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Published: Jan, 2015 | Pages:
133 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Amyloidosis - Pipeline Review, H1 2015 Summary Global Markets Direct's, 'Amyloidosis - Pipeline Review, H1 2015', provides an overview of the Amyloidosis's therapeutic pipeline. This report provides comprehensive information on the therapeutic development for Amyloidosis, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Amyloidosis and special features on late-stage and discontinued projects. Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct's proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis. The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products. Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease. Scope - The report provides a snapshot of the global therapeutic landscape of Amyloidosis - The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities - The report reviews key players involved in the therapeutics development for Amyloidosis and enlists all their major and minor projects - The report summarizes all the dormant and discontinued pipeline projects - A review of the Amyloidosis products under development by companies and universities/research institutes based on information derived from company and industry-specific sources - Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages - A detailed assessment of monotherapy and combination therapy pipeline projects - Coverage of the Amyloidosis pipeline on the basis of target, MoA, route of administration and molecule type - Latest news and deals relating related to pipeline products Reasons to buy - Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies - Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage - Develop strategic initiatives by understanding the focus areas of leading companies - Identify and understand important and diverse types of therapeutics under development for Amyloidosis - Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline - Devise corrective measures for pipeline projects by understanding Amyloidosis 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 - Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that 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 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
List of Tables Number of Products under Development for Amyloidosis, H1 2015 10 Number of Products under Development for Amyloidosis - Comparative Analysis, H1 2015 11 Number of Products under Development by Companies, H1 2015 13 Number of Products under Investigation by Universities/Institutes, H1 2015 14 Comparative Analysis by Late Stage Development, H1 2015 15 Comparative Analysis by Clinical Stage Development, H1 2015 16 Comparative Analysis by Early Stage Development, H1 2015 17 Products under Development by Companies, H1 2015 18 Products under Development by Companies, H1 2015 (Contd..1) 19 Products under Investigation by Universities/Institutes, H1 2015 20 Amyloidosis - Pipeline by Alnylam Pharmaceuticals, Inc., H1 2015 22 Amyloidosis - Pipeline by Arcturus Therapeutics, Inc, H1 2015 23 Amyloidosis - Pipeline by Auven Therapeutics Management L.L.L.P, H1 2015 24 Amyloidosis - Pipeline by Bellus Health Inc., H1 2015 25 Amyloidosis - Pipeline by Bsim2, H1 2015 26 Amyloidosis - Pipeline by Celgene Corporation, H1 2015 27 Amyloidosis - Pipeline by GlaxoSmithKline plc, H1 2015 28 Amyloidosis - Pipeline by Immune System Therapeutics Ltd., H1 2015 29 Amyloidosis - Pipeline by Isis Pharmaceuticals, Inc., H1 2015 30 Amyloidosis - Pipeline by NeuroPhage Pharmaceuticals, Inc., H1 2015 31 Amyloidosis - Pipeline by Onyx Pharmaceuticals, Inc., H1 2015 32 Amyloidosis - Pipeline by Pfizer Inc., H1 2015 33 Amyloidosis - Pipeline by ProteoTech, Inc., H1 2015 34 Amyloidosis - Pipeline by Prothena Corporation plc, H1 2015 35 Amyloidosis - Pipeline by SOM Innovation Biotech SL, H1 2015 36 Amyloidosis - Pipeline by Takeda Oncology, H1 2015 37 Assessment by Monotherapy Products, H1 2015 38 Assessment by Combination Products, H1 2015 39 Number of Products by Stage and Target, H1 2015 41 Number of Products by Stage and Mechanism of Action, H1 2015 43 Number of Products by Stage and Route of Administration, H1 2015 45 Number of Products by Stage and Molecule Type, H1 2015 47 Amyloidosis Therapeutics - Recent Pipeline Updates, H1 2015 87 Amyloidosis - Dormant Projects, H1 2015 116
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