Proprotein convertase subtilisin kexin 9 inhibitors: next generation in lipid-lowering therapy

Bibliography

• Catapano AL, Reiner Z, De Backer G, et al. ESC/EAS Guidelines for the management of dyslipidaemias The Task Force for the management of dyslipidaemias of the European Society of Cardiology (ESC) and the European Atherosclerosis Society (EAS). Atherosclerosis 2011;217:3-46
   PubMed Web of Science ®Google Scholar
• Stone NJ, Robinson JG, Lichtenstein AH, et al. 2013 ACC/AHA Guideline on the Treatment of Blood Cholesterol to Reduce Atherosclerotic Cardiovascular Risk in Adults: a Report of the American College of Cardiology/American Heart Association Task Force on Practice Guidelines. Circulation 2014;129:S1-S45
   PubMed Web of Science ®Google Scholar
• Seidah NG, Prat A. The biology and therapeutic targeting of the proprotein convertases. Nat Rev Drug Discov 2012;11:367-83
   PubMed Web of Science ®Google Scholar
• Maxwell KN, Breslow JL. Adenoviral-mediated expression of PCSK9 in mice results in a low-density lipoprotein receptor knockout phenotype. Proc Natl Acad Sci USA 2004;101:7100-5
   PubMed Web of Science ®Google Scholar
• Park SW, Moon YA, Horton JD. Post-transcriptional regulation of low density lipoprotein receptor protein by proprotein convertase subtilisin/kexin type 9a in mouse liver. J Biol Chem 2004;279:50630-8
   PubMed Web of Science ®Google Scholar
• Cariou B, Le May C, Costet P. Clinical aspects of PCSK9. Atherosclerosis 2011;216:258-65
   PubMed Web of Science ®Google Scholar
• Abifadel M, Varret M, Rabes JP, et al. Mutations in PCSK9 cause autosomal dominant hypercholesterolemia. Nat Genet 2003;34:154-6
   PubMed Web of Science ®Google Scholar
• Cohen J, Pertsemlidis A, Kotowski IK, et al. Low LDL cholesterol in individuals of African descent resulting from frequent nonsense mutations in PCSK9. Nat Genet 2005;37:161-5
   PubMed Web of Science ®Google Scholar
• Levy E, Ben Djoudi Ouadda A, Spahis S, et al. PCSK9 plays a significant role in cholesterol homeostasis and lipid transport in intestinal epithelial cells. Atherosclerosis 2013;227:297-306
   PubMed Web of Science ®Google Scholar
• Mbikay M, Sirois F, Mayne J, et al. PCSK9-deficient mice exhibit impaired glucose tolerance and pancreatic islet abnormalities. FEBS Lett 2010;584:701-6
   PubMed Web of Science ®Google Scholar
• Seidah NG, Sadr MS, Chretien M, Mbikay M. The multifaceted proprotein convertases: their unique, redundant, complementary, and opposite functions. J Biol Chem 2013;288:21473-81
   PubMed Web of Science ®Google Scholar
• Farnier M. The role of proprotein convertase subtilisin/kexin type 9 in hyperlipidemia: focus on therapeutic implications. Am J Cardiovasc Drugs 2011;11:145-52
   PubMed Web of Science ®Google Scholar
• Farnier M. PCSK9: from discovery to therapeutic applications. Arch Cardiovasc Dis 2014;107:58-66
   PubMed Web of Science ®Google Scholar
• Hooper AJ, Burnett JR. Anti-PCSK9 therapies for the treatment of hypercholesterolemia. Expert Opin Biol Ther 2013;13:429-35
   PubMed Web of Science ®Google Scholar
• Frank-Kamenetsky M, Grefhorst A, Anderson NN, et al. Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates. Proc Natl Acad Sci USA 2008;105:11915-20
   PubMed Web of Science ®Google Scholar
• Graham MJ, Lemonidis KM, Whipple CP, et al. Antisense inhibition of proprotein convertase subtilisin/kexin type 9 reduces serum LDL in hyperlipidemic mice. J Lipid Res 2007;48:763-7
   PubMed Web of Science ®Google Scholar
• Catapano AL, Papadopoulos N. The safety of therapeutic monoclonal antibodies: implications for cardiovascular disease and targeting the PCSK9 pathway. Atherosclerosis 2013;228:18-28
   PubMed Web of Science ®Google Scholar
• Dragan S, Serban MC, Banach M. Proprotein convertase subtilisin/Kexin 9 inhibitors: an emerging lipid-lowering therapy? J Cardiovasc Pharmacol Ther 2014. [ Epub ahead of print]
   PubMed Web of Science ®Google Scholar
• Banach M, Rizzo M, Obradovic M, et al. PCSK9 inhibition - a novel mechanism to treat lipid disorders? Curr Pharm Des 2013;19:3869-77
   PubMed Web of Science ®Google Scholar
• Fitzgerald K, Frank-Kamenetsky M, Shulga-Morskaya S, et al. Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial. Lancet 2014;383:60-8
   PubMed Web of Science ®Google Scholar
• Chan JC, Piper DE, Cao Q, et al. A proprotein convertase subtilisin/kexin type 9 neutralizing antibody reduces serum cholesterol in mice and nonhuman primates. Proc Natl Acad Sci USA 2009;106:9820-5
   PubMed Web of Science ®Google Scholar
• Ni YG, Di Marco S, Condra JH, et al. A PCSK9-binding antibody that structurally mimics the EGF(A) domain of LDL-receptor reduces LDL cholesterol in vivo. J Lipid Res 2011;52:78-86
   PubMed Web of Science ®Google Scholar
• Sun X, Essalmani R, Day R, et al. Proprotein convertase subtilisin/kexin type 9 deficiency reduces melanoma metastasis in liver. Neoplasia 2012;14:1122-31
   PubMed Web of Science ®Google Scholar
• Dias CS, Shaywitz AJ, Wasserman SM, et al. Effects of AMG 145 on low-density lipoprotein cholesterol levels: results from 2 randomized, double-blind, placebo-controlled, ascending-dose phase 1 studies in healthy volunteers and hypercholesterolemic subjects on statins. J Am Coll Cardiol 2012;60:1888-98
   PubMed Web of Science ®Google Scholar
• Stein EA, Mellis S, Yancopoulos GD, et al. Effect of a. monoclonal antibody to PCSK9 on LDL cholesterol. N Engl J Med 2012;366:1108-18
   PubMed Web of Science ®Google Scholar
• Koren MJ, Giugliano RP, Raal FJ, et al. Efficacy and safety of longer-term administration of evolocumab (AMG 145) in patients with hypercholesterolemia: 52-week results from the Open-Label Study of Long-Term Evaluation Against LDL-C (OSLER) randomized trial. Circulation 2014;129:234-43
   PubMed Web of Science ®Google Scholar
• Koren MJ, Scott R, Kim JB, et al. Efficacy, safety, and tolerability of a. monoclonal antibody to proprotein convertase subtilisin/kexin type 9 as monotherapy in patients with hypercholesterolaemia (MENDEL): a randomised, double-blind, placebo-controlled, phase 2 study. Lancet 2012;380:1995-2006
   PubMed Web of Science ®Google Scholar
• Sullivan D, Olsson AG, Scott R, et al. Effect of a. monoclonal antibody to PCSK9 on low-density lipoprotein cholesterol levels in statin-intolerant patients: the GAUSS randomized trial. JAMA 2012;308:2497-506
   PubMed Web of Science ®Google Scholar
• Raal F, Scott R, Somaratne R, et al. Low-density lipoprotein cholesterol-lowering effects of AMG 145, a. monoclonal antibody to proprotein convertase subtilisin/kexin type 9 serine protease in patients with heterozygous familial hypercholesterolemia: the Reduction of LDL-C with PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder (RUTHERFORD) randomized trial. Circulation 2012;126:2408-17
   PubMed Web of Science ®Google Scholar
• Giugliano RP, Desai NR, Kohli P, et al. Efficacy, safety, and tolerability of a mAb to proprotein convertase subtilisin/kexin type 9 in combination with a statin in patients with hypercholesterolaemia (LAPLACE-TIMI 57): a randomised, placebo-controlled, dose-ranging, phase 2 study. Lancet 2012;380:2007-17
   PubMed Web of Science ®Google Scholar
• Raal FJ, Giugliano RP, Sabatine MS, et al. Reduction in lipoprotein(a) with PCSK9 monoclonal antibody evolocumab (AMG 145): a pooled analysis of more than 1,300 patients in 4 phase II trials. J Am Coll Cardiol 2014;63:1278-88
   PubMed Web of Science ®Google Scholar
• McKenney JM, Koren MJ, Kereiakes DJ, et al. Safety and efficacy of a monoclonal antibody to proprotein convertase subtilisin/kexin type 9 serine protease, SAR236553/REGN727, in patients with primary hypercholesterolemia receiving ongoing stable atorvastatin therapy. J Am Coll Cardiol 2012;59:2344-53
   PubMed Web of Science ®Google Scholar
• Roth EM, McKenney JM, Hanotin C, et al. Atorvastatin with or without an antibody to PCSK9 in primary hypercholesterolemia. N Engl J Med 2012;367:1891-900
   PubMed Web of Science ®Google Scholar
• Chaparro-Riggers J, Liang H, DeVay RM, et al. Increasing serum half-life and extending cholesterol lowering in vivo by engineering antibody with pH-sensitive binding to PCSK9. J Biol Chem 2012;287:11090-7
   PubMed Web of Science ®Google Scholar
• Weinreich M, Frishman WH. Antihyperlipidemic therapies targeting PCSK9. Cardiol Rev 2014;22:140-6
   PubMed Web of Science ®Google Scholar
• Stroes E, Colquhoun D, Sullivan D, et al. Anti-PCSK9 antibody effectively lowers cholesterol in patients with statin intolerance: the GAUSS-2 randomized, placebo-controlled phase 3 clinical trial of evolocumab. J Am Coll Cardiol 2014;63:2541-8
   PubMed Web of Science ®Google Scholar
• Robinson JG, Nedergaard BS, Rogers WJ, et al. Effect of evolocumab or ezetimibe added to moderate- or high-intensity statin therapy on LDL-C lowering in patients with hypercholesterolemia: the LAPLACE-2 randomized clinical trial. JAMA 2014;311:1870-82
   PubMed Web of Science ®Google Scholar
• Raal FJ, Stein EA, Dufour R, et al. PCSK9 inhibition with evolocumab (AMG 145) in heterozygous familial hypercholesterolaemia (RUTHERFORD-2): a randomised, double-blind, placebo-controlled trial. Lancet 2014; pii: S0140-6736(14)61399-4; 10.1016/S0140-6736(14)61399-4
   Web of Science ®Google Scholar
• Raal FJ, Honarpour N, Blom DJ, et al. Inhibition of PCSK9 with evolocumab in homozygous familial hypercholesterolaemia (TESLA Part B): a randomised, double-blind, placebo-controlled trial. Lancet 2014; pii: S0140-6736(14)61374-X; 10.1016/S0140-6736(14)61374-X
   Web of Science ®Google Scholar
• Blom DJ, Hala T, Bolognese M, et al. A 52-week placebo-controlled trial of evolocumab in hyperlipidemia. N Engl J Med 2014;370:1809-19
   PubMed Web of Science ®Google Scholar
• Koren MJ, Lundqvist P, Bolognese M, et al. Anti-PCSK9 monotherapy for hypercholesterolemia: the MENDEL-2 randomized, controlled phase III clinical trial of evolocumab. J Am Coll Cardiol 2014;63:2531-40
   PubMed Web of Science ®Google Scholar
• Roth EM, Taskinen MR, Ginsberg HN, et al. Monotherapy with the PCSK9 inhibitor alirocumab versus ezetimibe in patients with hypercholesterolemia: results of a 24week, double-blind, randomized Phase 3 trial. Int J Cardiol 2014;176(1):55-61
   PubMed Web of Science ®Google Scholar
• Cicero AF, Tartagni E, Ertek S. Safety and tolerability of injectable lipid-lowering drugs: a review of available clinical data. Expert Opin Drug Saf 2014;13:1023-30
   PubMed Web of Science ®Google Scholar
• Stein EA, Gipe D, Bergeron J, et al. Effect of a monoclonal antibody to PCSK9, REGN727/SAR236553, to reduce low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolaemia on stable statin dose with or without ezetimibe therapy: a phase 2 randomised controlled trial. Lancet 2012;380:29-36
   PubMed Web of Science ®Google Scholar
• Zhao Z, Tuakli-Wosornu Y, Lagace TA, et al. Molecular characterization of loss-of-function mutations in PCSK9 and identification of a compound heterozygote. Am J Hum Genet 2006;79:514-23
   PubMed Web of Science ®Google Scholar
• Rader DJ, Kastelein JJ. Lomitapide and mipomersen: two first-in-class drugs for reducing low-density lipoprotein cholesterol in patients with homozygous familial hypercholesterolemia. Circulation 2014;129:1022-32
   PubMed Web of Science ®Google Scholar
• Desai NR, Kohli P, Giugliano RP, et al. AMG145, a monoclonal antibody against proprotein convertase subtilisin kexin type 9, significantly reduces lipoprotein(a) in hypercholesterolemic patients receiving statin therapy: an analysis from the LDL-C Assessment with Proprotein Convertase Subtilisin Kexin Type 9 Monoclonal Antibody Inhibition Combined with Statin Therapy (LAPLACE)-Thrombolysis in Myocardial Infarction (TIMI) 57 trial. Circulation 2013;128:962-9
   PubMed Web of Science ®Google Scholar