Announcement • May 27
Esperion Presents Phase 2 Study Results of Bempedoic Acid in Children and Post Hoc Analysis from Clear Outcomes At EAS Congress 2026
Esperion had announced the presentation of results from a Phase 2 study of bempedoic acid in children, along with an additional post hoc analysis from CLEAR Outcomes describing patient traits associated with enhanced LDL-C lowering with bempedoic acid. These data were presented as moderated Science at a Glance sessions at the European Atherosclerosis Society Congress 2026 taking place on May 24-27, 2026, in Athens, Greece. The data from CLEAR Path 1 in pediatric patients with familial hypercholesterolemia gives promise for bempedoic acid as a potentially safe and effective additional lipid lowering agent for use in children who carry a greater lifetime cardiovascular risk and will further inform Phase 3 studies. The 30% or greater reduction in LDL-C more often observed in women, those with higher baseline LDL-C, and with concurrent baseline ezetimibe use as seen in the post-hoc analysis of CLEAR Outcomes confirms what was previously seen in the earlier bempedoic acid pooled hyperlipidemia trials and highlights the important role of bempedoic acid as both an adjunct and alternative lipid lowering therapy to statins. These new analyses add to the breadth of clinical evidence demonstrating the robust LDL cholesterol lowering of NEXLETOL (bempedoic acid) across a wide range of patient populations. These data further support use of the combination product NEXLIZET (bempedoic acid/ezetimibe) to help patients achieve their LDL-C goals and reduce cardiovascular risk. Data supporting the use of bempedoic acid in children with heterozygous familial hypercholesterolemia informs Phase 3 pediatric studies. The variable dosing of bempedoic acid, as determined by patient weight in this study, resulted in similar drug exposure (levels) to that in adults, and supports the doses of bempedoic acid to be used in further clinical studies. Bempedoic acid, when given as a tablet in addition to patient’s existing lipid lowering treatments, reduced LDL-C in pediatric patients (range 6-25%) and did not raise any new safety concerns. In CLEAR Outcomes, 42% of patients in the bempedoic acid arm demonstrated a =30% LDL-C reduction at 3 months, a reduction on par with moderate intensity statins. Patient factors associated with achieving a greater LDL-C reduction included female sex, higher starting LDL-C, and concurrent use of ezetimibe. Bempedoic acid, a component of NEXLIZET and NEXLETOL, is indicated to reduce the risk of major adverse cardiovascular events (cardiovascular death, myocardial infarction, stroke, or coronary revascularization) in adults at increased risk for these events who are unable to take recommended statin therapy (including those not taking a statin). NEXLIZET is indicated to reduce LDL-C in adults with hypercholesterolemia, including HeFH. NEXLETOL is indicated, in combination with other LDL-C lowering therapies or alone when concomitant LDL-C lowering therapy is not possible, to reduce LDL-C in adults with hypercholesterolemia, including HeFH. NEXLIZET and NEXLETOL are contraindicated in patients with a prior hypersensitivity to bempedoic acid or ezetimibe or any of the excipients. Serious hypersensitivity reactions including anaphylaxis, angioedema, rash, and urticaria have been reported. Bempedoic acid, a component of NEXLIZET and NEXLETOL, may increase blood uric acid levels, which may lead to gout. Monitor as clinically indicated and initiate treatment with urate-lowering drugs as appropriate. Bempedoic acid is associated with an increased risk of tendon rupture or injury. Tendon rupture occurred in 0.5% of patients treated with bempedoic acid in primary hypercholesterolemia trials, versus 0% on placebo. In the cardiovascular outcomes trial, the rates were 1.2% for bempedoic acid and 0.9% for placebo. Discontinue NEXLIZET or NEXLETOL at the first sign of tendon rupture. Consider alternative therapy in patients who have a history of tendon disorders or tendon rupture. The most common adverse reactions in the primary hypercholesterolemia trials of bempedoic acid in =2% of patients and greater than placebo were upper respiratory tract infection, muscle spasms, hyperuricemia, back pain, abdominal pain or discomfort, bronchitis, pain in extremity, anemia, and elevated liver enzymes. Adverse reactions reported in =2% of patients treated with ezetimibe (a component of NEXLIZET) and at an incidence greater than placebo in clinical trials were upper respiratory tract infection, diarrhea, arthralgia, sinusitis, pain in extremity, fatigue, and influenza. The most common adverse reactions (incidence =3% and greater than placebo) observed with NEXLIZET but not observed in clinical trials of bempedoic acid or ezetimibe, were urinary tract infection, nasopharyngitis, and constipation. The most common adverse reactions in the cardiovascular outcomes trial for bempedoic acid, at an incidence of =2% and 0.5% greater than placebo, were hyperuricemia, renal impairment, anemia, elevated liver enzymes, muscle spasms, gout, and cholelithiasis. Concomitant use of NEXLIZET or NEXLETOL with greater than 20 mg of simvastatin or 40 mg of pravastatin should be avoided due to the potential for increased risk of simvastatin- or pravastatin-related myopathy. Concomitant use with fibrates may increase triglycerides and decrease high-density lipoprotein cholesterol. Monitor and adjust therapies as recommended. Discontinue NEXLIZET or NEXLETOL when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus. The benefits of breastfeeding should be considered along with the mother’s clinical need for NEXLIZET or NEXLETOL and any potential adverse effects on the breastfed infant from NEXLIZET or NEXLETOL or from the underlying maternal condition.
