Amyloidosis - Pipeline Review, H2 2016

Published: Jul, 2016 | Pages: 152 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Amyloidosis - Pipeline Review, H2 2016

Summary

Global Markets Direct's, 'Amyloidosis - Pipeline Review, H2 2016', 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

 Table of Contents

Table of Contents 2
List of Tables 7
List of Figures 8
Introduction 9
Global Markets Direct Report Coverage 9
Amyloidosis Overview 10
Therapeutics Development 11
Pipeline Products for Amyloidosis - Overview 11
Pipeline Products for Amyloidosis - Comparative Analysis 12
Amyloidosis - Therapeutics under Development by Companies 13
Amyloidosis - Therapeutics under Investigation by Universities/Institutes 15
Amyloidosis - Pipeline Products Glance 16
Late Stage Products 16
Clinical Stage Products 17
Early Stage Products 18
Amyloidosis - Products under Development by Companies 19
Amyloidosis - Products under Investigation by Universities/Institutes 21
Amyloidosis - Companies Involved in Therapeutics Development 22
Alnylam Pharmaceuticals, Inc. 22
Amgen Inc. 23
Arcturus Therapeutics, Inc. 24
BELLUS Health Inc. 25
Bsim2 26
Celgene Corporation 27
GlaxoSmithKline Plc 28
Ionis Pharmaceuticals, Inc. 29
Johnson & Johnson 30
Neurimmune Holding AG 31
NeuroPhage Pharmaceuticals, Inc. 32
Oncopeptides AB 33
Pfizer Inc. 34
Prothena Corporation Plc 35
Regeneron Pharmaceuticals, Inc. 36
SOM Innovation Biotech SL 37
Takeda Pharmaceutical Company Limited 38
Amyloidosis - Therapeutics Assessment 39
Assessment by Monotherapy Products 39
Assessment by Combination Products 40
Assessment by Target 41
Assessment by Mechanism of Action 43
Assessment by Route of Administration 45
Assessment by Molecule Type 47
Drug Profiles 49
ALN-ANG - Drug Profile 49
Product Description 49
Mechanism Of Action 49
R&D Progress 49
ALN-TTRsc02 - Drug Profile 50
Product Description 50
Mechanism Of Action 50
R&D Progress 50
Antisense RNAi Oligonucleotides to Inhibit Amyloid Precursor Protein for Amyloidosis - Drug Profile 51
Product Description 51
Mechanism Of Action 51
R&D Progress 51
carfilzomib - Drug Profile 52
Product Description 52
Mechanism Of Action 52
R&D Progress 52
CLR-01 - Drug Profile 63
Product Description 63
Mechanism Of Action 63
R&D Progress 63
daratumumab - Drug Profile 64
Product Description 64
Mechanism Of Action 64
R&D Progress 64
doxycycline hyclate - Drug Profile 75
Product Description 75
Mechanism Of Action 75
R&D Progress 75
EDE-1307 - Drug Profile 76
Product Description 76
Mechanism Of Action 76
R&D Progress 76
GSK-2315698 + GSK-2398852 - Drug Profile 77
Product Description 77
Mechanism Of Action 77
R&D Progress 77
GSK-3039294 - Drug Profile 78
Product Description 78
Mechanism Of Action 78
R&D Progress 78
inotersen sodium - Drug Profile 79
Product Description 79
Mechanism Of Action 79
R&D Progress 79
ixazomib citrate - Drug Profile 83
Product Description 83
Mechanism Of Action 83
R&D Progress 83
LUNAR-TTR - Drug Profile 92
Product Description 92
Mechanism Of Action 92
R&D Progress 92
Melflufen - Drug Profile 93
Product Description 93
Mechanism Of Action 93
R&D Progress 93
Monoclonal Antibody for Amyloidosis - Drug Profile 95
Product Description 95
Mechanism Of Action 95
R&D Progress 95
Monoclonal Antibody to Inhibit ATTR for Familial Amyloid Polyneuropathy - Drug Profile 96
Product Description 96
Mechanism Of Action 96
R&D Progress 96
Monoclonal Antibody to Target CD66b for Blood Cancer and Metabolic Disorders - Drug Profile 97
Product Description 97
Mechanism Of Action 97
R&D Progress 97
NEOD-001 - Drug Profile 98
Product Description 98
Mechanism Of Action 98
R&D Progress 98
NI-301 - Drug Profile 103
Product Description 103
Mechanism Of Action 103
R&D Progress 103
NPT-005 - Drug Profile 104
Product Description 104
Mechanism Of Action 104
R&D Progress 104
Oligonucleotides to Inhibit Transthyretin for Amyloidosis - Drug Profile 105
Product Description 105
Mechanism Of Action 105
R&D Progress 105
patisiran - Drug Profile 106
Product Description 106
Mechanism Of Action 106
R&D Progress 106
pomalidomide - Drug Profile 113
Product Description 113
Mechanism Of Action 113
R&D Progress 113
PRX-004 - Drug Profile 120
Product Description 120
Mechanism Of Action 120
R&D Progress 120
revusiran - Drug Profile 121
Product Description 121
Mechanism Of Action 121
R&D Progress 121
Small Molecules for AL Amyloidosis - Drug Profile 126
Product Description 126
Mechanism Of Action 126
R&D Progress 126
Small Molecules to Inhibit Transthyretin for Familial Amyloid Polyneuropathy - Drug Profile 127
Product Description 127
Mechanism Of Action 127
R&D Progress 127
Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Cardiomyopathy - Drug Profile 128
Product Description 128
Mechanism Of Action 128
R&D Progress 128
Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Polyneuropathy - Drug Profile 129
Product Description 129
Mechanism Of Action 129
R&D Progress 129
tafamidis meglumine - Drug Profile 130
Product Description 130
Mechanism Of Action 130
R&D Progress 130
tolcapone - Drug Profile 133
Product Description 133
Mechanism Of Action 133
R&D Progress 133
Amyloidosis - Dormant Projects 135
Amyloidosis - Product Development Milestones 136
Featured News & Press Releases 136
Jul 07, 2016: Ionis Pharmaceuticals Reports Positive Clinical Data on IONIS-TTR Rx at the XV International Symposium on Amyloidosis 136
Jul 05, 2016: Prothena Presents New Data from Phase 1/2 Study of NEOD001 Demonstrating Improvements in Three Organ Systems in Previously-Treated Patients with AL Amyloidosis 137
Jul 01, 2016: Alnylam Reports New Results on patisiran at the XV International Symposium on Amyloidosis 140
Jul 01, 2016: Alnylam To Present New Results on Revusiran at the XV International Symposium on Amyloidosis 142
Jun 27, 2016: Alnylam to Report New Patisiran Results at the XV International Symposium on Amyloidosis 144
Jun 27, 2016: Alnylam to Present New Revusiran Results at the XV International Symposium on Amyloidosis 144
Jun 21, 2016: Prothena to Present at 15th International Symposium on Amyloidosis Showcasing Broad Commitment to Advancing New Therapies for Systemic Amyloidoses 145
Jun 09, 2016: Alnylam Initiates Phase 1 Clinical Trial for ALN-TTRsc02, an Investigational RNAi Therapeutic for the Treatment of TTR-Mediated Amyloidosis 147
May 26, 2016: Ionis Pharmaceuticals Provides Update on IONIS-TTR Rx Program 148
May 23, 2016: Prothena to Present Wide Range of Studies on Amyloidosis, Including New Clinical Data on NEOD001, at International Symposium on Amyloidosis 148
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) 149
Apr 07, 2016: Ionis Pharmaceuticals Provides Update on IONIS-TTRRX Program 150
Appendix 151
Methodology 151
Coverage 151
Secondary Research 151
Primary Research 151
Expert Panel Validation 151
Contact Us 151
Disclaimer 152

List of Tables

Number of Products under Development for Amyloidosis, H2 2016 11
Number of Products under Development for Amyloidosis - Comparative Analysis, H2 2016 12
Number of Products under Development by Companies, H2 2016 14
Number of Products under Investigation by Universities/Institutes, H2 2016 15
Comparative Analysis by Late Stage Development, H2 2016 16
Comparative Analysis by Clinical Stage Development, H2 2016 17
Comparative Analysis by Early Stage Development, H2 2016 18
Products under Development by Companies, H2 2016 19
Products under Development by Companies, H2 2016 (Contd..1) 20
Products under Investigation by Universities/Institutes, H2 2016 21
Amyloidosis - Pipeline by Alnylam Pharmaceuticals, Inc., H2 2016 22
Amyloidosis - Pipeline by Amgen Inc., H2 2016 23
Amyloidosis - Pipeline by Arcturus Therapeutics, Inc., H2 2016 24
Amyloidosis - Pipeline by BELLUS Health Inc., H2 2016 25
Amyloidosis - Pipeline by Bsim2, H2 2016 26
Amyloidosis - Pipeline by Celgene Corporation, H2 2016 27
Amyloidosis - Pipeline by GlaxoSmithKline Plc, H2 2016 28
Amyloidosis - Pipeline by Ionis Pharmaceuticals, Inc., H2 2016 29
Amyloidosis - Pipeline by Johnson & Johnson, H2 2016 30
Amyloidosis - Pipeline by Neurimmune Holding AG, H2 2016 31
Amyloidosis - Pipeline by NeuroPhage Pharmaceuticals, Inc., H2 2016 32
Amyloidosis - Pipeline by Oncopeptides AB, H2 2016 33
Amyloidosis - Pipeline by Pfizer Inc., H2 2016 34
Amyloidosis - Pipeline by Prothena Corporation Plc, H2 2016 35
Amyloidosis - Pipeline by Regeneron Pharmaceuticals, Inc., H2 2016 36
Amyloidosis - Pipeline by SOM Innovation Biotech SL, H2 2016 37
Amyloidosis - Pipeline by Takeda Pharmaceutical Company Limited, H2 2016 38
Assessment by Monotherapy Products, H2 2016 39
Assessment by Combination Products, H2 2016 40
Number of Products by Stage and Target, H2 2016 42
Number of Products by Stage and Mechanism of Action, H2 2016 44
Number of Products by Stage and Route of Administration, H2 2016 46
Number of Products by Stage and Molecule Type, H2 2016 48
Amyloidosis - Dormant Projects, H2 2016 135