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

Global Markets Direct’s, ‘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 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, 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 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

Companies mentioned

Alnylam Pharmaceuticals, Inc.

Arcturus Therapeutics, Inc.

Ionis Pharmaceuticals, Inc.

Neurimmune Holding AG

Pfizer Inc.

Prothena Corporation Plc

Regeneron Pharmaceuticals Inc

SOM Biotech SL

Table of Contents

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 d’Hebron 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

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

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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