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Amyloidosis - Pipeline Review, H1 2017

  • February 2017
  • -
  • Global Markets Direct
  • -
  • 160 pages

Amyloidosis - Pipeline Review, H1 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Amyloidosis – Pipeline Review, H1 2017, provides an overview of the Amyloidosis (Metabolic Disorders) pipeline landscape.

Abnormal accumulation of amyloid protein in the tissues results in a group of diseases known as amyloidosis. It may affect the shape and functioning of the organ eventually leading to organ failure. Predisposing factors are misfolding of abnormal protein called the light chain and its subsequent deposition in the body. It may also occur as a result of dialysis. Symptoms include fatigue, chest pain, swelling and breathing problems. Amyloidosis can be managed by transplant and, medication such as diuretics and chemotherapy drugs.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Amyloidosis – Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Amyloidosis (Metabolic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Amyloidosis (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Amyloidosis and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Filing rejected/Withdrawn, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 1, 4, 7, 7, 4 and 5 respectively. Similarly, the Universities portfolio in Phase II, Preclinical and Discovery stages comprises 1, 3 and 2 molecules, respectively.

Amyloidosis (Metabolic Disorders) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Amyloidosis (Metabolic Disorders).
- The pipeline guide reviews pipeline therapeutics for Amyloidosis (Metabolic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Amyloidosis (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Amyloidosis (Metabolic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Amyloidosis (Metabolic Disorders)

Reasons to buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Amyloidosis (Metabolic Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Amyloidosis (Metabolic Disorders) 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Table Of Contents

Amyloidosis - Pipeline Review, H1 2017
Table of Contents
Table of Contents 2
List of Tables 6
List of Figures 7
Introduction 8
Global Markets Direct Report Coverage 8
Amyloidosis - Overview 9
Amyloidosis - Therapeutics Development 10
Pipeline Overview 10
Pipeline by Companies 11
Pipeline by Universities/Institutes 14
Products under Development by Companies 15
Products under Development by Universities/Institutes 17
Amyloidosis - Therapeutics Assessment 18
Assessment by Target 18
Assessment by Mechanism of Action 20
Assessment by Route of Administration 22
Assessment by Molecule Type 24
Amyloidosis - Companies Involved in Therapeutics Development 26
Alnylam Pharmaceuticals Inc 26
Amgen Inc 26
Arcturus Therapeutics Inc 27
Bsim2 28
Celgene Corp 28
GlaxoSmithKline Plc 29
Ionis Pharmaceuticals Inc 29
Johnson and Johnson 30
Millennium Pharmaceuticals Inc 30
Neurimmune Holding AG 31
Novartis AG 31
Oncopeptides AB 32
Pfizer Inc 32
Proclara Biosciences Inc 33
Prothena Corp Plc 33
R Pharm 34
Regeneron Pharmaceuticals Inc 34
SOM Biotech SL 35
Spectrum Pharmaceuticals Inc 35
Amyloidosis - Drug Profiles 36
ALN-ANG - Drug Profile 36
Product Description 36
Mechanism Of Action 36
RandD Progress 36
ALN-TTRsc02 - Drug Profile 37
Product Description 37
Mechanism Of Action 37
RandD Progress 37
Antisense RNAi Oligonucleotides to Inhibit Amyloid Precursor Protein for Amyloidosis - Drug Profile 39
Product Description 39
Mechanism Of Action 39
RandD Progress 39
CAEL-101 - Drug Profile 40
Product Description 40
Mechanism Of Action 40
RandD Progress 40
canakinumab - Drug Profile 41
Product Description 41
Mechanism Of Action 41
RandD Progress 41
carfilzomib - Drug Profile 47
Product Description 47
Mechanism Of Action 47
RandD Progress 47
CLR-01 - Drug Profile 60
Product Description 60
Mechanism Of Action 60
RandD Progress 60
daratumumab - Drug Profile 62
Product Description 62
Mechanism Of Action 62
RandD Progress 62
dezamizumab - Drug Profile 76
Product Description 76
Mechanism Of Action 76
RandD Progress 76
dezamizumab + GSK-2315698 - Drug Profile 77
Product Description 77
Mechanism Of Action 77
RandD Progress 77
doxycycline hyclate - Drug Profile 78
Product Description 78
Mechanism Of Action 78
RandD Progress 78
EDE-1307 - Drug Profile 79
Product Description 79
Mechanism Of Action 79
RandD Progress 79
GSK-2315698 - Drug Profile 80
Product Description 80
Mechanism Of Action 80
RandD Progress 80
GSK-3039294 - Drug Profile 81
Product Description 81
Mechanism Of Action 81
RandD Progress 81
inotersen sodium - Drug Profile 82
Product Description 82
Mechanism Of Action 82
RandD Progress 82
ixazomib citrate - Drug Profile 86
Product Description 86
Mechanism Of Action 86
RandD Progress 86
LUNAR-TTR - Drug Profile 96
Product Description 96
Mechanism Of Action 96
RandD Progress 96
melphalan - Drug Profile 97
Product Description 97
Mechanism Of Action 97
RandD Progress 97
melphalan flufenamide - Drug Profile 101
Product Description 101
Mechanism Of Action 101
RandD Progress 101
Monoclonal Antibody to Inhibit ATTR for Familial Amyloid Polyneuropathy - Drug Profile 103
Product Description 103
Mechanism Of Action 103
RandD Progress 103
Monoclonal Antibody to Target CD66b for Blood Cancer and Metabolic Disorders - Drug Profile 104
Product Description 104
Mechanism Of Action 104
RandD Progress 104
NEOD-001 - Drug Profile 105
Product Description 105
Mechanism Of Action 105
RandD Progress 105
NI-301 - Drug Profile 110
Product Description 110
Mechanism Of Action 110
RandD Progress 110
NPT-289 - Drug Profile 111
Product Description 111
Mechanism Of Action 111
RandD Progress 111
Oligonucleotides to Inhibit Transthyretin for Amyloidosis - Drug Profile 112
Product Description 112
Mechanism Of Action 112
RandD Progress 112
patisiran - Drug Profile 113
Product Description 113
Mechanism Of Action 113
RandD Progress 113
pomalidomide - Drug Profile 121
Product Description 121
Mechanism Of Action 121
RandD Progress 121
PRX-004 - Drug Profile 128
Product Description 128
Mechanism Of Action 128
RandD Progress 128
RPH-104 - Drug Profile 129
Product Description 129
Mechanism Of Action 129
RandD Progress 129
Small Molecule to Inhibit Amyloid Beta Peptide for Alzheimer's Disease and Amyloidosis - Drug Profile 130
Product Description 130
Mechanism Of Action 130
RandD Progress 130
Small Molecules to Inhibit Transthyretin for Familial Amyloid Polyneuropathy - Drug Profile 131
Product Description 131
Mechanism Of Action 131
RandD Progress 131
Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Cardiomyopathy - Drug Profile 132
Product Description 132
Mechanism Of Action 132
RandD Progress 132
Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Polyneuropathy - Drug Profile 133
Product Description 133
Mechanism Of Action 133
RandD Progress 133
tafamidis meglumine - Drug Profile 134
Product Description 134
Mechanism Of Action 134
RandD Progress 134
tolcapone - Drug Profile 137
Product Description 137
Mechanism Of Action 137
RandD Progress 137
Amyloidosis - Dormant Projects 139
Amyloidosis - Discontinued Products 140
Amyloidosis - Product Development Milestones 141
Featured News and Press Releases 141
Jan 04, 2017: Fortress Biotech Forms New Subsidiary, Caelum Biosciences to Develop Novel Treatment for AL Amyloidosis 141
Dec 16, 2016: Novartis receives positive CHMP recommendation for Ilaris to treat rare diseases in adults and children called Periodic Fever Syndromes 142
Nov 07, 2016: VYNDAQEL (tafamidis) Receives Approval in Brazil by ANVISA for the Treatment of Transthyretin Familial Amyloid Polyneuropathy 142
Nov 03, 2016: Data for Prothena NEOD001 to be Presented at the 58th Annual American Society of Hematology Meeting 143
Oct 10, 2016: Data Monitoring Committee Recommends Continuation of APOLLO Phase 3 Clinical Trial of Patisiran for Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN) 144
Sep 23, 2016: Novartis receives three new FDA approvals for the expanded use of Ilaris treating rare Periodic Fever Syndrome conditions 145
Aug 08, 2016: Pfizer Announces Publication of New Analysis Showing Long-Term Therapy with VYNDAQEL (tafamidis) Slowed Progression of Rare Neurodegenerative Disease 145
Jul 07, 2016: Ionis Pharmaceuticals Reports Positive Clinical Data on IONIS-TTR Rx at the XV International Symposium on Amyloidosis 147
Jul 07, 2016: Ionis Pharmaceuticals Reports Positive Clinical Data on IONIS-TTR Rx at the XV International Symposium on Amyloidosis 148
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 150
Jul 01, 2016: Alnylam Reports New Results on patisiran at the XV International Symposium on Amyloidosis 153
Jun 27, 2016: Alnylam to Report New Patisiran Results at the XV International Symposium on Amyloidosis 155
Jun 21, 2016: Prothena to Present at 15th International Symposium on Amyloidosis Showcasing Broad Commitment to Advancing New Therapies for Systemic Amyloidoses 156
Jun 09, 2016: Alnylam Initiates Phase 1 Clinical Trial for ALN-TTRsc02, an Investigational RNAi Therapeutic for the Treatment of TTR-Mediated Amyloidosis 158
Appendix 159
Methodology 159
Coverage 159
Secondary Research 159
Primary Research 159
Expert Panel Validation 159
Contact Us 159
Disclaimer 160

List of Tables
Number of Products under Development for Amyloidosis, H1 2017 10
Number of Products under Development by Companies, H1 2017 12
Number of Products under Development by Companies, H1 2017 (Contd..1), H1 2017 13
Number of Products under Development by Universities/Institutes, H1 2017 14
Products under Development by Companies, H1 2017 15
Products under Development by Companies, H1 2017 (Contd..1), H1 2017 16
Products under Development by Universities/Institutes, H1 2017 17
Number of Products by Stage and Target, H1 2017 19
Number of Products by Stage and Mechanism of Action, H1 2017 21
Number of Products by Stage and Route of Administration, H1 2017 23
Number of Products by Stage and Molecule Type, H1 2017 25
Amyloidosis - Pipeline by Alnylam Pharmaceuticals Inc, H1 2017 26
Amyloidosis - Pipeline by Amgen Inc, H1 2017 27
Amyloidosis - Pipeline by Arcturus Therapeutics Inc, H1 2017 27
Amyloidosis - Pipeline by Bsim2, H1 2017 28
Amyloidosis - Pipeline by Celgene Corp, H1 2017 28
Amyloidosis - Pipeline by GlaxoSmithKline Plc, H1 2017 29
Amyloidosis - Pipeline by Ionis Pharmaceuticals Inc, H1 2017 29
Amyloidosis - Pipeline by Johnson and Johnson, H1 2017 30
Amyloidosis - Pipeline by Millennium Pharmaceuticals Inc, H1 2017 30
Amyloidosis - Pipeline by Neurimmune Holding AG, H1 2017 31
Amyloidosis - Pipeline by Novartis AG, H1 2017 31
Amyloidosis - Pipeline by Oncopeptides AB, H1 2017 32
Amyloidosis - Pipeline by Pfizer Inc, H1 2017 32
Amyloidosis - Pipeline by Proclara Biosciences Inc, H1 2017 33
Amyloidosis - Pipeline by Prothena Corp Plc, H1 2017 33
Amyloidosis - Pipeline by R Pharm, H1 2017 34
Amyloidosis - Pipeline by Regeneron Pharmaceuticals Inc, H1 2017 34
Amyloidosis - Pipeline by SOM Biotech SL, H1 2017 35
Amyloidosis - Pipeline by Spectrum Pharmaceuticals Inc, H1 2017 35
Amyloidosis - Dormant Projects, H1 2017 139
Amyloidosis - Discontinued Products, H1 2017 140

List of Figures
Number of Products under Development for Amyloidosis, H1 2017 10
Number of Products under Development by Companies, H1 2017 11
Number of Products under Development by Universities/Institutes, H1 2017 14
Number of Products by Top 10 Targets, H1 2017 18
Number of Products by Stage and Top 10 Targets, H1 2017 18
Number of Products by Top 10 Mechanism of Actions, H1 2017 20
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2017 20
Number of Products by Routes of Administration, H1 2017 22
Number of Products by Stage and Routes of Administration, H1 2017 22
Number of Products by Molecule Types, H1 2017 24
Number of Products by Stage and Molecule Types, H1 2017 24

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