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

Global Markets Direct’s, ‘Transthyretin (ATTR or Prealbumin or TBPA or TTR) – Pipeline Review, H2 2017’, provides in depth analysis on Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted pipeline therapeutics. The report provides comprehensive information 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. The report analyses the pipeline products from therapy areas Metabolic Disorders, Central Nervous System and Cardiovascular under development targeting Transthyretin (ATTR or Prealbumin or TBPA or TTR).

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 Corp Plc

Regeneron Pharmaceuticals Inc

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

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

List of Tables

List of Tables

Number of Products under Development by Stage of Development, H2 2017 8

Number of Products under Development by Therapy Areas, H2 2017 9

Number of Products under Development by Indication, H2 2017 10

Number of Products under Development by Companies, H2 2017 11

Products under Development by Companies, H2 2017 12

Products under Development by Companies, H2 2017 (Contd..1), H2 2017 13

Number of Products under Investigation by Universities/Institutes, H2 2017 14

Products under Investigation by Universities/Institutes, H2 2017 15

Number of Products by Stage and Mechanism of Actions, H2 2017 17

Number of Products by Stage and Route of Administration, H2 2017 19

Number of Products by Stage and Molecule Type, H2 2017 21

Pipeline by Alnylam Pharmaceuticals Inc, H2 2017 22

Pipeline by Arcturus Therapeutics Inc, H2 2017 22

Pipeline by Ionis Pharmaceuticals Inc, H2 2017 23

Pipeline by Neurimmune Holding AG, H2 2017 23

Pipeline by Pfizer Inc, H2 2017 24

Pipeline by Prothena Corp Plc, H2 2017 24

Pipeline by Regeneron Pharmaceuticals Inc, H2 2017 25

Dormant Projects, H2 2017 58

Discontinued Products, H2 2017 59

List of Figures

List of Figures

Number of Products under Development by Stage of Development, H2 2017 8

Number of Products under Development by Therapy Areas, H2 2017 9

Number of Products under Development by Top 10 Indications, H2 2017 10

Number of Products by Mechanism of Actions, H2 2017 16

Number of Products by Stage and Mechanism of Actions, H2 2017 16

Number of Products by Routes of Administration, H2 2017 18

Number of Products by Stage and Routes of Administration, H2 2017 18

Number of Products by Molecule Types, H2 2017 20

Number of Products by Stage and Molecule Types, H2 2017 20

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