Familial Amyloid Neuropathies – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Familial Amyloid Neuropathies – Pipeline Review, H2 2016’, provides an overview of the Familial Amyloid Neuropathies pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Familial Amyloid Neuropathies, complete with analysis by stage of development, drug target, 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 therapeutic development for Familial Amyloid Neuropathies 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.

Scope

The report provides a snapshot of the global therapeutic landscape of Familial Amyloid Neuropathies

The report reviews pipeline therapeutics for Familial Amyloid Neuropathies 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 Familial Amyloid Neuropathies therapeutics and enlists all their major and minor projects

The report assesses Familial Amyloid Neuropathies therapeutics based on drug target, 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 related to pipeline therapeutics for Familial Amyloid Neuropathies

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 important and diverse types of therapeutics under development for Familial Amyloid Neuropathies

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 Familial Amyloid Neuropathies 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

Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Companies mentioned

Alnylam Pharmaceuticals Inc

Bsim2

Ionis Pharmaceuticals Inc

Pfizer 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

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

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

List of Figures

List of Figures

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 Products, H2 2016 14

Assessment by Monotherapy Products, H2 2016 22

Number of Products by Targets, H2 2016 23

Number of Products by Stage and Targets, H2 2016 23

Number of Products by Mechanism of Actions, H2 2016 25

Number of Products by Stage and Mechanism of Actions, H2 2016 25

Number of Products by Routes of Administration, H2 2016 27

Number of Products by Stage and Routes of Administration, H2 2016 27

Number of Products by Molecule Types, H2 2016 29

Number of Products by Stage and Molecule Types, H2 2016 29

    Pricing

Discounts available for multiple purchases.

reportstore@globalmarketsdirect.com
+44 20 7947 2745

Saved reports