Amyloidosis – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Amyloidosis – Pipeline Review, H2 2017’, provides an overview of the Amyloidosis pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Amyloidosis, 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 Amyloidosis 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 Amyloidosis

The report reviews pipeline therapeutics for Amyloidosis 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 Amyloidosis therapeutics and enlists all their major and minor projects

The report assesses Amyloidosis 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 Amyloidosis

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 Amyloidosis

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 Amyloidosis 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

Amgen Inc

Arcturus Therapeutics Inc

Bsim2

Celgene Corp

GlaxoSmithKline Plc

Ionis Pharmaceuticals Inc

Johnson & Johnson

Millennium Pharmaceuticals Inc

Neurimmune Holding AG

Novartis AG

Oncopeptides AB

Pfizer Inc

Prothena Corp Plc

R Pharm

Regeneron Pharmaceuticals Inc

SOM Biotech SL

Spectrum Pharmaceuticals Inc

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 4

List of Figures 4

Introduction 5

Global Markets Direct Report Coverage 5

Amyloidosis - Overview 6

Amyloidosis - Therapeutics Development 7

Pipeline Overview 7

Pipeline by Companies 8

Pipeline by Universities/Institutes 11

Products under Development by Companies 12

Products under Development by Universities/Institutes 14

Amyloidosis - Therapeutics Assessment 15

Assessment by Target 15

Assessment by Mechanism of Action 17

Assessment by Route of Administration 19

Assessment by Molecule Type 21

Amyloidosis - Companies Involved in Therapeutics Development 23

Alnylam Pharmaceuticals Inc 23

Amgen Inc 23

Arcturus Therapeutics Inc 24

Bsim2 24

Celgene Corp 25

GlaxoSmithKline Plc 25

Ionis Pharmaceuticals Inc 26

Johnson & Johnson 26

Millennium Pharmaceuticals Inc 27

Neurimmune Holding AG 27

Novartis AG 28

Oncopeptides AB 28

Pfizer Inc 29

Prothena Corp Plc 29

R Pharm 30

Regeneron Pharmaceuticals Inc 30

SOM Biotech SL 31

Spectrum Pharmaceuticals Inc 31

Amyloidosis - Drug Profiles 32

AG-10 - Drug Profile 32

ALN-ANG - Drug Profile 33

ALN-TTRsc02 - Drug Profile 34

CAEL-101 - Drug Profile 35

canakinumab - Drug Profile 37

carfilzomib - Drug Profile 43

CLR-01 - Drug Profile 55

daratumumab - Drug Profile 57

dezamizumab - Drug Profile 73

dezamizumab + GSK-2315698 - Drug Profile 74

doxycycline hyclate - Drug Profile 75

EDE-1307 - Drug Profile 76

GSK-2315698 - Drug Profile 77

GSK-3039294 - Drug Profile 78

inotersen sodium - Drug Profile 79

ixazomib citrate - Drug Profile 83

LUNAR-TTR - Drug Profile 93

melphalan - Drug Profile 94

melphalan flufenamide - Drug Profile 98

Monoclonal Antibody to Inhibit ATTR for Familial Amyloid Polyneuropathy - Drug Profile 100

Monoclonal Antibody to Target CD66b for Blood Cancer and Metabolic Disorders - Drug Profile 101

NEOD-001 - Drug Profile 102

NI-301 - Drug Profile 107

NPT-289 - Drug Profile 108

Oligonucleotides to Inhibit Transthyretin for Amyloidosis - Drug Profile 109

patisiran - Drug Profile 110

pomalidomide - Drug Profile 118

PRX-004 - Drug Profile 125

RPH-104 - Drug Profile 126

Small Molecule to Inhibit Amyloid Beta Peptide for Alzheimer's Disease and Amyloidosis - Drug Profile 127

Small Molecules to Activate ATF6 for Amyloidosis - Drug Profile 128

Small Molecules to Inhibit Transthyretin for Familial Amyloid Polyneuropathy - Drug Profile 129

Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Cardiomyopathy - Drug Profile 130

Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Polyneuropathy - Drug Profile 131

tafamidis meglumine - Drug Profile 132

tolcapone - Drug Profile 135

Amyloidosis - Dormant Projects 137

Amyloidosis - Discontinued Products 138

Amyloidosis - Product Development Milestones 139

Featured News & Press Releases 139

Appendix 153

Methodology 153

Coverage 153

Secondary Research 153

Primary Research 153

Expert Panel Validation 153

Contact Us 153

Disclaimer 154

List of Tables

List of Tables

Number of Products under Development for Amyloidosis, H2 2017

Number of Products under Development by Companies, H2 2017

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

Number of Products under Development by Universities/Institutes, H2 2017

Products under Development by Companies, H2 2017

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

Products under Development by Universities/Institutes, H2 2017

Number of Products by Stage and Target, H2 2017

Number of Products by Stage and Mechanism of Action, H2 2017

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

Number of Products by Stage and Molecule Type, H2 2017

Amyloidosis – Pipeline by Alnylam Pharmaceuticals Inc, H2 2017

Amyloidosis – Pipeline by Amgen Inc, H2 2017

Amyloidosis – Pipeline by Arcturus Therapeutics Inc, H2 2017

Amyloidosis – Pipeline by Bsim2, H2 2017

Amyloidosis – Pipeline by Celgene Corp, H2 2017

Amyloidosis – Pipeline by GlaxoSmithKline Plc, H2 2017

Amyloidosis – Pipeline by Ionis Pharmaceuticals Inc, H2 2017

Amyloidosis – Pipeline by Johnson & Johnson, H2 2017

Amyloidosis – Pipeline by Millennium Pharmaceuticals Inc, H2 2017

Amyloidosis – Pipeline by Neurimmune Holding AG, H2 2017

Amyloidosis – Pipeline by Novartis AG, H2 2017

Amyloidosis – Pipeline by Oncopeptides AB, H2 2017

Amyloidosis – Pipeline by Pfizer Inc, H2 2017

Amyloidosis – Pipeline by Prothena Corp Plc, H2 2017

Amyloidosis – Pipeline by R Pharm, H2 2017

Amyloidosis – Pipeline by Regeneron Pharmaceuticals Inc, H2 2017

Amyloidosis – Pipeline by SOM Biotech SL, H2 2017

Amyloidosis – Pipeline by Spectrum Pharmaceuticals Inc, H2 2017

Amyloidosis – Dormant Projects, H2 2017

Amyloidosis – Discontinued Products, H2 2017

List of Figures

List of Figures

Number of Products under Development for Amyloidosis, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Number of Products by Top 10 Targets, H2 2017

Number of Products by Stage and Top 10 Targets, H2 2017

Number of Products by Top 10 Mechanism of Actions, H2 2017

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2017

Number of Products by Routes of Administration, H2 2017

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

Number of Products by Molecule Types, H2 2017

Number of Products by Stage and Molecule Types, H2 2017

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