Efficacy and Safety of Bempedoic Acid in Patients with Heterozygous Familial Hypercholesterolemia: Analysis of Pooled Patient-level Data from Phase 3 Clinical Trials

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Abstract

Background and Aims

Patients with heterozygous familial hypercholesterolemia (HeFH) have high cardiovascular risk due to lifelong elevated levels of low-density lipoprotein cholesterol (LDL-C), often requiring multidrug therapy to achieve sufficient LDL-C lowering. In patients with and without HeFH, we evaluated efficacy and safety of bempedoic acid (BA), an investigational, oral, once-daily, ATP-citrate lyase inhibitor, when added to existing lipid-lowering therapy (LLT).

Methods

Data were pooled from two phase 3 clinical trials that randomized (2:1) 3009 patients with atherosclerotic cardiovascular disease and/or HeFH receiving background maximally-tolerated statin therapy with or without other nonstatin LLTs to treatment with BA 180 mg or placebo once daily for 52 weeks. The primary efficacy endpoint was percent change from baseline to week 12 in LDL-C. Safety assessments included treatment-emergent adverse events (TEAEs).

Results

HeFH patients (n=112) had higher baseline LDL-C (mean [SD], BA: 3.7 [0.1] mmol/L; placebo: 4.0 [0.3] mmol/L) vs those without HeFH (n=2897) (BA: 2.7 [0.02] mmol/L; placebo: 2.7 [0.02] mmol/L). Mean LDL-C reductions from baseline to week 12 were significantly greater with BA vs placebo (P < .001) for patients with and without HeFH (placebo-corrected, HeFH: –22.3%; without HeFH: –18.3%). BA treatment improved secondary efficacy measures, regardless of HeFH status (Table). TEAE incidence was higher in patients with HeFH (BA: 81.6%; placebo: 91.7%) vs those without HeFH (BA: 76.1%; placebo: 76.1%), but was not increased with BA treatment relative to placebo.

Conclusions

BA significantly lowered LDL-C in patients with HeFH receiving background LLT and did not increase the risk of TEAEs compared with placebo.

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