Familial Amyloid Neuropathies - Pipeline Review, H2 2016

Published: Dec, 2016 | Pages: 72 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Familial Amyloid Neuropathies - Pipeline Review, H2 2016, provides an overview of the Familial Amyloid Neuropathies (Metabolic Disorders) pipeline landscape.

Familial amyloid neuropathy is a slowly progressive condition characterized by the buildup of abnormal deposits of a protein called amyloid (amyloidosis) in the body's organs and tissues. Symptoms include numbness, tingling, pins and needles in the feet and hands, weakness and pain in the arms and legs, loss of sensation, urinary retention, reduced sweating, ankle swelling, fatigue, nausea, weight loss, dizziness and fainting. Supportive treatment includes diuretics, calcium channel blockers, beta blockers, angiotensin receptor blockers and angiotensin converting enzyme (ACE) inhibitors.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Familial Amyloid Neuropathies - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Familial Amyloid Neuropathies (Metabolic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Familial Amyloid Neuropathies (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Familial Amyloid Neuropathies and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Filing rejected/Withdrawn, Phase III, Phase II and Discovery stages are 1, 2, 2 and 1 respectively. Similarly, the Universities portfolio in Phase II, Preclinical and Discovery stages comprises 1, 2 and 1 molecules, respectively.Familial Amyloid Neuropathies.

Familial Amyloid Neuropathies (Metabolic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Familial Amyloid Neuropathies (Metabolic Disorders).
- The pipeline guide reviews pipeline therapeutics for Familial Amyloid Neuropathies (Metabolic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Familial Amyloid Neuropathies (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Familial Amyloid Neuropathies (Metabolic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Familial Amyloid Neuropathies (Metabolic Disorders)

Reasons To Buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Familial Amyloid Neuropathies (Metabolic Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Familial Amyloid Neuropathies (Metabolic Disorders) 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

 Table of Contents

Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Familial Amyloid Neuropathies Overview 7
Therapeutics Development 8
Pipeline Products for Familial Amyloid Neuropathies - Overview 8
Pipeline Products for Familial Amyloid Neuropathies - Comparative Analysis 9
Familial Amyloid Neuropathies - Therapeutics under Development by Companies 10
Familial Amyloid Neuropathies - Therapeutics under Investigation by Universities/Institutes 11
Familial Amyloid Neuropathies - Pipeline Products Glance 12
Late Stage Products 12
Clinical Stage Products 13
Early Stage Products 14
Familial Amyloid Neuropathies - Products under Development by Companies 15
Familial Amyloid Neuropathies - Products under Investigation by Universities/Institutes 16
Familial Amyloid Neuropathies - Companies Involved in Therapeutics Development 17
Alnylam Pharmaceuticals Inc 17
Bsim2 18
Ionis Pharmaceuticals Inc 19
Pfizer Inc 20
SOM Biotech SL 21
Familial Amyloid Neuropathies - Therapeutics Assessment 22
Assessment by Monotherapy Products 22
Assessment by Target 23
Assessment by Mechanism of Action 25
Assessment by Route of Administration 27
Assessment by Molecule Type 29
Drug Profiles 31
CLR-01 - Drug Profile 31
Product Description 31
Mechanism Of Action 31
R&D Progress 31
doxycycline hyclate - Drug Profile 32
Product Description 32
Mechanism Of Action 32
R&D Progress 32
EDE-1307 - Drug Profile 33
Product Description 33
Mechanism Of Action 33
R&D Progress 33
inotersen sodium - 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 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
patisiran - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
Small Molecules to Inhibit Transthyretin for Familial Amyloid Polyneuropathy - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Polyneuropathy - Drug Profile 47
Product Description 47
Mechanism Of Action 47
R&D Progress 47
tafamidis meglumine - Drug Profile 48
Product Description 48
Mechanism Of Action 48
R&D Progress 48
tolcapone - Drug Profile 51
Product Description 51
Mechanism Of Action 51
R&D Progress 51
Familial Amyloid Neuropathies - Dormant Projects 52
Familial Amyloid Neuropathies - Discontinued Products 53
Familial Amyloid Neuropathies - Product Development Milestones 54
Featured News & Press Releases 54
Oct 10, 2016: Data Monitoring Committee Recommends Continuation of APOLLO Phase 3 Clinical Trial of Patisiran for Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN) 54
Jul 07, 2016: Ionis Pharmaceuticals Reports Positive Clinical Data on IONIS-TTR Rx at the XV International Symposium on Amyloidosis 54
Jul 01, 2016: Alnylam Reports New Results on patisiran at the XV International Symposium on Amyloidosis 56
Jun 27, 2016: Alnylam to Report New Patisiran Results at the XV International Symposium on Amyloidosis 58
May 26, 2016: Ionis Pharmaceuticals Provides Update on IONIS-TTR Rx Program 58
Apr 20, 2016: Alnylam Reports Complete 18-Month Data from Ongoing Phase 2 Open-Label Extension Study of Patisiran, an Investigational RNAi Therapeutic Targeting Transthyretin for the Treatment of Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN) 59
Apr 07, 2016: Ionis Pharmaceuticals Provides Update on IONIS-TTRRX Program 61
Feb 01, 2016: Alnylam Completes Enrollment in APOLLO Phase 3 Study with Patisiran, an Investigational RNAi Therapeutic for Patients with Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis) 61
Dec 17, 2015: Isis Pharmaceuticals Announces Initiation of an Investigator-Sponsored Phase 2 Study of ISIS-TTR Rx in Patients with TTR Cardiomyopathy Amyloidosis 62
Nov 03, 2015: Isis Pharmaceuticals Reports an Update on ISIS-TTR Rx, Including Positive Data from Multiple Clinical Studies, Presented at the EC-ATTR Meeting 63
Nov 03, 2015: Alnylam Reports Positive Results from Ongoing Phase 2 Open-Label Extension Studies for Patisiran in Development for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis) 65
Oct 27, 2015: Isis Pharmaceuticals to Host Webcast to Review ISIS-TTR Data Presented at 1st European Congress of Hereditary ATTR Amyloidosis and Provide Phase 3 Development Plan Update 66
Sep 28, 2015: Alnylam and Collaborators Report First-Ever Evidence that Patisiran Reduces Pathogenic, Misfolded Transthyretin Monomers and Oligomers in TTR-Mediated Amyloidosis Patients with Familial Amyloidotic Polyneuropathy 66
Jul 20, 2015: Alnylam Initiates Phase 3 Open Label Extension Study with Patisiran, an Investigational RNAi Therapeutic Targeting Transthyretin for the Treatment of TTR-Mediated Amyloidosis 68
Apr 22, 2015: Isis Pharmaceuticals Reports Positive Data From ISIS-TTR Rx in Patients With TTR Amyloidosis 69
Appendix 71
Methodology 71
Coverage 71
Secondary Research 71
Primary Research 71
Expert Panel Validation 71
Contact Us 71
Disclaimer 72

List of Tables

Number of Products under Development for Familial Amyloid Neuropathies, H2 2016 8
Number of Products under Development for Familial Amyloid Neuropathies - Comparative Analysis, H2 2016 9
Number of Products under Development by Companies, H2 2016 10
Number of Products under Investigation by Universities/Institutes, H2 2016 11
Comparative Analysis by Late Stage Development, H2 2016 12
Comparative Analysis by Clinical Stage Development, H2 2016 13
Comparative Analysis by Early Stage Development, H2 2016 14
Products under Development by Companies, H2 2016 15
Products under Investigation by Universities/Institutes, H2 2016 16
Familial Amyloid Neuropathies - Pipeline by Alnylam Pharmaceuticals Inc, H2 2016 17
Familial Amyloid Neuropathies - Pipeline by Bsim2, H2 2016 18
Familial Amyloid Neuropathies - Pipeline by Ionis Pharmaceuticals Inc, H2 2016 19
Familial Amyloid Neuropathies - Pipeline by Pfizer Inc, H2 2016 20
Familial Amyloid Neuropathies - Pipeline by SOM Biotech SL, H2 2016 21
Assessment by Monotherapy Products, H2 2016 22
Number of Products by Stage and Target, H2 2016 24
Number of Products by Stage and Mechanism of Action, H2 2016 26
Number of Products by Stage and Route of Administration, H2 2016 28
Number of Products by Stage and Molecule Type, H2 2016 30
Familial Amyloid Neuropathies - Dormant Projects, H2 2016 52
Familial Amyloid Neuropathies - Discontinued Products, H2 2016 53