Abstract 13130: Efficacy and Safety of Bempedoic Acid by Sex: Pooled Analyses From Phase 3 Trials

Abstract

Introduction: Bempedoic acid (BA), an ATP-citrate lyase inhibitor, lowers low-density lipoprotein cholesterol (LDL-C) in patients with hypercholesterolemia. Objective: To report efficacy and safety of BA by sex in patients with elevated LDL-C. Methods: Data were pooled from 4 phase 3 randomized (2:1), double-blind studies investigating oral BA (180 mg once daily) vs placebo for 12 weeks to 52 weeks in adults receiving maximally tolerated statins who required additional LDL-C lowering, analyzed by sex. The primary efficacy endpoint was % change in LDL-C from baseline to week 12. Results were analyzed in cohorts with atherosclerotic cardiovascular disease (ASCVD) and/or heterozygous familial hypercholesterolemia (“ASCVD/HeFH on statins pool”, n=3009) or history of statin intolerance (“statin intolerant pool”, n=614). Safety assessments included treatment-emergent adverse events (TEAEs). Results: Of all participants, 34.3% were women. Significant LDL-C lowering from baseline with BA vs placebo was demonstrated in both pools and sexes ( P < 0.001; Table 1 ), with greater lowering in women vs men in the ASCVD/HeFH on statins pool ( P = 0.04). Rates of TEAEs were similar across groups ( Table 2 ). Rates of common TEAEs were similar in both pools and sexes, except urinary tract infection (men: BA, 2.8%; placebo, 3.0%; women: BA, 8.0%; placebo, 10.3%) and pain in extremity (men: BA, 2.5%; placebo, 1.2%; women: BA, 4.2%; placebo, 2.9%), which occurred more frequently in women. Conclusion: BA lowered LDL-C significantly in both women and men, with greater effect in women vs men in both pools, and a safety profile generally comparable to placebo in both sexes.