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Amyloidosis - Pipeline Review, H1 2016

  • February 2016
  • -
  • Global Markets Direct
  • -
  • 141 pages

Amyloidosis - Pipeline Review, H1 2016

Summary

Global Markets Direct’s, ‘Amyloidosis - Pipeline Review, H1 2016’, provides an overview of the Amyloidosis pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Amyloidosis, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Amyloidosis and features dormant 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, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

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

Reasons to buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Identify and understand important and diverse types of therapeutics under development for Amyloidosis
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies
- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics
- 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

Amyloidosis - Pipeline Review, H1 2016
Table of Contents
Table of Contents 2
List of Tables 7
List of Figures 8
Introduction 9
Global Markets Direct Report Coverage 9
Amyloidosis Overview 10
Therapeutics Development 11
Pipeline Products for Amyloidosis - Overview 11
Pipeline Products for Amyloidosis - Comparative Analysis 12
Amyloidosis - Therapeutics under Development by Companies 13
Amyloidosis - Therapeutics under Investigation by Universities/Institutes 15
Amyloidosis - Pipeline Products Glance 16
Late Stage Products 16
Clinical Stage Products 17
Early Stage Products 18
Amyloidosis - Products under Development by Companies 19
Amyloidosis - Products under Investigation by Universities/Institutes 21
Amyloidosis - Companies Involved in Therapeutics Development 22
Alnylam Pharmaceuticals, Inc. 22
Arcturus Therapeutics, Inc 23
Bellus Health Inc. 24
Bsim2 25
Celgene Corporation 26
GlaxoSmithKline Plc 27
Neurimmune Holding AG 28
NeuroPhage Pharmaceuticals, Inc. 29
Onyx Pharmaceuticals, Inc. 30
Pfizer Inc. 31
Prothena Corporation Plc 32
SOM Innovation Biotech SL 33
Takeda Pharmaceutical Company Limited 34
Amyloidosis - Therapeutics Assessment 35
Assessment by Monotherapy Products 35
Assessment by Combination Products 36
Assessment by Target 37
Assessment by Mechanism of Action 39
Assessment by Route of Administration 41
Assessment by Molecule Type 43
Drug Profiles 45
ALN-ANG - Drug Profile 45
Product Description 45
Mechanism of Action 45
RandD Progress 45
ALN-TTRsc02 - Drug Profile 46
Product Description 46
Mechanism of Action 46
RandD Progress 46
Antisense RNAi Oligonucleotides to Inhibit Amyloid Precursor Protein for Amyloidosis - Drug Profile 47
Product Description 47
Mechanism of Action 47
RandD Progress 47
carfilzomib - Drug Profile 48
Product Description 48
Mechanism of Action 48
RandD Progress 48
CLR-01 - Drug Profile 52
Product Description 52
Mechanism of Action 52
RandD Progress 52
eprodisate disodium - Drug Profile 54
Product Description 54
Mechanism of Action 54
RandD Progress 54
GSK-2315698 + GSK-2398852 - Drug Profile 57
Product Description 57
Mechanism of Action 57
RandD Progress 57
GSK-3039294 - Drug Profile 58
Product Description 58
Mechanism of Action 58
RandD Progress 58
inotersen sodium - Drug Profile 59
Product Description 59
Mechanism of Action 59
RandD Progress 59
ixazomib citrate - Drug Profile 60
Product Description 60
Mechanism of Action 60
RandD Progress 60
LUNAR-101 - Drug Profile 65
Product Description 65
Mechanism of Action 65
RandD Progress 65
LUNAR-102 - Drug Profile 66
Product Description 66
Mechanism of Action 66
RandD Progress 66
LUNAR-TTR - Drug Profile 67
Product Description 67
Mechanism of Action 67
RandD Progress 67
Monoclonal Antibody for Amyloidosis - Drug Profile 68
Product Description 68
Mechanism of Action 68
RandD Progress 68
Monoclonal Antibody to Inhibit ATTR for Familial Amyloid Polyneuropathy - Drug Profile 69
Product Description 69
Mechanism of Action 69
RandD Progress 69
NEOD-001 - Drug Profile 70
Product Description 70
Mechanism of Action 70
RandD Progress 70
NI-301 - Drug Profile 72
Product Description 72
Mechanism of Action 72
RandD Progress 72
NPT-005 - Drug Profile 73
Product Description 73
Mechanism of Action 73
RandD Progress 73
patisiran - Drug Profile 74
Product Description 74
Mechanism of Action 74
RandD Progress 74
pomalidomide - Drug Profile 75
Product Description 75
Mechanism of Action 75
RandD Progress 75
PTI-110 - Drug Profile 79
Product Description 79
Mechanism of Action 79
RandD Progress 79
revusiran - Drug Profile 80
Product Description 80
Mechanism of Action 80
RandD Progress 80
Small Molecules for AL Amyloidosis - Drug Profile 83
Product Description 83
Mechanism of Action 83
RandD Progress 83
Small Molecules to Inhibit Transthyretin for Familial Amyloid Polyneuropathy - Drug Profile 84
Product Description 84
Mechanism of Action 84
RandD Progress 84
Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Cardiomyopathy - Drug Profile 85
Product Description 85
Mechanism of Action 85
RandD Progress 85
Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Polyneuropathy - Drug Profile 86
Product Description 86
Mechanism of Action 86
RandD Progress 86
tafamidis meglumine - Drug Profile 87
Product Description 87
Mechanism of Action 87
RandD Progress 87
tolcapone - Drug Profile 89
Product Description 89
Mechanism of Action 89
RandD Progress 89
Amyloidosis - Recent Pipeline Updates 91
Amyloidosis - Dormant Projects 128
Amyloidosis - Product Development Milestones 129
Featured News and Press Releases 129
Feb 09, 2016: Data From Prothena Ongoing Phase 1/2 Clinical Trial of NEOD001 in AL Amyloidosis Published in the Journal of Clinical Oncology 129
Feb 01, 2016: Alnylam Completes Enrollment in APOLLO Phase 3 Study with Patisiran, an Investigational RNAi Therapeutic for Patients with Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis) 130
Jan 21, 2016: Auven Therapeutics and Bellus Health Announce Completion of Pivotal Phase 3 Confirmatory Trial of KIACTA for the Treatment of Orphan Disease AA Amyloidosis 131
Dec 17, 2015: Isis Pharmaceuticals Announces Initiation of an Investigator-Sponsored Phase 2 Study of ISIS-TTR Rx in Patients with TTR Cardiomyopathy Amyloidosis 132
Nov 05, 2015: Prothena to Present New Preclinical Data for NEOD001 at 57th Annual American Society of Hematology Meeting 133
Nov 05, 2015: Prothena to Present New Preclinical Data for NEOD001 at 57th Annual American Society of Hematology Meeting 134
Nov 03, 2015: Isis Pharmaceuticals Reports an Update on ISIS-TTR Rx, Including Positive Data from Multiple Clinical Studies, Presented at the EC-ATTR Meeting 135
Nov 03, 2015: Alnylam Reports Positive Results from Ongoing Phase 2 Open-Label Extension Studies for Patisiran in Development for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis) 136
Nov 03, 2015: Alnylam Reports Positive Results from Ongoing Phase 2 Open-Label Extension Studies for Revusiran, in Development for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis 137
Oct 27, 2015: Isis Pharmaceuticals to Host Webcast to Review ISIS-TTR Data Presented at 1st European Congress of Hereditary ATTR Amyloidosis and Provide Phase 3 Development Plan Update 139
Appendix 140
Methodology 140
Coverage 140
Secondary Research 140
Primary Research 140
Expert Panel Validation 140
Contact Us 140
Disclaimer 141

List of Tables
Number of Products under Development for Amyloidosis, H1 2016 11
Number of Products under Development for Amyloidosis - Comparative Analysis, H1 2016 12
Number of Products under Development by Companies, H1 2016 14
Number of Products under Investigation by Universities/Institutes, H1 2016 15
Comparative Analysis by Late Stage Development, H1 2016 16
Comparative Analysis by Clinical Stage Development, H1 2016 17
Comparative Analysis by Early Stage Development, H1 2016 18
Products under Development by Companies, H1 2016 19
Products under Development by Companies, H1 2016 (Contd..1) 20
Products under Investigation by Universities/Institutes, H1 2016 21
Amyloidosis - Pipeline by Alnylam Pharmaceuticals, Inc., H1 2016 22
Amyloidosis - Pipeline by Arcturus Therapeutics, Inc, H1 2016 23
Amyloidosis - Pipeline by Bellus Health Inc., H1 2016 24
Amyloidosis - Pipeline by Bsim2, H1 2016 25
Amyloidosis - Pipeline by Celgene Corporation, H1 2016 26
Amyloidosis - Pipeline by GlaxoSmithKline Plc, H1 2016 27
Amyloidosis - Pipeline by Neurimmune Holding AG, H1 2016 28
Amyloidosis - Pipeline by NeuroPhage Pharmaceuticals, Inc., H1 2016 29
Amyloidosis - Pipeline by Onyx Pharmaceuticals, Inc., H1 2016 30
Amyloidosis - Pipeline by Pfizer Inc., H1 2016 31
Amyloidosis - Pipeline by Prothena Corporation Plc, H1 2016 32
Amyloidosis - Pipeline by SOM Innovation Biotech SL, H1 2016 33
Amyloidosis - Pipeline by Takeda Pharmaceutical Company Limited, H1 2016 34
Assessment by Monotherapy Products, H1 2016 35
Assessment by Combination Products, H1 2016 36
Number of Products by Stage and Target, H1 2016 38
Number of Products by Stage and Mechanism of Action, H1 2016 40
Number of Products by Stage and Route of Administration, H1 2016 42
Number of Products by Stage and Molecule Type, H1 2016 44
Amyloidosis Therapeutics - Recent Pipeline Updates, H1 2016 91
Amyloidosis - Dormant Projects, H1 2016 128

List of Figures
Number of Products under Development for Amyloidosis, H1 2016 11
Number of Products under Development for Amyloidosis - Comparative Analysis, H1 2016 12
Number of Products under Development by Companies, H1 2016 13
Number of Products under Investigation by Universities/Institutes, H1 2016 15
Comparative Analysis by Late Stage Development, H1 2016 16
Comparative Analysis by Clinical Stage Development, H1 2016 17
Comparative Analysis by Early Stage Products, H1 2016 18
Assessment by Monotherapy Products, H1 2016 35
Number of Products by Top 10 Targets, H1 2016 37
Number of Products by Stage and Top 10 Targets, H1 2016 37
Number of Products by Top 10 Mechanism of Actions, H1 2016 39
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2016 39
Number of Products by Routes of Administration, H1 2016 41
Number of Products by Stage and Routes of Administration, H1 2016 41
Number of Products by Molecule Types, H1 2016 43
Number of Products by Stage and Molecule Types, H1 2016 43

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