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Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2016

Published By :

Global Markets Direct

Published Date : Oct 2016

Category :

Pharmaceutical

No. of Pages : 77 Pages

Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2016

Summary

Global Markets Directs, Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2016, provides in depth analysis on Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted pipeline therapeutics.

The report provides comprehensive information on the Transthyretin (ATTR or Prealbumin or TBPA or TTR) , targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted therapeutics development and features dormant and discontinued projects.

Global Markets Directs 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 Directs 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note:
*Certain sections in the report may be removed or altered based on the availability and relevance of data.
*Updated report will be delivered in 48 hours of order confirmation.

Scope

- The report provides a snapshot of the global therapeutic landscape for Transthyretin (ATTR or Prealbumin or TBPA or TTR)
- The report reviews Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
- The report reviews key players involved in Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted therapeutics and enlists all their major and minor projects
- The report assesses Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted therapeutics based on mechanism of action (MoA), route of administration (RoA) and molecule type
- The report summarizes all the dormant and discontinued pipeline projects
- The report reviews latest news and deals related to Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted therapeutics

Reasons to buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Identify and understand the targeted therapy areas and indications for Transthyretin (ATTR or Prealbumin or TBPA or TTR)
- Identify the use of drugs for target identification and drug repurposing
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies
- Plan mergers and acquisitions effectively by identifying key players and its most promising pipeline therapeutics
- Devise corrective measures for pipeline projects by understanding Transthyretin (ATTR or Prealbumin or TBPA or TTR) development landscape
- 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
Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Transthyretin (ATTR or Prealbumin or TBPA or TTR) Overview 7
Therapeutics Development 8
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Products under Development by Stage of Development 8
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Products under Development by Therapy Area 9
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Products under Development by Indication 10
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Products Glance 11
Late Stage Products 11
Early Stage Products 12
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Products under Development by Companies 13
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Products under Development by Universities/Institutes 15
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Therapeutics Assessment 17
Assessment by Monotherapy/Combination Products 17
Assessment by Mechanism of Action 18
Assessment by Route of Administration 19
Assessment by Molecule Type 21
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Companies Involved in Therapeutics Development 23
Alnylam Pharmaceuticals, Inc. 23
Arcturus Therapeutics, Inc. 24
Ionis Pharmaceuticals, Inc. 25
Neurimmune Holding AG 26
Pfizer Inc. 27
Prothena Corporation Plc 28
Regeneron Pharmaceuticals Inc 29
SOM Biotech SL 30
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Drug Profiles 31
ALN-TTRsc02 - 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
LUNAR-TTR - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
Monoclonal Antibody to Inhibit ATTR for Familial Amyloid Polyneuropathy - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
NI-301 - Drug Profile 40
Product Description 40
Mechanism Of Action 40
R&D Progress 40
Oligonucleotides to Inhibit Transthyretin for Amyloidosis - Drug Profile 41
Product Description 41
Mechanism Of Action 41
R&D Progress 41
patisiran - Drug Profile 42
Product Description 42
Mechanism Of Action 42
R&D Progress 42
PRX-004 - Drug Profile 50
Product Description 50
Mechanism Of Action 50
R&D Progress 50
Small Molecules to Inhibit Transthyretin for Familial Amyloid Polyneuropathy - Drug Profile 51
Product Description 51
Mechanism Of Action 51
R&D Progress 51
tafamidis meglumine - Drug Profile 52
Product Description 52
Mechanism Of Action 52
R&D Progress 52
tolcapone - Drug Profile 55
Product Description 55
Mechanism Of Action 55
R&D Progress 55
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Dormant Projects 57
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Discontinued Products 58
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Featured News & Press Releases 59
Oct 10, 2016: Data Monitoring Committee Recommends Continuation of APOLLO Phase 3 Clinical Trial of Patisiran for Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN) 59
Aug 08, 2016: Pfizer Announces Publication of New Analysis Showing Long-Term Therapy with VYNDAQEL (tafamidis) Slowed Progression of Rare Neurodegenerative Disease 59
Jul 07, 2016: Ionis Pharmaceuticals Reports Positive Clinical Data on IONIS-TTR Rx at the XV International Symposium on Amyloidosis 60
Jul 01, 2016: Alnylam Reports New Results on patisiran at the XV International Symposium on Amyloidosis 62
Jun 27, 2016: Alnylam to Report New Patisiran Results at the XV International Symposium on Amyloidosis 64
Jun 09, 2016: Alnylam Initiates Phase 1 Clinical Trial for ALN-TTRsc02, an Investigational RNAi Therapeutic for the Treatment of TTR-Mediated Amyloidosis 65
May 26, 2016: Ionis Pharmaceuticals Provides Update on IONIS-TTR Rx Program 65
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) 66
Apr 07, 2016: Ionis Pharmaceuticals Provides Update on IONIS-TTRRX Program 68
Mar 16, 2016: Data from Prothena's Preclinical Program on Conformation-Specific Antibodies Against Misfolded Transthyretin Published in Amyloid 69
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) 69
Dec 17, 2015: Isis Pharmaceuticals Announces Initiation of an Investigator-Sponsored Phase 2 Study of ISIS-TTR Rx in Patients with TTR Cardiomyopathy Amyloidosis 70
Nov 12, 2015: SOM Biotech and Vall dHebron successfully complete a clinical trial in patients with TTR amyloidosis 71
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) 72
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 74
Appendix 76
Methodology 76
Coverage 76
Secondary Research 76
Primary Research 76
Expert Panel Validation 76
Contact Us 76
Disclaimer 77

List of Tables
Number of Products under Development for, H2 2016 8
Number of Products under Development by Therapy Area, H2 2016 9
Number of Products under Development by Indication, H2 2016 10
Comparative Analysis by Late Stage Development, H2 2016 11
Comparative Analysis by Early Stage Products, H2 2016 12
Number of Products under Development by Companies, H2 2016 13
Products under Development by Companies, H2 2016 14
Number of Products under Investigation by Universities/Institutes, H2 2016 15
Products under Investigation by Universities/Institutes, H2 2016 16
Assessment by Monotherapy/Combination Products, H2 2016 17
Number of Products by Stage and Mechanism of Action, H2 2016 18
Number of Products by Stage and Route of Administration, H2 2016 20
Number of Products by Stage and Molecule Type, H2 2016 22
Pipeline by Alnylam Pharmaceuticals, Inc., H2 2016 23
Pipeline by Arcturus Therapeutics, Inc., H2 2016 24
Pipeline by Ionis Pharmaceuticals, Inc., H2 2016 25
Pipeline by Neurimmune Holding AG, H2 2016 26
Pipeline by Pfizer Inc., H2 2016 27
Pipeline by Prothena Corporation Plc, H2 2016 28
Pipeline by Regeneron Pharmaceuticals Inc, H2 2016 29
Pipeline by SOM Biotech SL, H2 2016 30
Dormant Projects, H2 2016 57
Discontinued Products, H2 2016 58

List of Figures
Number of Products under Development for, H2 2016 8
Number of Products under Development by Therapy Area, H2 2016 9
Number of Products under Development by Indication, H2 2016 10
Comparative Analysis by Late Stage Development, H2 2016 11
Comparative Analysis by Early Stage Products, H2 2016 12
Assessment by Monotherapy/Combination Products, H2 2016 17
Number of Products by Stage and Mechanism of Actions, H2 2016 18
Number of Products by Routes of Administration, H2 2016 19
Number of Products by Stage and Routes of Administration, H2 2016 19
Number of Products by Molecule Types, H2 2016 21
Number of Products by Stage and Molecule Type, H2 2016 21

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