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Amyloidosis - Pipeline Review, H2 2013

Published By :

Global Markets Direct

Published Date : Sep 2013

Category :

Metabolic Disorders

No. of Pages : 59 Pages


Global Markets Directs, \'Amyloidosis Pipeline Review, H2 2013\', provides an overview of the indications therapeutic pipeline. This report provides information on the therapeutic development for Amyloidosis, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Amyloidosis. Amyloidosis Pipeline Review, Half Year 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.

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

Scope

  • A snapshot of the global therapeutic scenario for Amyloidosis.
  • A review of the Amyloidosis products under development by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • Coverage of products based on various stages of development ranging from discovery till registration stages.
  • A feature on pipeline projects on the basis of monotherapy and combined therapeutics.
  • Coverage of the Amyloidosis pipeline on the basis of route of administration and molecule type.
  • Key discontinued pipeline projects.
  • Latest news and deals relating to the products.

Reasons to buy

  • Identify and understand important and diverse types of therapeutics under development for Amyloidosis.
  • Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.
  • Plan mergers and acquisitions effectively by identifying 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 4
Introduction 5
Global Markets Direct Report Coverage 5
Amyloidosis Overview 6
Therapeutics Development 7
An Overview of Pipeline Products for Amyloidosis 7
Amyloidosis Therapeutics under Development by Companies 9
Amyloidosis Therapeutics under Investigation by Universities/Institutes 10
Late Stage Products 11
Comparative Analysis 11
Early Clinical Stage Products 12
Comparative Analysis 12
Discovery and Pre-Clinical Stage Products 13
Comparative Analysis 13
Amyloidosis Therapeutics - Products under Development by Companies 14
Amyloidosis Therapeutics - Products under Investigation by Universities/Institutes 15
Companies Involved in Amyloidosis Therapeutics Development 16
GlaxoSmithKline plc 16
Onyx Pharmaceuticals, Inc. 17
ProteoTech, Inc. 18
Immune System Therapeutics Ltd. 19
TheraCarb Inc. 20
Amyloidosis - Therapeutics Assessment 21
Assessment by Monotherapy Products 21
Assessment by Route of Administration 22
Assessment by Molecule Type 24
Drug Profiles 26
MDX-1097 - Drug Profile 26
Product Description 26
Mechanism of Action 26
R&D Progress 26
carfilzomib - Drug Profile 27
Product Description 27
Mechanism of Action 27
R&D Progress 27
eprodisate disodium - Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
Systebryl - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
GSK-2315698 - Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
Drug For AA Amyloidosis - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
NPT-005 - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
Peptides For Amyloidoses - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
GSK-2398852 - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
Amyloidosis Therapeutics - Drug Profile Updates 38
Amyloidosis Therapeutics - Dormant Products 46
Amyloidosis - Product Development Milestones 47
Featured News & Press Releases 47
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 47
Jul 23, 2013: KIACTA Granted Orphan Drug Status in Japan 49
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 49
Jun 30, 2013: Alnylam Reports Positive Phase II Data for ALN-TTR02 for the Treatment of TTR-Mediated Amyloidosis 51
Apr 23, 2013: Prothena Announces Dosing Of First Patient In Phase I Study Of Therapeutic Antibody For Treatment Of AL Amyloidosis 53
Feb 27, 2013: Prothena Achieves Orphan Drug Status In EU For Lead Program NEOD001 53
Nov 05, 2012: Millennium To Present Data On MLN9708 At American Society Of Hematology Annual Meeting 54
Oct 19, 2012: Millennium Initiates Global TOURMALINE-AL1 Pivotal Phase III Trial Of MLN9708 In Patients With Relapsed Or Refractory Primary Amyloidosis 55
May 10, 2012: Alnylam Presents Final Results From Phase I Clinical Trial Of ALN-TTR01 For Treatment Of Transthyretin-Mediated Amyloidosis 55
May 10, 2012: Bellus Health Announces Update On Phase III Trial Of KIACTA In AA Amyloidosis At XIII International Symposium On Amyloidosis 57

Appendix 58
Methodology 58
Coverage 58
Secondary Research 58
Primary Research 58
Expert Panel Validation 58
Contact Us 59
Disclaimer 59

List of Table


Number of Products Under Development for Amyloidosis, H2 2013 7
Products under Development for Amyloidosis - Comparative Analysis, H2 2013 8
Number of Products under Development by Companies, H2 2013 9
Number of Products under Investigation by Universities/Institutes, H2 2013 10
Comparative Analysis by Late Stage Development, H2 2013 11
Comparative Analysis by Early Clinical Stage Development, H2 2013 12
Comparative Analysis by Discovery and Pre-Clinical Stage Development, H2 2013 13
Products under Development by Companies, H2 2013 14
Products under Investigation by Universities/Institutes, H2 2013 15
GlaxoSmithKline plc, H2 2013 16
Onyx Pharmaceuticals, Inc., H2 2013 17
ProteoTech, Inc., H2 2013 18
Immune System Therapeutics Ltd., H2 2013 19
TheraCarb Inc., H2 2013 20
Assessment by Monotherapy Products, H2 2013 21
Assessment by Stage and Route of Administration, H2 2013 23
Assessment by Stage and Molecule Type, H2 2013 25
Amyloidosis Therapeutics - Drug Profile Updates 38
Amyloidosis Therapeutics - Dormant Products 46

List of Chart


Number of Products under Development for Amyloidosis, H2 2013 7
Products under Development for Amyloidosis - Comparative Analysis, H2 2013 8
Products under Development by Companies, H2 2013 9
Products under Investigation by Universities/Institutes, H2 2013 10
Late Stage Products, H2 2013 11
Early Clinical Stage Products, H2 2013 12
Discovery and Pre-Clinical Stage Products, H2 2013 13
Assessment by Monotherapy Products, H2 2013 21
Assessment by Route of Administration, H2 2013 22
Assessment by Stage and Route of Administration, H2 2013 23
Assessment by Molecule Type, H2 2013 24
Assessment by Stage and Molecule Type, H2 2013 25

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