Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Pipeline Review, H1 2014

Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Pipeline Review, H1 2014 Cover

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Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Pipeline Review, H1 2014

Summary

Global Markets Direct’s, ‘Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Pipeline Review, H1 2014’, provides an overview of the indication’s therapeutic pipeline. This report provides information on the therapeutic development for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease), complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease). Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Pipeline Review, Half Year 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.

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 Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease).
– A review of the Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) 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 Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) 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 Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease).
– 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 Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) 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
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) Overview 7
Therapeutics Development 8
Pipeline Products for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Overview 8
Pipeline Products for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Comparative Analysis 9
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Therapeutics under Development by Companies 10
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Therapeutics under Investigation by Universities/Institutes 11
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Pipeline Products Glance 12
Late Stage Products 12
Clinical Stage Products 13
Early Stage Products 14
Unknown Stage Products 15
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Products under Development by Companies 16
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Products under Investigation by Universities/Institutes 17
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Companies Involved in Therapeutics Development 18
Isis Pharmaceuticals, Inc. 18
Pfizer Inc. 19
Alnylam Pharmaceuticals, Inc. 20
SOM Biotech SL 21
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Therapeutics Assessment 22
Assessment by Monotherapy Products 22
Assessment by Target 23
Assessment by Mechanism of Action 24
Assessment by Route of Administration 26
Assessment by Molecule Type 28
Assessment by Therapeutic Class 30
Drug Profiles 32
tafamidis meglumine – Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
patisiran – Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
ISIS-TTRRx – Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
patisiran – Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
SOM-0226 – Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
ALN-TTR-01 – Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
Antibody Against Amyloidogenic Transthyretin – Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
patisiran – Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Recent Pipeline Updates 42
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Dormant Projects 52
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Product Development Milestones 53
Featured News & Press Releases 53
Jan 10, 2014: SOM Biotech announces Orphan Drug Designation in US for SOM0226, a compound for the treatment of Transthyretin Amyloidosis 53
Dec 16, 2013: Tekmira Receives $5 Million Milestone Payment for Phase III Initiation of LNP-Enabled Patisiran 53
Dec 02, 2013: Isis Pharmaceuticals Earns $2 Million from GSK for the Advancement of ISIS-TTR Rx 54
Sep 23, 2013: Alnylam Reports Positive Clinical Results – with up to 94% Knockdown of Serum Transthyretin – for ALN-TTRsc, a Subcutaneously Delivered RNAi Therapeutic for the Treatment of TTR-Mediated Amyloidosis 54
Jul 30, 2013: GSK makes milestone payment to Isis for progressing ISIS-TTRRx study 56
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 56
Mar 18, 2013: Alnylam Initiates Phase I Clinical Study For ALN-TTRsc For Treatment Of TTR-Mediated Amyloidosis 58
Feb 19, 2013: Isis Pharma Initiates Phase II/III Study Of ISIS-TTR Rx And Earns $7.5m Milestone Payment From GlaxoSmithKline 58
Jan 03, 2013: Alnylam Pharma Files Clinical Trial Application To Initiate Phase I Study For ALN-TTRsc To Treat TTR-Mediated Amyloidosis 59
Dec 13, 2012: Isis Pharma Announces Fast Track Designation For ISIS-TTR Rx For Treatment Of Patients With FAP 61
Appendix 62
Methodology 62
Coverage 62
Secondary Research 62
Primary Research 62
Expert Panel Validation 62
Contact Us 63
Disclaimer 63

List of Tables
Number of Products under Development for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease), H1 2014 8
Number of Products under Development for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Comparative Analysis, H1 2014 9
Number of Products under Development by Companies, H1 2014 10
Number of Products under Investigation by Universities/Institutes, H1 2014 11
Comparative Analysis by Late Stage Development, H1 2014 12
Comparative Analysis by Clinical Stage Development, H1 2014 13
Comparative Analysis by Early Stage Development, H1 2014 14
Comparative Analysis by Unknown Stage Development, H1 2014 15
Products under Development by Companies, H1 2014 16
Products under Investigation by Universities/Institutes, H1 2014 17
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Pipeline by Isis Pharmaceuticals, Inc., H1 2014 18
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Pipeline by Pfizer Inc., H1 2014 19
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Pipeline by Alnylam Pharmaceuticals, Inc., H1 2014 20
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Pipeline by SOM Biotech SL, H1 2014 21
Assessment by Monotherapy Products, H1 2014 22
Number of Products by Stage and Target, H1 2014 23
Number of Products by Stage and Mechanism of Action, H1 2014 25
Number of Products by Stage and Route of Administration, H1 2014 27
Number of Products by Stage and Molecule Type, H1 2014 29
Number of Products by Stage and Therapeutic Class, H1 2014 31
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) Therapeutics – Recent Pipeline Updates, H1 2014 42
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Dormant Projects, H1 2014 52

List of Figures
Number of Products under Development for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease), H1 2014 8
Number of Products under Development for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Comparative Analysis, H1 2014 9
Number of Products under Development by Companies, H1 2014 10
Comparative Analysis by Late Stage Development, H1 2014 12
Comparative Analysis by Clinical Stage Development, H1 2014 13
Assessment by Monotherapy Products, H1 2014 22
Number of Products by Stage and Top 10 Target, H1 2014 23
Number of Products by Top 10 Mechanism of Action, H1 2014 24
Number of Products by Stage and Top 10 Mechanism of Action, H1 2014 25
Number of Products by Top 10 Route of Administration, H1 2014 26
Number of Products by Stage and Top 10 Route of Administration, H1 2014 27
Number of Products by Top 10 Molecule Type, H1 2014 28
Number of Products by Stage and Top 10 Molecule Type, H1 2014 29
Number of Products by Top 10 Therapeutic Class, H1 2014 30
Number of Products by Stage and Top 10 Therapeutic Class, H1 2014 31

 

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