Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline Review, H2 2012

Global Markets Direct, August 2012, Pages: 52

Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) – Pipeline Review, H2 2012

Summary

Global Markets Direct’s, 'Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline Review, H2 2012', provides an overview of the Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) 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, H2 2012' 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.
- Profiles of late-stage pipeline products featuring sections on product description, mechanism of action and research & development progress.
- 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 with 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 Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) 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.

Keywords

Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) Therapeutic Products under Development, Key Players in Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) Therapeutics, Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) Pipeline Overview, Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) Pipeline, Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) Pipeline Assessment

2
List of Tables 4
List of Figures 5
Introduction 6
REPORT COVERAGE 6
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) Overview 7
Therapeutics Development 8
An Overview of Pipeline Products for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) 8
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
Late Stage Products 12
Comparative Analysis 12
Mid Clinical Stage Products 13
Comparative Analysis 13
Early Clinical Stage Products 14
Comparative Analysis 14
Discovery and Pre-Clinical Stage Products 15
Comparative Analysis 15
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) Therapeutics – Products under Development by Companies 16
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) Therapeutics – Products under Investigation by Universities/Institutes 17
Companies Involved in Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) Therapeutics Development 18
Isis Pharmaceuticals, Inc. 18
Pfizer Inc. 19
Alnylam Pharmaceuticals, Inc 20
BIOALVO S.A. 21
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) – Therapeutics Assessment 22
Assessment by Monotherapy Products 22
Assessment by Combination Products 23
Assessment by Route of Administration 24
Assessment by Molecule Type 26
Drug Profiles 28
SDGI-TT200801 - Drug Profile 28
Product Description 28
Mechanism of Action 28
R&D Progress 28
ALN-TTR01 - Drug Profile 29
Product Description 29
Mechanism of Action 29
R&D Progress 29
ALN-TTR02 - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
Vyndaqel - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
Diflunisal - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
ISIS-TTR - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
Doxycycline + Tauroursodeoxycholic Acid - 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
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) Therapeutics – Drug Profile Updates 38
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) – Product Development Milestones 43
Featured News & Press Releases 43
Aug 06, 2012: Alnylam Pharma Presents New Preclinical Data From ‘Alnylam5x15’ Programs Including ALN-TTRsc For Treatment Of Transthyretin-Mediated Amyloidosis 43
Jul 16, 2012: Alnylam Reports Positive Phase I Trial Results Of ALN-TTR02 For Treatment Of TTR-Mediated Amyloidosis 43
Jul 16, 2012: Tekmira's LNP Technology Enables Alnylam's Positive ALN-TTR02 Clinical Data 45
Jun 19, 2012: Alnylam's ALN-TTR02 Receives US Orphan Drug Designation For Treatment Of Familial Amyloidotic- Polyneuropathy 46
Jun 18, 2012: FDA Issues Complete Response Letter For Pfizer’s Tafamidis Meglumine New Drug Application 46
Jun 07, 2012: Alnylam Completes Enrollment In Phase I Clinical Trial And Initiates Phase II Clinical Trial Of ALN-TTR02 For Treatment Of Transthyretin-Mediated Amyloidosis 47
May 24, 2012: FDA Panel Votes Pfizer's Tafamidis Meglumine To Treat Transthyretin Familial Amyloid Polyneuropathy 47
Mar 19, 2012: Alnylam Initiates Phase I Clinical Study Of ALN-TTR02 For Treatment Of TTR-Mediated Amyloidosis 48
Feb 15, 2012: FDA Accepts Pfizer’s New Drug Application For Tafamidis Meglumine To Treat Transthyretin Familial Amyloid Polyneuropathy 49
Jan 06, 2012: Alnylam Files Clinical Trial Application For ALN-TTR02 For Treatment Of Transthyretin-Mediated Amyloidosis 49
Appendix 51
Methodology 51
Coverage 51
Secondary Research 51
Primary Research 51
Expert Panel Validation 51
Contact Us 52
Disclaimer 52

List of Tables
Number of Products Under Development for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease), H2 2012 8
Products under Development for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) – Comparative Analysis, H2 2012 9
Number of Products under Development by Companies, H2 2012 10
Number of Products under Investigation by Universities/Institutes, H2 2012 11
Comparative Analysis by Late Stage Development, H2 2012 12
Comparative Analysis by Mid Clinical Stage Development, H2 2012 13
Comparative Analysis by Early Clinical Stage Development, H2 2012 14
Comparative Analysis by Discovery and Pre-Clinical Stage Development, H2 2012 15
Products under Development by Companies, H2 2012 16
Products under Investigation by Universities/Institutes, H2 2012 17
Isis Pharmaceuticals, Inc., H2 2012 18
Pfizer Inc., H2 2012 19
Alnylam Pharmaceuticals, Inc, H2 2012 20
BIOALVO S.A., H2 2012 21
Assessment by Monotherapy Products, H2 2012 22
Assessment by Combination Products, H2 2012 23
Assessment by Stage and Route of Administration, H2 2012 25
Assessment by Stage and Molecule Type, H2 2012 27
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) Therapeutics – Drug Profile Updates 38

List of Figures
Number of Products under Development for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease), H2 2012 8
Products under Development for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) – Comparative Analysis, H2 2012 9
Products under Development by Companies, H2 2012 10
Products under Investigation by Universities/Institutes, H2 2012 11
Late Stage Products, H2 2012 12
Mid Clinical Stage Products, H2 2012 13
Early Clinical Stage Products, H2 2012 14
Discovery and Pre-Clinical Stage Products, H2 2012 15
Assessment by Monotherapy Products, H2 2012 22
Assessment by Combination Products, H2 2012 23
Assessment by Route of Administration, H2 2012 24
Assessment by Stage and Route of Administration, H2 2012 25
Assessment by Molecule Type, H2 2012 26
Assessment by Stage and Molecule Type, H2 2012 27

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