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アミロイドーシス:パイプライン製品の分析

Amyloidosis - Pipeline Review, H1 2017

発行 Global Markets Direct 商品コード 192465
出版日 ページ情報 英文 160 Pages
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アミロイドーシス:パイプライン製品の分析 Amyloidosis - Pipeline Review, H1 2017
出版日: 2017年02月28日 ページ情報: 英文 160 Pages
概要

アミロイドーシスとして知られる疾患群は、アミロイドタンパク質が体内の組織に異常に蓄積することから生じます。臓器の形状や機能に影響を与えるため、最終的に臓器不全に至ることもあります。素因として、L鎖と呼ばれる異常タンパク質のミスフォールディング、それに続く体内での蓄積が挙げられます。透析の結果、生じる場合もあります。症状は、倦怠感、胸痛、腫脹、呼吸障害などが見られます。治療には、移植、投薬 (利尿薬、化学療法薬) などが含まれます。

当レポートでは、アミロイドーシスに対する治療薬の開発状況について調査分析し、パイプライン製品の概要、治験の段階別の製品の概要、主要企業プロファイル、薬剤のプロファイル、パイプライン製品の最新動向などについて、体系的な情報を提供しています。

目次

イントロダクション

アミロイドーシスの概要

治療薬の開発

  • パイプライン製品:概要
  • パイプライン製品:比較分析

開発中の治療薬:企業別

調査中の治療薬:大学/研究機関別

パイプライン製品の概要

  • 後期段階の製品
  • 治験段階の製品
  • 初期段階の製品

開発中の製品:企業別

調査中の製品:大学/研究機関別

治療薬の開発に従事している企業

  • Alnylam Pharmaceuticals, Inc.
  • Arcturus Therapeutics, Inc
  • Bellus Health Inc.
  • Bsim2
  • Celgene Corporation
  • GlaxoSmithKline Plc
  • Isis Pharmaceuticals, Inc.
  • Millennium Pharmaceuticals, Inc.
  • Onyx Pharmaceuticals, Inc.
  • Pfizer Inc.
  • ProteoTech, Inc.
  • Prothena Corporation Plc
  • SOM Innovation Biotech SL

治療薬の評価

  • 単独療法の場合
  • 併用療法の場合
  • 標的別
  • 作用機序別
  • 投与経路別
  • 分子タイプ別

薬剤のプロファイル

  • ALN-TTRsc02
  • Antisense RNAi Oligonucleotides to Inhibit Amyloid Precursor Protein for Amyloidosis
  • carfilzomib
  • CLR-01
  • eprodisate disodium
  • GSK-2315698 + GSK-2398852
  • GSK-2398852
  • ixazomib citrate
  • LUNAR-101
  • LUNAR-102
  • Monoclonal Antibody for Amyloidosis
  • Monoclonal Antibody to Inhibit ATTR for Familial Amyloid Polyneuropathy
  • NEOD-001
  • NPT-005
  • patisiran
  • pomalidomide
  • PTI-110
  • revusiran
  • Small Molecules for AL Amyloidosis
  • Small Molecules to Inhibit Transthyretin for Familial Amyloid Polyneuropathy
  • Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Cardiomyopathy
  • Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Polyneuropathy
  • tafamidis meglumine
  • tolcapone

パイプライン製品の最新動向

休止状態のプロジェクト

開発が中止された製品

製品開発のマイルストーン

  • 最新ニュースとプレスリリース

付録

図表

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目次
Product Code: GMDHC9004IDB

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Amyloidosis - Pipeline Review, H1 2017, provides an overview of the Amyloidosis (Metabolic Disorders) pipeline landscape.

Abnormal accumulation of amyloid protein in the tissues results in a group of diseases known as amyloidosis. It may affect the shape and functioning of the organ eventually leading to organ failure. Predisposing factors are misfolding of abnormal protein called the light chain and its subsequent deposition in the body. It may also occur as a result of dialysis. Symptoms include fatigue, chest pain, swelling and breathing problems. Amyloidosis can be managed by transplant and, medication such as diuretics and chemotherapy drugs.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Amyloidosis - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Amyloidosis (Metabolic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Amyloidosis (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Amyloidosis and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Filing rejected/Withdrawn, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 1, 4, 7, 7, 4 and 5 respectively. Similarly, the Universities portfolio in Phase II, Preclinical and Discovery stages comprises 1, 3 and 2 molecules, respectively.

Amyloidosis (Metabolic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Amyloidosis (Metabolic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Amyloidosis (Metabolic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Amyloidosis (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Amyloidosis (Metabolic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews latest news related to pipeline therapeutics for Amyloidosis (Metabolic Disorders)

Reasons to buy

  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Amyloidosis (Metabolic Disorders).
  • Classify potential new clients or partners in the target demographic.
  • Develop tactical initiatives by understanding the focus areas of leading companies.
  • Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Amyloidosis (Metabolic Disorders) pipeline depth and focus of Indication therapeutics.
  • Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

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
  • Amyloidosis - Therapeutics Development 10
  • Pipeline Overview 10
  • Pipeline by Companies 11
  • Pipeline by Universities/Institutes 14
  • Products under Development by Companies 15
  • Products under Development by Universities/Institutes 17
  • Amyloidosis - Therapeutics Assessment 18
  • Assessment by Target 18
  • Assessment by Mechanism of Action 20
  • Assessment by Route of Administration 22
  • Assessment by Molecule Type 24
  • Amyloidosis - Companies Involved in Therapeutics Development 26
  • Alnylam Pharmaceuticals Inc 26
  • Amgen Inc 26
  • Arcturus Therapeutics Inc 27
  • Bsim2 28
  • Celgene Corp 28
  • GlaxoSmithKline Plc 29
  • Ionis Pharmaceuticals Inc 29
  • Johnson & Johnson 30
  • Millennium Pharmaceuticals Inc 30
  • Neurimmune Holding AG 31
  • Novartis AG 31
  • Oncopeptides AB 32
  • Pfizer Inc 32
  • Proclara Biosciences Inc 33
  • Prothena Corp Plc 33
  • R Pharm 34
  • Regeneron Pharmaceuticals Inc 34
  • SOM Biotech SL 35
  • Spectrum Pharmaceuticals Inc 35
  • Amyloidosis - Drug Profiles 36
  • ALN-ANG - Drug Profile 36
  • Product Description 36
  • Mechanism Of Action 36
  • R&D Progress 36
  • ALN-TTRsc02 - Drug Profile 37
  • Product Description 37
  • Mechanism Of Action 37
  • R&D Progress 37
  • Antisense RNAi Oligonucleotides to Inhibit Amyloid Precursor Protein for Amyloidosis - Drug Profile 39
  • Product Description 39
  • Mechanism Of Action 39
  • R&D Progress 39
  • CAEL-101 - Drug Profile 40
  • Product Description 40
  • Mechanism Of Action 40
  • R&D Progress 40
  • canakinumab - Drug Profile 41
  • Product Description 41
  • Mechanism Of Action 41
  • R&D Progress 41
  • carfilzomib - Drug Profile 47
  • Product Description 47
  • Mechanism Of Action 47
  • R&D Progress 47
  • CLR-01 - Drug Profile 60
  • Product Description 60
  • Mechanism Of Action 60
  • R&D Progress 60
  • daratumumab - Drug Profile 62
  • Product Description 62
  • Mechanism Of Action 62
  • R&D Progress 62
  • dezamizumab - Drug Profile 76
  • Product Description 76
  • Mechanism Of Action 76
  • R&D Progress 76
  • dezamizumab + GSK-2315698 - Drug Profile 77
  • Product Description 77
  • Mechanism Of Action 77
  • R&D Progress 77
  • doxycycline hyclate - Drug Profile 78
  • Product Description 78
  • Mechanism Of Action 78
  • R&D Progress 78
  • EDE-1307 - Drug Profile 79
  • Product Description 79
  • Mechanism Of Action 79
  • R&D Progress 79
  • GSK-2315698 - Drug Profile 80
  • Product Description 80
  • Mechanism Of Action 80
  • R&D Progress 80
  • GSK-3039294 - Drug Profile 81
  • Product Description 81
  • Mechanism Of Action 81
  • R&D Progress 81
  • inotersen sodium - Drug Profile 82
  • Product Description 82
  • Mechanism Of Action 82
  • R&D Progress 82
  • ixazomib citrate - Drug Profile 86
  • Product Description 86
  • Mechanism Of Action 86
  • R&D Progress 86
  • LUNAR-TTR - Drug Profile 96
  • Product Description 96
  • Mechanism Of Action 96
  • R&D Progress 96
  • melphalan - Drug Profile 97
  • Product Description 97
  • Mechanism Of Action 97
  • R&D Progress 97
  • melphalan flufenamide - Drug Profile 101
  • Product Description 101
  • Mechanism Of Action 101
  • R&D Progress 101
  • Monoclonal Antibody to Inhibit ATTR for Familial Amyloid Polyneuropathy - Drug Profile 103
  • Product Description 103
  • Mechanism Of Action 103
  • R&D Progress 103
  • Monoclonal Antibody to Target CD66b for Blood Cancer and Metabolic Disorders - Drug Profile 104
  • Product Description 104
  • Mechanism Of Action 104
  • R&D Progress 104
  • NEOD-001 - Drug Profile 105
  • Product Description 105
  • Mechanism Of Action 105
  • R&D Progress 105
  • NI-301 - Drug Profile 110
  • Product Description 110
  • Mechanism Of Action 110
  • R&D Progress 110
  • NPT-289 - Drug Profile 111
  • Product Description 111
  • Mechanism Of Action 111
  • R&D Progress 111
  • Oligonucleotides to Inhibit Transthyretin for Amyloidosis - Drug Profile 112
  • Product Description 112
  • Mechanism Of Action 112
  • R&D Progress 112
  • patisiran - Drug Profile 113
  • Product Description 113
  • Mechanism Of Action 113
  • R&D Progress 113
  • pomalidomide - Drug Profile 121
  • Product Description 121
  • Mechanism Of Action 121
  • R&D Progress 121
  • PRX-004 - Drug Profile 128
  • Product Description 128
  • Mechanism Of Action 128
  • R&D Progress 128
  • RPH-104 - Drug Profile 129
  • Product Description 129
  • Mechanism Of Action 129
  • R&D Progress 129
  • Small Molecule to Inhibit Amyloid Beta Peptide for Alzheimer's Disease and Amyloidosis - Drug Profile 130
  • Product Description 130
  • Mechanism Of Action 130
  • R&D Progress 130
  • Small Molecules to Inhibit Transthyretin for Familial Amyloid Polyneuropathy - Drug Profile 131
  • Product Description 131
  • Mechanism Of Action 131
  • R&D Progress 131
  • Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Cardiomyopathy - Drug Profile 132
  • Product Description 132
  • Mechanism Of Action 132
  • R&D Progress 132
  • Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Polyneuropathy - Drug Profile 133
  • Product Description 133
  • Mechanism Of Action 133
  • R&D Progress 133
  • tafamidis meglumine - Drug Profile 134
  • Product Description 134
  • Mechanism Of Action 134
  • R&D Progress 134
  • tolcapone - Drug Profile 137
  • Product Description 137
  • Mechanism Of Action 137
  • R&D Progress 137
  • Amyloidosis - Dormant Projects 139
  • Amyloidosis - Discontinued Products 140
  • Amyloidosis - Product Development Milestones 141
  • Featured News & Press Releases 141
  • Jan 04, 2017: Fortress Biotech Forms New Subsidiary, Caelum Biosciences to Develop Novel Treatment for AL Amyloidosis 141
  • Dec 16, 2016: Novartis receives positive CHMP recommendation for Ilaris to treat rare diseases in adults and children called Periodic Fever Syndromes 142
  • Nov 07, 2016: VYNDAQEL (tafamidis) Receives Approval in Brazil by ANVISA for the Treatment of Transthyretin Familial Amyloid Polyneuropathy 142
  • Nov 03, 2016: Data for Prothena NEOD001 to be Presented at the 58th Annual American Society of Hematology Meeting 143
  • Oct 10, 2016: Data Monitoring Committee Recommends Continuation of APOLLO Phase 3 Clinical Trial of Patisiran for Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN) 144
  • Sep 23, 2016: Novartis receives three new FDA approvals for the expanded use of Ilaris treating rare Periodic Fever Syndrome conditions 145
  • Aug 08, 2016: Pfizer Announces Publication of New Analysis Showing Long-Term Therapy with VYNDAQEL (tafamidis) Slowed Progression of Rare Neurodegenerative Disease 145
  • Jul 07, 2016: Ionis Pharmaceuticals Reports Positive Clinical Data on IONIS-TTR Rx at the XV International Symposium on Amyloidosis 147
  • Jul 07, 2016: Ionis Pharmaceuticals Reports Positive Clinical Data on IONIS-TTR Rx at the XV International Symposium on Amyloidosis 148
  • Jul 05, 2016: Prothena Presents New Data from Phase 1/2 Study of NEOD001 Demonstrating Improvements in Three Organ Systems in Previously-Treated Patients with AL Amyloidosis 150
  • Jul 01, 2016: Alnylam Reports New Results on patisiran at the XV International Symposium on Amyloidosis 153
  • Jun 27, 2016: Alnylam to Report New Patisiran Results at the XV International Symposium on Amyloidosis 155
  • Jun 21, 2016: Prothena to Present at 15th International Symposium on Amyloidosis Showcasing Broad Commitment to Advancing New Therapies for Systemic Amyloidoses 156
  • Jun 09, 2016: Alnylam Initiates Phase 1 Clinical Trial for ALN-TTRsc02, an Investigational RNAi Therapeutic for the Treatment of TTR-Mediated Amyloidosis 158
  • Appendix 159
  • Methodology 159
  • Coverage 159
  • Secondary Research 159
  • Primary Research 159
  • Expert Panel Validation 159
  • Contact Us 159
  • Disclaimer 160

List of Tables

  • Number of Products under Development for Amyloidosis, H1 2017 10
  • Number of Products under Development by Companies, H1 2017 12
  • Number of Products under Development by Companies, H1 2017 (Contd..1), H1 2017 13
  • Number of Products under Development by Universities/Institutes, H1 2017 14
  • Products under Development by Companies, H1 2017 15
  • Products under Development by Companies, H1 2017 (Contd..1), H1 2017 16
  • Products under Development by Universities/Institutes, H1 2017 17
  • Number of Products by Stage and Target, H1 2017 19
  • Number of Products by Stage and Mechanism of Action, H1 2017 21
  • Number of Products by Stage and Route of Administration, H1 2017 23
  • Number of Products by Stage and Molecule Type, H1 2017 25
  • Amyloidosis - Pipeline by Alnylam Pharmaceuticals Inc, H1 2017 26
  • Amyloidosis - Pipeline by Amgen Inc, H1 2017 27
  • Amyloidosis - Pipeline by Arcturus Therapeutics Inc, H1 2017 27
  • Amyloidosis - Pipeline by Bsim2, H1 2017 28
  • Amyloidosis - Pipeline by Celgene Corp, H1 2017 28
  • Amyloidosis - Pipeline by GlaxoSmithKline Plc, H1 2017 29
  • Amyloidosis - Pipeline by Ionis Pharmaceuticals Inc, H1 2017 29
  • Amyloidosis - Pipeline by Johnson & Johnson, H1 2017 30
  • Amyloidosis - Pipeline by Millennium Pharmaceuticals Inc, H1 2017 30
  • Amyloidosis - Pipeline by Neurimmune Holding AG, H1 2017 31
  • Amyloidosis - Pipeline by Novartis AG, H1 2017 31
  • Amyloidosis - Pipeline by Oncopeptides AB, H1 2017 32
  • Amyloidosis - Pipeline by Pfizer Inc, H1 2017 32
  • Amyloidosis - Pipeline by Proclara Biosciences Inc, H1 2017 33
  • Amyloidosis - Pipeline by Prothena Corp Plc, H1 2017 33
  • Amyloidosis - Pipeline by R Pharm, H1 2017 34
  • Amyloidosis - Pipeline by Regeneron Pharmaceuticals Inc, H1 2017 34
  • Amyloidosis - Pipeline by SOM Biotech SL, H1 2017 35
  • Amyloidosis - Pipeline by Spectrum Pharmaceuticals Inc, H1 2017 35
  • Amyloidosis - Dormant Projects, H1 2017 139
  • Amyloidosis - Discontinued Products, H1 2017 140

List of Figures

  • Number of Products under Development for Amyloidosis, H1 2017 10
  • Number of Products under Development by Companies, H1 2017 11
  • Number of Products under Development by Universities/Institutes, H1 2017 14
  • Number of Products by Top 10 Targets, H1 2017 18
  • Number of Products by Stage and Top 10 Targets, H1 2017 18
  • Number of Products by Top 10 Mechanism of Actions, H1 2017 20
  • Number of Products by Stage and Top 10 Mechanism of Actions, H1 2017 20
  • Number of Products by Routes of Administration, H1 2017 22
  • Number of Products by Stage and Routes of Administration, H1 2017 22
  • Number of Products by Molecule Types, H1 2017 24
  • Number of Products by Stage and Molecule Types, H1 2017 24
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