Amyloidosis - Pipeline Review, H2 2012

Global Markets Direct, September 2012, Pages: 137

Amyloidosis – Pipeline Review, H2 2012

Summary

Global Markets Direct’s, 'Amyloidosis - Pipeline Review, H2 2012', provides an overview of the indication’s therapeutic pipeline. This report provides information on the therapeutic development for Amyloidosis, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Amyloidosis. Amyloidosis - Pipeline Review, Half Year is built using data and information sourced from Global Markets Direct’s proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct’s team.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

- A snapshot of the global therapeutic scenario for Amyloidosis.
- A review of the Amyloidosis products under development by companies and universities/research institutes based on information derived from company and industry-specific sources.
- Coverage of products based on various stages of development ranging from discovery till registration stages.
- A feature on pipeline projects on the basis of monotherapy and combined therapeutics.
- Coverage of the Amyloidosis pipeline on the basis of route of administration and molecule type.
- Key discontinued pipeline projects.
- Latest news and deals relating to the products.

Reasons to buy

- Identify and understand important and diverse types of therapeutics under development for Amyloidosis.
- Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.
- Plan mergers and acquisitions effectively by identifying players of the most promising pipeline.
- 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.

2
List of Tables 8
List of Figures 9
Introduction 10
REPORT COVERAGE 10
Amyloidosis Overview 11
Therapeutics Development 12
An Overview of Pipeline Products for Amyloidosis 12
Amyloidosis Therapeutics under Development by Companies 14
Amyloidosis Therapeutics under Investigation by Universities/Institutes 16
Late Stage Products 19
Comparative Analysis 19
Mid Clinical Stage Products 20
Comparative Analysis 20
Early Clinical Stage Products 21
Comparative Analysis 21
Discovery and Pre-Clinical Stage Products 22
Comparative Analysis 22
Amyloidosis Therapeutics – Products under Development by Companies 23
Amyloidosis Therapeutics – Products under Investigation by Universities/Institutes 24
Companies Involved in Amyloidosis Therapeutics Development 26
GlaxoSmithKline plc 26
Isis Pharmaceuticals, Inc. 27
Millennium Pharmaceuticals, Inc. 28
Elan Corporation, plc 29
Pfizer Inc. 30
Alnylam Pharmaceuticals, Inc 31
ProteoTech, Inc. 32
BIOALVO S.A. 33
Immune System Therapeutics Ltd. 34
Celtic Therapeutics Holdings L.P. 35
Amyloidosis – Therapeutics Assessment 36
Assessment by Monotherapy Products 36
Assessment by Combination Products 37
Assessment by Route of Administration 38
Assessment by Molecule Type 40
Drug Profiles 43
ixazomib citrate - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
SDGI-TT200801 - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
MDX-1097 - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
eprodisate disodium - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
systebryl - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
Lenalidomide + Dexamethasone + Cyclophosphamide - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
ALN-TTR-01 - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
ALN-TTR-02 - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
Iododoxorubicin - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
tafamidis meglumine - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
tafamidis meglumine - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
Velcade + Melphalan + Dexamethasone - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
Velcade + Dexamethasone + Melphalan + Stem Cell Transplantation - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 63
Cyclophosphamide + Lenalidomide + Dexamethasone - Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
Revlimid + Cyclophosphamide + Dexamethasone - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
Filgrastim + Melphalan - Drug Profile 68
Product Description 68
Mechanism of Action 68
R&D Progress 68
Filgrastim + Melphalan - Drug Profile 69
Product Description 69
Mechanism of Action 69
R&D Progress 69
Dexamethasone + CC-4047 - Drug Profile 70
Product Description 70
Mechanism of Action 70
R&D Progress 70
Melphalan + Lenalidomide + Dexamethasone - Drug Profile 71
Product Description 71
Mechanism of Action 71
R&D Progress 71
Bortezomib + Dexamethasone + Melphalan - Drug Profile 73
Product Description 73
Mechanism of Action 73
R&D Progress 73
Bortezomib + Cyclophosphamide + Dexamethasone - Drug Profile 75
Product Description 75
Mechanism of Action 75
R&D Progress 75
Melphalan + Filgrastim + Autologous Hematopoietic Stem Cell Transplantation - Drug Profile 77
Product Description 77
Mechanism of Action 77
R&D Progress 77
Interleukin-2 + GM-CSF - Drug Profile 79
Product Description 79
Mechanism of Action 79
R&D Progress 79
Dexamethasone + Melphalan + Thalidomide + Filgrastim - Drug Profile 80
Product Description 80
Mechanism of Action 80
R&D Progress 80
lenalidomide - Drug Profile 82
Product Description 82
Mechanism of Action 82
R&D Progress 82
Melphalan + Autologous Stem Cell Transplantation + Bortezomib + Dexamethasone - Drug Profile 83
Product Description 83
Mechanism of Action 83
R&D Progress 83
Filgrastim + Melphalan + Cyclophosphamide + Dexamethasone + Thalidomide + Peripheral Blood Stem Cell Transplantation - Drug Profile 85
Product Description 85
Mechanism of Action 85
R&D Progress 86
Interferon Alfa + Dexamethasone - Drug Profile 87
Product Description 87
Mechanism of Action 87
R&D Progress 87
Bortezomib + Dexamethasone + Melphalan - Drug Profile 89
Product Description 89
Mechanism of Action 89
R&D Progress 89
Botezomib + Melphalan + Filgrastim + Autologous Hematopoietic Stem Cell Transplantation - Drug Profile 91
Product Description 91
Mechanism of Action 91
R&D Progress 92
diflunisal - Drug Profile 93
Product Description 93
Mechanism of Action 93
R&D Progress 93
Melphalan + Thalidomide + Dexamethasone - Drug Profile 94
Product Description 94
Mechanism of Action 94
R&D Progress 94
Velcade + Adriamycin + Dexamethasone - Drug Profile 96
Product Description 96
Mechanism of Action 96
R&D Progress 96
Velcade + Cyclophosphamide + Dexamethasone - Drug Profile 98
Product Description 98
Mechanism of Action 98
R&D Progress 98
Melpharan + Dexamethasone + Bortezomib - Drug Profile 100
Product Description 100
Mechanism of Action 100
R&D Progress 100
ISIS-TTRRx - Drug Profile 101
Product Description 101
Mechanism of Action 101
R&D Progress 101
Lendexal + Cyclophosphamide + Dexamethasone - Drug Profile 102
Product Description 102
Mechanism of Action 102
R&D Progress 102
Doxycycline + Tauroursodeoxycholic Acid - Drug Profile 104
Product Description 104
Mechanism of Action 104
R&D Progress 104
Melphalan + Peripheral Blood Stem Cell Transplantation + Filgrastim - Drug Profile 105
Product Description 105
Mechanism of Action 105
R&D Progress 105
Filgrastim + Melphalan + Stem Cell Transplantation - Drug Profile 106
Product Description 106
Mechanism of Action 106
R&D Progress 106
GSK-2315698 - Drug Profile 108
Product Description 108
Mechanism of Action 108
R&D Progress 108
High Dose Chemotherapy + Autologous Hematopoietic Cell Transplantation - Drug Profile 109
Product Description 109
Mechanism of Action 109
R&D Progress 109
Bortezomib + Dexamethasone + Melphalan - Drug Profile 110
Product Description 110
Mechanism of Action 110
R&D Progress 110
Vincristine + Adriamycin + Dexamethasone + G-CSF + Melphalan + Peripheral Blood Stem Cell Transplantation - Drug Profile 111
Product Description 111
Mechanism of Action 111
R&D Progress 111
NEOD-001 - Drug Profile 113
Product Description 113
Mechanism of Action 113
R&D Progress 113
SOM-0226 - Drug Profile 114
Product Description 114
Mechanism of Action 114
R&D Progress 114
Amyloidosis Therapeutics – Drug Profile Updates 115
Amyloidosis Therapeutics – Discontinued Products 126
Amyloidosis Therapeutics - Dormant Products 127
Amyloidosis – Product Development Milestones 128
Featured News & Press Releases 128
Aug 06, 2012: Alnylam Pharma Presents New Preclinical Data From ‘Alnylam5x15’ Programs Including ALN-TTRsc For Treatment Of Transthyretin-Mediated Amyloidosis 128
Jul 16, 2012: Alnylam Reports Positive Phase I Trial Results Of ALN-TTR02 For Treatment Of TTR-Mediated Amyloidosis 128
Jul 16, 2012: Tekmira's LNP Technology Enables Alnylam's Positive ALN-TTR02 Clinical Data 130
Jun 19, 2012: Alnylam's ALN-TTR02 Receives US Orphan Drug Designation For Treatment Of Familial Amyloidotic- Polyneuropathy 131
Jun 18, 2012: FDA Issues Complete Response Letter For Pfizer’s Tafamidis Meglumine New Drug Application 131
Jun 07, 2012: Alnylam Completes Enrollment In Phase I Clinical Trial And Initiates Phase II Clinical Trial Of ALN-TTR02 For Treatment Of Transthyretin-Mediated Amyloidosis 132
May 24, 2012: FDA Panel Votes Pfizer's Tafamidis Meglumine To Treat Transthyretin Familial Amyloid Polyneuropathy 132
May 10, 2012: Alnylam Presents Final Results From Phase I Clinical Trial Of ALN-TTR01 For Treatment Of Transthyretin-Mediated Amyloidosis 133
May 10, 2012: Bellus Health Announces Update On Phase III Trial Of KIACTA In AA Amyloidosis At XIII International Symposium On Amyloidosis 134
Apr 23, 2012: ProteoTech To Present New Data On Systebryl For Treatment Of Systemic AA Amyloidosis At XIIIth International Symposium On Amyloidosis 135
Appendix 136
Methodology 136
Coverage 136
Secondary Research 136
Primary Research 136
Expert Panel Validation 136
Contact Us 137
Disclaimer 137

List of Tables
Number of Products Under Development for Amyloidosis, H2 2012 12
Products under Development for Amyloidosis – Comparative Analysis, H2 2012 13
Number of Products under Development by Companies, H2 2012 15
Number of Products under Investigation by Universities/Institutes, H2 2012 17
Number of Products under Investigation by Universities/Institutes, H2 2012 (Contd..1) 18
Comparative Analysis by Late Stage Development, H2 2012 19
Comparative Analysis by Mid Clinical Stage Development, H2 2012 20
Comparative Analysis by Early Clinical Stage Development, H2 2012 21
Comparative Analysis by Discovery and Pre-Clinical Stage Development, H2 2012 22
Products under Development by Companies, H2 2012 23
Products under Investigation by Universities/Institutes, H2 2012 24
Products under Investigation by Universities/Institutes, H2 2012 (Contd..1) 25
GlaxoSmithKline plc, H2 2012 26
Isis Pharmaceuticals, Inc., H2 2012 27
Millennium Pharmaceuticals, Inc., H2 2012 28
Elan Corporation, plc, H2 2012 29
Pfizer Inc., H2 2012 30
Alnylam Pharmaceuticals, Inc, H2 2012 31
ProteoTech, Inc., H2 2012 32
BIOALVO S.A., H2 2012 33
Immune System Therapeutics Ltd., H2 2012 34
Celtic Therapeutics Holdings L.P., H2 2012 35
Assessment by Monotherapy Products, H2 2012 36
Assessment by Combination Products, H2 2012 37
Assessment by Stage and Route of Administration, H2 2012 39
Assessment by Stage and Molecule Type, H2 2012 42
Amyloidosis Therapeutics – Drug Profile Updates 115
Amyloidosis Therapeutics – Discontinued Products 126
Amyloidosis Therapeutics – Dormant Products 127

List of Figures
Number of Products under Development for Amyloidosis, H2 2012 12
Products under Development for Amyloidosis – Comparative Analysis, H2 2012 13
Products under Development by Companies, H2 2012 14
Products under Investigation by Universities/Institutes, H2 2012 16
Late Stage Products, H2 2012 19
Mid Clinical Stage Products, H2 2012 20
Early Clinical Stage Products, H2 2012 21
Discovery and Pre-Clinical Stage Products, H2 2012 22
Assessment by Monotherapy Products, H2 2012 36
Assessment by Combination Products, H2 2012 37
Assessment by Route of Administration, H2 2012 38
Assessment by Stage and Route of Administration, H2 2012 39
Assessment by Molecule Type, H2 2012 40
Assessment by Stage and Molecule Type, H2 2012 41

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