家族性高コレステロール血症(II型高リポ蛋白血症)(Familial Hypercholesterolemia)：治療薬開発パイプライン動向（2015年上半期版）...市場調査レポートについてご紹介

Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) Overview 8
Therapeutics Development 9
Pipeline Products for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) – Overview 9
Pipeline Products for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) – Comparative Analysis 10
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) – Therapeutics under Development by Companies 11
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) – Therapeutics under Investigation by Universities/Institutes 13
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) – Pipeline Products Glance 14
Late Stage Products 14
Clinical Stage Products 15
Early Stage Products 16
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) – Products under Development by Companies 17
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) – Products under Investigation by Universities/Institutes 19
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) – Companies Involved in Therapeutics Development 20
Aegerion Pharmaceuticals, Inc. 20
Amgen Inc. 21
Cerenis Therapeutics Holding SA 22
CymaBay Therapeutics, Inc. 23
Merck & Co., Inc. 24
Pfizer Inc. 25
Regeneron Pharmaceuticals, Inc. 26
ReGenX Biosciences, LLC 27
Sanofi 28
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) – Therapeutics Assessment 29
Assessment by Monotherapy Products 29
Assessment by Target 30
Assessment by Mechanism of Action 32
Assessment by Route of Administration 34
Assessment by Molecule Type 36
Drug Profiles 38
AEM-28 – Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
alirocumab – Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
AM-0010 – Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
anacetrapib – Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
Antisense Oligonucleotides to Inhibit ApoB100 for Familial Hypercholesterolemia – Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
bococizumab – Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
BSN-272 – Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
CER-001 – Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
evinacumab – Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
evolocumab – Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
Gene Therapy to Activate LDL Receptor for Homozygous Familial Hypercholesterolemia – Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
lomitapide mesylate – Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
MBX-8025 – Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
mipomersen sodium – Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) – Recent Pipeline Updates 62
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) – Dormant Projects 87
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) – Discontinued Products 88
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) – Product Development Milestones 89
Featured News & Press Releases 89
Jan 28, 2015: Aegerion Investigated by Former Louisiana Attorney General and Special Prosecutor Richard P. Ieyoub and the Firm of Ieyoub O’Bell & Myers 89
Nov 18, 2014: Genzyme And Isis Present KYNAMRO Clinical Data At The American Heart Association 89
Sep 02, 2014: Isis Pharmaceuticals Presents Data On KYNAMRO At European Society of Cardiology Congress 2014 90
Sep 02, 2014: Isis Pharmaceuticals Presents Data On KYNAMRO At European Society of Cardiology Congress 2014 91
Aug 27, 2014: Aegerion Pharmaceuticals Announces Resolution of Warning Letter for JUXTAPID Capsules 92
Jun 02, 2014: Cerenis Reports Two Positive Phase II Studies for HDL Mimetic CER-001 at EAS 92
May 08, 2014: Genzyme and Isis Receive Corporate Award from the National Organization for Rare Disorders for the Development of KYNAMRO 93
Apr 09, 2014: Aegerion Pharmaceuticals Initiates Phase 3 Clinical Trial in Japan to Evaluate Lomitapide for Treatment of Homozygous Familial Hypercholesterolemia 94
Mar 30, 2014: Data From Phase 3 Pivotal Studies Show Amgen’s Novel Investigational Cholesterol-Lowering Medication Evolocumab Significantly Reduced LDL Cholesterol In Statin Intolerant Patients And In Patients On Statins 95
Mar 27, 2014: Sanofi and Regeneron To Present Alirocumab Clinical Data at the American College of Cardiology’s 63rd Annual Scientific Session 98
Appendix 100
Methodology 100
Coverage 100
Secondary Research 100
Primary Research 100
Expert Panel Validation 100
Contact Us 100
Disclaimer 101