Amyloidosis - Pipeline Review, H2 2015 Now Available at iData Insights Amyloidosis - Pipeline Review, H2 2015 Summary Global Markets Direct s, Amyloidosis - Pipeline Review, H2 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 Directs 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 Directs 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 Directs 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-inclass 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 1
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 Browse Complete Report with TOC @ http://www.idatainsights.com/reports-landingpage.php?id=124213/amyloidosis-pipeline-review-h2-2015 To Get Sample Copy of Report visit @ http://www.idatainsights.com/reports-landingpage.php?id=124213/amyloidosis-pipeline-review-h2-2015 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 22 Bellus Health Inc. 23 2
Bsim2 24 Celgene Corporation 25 GlaxoSmithKline Plc 26 Isis Pharmaceuticals, Inc. 27 Millennium Pharmaceuticals, Inc. 28 Onyx Pharmaceuticals, Inc. 29 Pfizer Inc. 30 ProteoTech, Inc. 31 Prothena Corporation Plc 32 SOM Innovation Biotech SL 33 Amyloidosis - Therapeutics Assessment 34 Assessment by Monotherapy Products 34 Assessment by Combination Products 35 Assessment by Target 36 Assessment by Mechanism of Action 38 Assessment by Route of Administration 40 Assessment by Molecule Type 42 Drug Profiles 44 ALN-TTRsc02 - Drug Profile 44 Product Description 44 Mechanism of Action 44 R&D Progress 44 Antisense RNAi Oligonucleotides to Inhibit Amyloid Precursor Protein for Amyloidosis - Drug Profile 45 Product Description 45 Mechanism of Action 45 R&D Progress 45
3
carfilzomib - Drug Profile 46 Product Description 46 Mechanism of Action 46 R&D Progress 46 CLR-01 - Drug Profile 50 Product Description 50 Mechanism of Action 50 R&D Progress 50 eprodisate disodium - Drug Profile 52 Product Description 52 Mechanism of Action 52 R&D Progress 52 GSK-2315698 + GSK-2398852 - Drug Profile 55 Product Description 55 Mechanism of Action 55 R&D Progress 55 GSK-2398852 - Drug Profile 56 Product Description 56 Mechanism of Action 56 R&D Progress 56 ISIS-TTRRx - Drug Profile 57 Product Description 57 Mechanism of Action 57 R&D Progress 57 ixazomib citrate - Drug Profile 58 Product Description 58 Mechanism of Action 58 4
R&D Progress 58 LUNAR-101 - Drug Profile 62 Product Description 62 Mechanism of Action 62 R&D Progress 62 LUNAR-102 - Drug Profile 63 Product Description 63 Mechanism of Action 63 R&D Progress 63 Monoclonal Antibody for Amyloidosis - Drug Profile 64 Product Description 64 Mechanism of Action 64 R&D Progress 64 Monoclonal Antibody to Inhibit ATTR for Familial Amyloid Polyneuropathy - Drug Profile 65 Product Description 65 Mechanism of Action 65 R&D Progress 65 NEOD-001 - Drug Profile 66 Product Description 66 Mechanism of Action 66 R&D Progress 66 NPT-005 - Drug Profile 67 Product Description 67 Mechanism of Action 67 R&D Progress 67 patisiran - Drug Profile 68
5
Product Description 68 Mechanism of Action 68 R&D Progress 68 pomalidomide - Drug Profile 69 Product Description 69 Mechanism of Action 69 R&D Progress 69 PTI-110 - Drug Profile 73 Product Description 73 Mechanism of Action 73 R&D Progress 73 revusiran - Drug Profile 74 Product Description 74 Mechanism of Action 74 R&D Progress 74 Small Molecules for AL Amyloidosis - Drug Profile 76 Product Description 76 Mechanism of Action 76 R&D Progress 76 Small Molecules to Inhibit Transthyretin for Familial Amyloid Polyneuropathy - Drug Profile 77 Product Description 77 Mechanism of Action 77 R&D Progress 77 Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Cardiomyopathy - Drug Profile 78 Product Description 78 Mechanism of Action 78
6
R&D Progress 78 Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Polyneuropathy - Drug Profile 79 Product Description 79 Mechanism of Action 79 R&D Progress 79 tafamidis meglumine - Drug Profile 80 Product Description 80 Mechanism of Action 80 R&D Progress 80 tolcapone - Drug Profile 82 Product Description 82 Mechanism of Action 82 R&D Progress 82 Amyloidosis - Recent Pipeline Updates 83 Amyloidosis - Dormant Projects 117 Amyloidosis - Product Development Milestones 118 Featured News & Press Releases 118 Sep 28, 2015: Alnylam and Collaborators Report First-Ever Evidence that Patisiran Reduces Pathogenic, Misfolded Transthyretin Monomers and Oligomers in TTR-Mediated Amyloidosis Patients with Familial Amyloidotic Polyneuropathy 118 Aug 12, 2015: BELLUS Health narrows timelines for the Phase III study for KIACTA 120 Jul 20, 2015: Alnylam Initiates Phase 3 Open Label Extension Study with Patisiran, an Investigational RNAi Therapeutic Targeting Transthyretin for the Treatment of TTR-Mediated Amyloidosis 121 Jun 12, 2015: Opportunity to Improve Diagnosis and Treatment of Systemic Amyloidosis Highlighted in Patient Experience Survey Presented at the 20th Congress of the European Hematology Association 122 Jun 12, 2015: Prothena's NEOD001 Protein Immunotherapy Clinical Data Highlighted in Oral Presentation at the 20th Congress of the European Hematology Association 123 Jun 02, 2015: Prothena Presents Compelling New Data From Ongoing Phase 1/2 Study of NEOD001 in Patients With AL Amyloidosis at 2015 ASCO Annual Meeting 124
7
May 21, 2015: Prothena Announces Presentation of Clinical Data From the Ongoing Phase 1/2 NEOD001 Study and Systemic Amyloidosis Patient Survey Results at 20th Congress of the European Hematology Association 126 May 20, 2015: Alnylam Receives Orphan Drug Designation from the United States Food & Drug Administration for Revusiran, an Investigational RNAi Therapeutic for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis 127 May 13, 2015: Prothena to Present Updated Clinical Data From Ongoing Phase 1/2 Study of NEOD001 at 2015 ASCO Annual Meeting 128 Apr 22, 2015: Isis Pharmaceuticals Reports Positive Data From ISIS-TTR Rx in Patients With TTR Amyloidosis 129 Appendix 131 Methodology 131 Coverage 131 Secondary Research 131 Primary Research 131 Expert Panel Validation 131 Contact Us 131 Disclaimer 132 Read More http://www.idatainsights.com/reports-landing-page.php?id=124213/amyloidosis-pipelinereview-h2-2015 About Us: iData Insights which operates under Precision Research and Consulting Private Limited is a leading market research information aggregator and marketing research consulting firm. We conduct both primary and secondary research. Our work does not end with research it is also involved in distributing reports for different companies . We provide actionable recommendations and provide our expertise for business success today and tomorrow. It is our goal to be a partner to our clients in the exploration and discovery, and then be their guide in the implementation of changes that will make a difference to their bottom line. ContactUs: iData Insights Tel: 1-866-237-2965 Web: http://www.idatainsights.com Email: info@idatainsights.com About iData Insights We work around the clock until our clients are completely satisfied with their promised results. It is our mission to provide timely services coupled with acute client care to create a long-lasting relationship 8
with you. We deliver on our promise to provide results that are essential to the success of your company. We believe that by doing so, we will earn the trust of our clients for future projects
9