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Amyloidosis - Pipeline Review, H1 2014

Published By :

Global Markets Direct

Published Date : Mar 2014

Category :

Metabolic Disorders

No. of Pages : 82 Pages

Amyloidosis - Pipeline Review, H1 2014

Summary

Global Markets Directs, Amyloidosis - Pipeline Review, H1 2014, provides an overview of the Amyloidosiss 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-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 4
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Amyloidosis Overview 7
Therapeutics Development 8
Pipeline Products for Amyloidosis Overview 8
Pipeline Products for Amyloidosis Comparative Analysis 9
Amyloidosis Therapeutics under Development by Companies 10
Amyloidosis Therapeutics under Investigation by Universities/Institutes 12
Amyloidosis Pipeline Products Glance 13
Late Stage Products 13
Clinical Stage Products 14
Early Stage Products 15
Amyloidosis Products under Development by Companies 16
Amyloidosis Products under Investigation by Universities/Institutes 17
Amyloidosis Companies Involved in Therapeutics Development 18
GlaxoSmithKline plc 18
Celgene Corporation 19
Onyx Pharmaceuticals, Inc. 20
Bellus Health Inc. 21
ProteoTech, Inc. 22
Immune System Therapeutics Ltd. 23
Auven Therapeutics Management L.L.L.P 24
NeuroPhage Pharmaceuticals, Inc. 25
SOM Biotech SL 26
Amyloidosis Therapeutics Assessment 27
Assessment by Monotherapy Products 27
Assessment by Target 28
Assessment by Mechanism of Action 30
Assessment by Route of Administration 33
Assessment by Molecule Type 35
Drug Profiles 37
eprodisate disodium - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
SOM-0226 - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
pomalidomide - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
carfilzomib - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
Systebryl - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
GSK-2315698 - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
GSK-2398852 - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
Peptides For Amyloidoses - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
IST-1097 - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
NPT-005 - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
Small Molecule For AL Amyloidosis - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
Amyloidosis Recent Pipeline Updates 53
Amyloidosis Dormant Projects 66
Amyloidosis Product Development Milestones 67
Featured News & Press Releases 67
Jan 09, 2014: BELLUS Health provides update on KIACTA Phase III orphan drug study 67
Dec 16, 2013: Alnylam Initiates Phase II Clinical Trial with ALN-TTRsc, a Subcutaneously Delivered RNAi Therapeutic Targeting Transthyretin in Development for the Treatment of TTR-Mediated Amyloidosis 67
Nov 25, 2013: Alnylam Earns $7 Million Milestone Payment from Genzyme for Phase II Success with Patisiran (ALN-TTR02), an RNAi Therapeutic Targeting Transthyretin for the Treatment of TTR-Mediated Amyloidosis 69
Nov 11, 2013: Alnylam Receives Fast Track Designation for Patisiran (ALN-TTR02), an RNAi Therapeutic Targeting Transthyretin for the Treatment of TTR-Mediated Amyloidosis 69
Nov 10, 2013: Alnylam Reports Positive Phase II Data for Patisiran, an RNAi Therapeutic Targeting Transthyretin for the Treatment of TTR-Mediated Amyloidosis, and Initiates Phase III Trial 70
Sep 30, 2013: Alnylam Completes Enrollment of Phase II Clinical Trial and Initiates Open-Label Extension (OLE) Study with ALN-TTR02, an RNAi Therapeutic Targeting Transthyretin for the Treatment of Transthyretin-Mediated Amyloidos 72
Aug 28, 2013: Alnylam and Collaborators Publish Clinical Trial Results with RNAi Therapeutics Targeting Transthyretin for the Treatment of TTR-Mediated Amyloidosis in the New England Journal of Medicine 74
Jul 23, 2013: KIACTA Granted Orphan Drug Status in Japan 76
Jul 11, 2013: Alnylam Reports Positive Top-Line Results for ALN-TTRsc, a Subcutaneously Administered RNAi Therapeutic Targeting Transthyretin for the Treatment of TTR-Mediated Amyloidosis 77
Jun 30, 2013: Alnylam Reports Positive Phase II Data for ALN-TTR02 for the Treatment of TTR-Mediated Amyloidosis 78
Appendix 81
Methodology 81
Coverage 81
Secondary Research 81
Primary Research 81
Expert Panel Validation 81
Contact Us 82
Disclaimer 82

List of Table

List of Tables
Number of Products under Development for Amyloidosis, H1 2014 8
Number of Products under Development for Amyloidosis Comparative Analysis, H1 2014 9
Number of Products under Development by Companies, H1 2014 11
Number of Products under Investigation by Universities/Institutes, H1 2014 12
Comparative Analysis by Late Stage Development, H1 2014 13
Comparative Analysis by Clinical Stage Development, H1 2014 14
Comparative Analysis by Early Stage Development, H1 2014 15
Products under Development by Companies, H1 2014 16
Products under Investigation by Universities/Institutes, H1 2014 17
Amyloidosis Pipeline by GlaxoSmithKline plc, H1 2014 18
Amyloidosis Pipeline by Celgene Corporation., H1 2014 19
Amyloidosis Pipeline by Onyx Pharmaceuticals, Inc., H1 2014 20
Amyloidosis Pipeline by Bellus Health Inc., H1 2014 21
Amyloidosis Pipeline by ProteoTech, Inc., H1 2014 22
Amyloidosis Pipeline by Immune System Therapeutics Ltd., H1 2014 23
Amyloidosis Pipeline by Auven Therapeutics Management L.L.L.P, H1 2014 24
Amyloidosis Pipeline by NeuroPhage Pharmaceuticals, Inc., H1 2014 25
Amyloidosis Pipeline by SOM Biotech SL, H1 2014 26
Assessment by Monotherapy Products, H1 2014 27
Number of Products by Stage and Target, H1 2014 29
Number of Products by Stage and Mechanism of Action, H1 2014 32
Number of Products by Stage and Route of Administration, H1 2014 34
Number of Products by Stage and Molecule Type, H1 2014 36
Amyloidosis Therapeutics Recent Pipeline Updates, H1 2014 53
Amyloidosis Dormant Projects, H1 2014 66

List of Chart

List of Figures
Number of Products under Development for Amyloidosis, H1 2014 8
Number of Products under Development for Amyloidosis Comparative Analysis, H1 2014 9
Number of Products under Development by Companies, H1 2014 10
Comparative Analysis by Clinical Stage Development, H1 2014 14
Comparative Analysis by Early Stage Products, H1 2014 15
Assessment by Monotherapy Products, H1 2014 27
Number of Products by Top 10 Target, H1 2014 28
Number of Products by Stage and Top 10 Target, H1 2014 29
Number of Products by Top 10 Mechanism of Action, H1 2014 30
Number of Products by Stage and Top 10 Mechanism of Action, H1 2014 31
Number of Products by Top 10 Route of Administration, H1 2014 33
Number of Products by Stage and Top 10 Route of Administration, H1 2014 34
Number of Products by Top 10 Molecule Type, H1 2014 35
Number of Products by Stage and Top 10 Molecule Type, H1 2014 36

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