Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrades Disease) - Pipeline Review, H1 2015

Published: Mar, 2015 | Pages: 59 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline Review, H1 2015

Summary

Global Markets Direct’s, ‘Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline Review, H1 2015’, 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
Alnylam Pharmaceuticals, Inc. 17
Arcturus Therapeutics, Inc 18
Bsim2 19
GlaxoSmithKline plc 20
Isis Pharmaceuticals, Inc. 21
Pfizer Inc. 22
SOM Innovation Biotech SL 23
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Therapeutics Assessment 24
Assessment by Monotherapy Products 24
Assessment by Target 25
Assessment by Mechanism of Action 26
Assessment by Route of Administration 28
Assessment by Molecule Type 30
Drug Profiles 32
Antisense Oligonucleotide for Transthyretin Amyloidosis - 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
LUNAR-102 - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
Monoclonal Antibody to Inhibit ATTR for Familial Amyloid Polyneuropathy - 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
Small Molecules to Inhibit Transthyretin for Familial Amyloid Polyneuropathy - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Polyneuropathy - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
tafamidis meglumine - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
tolcapone - 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 51
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Product Development Milestones 52
Featured News & Press Releases 52
Mar 02, 2015: Isis Pharmaceuticals Earns $15 Million From GSK for Advancing ISIS-TTR 52
Oct 13, 2014: Arcturus Therapeutics to Present Gene Knockdown Data in Non-Human Primates, Showing up to 94% Reduction in Gene Expression with a Single Low Dose 52
Oct 13, 2014: Alnylam Reports Six-Month Clinical Data from Patisiran Phase 2 Open-Label Extension (OLE) Study in Patients with Familial Amyloidotic Polyneuropathy (FAP) 52
Oct 09, 2014: Isis Pharmaceuticals Earns $18 Million From GSK for Advancing ISIS-TTR Rx 54
Jul 16, 2014: Isis Pharmaceuticals Earns $1 Million from GSK for Advancing ISIS-TTR Rx 54
Jun 24, 2014: Isis Pharmaceuticals Earns $1 Million From GSK for Advancing ISIS-TTR Rx 55
May 15, 2014: Isis Pharmaceuticals Earns $3.0 Million from GSK 55
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 56
Feb 14, 2014: Isis Announces Positive Opinion on European Orphan Drug Designation for ISIS-TTR Rx 56
Jan 10, 2014: SOM Biotech announces Orphan Drug Designation in US for SOM0226, a compound for the treatment of Transthyretin Amyloidosis 57
Appendix 58
Methodology 58
Coverage 58
Secondary Research 58
Primary Research 58
Expert Panel Validation 58
Contact Us 58
Disclaimer 59

List of Tables

Number of Products under Development for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease), H1 2015 8
Number of Products under Development for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Comparative Analysis, H1 2015 9
Number of Products under Development by Companies, H1 2015 10
Number of Products under Investigation by Universities/Institutes, H1 2015 11
Comparative Analysis by Late Stage Development, H1 2015 12
Comparative Analysis by Clinical Stage Development, H1 2015 13
Comparative Analysis by Early Stage Development, H1 2015 14
Products under Development by Companies, H1 2015 15
Products under Investigation by Universities/Institutes, H1 2015 16
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline by Alnylam Pharmaceuticals, Inc., H1 2015 17
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline by Arcturus Therapeutics, Inc, H1 2015 18
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline by Bsim2, H1 2015 19
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline by GlaxoSmithKline plc, H1 2015 20
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline by Isis Pharmaceuticals, Inc., H1 2015 21
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline by Pfizer Inc., H1 2015 22
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline by SOM Innovation Biotech SL, H1 2015 23
Assessment by Monotherapy Products, H1 2015 24
Number of Products by Stage and Target, H1 2015 25
Number of Products by Stage and Mechanism of Action, H1 2015 27
Number of Products by Stage and Route of Administration, H1 2015 29
Number of Products by Stage and Molecule Type, H1 2015 31
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) Therapeutics - Recent Pipeline Updates, H1 2015 42
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Dormant Projects, H1 2015 51