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    Amyloidosis - Pipeline Review, H1 2017

    Amyloidosis - Pipeline Review, H1 2017

    • Report Code ID: RW0001689436
    • Category Healthcare
    • No. of Pages 160
    • Publication Month Feb-17
    • Publisher Name Global Markets Direct
    Report Summary

    Publisher'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

    Publisher'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by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Filing rejected/Withdrawn, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 1, 4, 7, 7, 4 and 5 respectively.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 Publisher’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.

    Report 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 2
    List of Tables 6
    List of Figures 7
    Introduction 8
    Publisher 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
    Alnylam Pharmaceuticals Inc
    Amgen Inc
    Arcturus Therapeutics Inc
    Bsim2
    Celgene Corp
    GlaxoSmithKline Plc
    Ionis Pharmaceuticals Inc
    Johnson & Johnson
    Millennium Pharmaceuticals Inc
    Neurimmune Holding AG
    Novartis AG
    Oncopeptides AB
    Pfizer Inc
    Proclara Biosciences Inc
    Prothena Corp Plc
    R Pharm
    Regeneron Pharmaceuticals Inc
    SOM Biotech SL
    Spectrum Pharmaceuticals Inc

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