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Bempedoic acid

Phase 3

Hypercholesterolemia | Small molecule | Metabolic |Esperion Therapeutics, Inc.|Last Updated: Aug 8, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindCONTROLLEDDMCBiomarker
Total Trials7
Total Enrollment3,008
FDA Designations
No designations recorded
Clinical Trials (7)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03067441Assessment of the Long-Term Safety and Efficacy of Bempedoic Acid (CLEAR Harmony OLE)PHASE3 COMPLETED 1,462Feb 3, 2017Nov 5, 2019Mar 1, 20213 United States
NCT03001076Evaluation of the Efficacy and Safety of Bempedoic Acid (ETC-1002) as Add-on to Ezetimibe Therapy in Patients With Elevated LDL-C (CLEAR Tranquility)PHASE3 COMPLETED 269Nov 29, 2016Feb 12, 2018May 11, 20201 United States
NCT02991118Evaluation of Long-Term Efficacy of Bempedoic Acid (ETC-1002) in Patients With Hyperlipidemia at High Cardiovascular RiskPHASE3 COMPLETED 779Nov 18, 2016Aug 22, 2018Apr 27, 20202 United States
NCT02988115Evaluation of the Efficacy and Safety of Bempedoic Acid (ETC-1002) in Patients With Hyperlipidemia and Statin IntolerantPHASE3 COMPLETED 345Nov 16, 2016Mar 16, 2018Apr 3, 202067 United States, Canada
NCT05694260A Clinical Study in Children With Heterozygous Familial Hypercholesterolemia (HeFH) Aged 6 to 17 Treated Once Daily With Bempedoic Acid Oral Dosing (CLEAR Path 1)PHASE2 COMPLETED 31Jan 12, 2023Jun 4, 2025Aug 8, 202524 United States, Canada +4
NCT03193047Evaluation of the Efficacy and Safety of Bempedoic Acid (ETC-1002) 180mg When Added to PCSK9 Inhibitor TherapyPHASE2 COMPLETED 59Apr 7, 2017Feb 19, 2018Apr 3, 20201 United States
NCT03051100Evaluation of the Efficacy and Safety of Bempedoic Acid (ETC-1002) 180mg, Ezetimibe 10mg, and Atorvastatin 20 mg Triplet Therapy in Patients With Elevated LDL-CPHASE2 COMPLETED 63Jan 19, 2017Jul 5, 2017Apr 3, 202011 United States
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Study Endpoints
Primary Endpoints
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Up to Week 82

TEAEs are defined as adverse events that began or worsened in severity after the first dose of investigational medicinal product (IMP) until 30 days after the last dose in the Open-Label Extension (OLE) Study.

Percent Change From Baseline to Week 12 in Low-Density Lipoprotein Cholesterol (LDL-C)
Week 12

Blood samples were drawn after a minimum 10-hour fast (water was allowed) at pre-specified intervals. Samples were collected and analyzed for LDL-C. Baseline was defined as the mean of the LDL-C values from the last two non-missing values on or prior to Day 1. Percent change from baseline was calculated as: (\[LDL-C value at Week 12 minus Baseline value\] divided by \[Baseline Value\]) multiplied by 100. Bempedoic Acid = BA. Percent change from Baseline in LDL-C was analyzed using an analysis of covariance (ANCOVA) model with percent change from Baseline as the dependent variable, treatment as a fixed effects and Baseline as a covariate. In the ANCOVA model, missing LDL-C data at Week 12 were imputed using multiple imputation method taking into account adherence to treatment.

Percent Change From Baseline (PCFB) in Low-density Lipoprotein Cholesterol (LDL-C) at Week 12
Baseline; Week 12

PCFB was calculated as the (\[post-Baseline (BL) value minus the BL value\] divided by the BL value ) x 100. BL was defined as the mean of the last two non-missing values on or prior to Day 1. If only one value was available, that single value was used as BL. PCFB in LDL-C was analyzed using analysis of covariance (ANCOVA), with treatment group and stratification factor (primary prevention; secondary prevention) as fixed effects and BL as a covariate. For participants with missing lipid data at Week 12 who were no longer taking study treatment, missing values were imputed using multiple imputation via a regression-based model including stratification and BL data from placebo participants only. In this imputation model, treatment group was not included. For participants with missing lipid data at Week 12 who were still taking study treatment, missing values were imputed using multiple imputation via a regression-based model including treatment, stratification and BL value.

Plasma trough concentration of ETC-1002
8 weeks of steady-state dosing
Area under the plasma concentration-time curve (AUC,ss) of ETC-1002
8 weeks of steady-state dosing
Average plasma concentration (Cavg,ss) of ETC-1002
8 weeks of steady-state dosing
Maximum plasma concentration (Cmax,ss) of ETC-1002
8 weeks of steady-state dosing
Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Month 2
Baseline; Month 2

Percent change from Baseline is calculated as the (\[post-Baseline value minus the Baseline value\] divided by the Baseline value ) x 100. Baseline is defined as the average of the last two non-missing values within Month -1 (Screening Visit 4) and Day 1 (Treatment Visit 1) values (including unscheduled assessments). If only one value was available, then that single value was used at Baseline. Percent change from Baseline was analyzed using analysis of covariance (ANCOVA), with treatment group as a factor and Baseline as a covariate. Missing data were imputed using last observation carried forward (LOCF) (only post-Baseline values were carried forward).

Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Week 6
Baseline; Week 6

Percent change from Baseline is calculated as the (\[post-Baseline value minus the Baseline value\] divided by the Baseline value ) x 100. Baseline is defined as the mean of the values from Week -1 (Screening Visit 2) and predose Day 1/Week 0 (Treatment Visit 1). Percent change from Baseline in LDL-C was analyzed using analysis of covariance (ANCOVA) with treatment group as a factor and Baseline value as a covariate. Missing LDL-C values were imputed using last observation carried forward (LOCF), with only post-Baseline values carried forward. If LDL-C was measured (i.e., if TG was \>400 mg/dL or LDL-C was \<50 mg/dL), the measured values were used in the analysis.

Secondary Endpoints
Percent Change From Parent Study Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Weeks 52 and 78
Baseline; Week 52 and Week 78
Mean Change From Parent Study Baseline in LDL-C at Weeks 52 and 78
Baseline; Week 52 and Week 78
Percent Change From Parent Study Baseline in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Weeks 52 and 78
Baseline; Week 52 and Week 72
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Open-Label bempedoic acidEXPERIMENTALbempedoic acid 180 mg tablet
bempedoic acidEXPERIMENTALbempedoic acid 180 mg tablet taken orally, daily. Patients remain on ongoing ezetimibe therapy (study provided)
placeboPLACEBO_COMPARATORMatching placebo tablet taken orally, daily. Patients remain on ongoing ezetimibe therapy (study provided)
Cohort 1EXPERIMENTALParticipants at 16 to \<30 kilograms (kg) body weight at screening receiving once daily 60 milligrams (mg) bempedoic acid for 8 weeks followed by 90 mg bempedoic acid for 8 weeks.
Cohort 2EXPERIMENTALParticipants at 30 to 60 kg body weight at screening receiving once daily120 mg bempedoic acid for 8 weeks followed by 150 mg bempedoic acid for 8 weeks.
Cohort 3EXPERIMENTALParticipants at greater than 60 kg body weight at screening receiving once daily 180 mg bempedoic acid for 8 weeks.
Triplet TherapyEXPERIMENTALBempedoic acid 180 mg, ezetimibe 10 mg, and atorvastatin 20 mg taken orally, daily.
Interventions
NameTypeDescription
bempedoic acidDRUGbempedoic acid 180 mg tablets taken orally, once per day.
EzetimibeDRUGezetimibe 10 mg tablet
PlaceboOTHERmatching placebo tablet
bempedoic acid 180mgDRUGDaily bempedoic acid 180mg tablet in addition to monthly PCSK9i (evolocumab) background therapy
evolocumabDRUGMonthly PCSK9i (evolocumab) background therapy
Ezetimibe 10mgDRUGezetimibe 10 mg
Atorvastatin 20mgDRUGatorvastatin 20 mg
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: * Successfully completed CLEAR Harmony (1002-040) parent study Exclusion Criteria: * Experienced a treatment-related SAE that led to study drug discontinuation in the CLEAR Harmony (1002-040) parent study. * Medical condition requires lipid measurement and/or adjustment of bac...

Countries:United StatesCanadaDenmarkGermanyNetherlandsSpain
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