (CLEAR Path 1)
NCT01485146PHASE1COMPLETEDA Multiple Ascending Dose Study of ETC-1002 in Healthy Subjects
NCT06742853PHASE1COMPLETEDA Study to Investigate the Pharmacokinetics, Safety, Tolerability, and Efficacy of AZD0780 With Ezetimibe Combinations in Healthy Adults With Elevated LDL-C.
NCT07182383PHASE1COMPLETEDEvaluating Bioequivalence of a Fixed Dose Combination Versus Individual Tablets of Bempedoic Acid, Ezetimibe, and Rosuvastatin
NCT07201545PHASE1COMPLETEDEvaluating Bioequivalence of a Fixed Dose Combination Versus Tablets of Bempedoic Acid / Ezetimibe and Rosuvastatin
NCT07235189PHASE1COMPLETEDA Study Evaluating Bioequivalence of a Fixed Dose Combination Versus Individual Tablets of Bempedoic Acid, Ezetimibe, and Atorvastatin
NCT07268625PHASE1COMPLETEDEvaluating Bioequivalence of a Fixed Dose Combination Versus Individual Tablets of Bempedoic Acid / Ezetimibe, and Atorvastatin
NCT05103254Not specifiedRECRUITINGBempedoic Acid Pregnancy Surveillance Program
NCT05587621Not specifiedNOT_YET_RECRUITINGIntensive Lipid-lowering in Patients With STEMI and NSTEMI (Germany on Target)
NCT06035874Not specifiedRECRUITINGEffect of Bempedoic Acid on Liver Fat in Individuals With Nonalcoholic Fatty Liver Disease and Type 2 Diabetes
NCT06686615Not specifiedRECRUITINGA Study of Bempedoic Acid in Combination With Ezetimibe and Either Rosuvastatin or Atorvastatin in Patients With Primary Hypercholesterolemia or Mixed Dyslipidemia
NCT07256171Not specifiedNOT_YET_RECRUITINGCOAST Study - Cholesterol Optimization After Stroke
NCT07374861Not specifiedRECRUITINGMulticenter Study on the Evaluation of Adherence, Persistence and Efficacy of Treatment With Bempedoic Acid in Italy

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.

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).