Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrades Disease) - Pipeline Review, H2 2014

Published: Jul, 2014 | Pages: 59 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)

Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline Review, H2 2014

Summary

Global Markets Direct's, 'Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline Review, H2 2014', provides an overview of the Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease)'s therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease), 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 Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) 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.

Scope

- The report provides a snapshot of the global therapeutic landscape of Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease)
- 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 Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects 
- 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
- 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 Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) 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 Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease)
- Plan mergers and acquisitions effectively by identifying key 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
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Products under Development by Companies 15
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Products under Investigation by Universities/Institutes 16
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Companies Involved in Therapeutics Development 17
GlaxoSmithKline plc 17
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
Drug Profiles 30
tafamidis meglumine - Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
patisiran - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
ISIS-TTRRx - Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
SOM-0226 - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
Antisense Oligonucleotide for Transthyretin Amyloidosis - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
ALNTTR-01 - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
Monoclonal Antibody to Inhibit ATTR for Familial Amyloid Polyneuropathy - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
LUNAR-102 - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Recent Pipeline Updates 40
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Dormant Projects 50
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Product Development Milestones 51
Featured News & Press Releases 51
Jun 24, 2014: Isis Pharmaceuticals Earns $1 Million From GSK for Advancing ISIS-TTR Rx 51
May 15, 2014: Isis Pharmaceuticals Earns $3.0 Million from GSK 51
Apr 02, 2014: Alnylam Receives Positive Opinion for Orphan Drug Designation in the European Union for ALN-TTRsc, a Subcutaneously Delivered RNAi Therapeutic for the Treatment of Transthyretin-Mediated Amyloidosis 52
Mar 21, 2014: Alnylam to Present Data from its RNAi Therapeutic Programs Targeting Transthyretin for the Treatment of TTR-Mediated Amyloidosis at XIVth International Symposium on Amyloidosis 52
Feb 14, 2014: Isis Announces Positive Opinion on European Orphan Drug Designation for ISIS-TTR Rx 53
Jan 10, 2014: SOM Biotech announces Orphan Drug Designation in US for SOM0226, a compound for the treatment of Transthyretin Amyloidosis 54
Dec 16, 2013: Tekmira Receives $5 Million Milestone Payment for Phase III Initiation of LNP-Enabled Patisiran 54
Dec 02, 2013: Isis Pharmaceuticals Earns $2 Million from GSK for the Advancement of ISIS-TTR Rx 55
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 55
Jul 30, 2013: GSK makes milestone payment to Isis for progressing ISIS-TTRRx study 57
Appendix 58
Methodology 58
Coverage 58
Secondary Research 58
Primary Research 58
Expert Panel Validation 58
Contact Us 59
Disclaimer 59

List of Tables
Number of Products under Development for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease), H2 2014 8
Number of Products under Development for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Comparative Analysis, H2 2014 9
Number of Products under Development by Companies, H2 2014 10
Number of Products under Investigation by Universities/Institutes, H2 2014 11
Comparative Analysis by Late Stage Development, H2 2014 12
Comparative Analysis by Clinical Stage Development, H2 2014 13
Comparative Analysis by Early Stage Development, H2 2014 14
Products under Development by Companies, H2 2014 15
Products under Investigation by Universities/Institutes, H2 2014 16
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline by GlaxoSmithKline plc, H2 2014 17
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline by Isis Pharmaceuticals, Inc., H2 2014 18
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline by Pfizer Inc., H2 2014 19
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline by Alnylam Pharmaceuticals, Inc., H2 2014 20
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline by SOM Biotech SL, H2 2014 21
Assessment by Monotherapy Products, H2 2014 22
Number of Products by Stage and Target, H2 2014 23
Number of Products by Stage and Mechanism of Action, H2 2014 25
Number of Products by Stage and Route of Administration, H2 2014 27
Number of Products by Stage and Molecule Type, H2 2014 29
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) Therapeutics - Recent Pipeline Updates, H2 2014 40
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Dormant Projects, H2 2014 50