Amyloidosis – Pipeline Review, H1 2018

Global Markets Direct’s, ‘Amyloidosis – Pipeline Review, H1 2018’, 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 Ltd

Bsim2

Celgene Corp

Chugai Pharmaceutical Co Ltd

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

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 15

Amyloidosis - Therapeutics Assessment 16

Assessment by Target 16

Assessment by Mechanism of Action 18

Assessment by Route of Administration 20

Assessment by Molecule Type 22

Amyloidosis - Companies Involved in Therapeutics Development 24

Alnylam Pharmaceuticals Inc 24

Amgen Inc 24

Arcturus Therapeutics Ltd 25

Bsim2 25

Celgene Corp 26

Chugai Pharmaceutical Co Ltd 26

GlaxoSmithKline Plc 27

Ionis Pharmaceuticals Inc 27

Johnson & Johnson 28

Millennium Pharmaceuticals Inc 28

Neurimmune Holding AG 29

Novartis AG 29

Oncopeptides AB 30

Pfizer Inc 30

Prothena Corp Plc 31

R Pharm 31

Regeneron Pharmaceuticals Inc 32

Spectrum Pharmaceuticals Inc 32

Amyloidosis - Drug Profiles 33

AG-10 - Drug Profile 33

ALN-TTRsc02 - Drug Profile 35

birtamimab - Drug Profile 37

CAEL-101 - Drug Profile 44

canakinumab - Drug Profile 46

carfilzomib - Drug Profile 54

Cellular Immunotherapy to Target CS1 for Metabolic Disorders and Oncology - Drug Profile 68

CLR-01 - Drug Profile 69

CRX-1008 - Drug Profile 71

daratumumab - Drug Profile 73

dezamizumab + GSK-2315698 - Drug Profile 91

doxycycline hyclate - Drug Profile 92

EDE-1307 - Drug Profile 93

GSK-2315698 - Drug Profile 94

GSK-3039294 - Drug Profile 95

inotersen sodium - Drug Profile 96

ixazomib citrate - Drug Profile 102

LUNAR-TTR - Drug Profile 113

melphalan - Drug Profile 114

melphalan flufenamide - Drug Profile 118

Monoclonal Antibody to Target CD66b for Blood Cancer and Metabolic Disorders - Drug Profile 121

NI-301 - Drug Profile 122

NPT-189 - Drug Profile 123

Oligonucleotides to Inhibit Transthyretin for Amyloidosis - Drug Profile 124

patisiran - Drug Profile 125

pomalidomide - Drug Profile 135

PRX-004 - Drug Profile 142

RPH-104 - Drug Profile 143

Small Molecule to Inhibit Amyloid Beta Peptide for Alzheimer's Disease and Amyloidosis - Drug Profile 144

Small Molecules to Activate ATF6 for Amyloidosis - Drug Profile 145

Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Cardiomyopathy - Drug Profile 146

Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Polyneuropathy - Drug Profile 147

tafamidis meglumine - Drug Profile 148

tocilizumab - Drug Profile 152

VERU-111 - Drug Profile 163

VERU-112 - Drug Profile 164

Amyloidosis - Dormant Projects 165

Amyloidosis - Discontinued Products 166

Amyloidosis - Product Development Milestones 167

Featured News & Press Releases 167

Appendix 178

Methodology 178

Coverage 178

Secondary Research 178

Primary Research 178

Expert Panel Validation 178

Contact Us 178

Disclaimer 179

List of Tables

List of Tables

Number of Products under Development for Amyloidosis, H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Companies, H1 2018 (Contd..1), H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Products under Development by Companies, H1 2018

Products under Development by Companies, H1 2018 (Contd..1), H1 2018

Products under Development by Companies, H1 2018 (Contd..2), H1 2018

Products under Development by Universities/Institutes, H1 2018

Number of Products by Stage and Target, H1 2018

Number of Products by Stage and Mechanism of Action, H1 2018

Number of Products by Stage and Route of Administration, H1 2018

Number of Products by Stage and Molecule Type, H1 2018

Amyloidosis – Pipeline by Alnylam Pharmaceuticals Inc, H1 2018

Amyloidosis – Pipeline by Amgen Inc, H1 2018

Amyloidosis – Pipeline by Arcturus Therapeutics Ltd, H1 2018

Amyloidosis – Pipeline by Bsim2, H1 2018

Amyloidosis – Pipeline by Celgene Corp, H1 2018

Amyloidosis – Pipeline by Chugai Pharmaceutical Co Ltd, H1 2018

Amyloidosis – Pipeline by GlaxoSmithKline Plc, H1 2018

Amyloidosis – Pipeline by Ionis Pharmaceuticals Inc, H1 2018

Amyloidosis – Pipeline by Johnson & Johnson, H1 2018

Amyloidosis – Pipeline by Millennium Pharmaceuticals Inc, H1 2018

Amyloidosis – Pipeline by Neurimmune Holding AG, H1 2018

Amyloidosis – Pipeline by Novartis AG, H1 2018

Amyloidosis – Pipeline by Oncopeptides AB, H1 2018

Amyloidosis – Pipeline by Pfizer Inc, H1 2018

Amyloidosis – Pipeline by Prothena Corp Plc, H1 2018

Amyloidosis – Pipeline by R Pharm, H1 2018

Amyloidosis – Pipeline by Regeneron Pharmaceuticals Inc, H1 2018

Amyloidosis – Pipeline by Spectrum Pharmaceuticals Inc, H1 2018

Amyloidosis – Dormant Projects, H1 2018

Amyloidosis – Discontinued Products, H1 2018

List of Figures

List of Figures

Number of Products under Development for Amyloidosis, H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Number of Products by Top 10 Targets, H1 2018

Number of Products by Stage and Top 10 Targets, H1 2018

Number of Products by Top 10 Mechanism of Actions, H1 2018

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2018

Number of Products by Routes of Administration, H1 2018

Number of Products by Stage and Routes of Administration, H1 2018

Number of Products by Molecule Types, H1 2018

Number of Products by Stage and Molecule Types, H1 2018

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