PCSK9 inhibitors trials

28 studies from 2014 to 2026, with 111,238 people taking part. Results: 3 fewer heart events or deaths, 1 no clear benefit, 22 lipid or plaque goal met, 2 safety, side effects or genetics. Every summary comes from the trial's PubMed abstract.

Fewer heart events or deaths
No clear benefit
Lipid or plaque goal met
Safety, side effects or genetics

1984 to 202628 studies, 111,238 people in trials

Circle size shows how many people took part. Tap a circle to open the trial; on a phone, swipe sideways for earlier years. The people count leaves out meta-analyses and genetic studies, which pool other studies.

All 28 studies

CORALreef HeFH2026 · Enlicitide · 303 people
In patients with heterozygous familial hypercholesterolemia already on statin therapy, adding oral enlicitide substantially and safely lowered LDL-C and other atherogenic lipid measures compared with placebo.
Primary lipid endpoint metPCSK9 inhibitorsUS-approved
CORALreef Lipids2026 · Enlicitide · 2,909 people
In patients with or at risk for atherosclerotic cardiovascular disease, the oral PCSK9 inhibitor enlicitide substantially lowered LDL cholesterol and other atherogenic lipid markers compared with placebo, with adverse event rates similar between groups.
Primary lipid endpoint metPCSK9 inhibitorsUS-approved
VESALIUS-CV2026 · Evolocumab · 12,257 people
In patients with atherosclerosis or diabetes but no prior MI or stroke, adding evolocumab to therapy significantly lowered the risk of major cardiovascular events compared with placebo, with no apparent safety concerns.
BenefitPCSK9 inhibitorsUS-approved
PURSUIT2025 · AZD0780 · 428 people
In statin-treated patients with hypercholesterolemia, the oral PCSK9 inhibitor AZD0780 produced robust, dose-dependent LDL-C reductions over 12 weeks with an adverse event rate similar to placebo.
Primary lipid endpoint metPCSK9 inhibitorsInvestigational
LIBerate-HR2024 · Lerodalcibep · 922 people
Monthly lerodalcibep added to maximally tolerated statins substantially lowers LDL-C and helps most high-risk patients reach guideline LDL-C targets, with a safety profile similar to placebo aside from mild/moderate injection site reactions.
Primary lipid endpoint metPCSK9 inhibitorsUS-approved
LIBerate-HeFH2023 · Lerodalcibep · 478 people
In patients with heterozygous familial hypercholesterolaemia needing more LDL-C lowering, adding monthly subcutaneous lerodalcibep to therapy produced a large, highly significant LDL-C reduction versus placebo over 24 weeks with a safety profile similar to placebo aside from mild injection site reactions.
Primary lipid endpoint metPCSK9 inhibitorsUS-approved
MK-0616 phase 2b2023 · Enlicitide (MK-0616) · 381 people
In this phase 2b trial, the oral PCSK9 inhibitor MK-0616 produced large, dose-dependent LDL-C reductions compared with placebo and was well tolerated over 8 weeks of treatment plus 8 weeks of follow-up.
Primary lipid endpoint metPCSK9 inhibitorsUS-approved 2026 (as enlicitide)
ORION-32023 · Inclisiran · 497 people
Twice-yearly inclisiran produced durable, substantial LDL cholesterol and PCSK9 lowering over 4 years of repeated hepatic dosing and was well tolerated with low rates of serious drug-related adverse events.
Sustained effectPCSK9 inhibitorsUS-approved
FOURIER-OLE2022 · Evolocumab · 6,635 people
Long-term evolocumab treatment after FOURIER maintained very low LDL-C with a persistently favorable safety profile over more than 8 years, and earlier initiation of evolocumab was associated with fewer cardiovascular events, including cardiovascular death, compared with delayed treatment start.
Long-term safety; benefit sustainedPCSK9 inhibitorsUS-approved
HUYGENS2022 · Evolocumab · 161 people
Adding evolocumab to statin therapy after NSTEMI led to greater regression and stabilization of coronary plaque on imaging (thicker fibrous caps, smaller lipid cores, less plaque burden) compared with placebo.
Plaque regression / favourable changePCSK9 inhibitorsUS-approved
PACMAN-AMI2022 · Alirocumab · 300 people
Adding alirocumab to high-intensity statin therapy after acute MI led to significantly greater regression of coronary plaque burden, lipid content, and thickening of fibrous caps over 52 weeks, with similar adverse event rates to placebo.
Plaque regression / favourable changePCSK9 inhibitorsUS-approved
HAUSER-RCT2020 · Evolocumab · 157 people
In pediatric patients with heterozygous familial hypercholesterolemia already on lipid-lowering therapy, adding monthly evolocumab substantially lowered LDL cholesterol compared with placebo with a similar adverse event rate.
Primary lipid endpoint metPCSK9 inhibitorsUS-approved
ORION-10/112020 · Inclisiran · 3,178 people
Twice-yearly inclisiran injections lowered LDL cholesterol by about 50% compared with placebo in statin-treated patients with atherosclerotic cardiovascular disease, with mostly mild injection-site reactions as the main safety difference.
Primary lipid endpoint metPCSK9 inhibitorsUS-approved
ORION-92020 · Inclisiran · 482 people
In patients with heterozygous familial hypercholesterolemia, twice-yearly inclisiran injections significantly lowered LDL cholesterol compared with placebo, with a similar safety profile and a convenient infrequent dosing schedule.
Primary lipid endpoint metPCSK9 inhibitorsUS-approved
ODYSSEY OUTCOMES2018 · Alirocumab · 18,924 people
In post-ACS patients already on high-intensity statins, adding alirocumab reduced recurrent cardiovascular events and all-cause death compared with placebo.
BenefitPCSK9 inhibitorsUS-approved
EBBINGHAUS2017 · Evolocumab · 1,204 people
Adding evolocumab to statin therapy did not worsen cognitive function compared with placebo over a median follow-up of 19 months.
No excess harm vs placeboPCSK9 inhibitorsUS-approved
FOURIER2017 · Evolocumab · 27,564 people
Adding evolocumab to statin therapy substantially lowers LDL cholesterol and reduces cardiovascular events in patients with atherosclerotic cardiovascular disease, with injection-site reactions being the main added safety concern.
BenefitPCSK9 inhibitorsUS-approved
ORION-12017 · Inclisiran · 501 people
In this phase 2 trial, inclisiran produced substantial, dose-dependent, and durable reductions in LDL cholesterol and PCSK9 levels in high cardiovascular risk patients, with an acceptable safety profile, but the trial was not designed to assess cardiovascular outcomes.
Primary lipid endpoint metPCSK9 inhibitorsUS-approved
SPIRE-1/22017 · Bococizumab · 27,438 people
Bococizumab substantially lowered LDL cholesterol but showed no significant reduction in major cardiovascular events overall, with benefit seen only in the higher-risk trial, and the drug's development was discontinued due to high rates of antidrug antibodies and injection-site reactions.
No significant benefit (program stopped)PCSK9 inhibitorsDiscontinued
GAUSS-32016 · Evolocumab · 218 people
In statin-intolerant patients, evolocumab produced substantially greater LDL-C lowering than ezetimibe over 24 weeks, with fewer treatment discontinuations for muscle symptoms.
Primary lipid endpoint metPCSK9 inhibitorsUS-approved
GLAGOV2016 · Evolocumab · 968 people
Adding evolocumab to statin therapy in patients with coronary artery disease caused regression of coronary atherosclerosis on IVUS imaging compared with placebo, though its effect on clinical cardiovascular outcomes was not assessed in this trial.
Plaque regression / favourable changePCSK9 inhibitorsUS-approved
ODYSSEY ALTERNATIVE2015 · Alirocumab · 361 people
In statin-intolerant patients, alirocumab lowered LDL-C far more than ezetimibe and caused fewer muscle-related adverse events than statin rechallenge with atorvastatin.
Primary lipid endpoint metPCSK9 inhibitorsUS-approved
ODYSSEY FH I and II2015 · Alirocumab · 735 people
In patients with heterozygous familial hypercholesterolemia inadequately controlled on maximally tolerated lipid-lowering therapy, adding alirocumab produced large, durable LDL-C reductions through 78 weeks and was generally well tolerated.
Primary lipid endpoint metPCSK9 inhibitorsUS-approved
ODYSSEY LONG TERM2015 · Alirocumab · 2,341 people
Adding alirocumab to maximally tolerated statin therapy markedly lowered LDL cholesterol and was associated with a post hoc reduction in cardiovascular events, though with somewhat higher rates of injection-site reactions, myalgia, neurocognitive, and ophthalmologic events.
Primary lipid endpoint metPCSK9 inhibitorsUS-approved
RUTHERFORD-22015 · Evolocumab · 331 people
In patients with heterozygous familial hypercholesterolaemia on stable lipid-lowering therapy, adding evolocumab produced rapid, large LDL cholesterol reductions of about 60% compared with placebo with a tolerability profile similar to placebo.
Primary lipid endpoint metPCSK9 inhibitorsUS-approved
TESLA Part B2015 · Evolocumab · 50 people
In patients with homozygous familial hypercholesterolaemia on stable background therapy, adding evolocumab 420 mg every 4 weeks significantly lowered LDL cholesterol compared with placebo and was well tolerated over 12 weeks.
Primary lipid endpoint metPCSK9 inhibitorsUS-approved
DESCARTES2014 · Evolocumab · 901 people
In patients with hyperlipidemia across a range of background lipid therapies, adding evolocumab for 52 weeks substantially lowered LDL cholesterol compared with placebo.
Primary lipid endpoint metPCSK9 inhibitorsUS-approved
MENDEL-22014 · Evolocumab · 614 people
In people with moderate hypercholesterolemia and low cardiovascular risk, evolocumab monotherapy (biweekly or monthly) lowered LDL-C far more than placebo or ezetimibe and had a similar rate of side effects as placebo/ezetimibe.
Primary lipid endpoint metPCSK9 inhibitorsUS-approved

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