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Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2017 [Report Updated: 10-10-2017]

Published by Global Markets Direct: 10 Oct 2017 | 139662 | In Stock

Introduction

Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2017

Summary

According to the recently published report 'Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2017'; Transthyretin (ATTR or Prealbumin or TBPA or TTR) pipeline Target constitutes close to 15 molecules. Out of which approximately 12 molecules are developed by companies and remaining by the universities/institutes.

Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Transthyretin is a transport protein. It transports thyroid hormones in the plasma and cerebrospinal fluid, and also transports retinol (vitamin A) in the plasma. The diseases caused by mutations include amyloidotic polyneuropathy, euthyroid hyperthyroxinaemia, amyloidotic vitreous opacities, cardiomyopathy, oculoleptomeningeal amyloidosis, meningocerebrovascular amyloidosis and carpal tunnel syndrome.

The report 'Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2017' outlays 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; that are being developed by Companies / Universities.

It also reviews key players involved in Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted therapeutics development with respective active and dormant or discontinued projects. Currently, The molecules developed by companies in Filing rejected/Withdrawn, Phase III, Phase II, Phase I and Preclinical stages are 1, 2, 2, 2 and 5 respectively. Similarly, the universities portfolio in Phase II, Preclinical and Discovery stages comprises 1, 1 and 1 molecules, respectively. Report covers products from therapy areas Metabolic Disorders, Central Nervous System and Cardiovascular which include indications Familial Amyloid Neuropathies, Amyloidosis, Amyloid Cardiomyopathy, Familial Amyloid Cardiomyopathy, Alzheimer's Disease, Cardiomyopathy and Neuropathy.

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

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 it’s 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
for Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2017 [Report Updated: 10-10-2017]

  • 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

    Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Therapeutics Development 8

    Products under Development by Stage of Development 8

    Products under Development by Therapy Area 9

    Products under Development by Indication 10

    Products under Development by Companies 11

    Products under Development by Universities/Institutes 14

    Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Therapeutics Assessment 16

    Assessment by Mechanism of Action 16

    Assessment by Route of Administration 18

    Assessment by Molecule Type 20

    Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Companies Involved in Therapeutics Development 22

    Alnylam Pharmaceuticals Inc 22

    Arcturus Therapeutics Inc 22

    Ionis Pharmaceuticals Inc 23

    Neurimmune Holding AG 23

    Pfizer Inc 24

    Prothena Corp Plc 24

    Regeneron Pharmaceuticals Inc 24

    Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Drug Profiles 26

    AG-10 - Drug Profile 26

    Product Description 26

    Mechanism Of Action 26

    R&D Progress 26

    ALN-TTRsc02 - Drug Profile 27

    Product Description 27

    Mechanism Of Action 27

    R&D Progress 27

    CRX-1008 - Drug Profile 29

    Product Description 29

    Mechanism Of Action 29

    R&D Progress 29

    doxycycline hyclate - Drug Profile 31

    Product Description 31

    Mechanism Of Action 31

    R&D Progress 31

    EDE-1307 - Drug Profile 32

    Product Description 32

    Mechanism Of Action 32

    R&D Progress 32

    inotersen sodium - Drug Profile 33

    Product Description 33

    Mechanism Of Action 33

    R&D Progress 33

    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

    NPT-189 - Drug Profile 41

    Product Description 41

    Mechanism Of Action 41

    R&D Progress 41

    Oligonucleotides to Inhibit Transthyretin for Amyloidosis - Drug Profile 42

    Product Description 42

    Mechanism Of Action 42

    R&D Progress 42

    patisiran - Drug Profile 43

    Product Description 43

    Mechanism Of Action 43

    R&D Progress 43

    PRX-004 - Drug Profile 52

    Product Description 52

    Mechanism Of Action 52

    R&D Progress 52

    Small Molecules to Inhibit Transthyretin for Familial Amyloid Polyneuropathy - Drug Profile 53

    Product Description 53

    Mechanism Of Action 53

    R&D Progress 53

    tafamidis meglumine - Drug Profile 54

    Product Description 54

    Mechanism Of Action 54

    R&D Progress 54

    Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Dormant Products 58

    Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Discontinued Products 59

    Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Product Development Milestones 60

    Featured News & Press Releases 60

    Sep 28, 2017: Eidos Therapeutics Initiates First Clinical Study for AG10 Targeting Transthyretin Amyloidosis, Appoints Camille Landis as Chief Business Officer 60

    Sep 20, 2017: Arbutus’ LNP Licensee Alnylam Announces Positive Phase 3 Results for LNP-Enabled Patisiran Program 60

    Sep 20, 2017: Sanofi and Alnylam Report Positive Topline Results from APOLLO Phase 3 Study of Patisiran in Hereditary ATTR (hATTR) Amyloidosis Patients with Polyneuropathy 61

    Aug 31, 2017: Amyloid Publishes Long-term Data Analysis From Pfizer Suggesting Tafamidis Delays Progression of TTR-FAP, a Rare Disease 62

    Jul 18, 2017: Proclara Biosciences Presents New Preclinical Data Supporting the Development of NPT189 at Alzheimer’s Association International Conference (AAIC) 2017 64

    Jul 17, 2017: Proclara Biosciences Provides Update on NPT189 65

    Jul 10, 2017: New Data Presented at Peripheral Nerve Society Meeting Further Support Potential Benefit of Inotersen 65

    Jun 06, 2017: Pfizer Receives FDA Fast Track Designation for Tafamidis for Transthyretin Cardiomyopathy 66

    May 15, 2017: Ionis Pharmaceuticals Announces Phase 3 NEURO-TTR Study of Inotersen (IONIS-TTR Rx) Meets Both Primary Endpoints 67

    Apr 27, 2017: BridgeBio Pharma Launches Eidos Therapeutics to Develop Targeted Therapy for Fatal Heart Disease 68

    Apr 26, 2017: Alnylam Reports Final 24-Month Results from Phase 2 Open-Label Extension Study of Patisiran, an Investigational RNAi Therapeutic in Development for the Treatment of Hereditary ATTR (hATTR) Amyloidosis 69

    Mar 10, 2017: Proclara Biosciences Australia, a Subsidiary of Proclara Biosciences, Awarded Grant by Biopharmaceuticals Australia to Help Develop New Biotherapy with Blockbuster Potential 70

    Nov 07, 2016: VYNDAQEL (tafamidis) Receives Approval in Brazil by ANVISA for the Treatment of Transthyretin Familial Amyloid Polyneuropathy 70

    Oct 10, 2016: Data Monitoring Committee Recommends Continuation of APOLLO Phase 3 Clinical Trial of Patisiran for Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN) 71

    Aug 08, 2016: Pfizer Announces Publication of New Analysis Showing Long-Term Therapy with VYNDAQEL (tafamidis) Slowed Progression of Rare Neurodegenerative Disease 71

    Appendix 73

    Methodology 73

    Coverage 73

    Secondary Research 73

    Primary Research 73

    Expert Panel Validation 73

    Contact Us 73

    Disclaimer 74

Additional Details

Publisher

Global Markets Direct

Publisher Information

Reference

139662 | GMDHC1052TDB

Number of Pages

74

Report Format

PDF

Global Markets Direct Reports

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