アミロイドーシス(Amyloidosis)の治療薬パイプライン動向（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 6
List of Figures 7
Introduction 8
Global Markets Direct Report Coverage 8
Amyloidosis Overview 9
Therapeutics Development 10
Pipeline Products for Amyloidosis – Overview 10
Pipeline Products for Amyloidosis – Comparative Analysis 11
Amyloidosis – Therapeutics under Development by Companies 12
Amyloidosis – Therapeutics under Investigation by Universities/Institutes 14
Amyloidosis – Pipeline Products Glance 15
Late Stage Products 15
Clinical Stage Products 16
Early Stage Products 17
Amyloidosis – Products under Development by Companies 18
Amyloidosis – Products under Investigation by Universities/Institutes 20
Amyloidosis – Companies Involved in Therapeutics Development 21
Alnylam Pharmaceuticals, Inc. 21
Arcturus Therapeutics, Inc 23
Auven Therapeutics Management L.L.L.P 24
Bellus Health Inc. 25
Bsim2 26
Celgene Corporation 27
GlaxoSmithKline plc 28
Immune System Therapeutics Ltd. 29
Isis Pharmaceuticals, Inc. 30
NeuroPhage Pharmaceuticals, Inc. 31
Onyx Pharmaceuticals, Inc. 32
Pfizer Inc. 33
ProteoTech, Inc. 34
Prothena Corporation plc 35
SOM Innovation Biotech SL 36
Takeda Oncology 37
Amyloidosis – Therapeutics Assessment 38
Assessment by Monotherapy Products 38
Assessment by Combination Products 39
Assessment by Target 40
Assessment by Mechanism of Action 42
Assessment by Route of Administration 44
Assessment by Molecule Type 46
Drug Profiles 48
ALN-TTRsc – Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
ALNTTR-01 – Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
Antisense Oligonucleotide for Transthyretin Amyloidosis – Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
carfilzomib – Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
eprodisate disodium – Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
GSK-2315698 – Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
GSK-2315698 + GSK-2398852 – Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
GSK-2398852 – Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
ISIS-TTRRx – Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
IST-1097 – Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
ixazomib citrate – Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
LUNAR-102 – Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
Monoclonal Antibody for Amyloidosis – Drug Profile 68
Product Description 68
Mechanism of Action 68
R&D Progress 68
Monoclonal Antibody to Inhibit ATTR for Familial Amyloid Polyneuropathy – Drug Profile 69
Product Description 69
Mechanism of Action 69
R&D Progress 69
NEOD-001 – Drug Profile 70
Product Description 70
Mechanism of Action 70
R&D Progress 70
NPT-005 – Drug Profile 72
Product Description 72
Mechanism of Action 72
R&D Progress 72
patisiran – Drug Profile 73
Product Description 73
Mechanism of Action 73
R&D Progress 73
pomalidomide – Drug Profile 74
Product Description 74
Mechanism of Action 74
R&D Progress 74
PTI-110 – Drug Profile 78
Product Description 78
Mechanism of Action 78
R&D Progress 78
RNAi Oligonucleotides to Inhibit Amyloid Precursor Protein for Amyloidosis – Drug Profile 79
Product Description 79
Mechanism of Action 79
R&D Progress 79
Small Molecule for AL Amyloidosis – Drug Profile 80
Product Description 80
Mechanism of Action 80
R&D Progress 80
Small Molecules to Inhibit Transthyretin for Familial Amyloid Polyneuropathy – Drug Profile 81
Product Description 81
Mechanism of Action 81
R&D Progress 81
Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Cardiomyopathy – Drug Profile 82
Product Description 82
Mechanism of Action 82
R&D Progress 82
Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Polyneuropathy – Drug Profile 83
Product Description 83
Mechanism of Action 83
R&D Progress 83
tafamidis meglumine – Drug Profile 84
Product Description 84
Mechanism of Action 84
R&D Progress 84
tolcapone – Drug Profile 86
Product Description 86
Mechanism of Action 86
R&D Progress 86
Amyloidosis – Recent Pipeline Updates 87
Amyloidosis – Dormant Projects 116
Amyloidosis – Product Development Milestones 117
Featured News & Press Releases 117
Dec 15, 2014: Prothena Receives FDA Fast Track Designation for NEOD001, a Monoclonal Antibody for the Treatment of Patients With AL Amyloidosis 117
Dec 11, 2014: Alnylam Initiates ENDEAVOUR Phase 3 Clinical Trial with Revusiran (ALN-TTRsc), an Investigational RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR Cardiac Amyloidosis 118
Dec 11, 2014: Alnylam Initiates ENDEAVOUR Phase 3 Clinical Trial with Revusiran (ALN-TTRsc), an Investigational RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR Cardiac Amyloidosis 119
Dec 02, 2014: Prothena Initiates NEOD001 Global Phase 3 Registrational Trial Based on Positive Results in Ongoing Phase 1/2 Study of NEOD001 in Patients With AL Amyloidosis 121
Dec 01, 2014: Takeda’s Investigational, Oral Proteasome Inhibitor Ixazomib Granted Breakthrough Therapy Designation by U.S. FDA for Relapsed or Refractory Systemic Light-chain Amyloidosis 123
Nov 14, 2014: Alnylam Announces Positive Initial Phase 2 Data with Revusiran 124
Nov 05, 2014: Alnylam Completes Phase 2 Enrollment and Initiates Open-Label Extension Study with Revusiran (ALN-TTRsc), an Investigational RNAi Therapeutic Targeting Transthyretin for the Treatment of TTR Cardiac Amyloidosis 126
Oct 14, 2014: Alnylam Presents Additional Data From Phase 1 Trial With ALN-TTRsc At 10th Annual Meeting of the Oligonucleotide Therapeutics Society 128
Oct 13, 2014: Arcturus Therapeutics to Present Gene Knockdown Data in Non-Human Primates, Showing up to 94% Reduction in Gene Expression with a Single Low Dose 129
Oct 13, 2014: Alnylam Reports Six-Month Clinical Data from Patisiran Phase 2 Open-Label Extension (OLE) Study in Patients with Familial Amyloidotic Polyneuropathy (FAP) 129
Appendix 132
Methodology 132
Coverage 132
Secondary Research 132
Primary Research 132
Expert Panel Validation 132
Contact Us 133
Disclaimer 133