TABLE 3

Clinical trials of PCSK9 inhibitors

Study	Drug	Description	No. patients	Weeks	Baseline LDL	Mean % LDL lowering
Phase 3 efficacy trials
MENDEL-2³⁵	Evolocumab	Monotherapy vs ezetimibe and placebo	614	12	140–144	55–57
DESCARTES³⁶	Evolocumab	Long-term tolerability/efficacy atorvastatin 10–80 ± ezetimibe	901	52	104 (95–120)	55–57
RUTHERFORD-2³⁷	Evolocumab	LDL-C goal achievement in HeFH on statin	331	12	151–161	59–61
LAPLACE-2³⁸	Evolocumab	Combined with different statins vs ezetimibe and placebo	2,067	12	108	55–76
GAUSS-2³⁹	Evolocumab	Statin intolerant vs ezetimibe	307	12	192–195	53–56
GAUSS-3⁴⁰	Evolocumab	Statin intolerant vs ezetimibe	511	24	212–219	53
TAUSSIG⁴¹	Evolocumab	Homozygous FH statin ± ezetimibe, open label	94	12	321	20.9
ODYSSEY FH I⁴²	Alirocumab	HeFH vs ezetimibe	486	24	145	58
ODYSSEY FH II⁴²	Alirocumab	HeFH vs ezetimibe	249	24	135	51
ODYSSEY-High FH⁴³	Alirocumab	HeFH on statin vs placebo	106	24	196–201	46
ODYSSEY-COMBO I⁴⁴	Alirocumab	Hypercholesterol vs placebo	316	24	95–100	48
ODYSSEY-COMBO II⁴⁵	Alirocumab	High CVD risk with ezetimibe vs placebo/ezetimibe	707	24	105–109	51
ODYSSEY CHOICE I⁴⁶	Alirocumab	Maximum statin or statin intolerant vs placebo	803	24	112–148	52 (no statin) 59 (+ statin)
ODYSSEY CHOICE II⁴⁷	Alirocumab	Combined with ezetimibe or fenofibrate or as monotherapy vs placebo	233	24	154–164	56
Phase 2 trials
NCT01592240⁴⁸	Bococizumab	Dose ranging, added to statins	250	24	105–118	34–53

CVD = cardiovascular disease; DESCARTES = Durable Effect of PCSK9 Antibody Compared With Placebo Study; GAUSS-2 = Goal Achievement After Utilizing an Anti-PCSK9 Antibody in Statin Intolerant Subjects-2; GAUSS-3 = Goal Achievement After Utilizing an Anti-PCSK9 Antibody in Statin Intolerant Subjects-3; HeFH = heterozygous familial hypercholesterolemia; LAPLACE-2 = LDL-C Assessment With PCSK9 Monoclonal Antibody Inhibition Combined With Statin Therapy-2; LDL-C = low-density lipoprotein cholesterol; MENDEL-2 = Monoclonal Antibody Against PCSK9 to Reduce Elevated LDL-C in Subjects Currently Not Receiving Drug Therapy for Easing Lipid Levels-2; ODYSSEY CHOICE I = Study to Evaluate the Efficacy and Safety of an Every Four Weeks Treatment Regimen of Alirocumab (REGN727/ SAR236553) in Patients With Primary Hypercholesterolemia; ODYSSEY CHOICE II = Phase III Study To Evaluate Alirocumab in Patients With Hypercholesterolemia Not Treated With a Statin; ODYSSEY COMBO I = Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With High Cardiovascular Risk and Hypercholesterolemia; ODYSSEY COMBO II = Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Ezetimibe on Top of Statin in High Cardiovascular Risk Patients With Hypercholesterolemia; ODYSSEY FH = Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With Heterozygous Familial Hypercholesterolemia Not Adequately Controlled With Their Lipid-Modifying Therapy; ODYSSEY-High FH = Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With Heterozygous Familial Hypercholesterolemia; RUTHERFORD-2 = Reduction of LDL-C With PCSK9 Inhibition in Heterozygous Familial Hypercholester-olemia Disorder Study-2; TAUSSIG = Trial Assessing Long Term Use of PCSK9 Inhibition in Subjects With Genetic LDL Disorders