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Amyloidosis - Pipeline Review, H1 2015

Published: Jan, 2015 | Pages: 133 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Amyloidosis - Pipeline Review, H1 2015


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.


- 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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