【レポートの概要（一部）】
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
【レポート販売概要】
■ タイトル：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）■ 英文：Amyloidosis - Pipeline Review, H1 2015
■ 発行日：2015年1月22日
■ 調査会社：Global Markets Direct
■ 商品コード：GMDHC6041IDB
■ 調査対象地域：グローバル
- 世界のヨット市場2015The Global Yacht Industry 2015 Market Research Report is a professional and in-depth study on the current state of the Yacht industry. The report provides a basic overview of the industry including definitions, classifications, applications and industry chain structure.The Yacht market analysis is provided for the international markets including development trends, competitive landscape analysis, …
- 韓国の心臓律動管理（CRM）装置市場見通しSouth Korea Cardiac Rhythm Management (CRM) Procedures Outlook to 2021 Summary GlobalData’s new report, "South Korea Cardiac Rhythm Management (CRM) Procedures Outlook to 2021", provides key procedures data on the South Korea Cardiac Rhythm Management (CRM) Procedures. The report provides procedure volumes within market segments - Implantable Loop Recorder (ILR) Procedures, Cardiac Resynchronisati …
- 資産管理システムの世界市場予測（～2022）：ソリューション別、資産種類別（電子的資産、リターナブル輸送資産、輸送中機器、製造資産、人材）、産業別、地域別The asset management system market is expected to reach USD 25.55 Billion by 2022, at a CAGR of 15.17% between 2016 and 2022. The growth of the asset management system market is propelled by factors such as optimum resource utilization offered by efficient asset tracking and management and reduced maintenance downtime owing to deployment of relevant solutions such as Real-Time Location System (RTL …
- 先進CO2センサーの世界市場：NDIR式CO2センサー、化学CO2センサーAdvanced CO2 sensors help to monitor the intensity of CO2 in different mediums like air or water and can adjust automatically to changes in temperature, humidity, and altitude. This market is an integral part of the global sensors market and is heavily influenced by the augmented demand for location-based advanced CO2 sensors, which results in its impressive CAGR of nearly 42% by 2019. Technavio's …
- プレキャスト建設の世界市場予測（～2021年）MarketsandMarkets projects that the precast construction market size will grow from USD 124.77 Billion in 2015 to USD 177.09 Billion by 2021, at an estimated CAGR of 6.10%. The precast construction market is expected to witness high growth as a result of the rise in urbanization, large-scale investments in infrastructure & industrial sectors, and growth in construction activities in emerging econo …
- ビックデータ及びテレコム分析市場：事例、市場分析、予測2014-2019（Big Data and Telecom Analytics Market: Business Case, Market Analysis & Forecasts 2014 – 2019）Big Data refers to a massive volume of both structured and unstructured data that is so large that it is difficult to process using traditional database and software techniques. While the presence of such datasets is not something new, the past few years have witnessed immense commercial investments in solutions that address the processing and analysis of Big Data. Big Data opens a vast array of a …
- VAALCO Energy, Inc.：石油・ガスバリューチェーン分析レポート2013VAALCO Energy, Inc. Analysis Across the Oil and Gas Value Chain Report, 2013 Update Summary "VAALCO Energy, Inc." Analysis Across the Oil and Gas Value Chain is an essential source for data, analysis and strategic insight into “VAALCO Energy, Inc.”. The report provides key information relating to oil and gas assets of the company along with its operations across the value chain. The report examine …
- 世界の放射線治療装置市場動向（2014-2018）About Radiation Therapy Equipment Radiation therapy equipment is used to perform radiation therapy, also known as radiotherapy or radiation oncology, which is a procedure used in the treatment of cancer. It involves delivering high doses of radiation consisting of photons, electrons or gamma rays to kill cancer cells and reduce the rate at which the cancerous cells multiply by damaging the DNA ins …
- 海中断熱材料の世界市場：ポリウレタン、ポリプロピレン、シリコーンゴム、エポキシ、エーロゲルThe Subsea thermal insulation materials market is projected to register a CAGR of 4.80% between 2014 and 2019 to reach $142.30 million by 2019. Europe was estimated to hold the largest share in 2013 with the U.S. being the leading country. In the less explored markets of Middle East & Africa, and Latin America, a high growth is expected while the highly developed North American region is predicted …
- 自動車用レーダーの世界市場予測（～2021年）The automotive RADAR market size is projected to grow at a CAGR of 23.81% during the forecast period, to reach USD 6.61 billion by 2021. Rising concerns pertaining to vehicle, driver, passenger, and pedestrian safety along with increased involvement of players across the ecosystem have fueled the growth of the automotive RADAR market globally. The growing trend of autonomous vehicles is also a maj …
- 自動車用サラウンドビューシステムの世界市場2016-2020About the Automotive Surround View Systems Market The automotive surround view system is an emerging automotive ADAS (advanced driver assistance system) technology that assist in low speed vehicle manoeuvres such as while parking the vehicle or slow curb side driving. The system helps the driver by allowing him/her to see a top-down view of the 360-degree surroundings of the vehicle. It normal con …
- 液圧プレス機械の世界市場2017-2021About Hydraulic Press Machines Press machine is a type of machine, which is crucial to industrial manufacturing processes. Presses provide energy by exerting a force, which acts over a stroke. Hydraulic press machines are widely used in the metal forging industry to perform blanking, stamping, coining and embossing. Press machines are also used in the metal fabrication and metal extrusion processe …
- 指関節用インプラントの世界市場2019-2023Global Digit Joint Implants Market: About this market Digit joint implants are medical devices used to replace the damaged or diseased bone and joints of body parts. Technavio’s digit joint implants market analysis considers sales from MCP and PIP joint implants, Trapeziometacarpal joint implants, Toe implants, Others. Our analysis also considers the sales of digit joint implants in Asia, Europe, …
- 内視鏡の世界市場分析および予測MediPoint: Endoscopy - Global Analysis and Market Forecasts Summary The global endoscopy market is a dynamic, billion-dollar market that is expected to grow through 2021. The market is also very saturated with intense competition due to the availability of similar product offerings across market players. In the face of increasing hospital consolidation into one network and the trend of oligopoly p …
- 世界の製薬産業分析2015The global presence of the pharmaceutical industry is evident with the roll out of continent specific R&D programs and drugs which help companies maximize penetration of markets and garner increased revenues with intercontinental treatment demand of patients being serviced from often one or two mega research centers in the continent. By contribution of size, the pharmaceutical industry is majorly …
