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Hemophilia - Pipeline Review, H2 2013

  • August 2013
  • -
  • Global Markets Direct
  • -
  • 220 pages

Hemophilia - Pipeline Review, H2 2013

Summary

Global Markets Direct’s, 'Hemophilia - Pipeline Review, H2 2013', provides an overview of the indication’s therapeutic pipeline. This report provides information on the therapeutic development for Hemophilia, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Hemophilia. Hemophilia - 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 Hemophilia.
- A review of the Hemophilia 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 Hemophilia 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 Hemophilia.
- 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 Hemophilia 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

Hemophilia - Pipeline Review, H2 2013
Table of Contents

Table of Contents 2
List of Tables 6
List of Figures 7
Introduction 8
Global Markets Direct Report Coverage 8
Hemophilia Overview 9
Therapeutics Development 10
An Overview of Pipeline Products for Hemophilia 10
Hemophilia Therapeutics under Development by Companies 12
Hemophilia Therapeutics under Investigation by Universities/Institutes 16
Late Stage Products 17
Comparative Analysis 17
Mid Clinical Stage Products 18
Comparative Analysis 18
Early Clinical Stage Products 19
Comparative Analysis 19
Discovery and Pre-Clinical Stage Products 20
Comparative Analysis 20
Hemophilia Therapeutics - Products under Development by Companies 21
Hemophilia Therapeutics - Products under Investigation by Universities/Institutes 26
Companies Involved in Hemophilia Therapeutics Development 27
Baxter International Inc. 27
Biogen Idec Inc. 28
AstraZeneca PLC 29
Nektar Therapeutics 30
Cangene Corporation 31
Lentigen Corporation 32
Sangamo BioSciences, Inc. 33
Pharming Group N.V. 34
Novo Nordisk A/S 35
BioMarin Pharmaceutical Inc. 36
Chugai Pharmaceutical Co. Ltd 37
Dong-A Pharmaceutical Co., Ltd. 38
GTC Biotherapeutics, Inc. 39
Octapharma AG 40
Pain Therapeutics, Inc. 41
Pfizer Inc. 42
Bayer AG 43
Living Cell Technologies Limited 44
PROLOR Biotech, Inc. 45
ReGenX Biosciences, LLC 46
Alnylam Pharmaceuticals, Inc 47
CSL Limited 48
Green Cross Holdings Corporation 49
Catalyst Biosciences, Inc. 50
NanoCarrier Co., Ltd. 51
Recoly N.V. 52
Amarna Therapeutics B.V. 53
EpiVax, Inc. 54
Expression Therapeutics LLC 55
Asklepios BioPharmaceutical, Inc. 56
REGiMMUNE Corporation 57
China Biologic Products, Inc. 58
Amunix, Inc. 59
LFB Biotechnologies, S.A.S.U. 60
Ascendis Pharma A/S 61
PolyTherics Limited 62
Celtic Pharmaceutical Holdings L.P. 63
Hemophilia - Therapeutics Assessment 64
Assessment by Monotherapy Products 64
Assessment by Route of Administration 65
Assessment by Molecule Type 67
Drug Profiles 69
turoctocog alfa - Drug Profile 69
BIIB-031 - Drug Profile 71
Factor IX Gene Therapy - Drug Profile 73
OBI-1 - Drug Profile 74
beroctocog alfa - Drug Profile 76
beroctocog alfa - Drug Profile 78
CSL-689 - Drug Profile 80
Recombinant Coagulation Factors - Drug Profile 82
BAX-855 - Drug Profile 83
Factor IX - Drug Profile 84
ZFP Transcription Factors For Hemophilia - Drug Profile 85
hcl-rFVIII - Drug Profile 86
LG-889 - Drug Profile 88
BAX-826 - Drug Profile 89
BIIB-029 - Drug Profile 90
BIIB-029 - Drug Profile 91
oprelvekin - Drug Profile 92
BAY-818973 - Drug Profile 93
trenonacog alfa - Drug Profile 95
trenonacog alfa - Drug Profile 97
PF-05280602 - Drug Profile 99
Hemophilia A Gene Therapy - Drug Profile 100
CSL-654 - Drug Profile 101
NN-7088 - Drug Profile 103
AAV2-hFIX - Drug Profile 104
NN-7999 - Drug Profile 105
BAX 326 - Drug Profile 106
NN-7415 - Drug Profile 108
Drug For Hemophilia B - Drug Profile 109
BAY-949027 - Drug Profile 110
BAX-513 - Drug Profile 111
IB1-007 - Drug Profile 112
IB1-008 - Drug Profile 113
DA-3808 - Drug Profile 114
MOD-9017 - Drug Profile 115
HM-12260B - Drug Profile 116
Recombinant Factor VIIa - Drug Profile 117
PEG-rFVIII-PI Complex - Drug Profile 119
Longer Acting Therapeutic Clotting Proteins - Drug Profile 120
ALN-APC Program - Drug Profile 121
MOD-5017 - Drug Profile 122
CSL-627 - Drug Profile 123
Human Coagulation Factor-VIII - Drug Profile 124
Deimmunized FVIII - Drug Profile 125
CSL-650 - Drug Profile 126
Gene Therapy For Hemophilia A - Drug Profile 127
Factor VIII Program - Drug Profile 128
Factor VIIa Program - Drug Profile 129
Factor IX Program - Drug Profile 130
Long Acting Blood Coagulation Factor IX - Drug Profile 131
Long Acting Coagulation Factor VIIa - Drug Profile 132
ALN-AT3 - Drug Profile 134
rhFactor VIII - Drug Profile 135
Human Prothrombin Complex Concentrate - Drug Profile 136
Gene Therapy for Hemophilia A - Drug Profile 137
beroctocog alfa - Drug Profile 138
beroctocog alfa - Drug Profile 139
Fac8Cell - Drug Profile 140
ACE-910 - Drug Profile 141
BNP-FVIII - Drug Profile 142
BAX-817 - Drug Profile 143
Protein Micelle - Drug Profile 145
RGI-5000 - Drug Profile 146
NBP-604 - Drug Profile 147
NBP-611 - Drug Profile 148
NBP-603 - Drug Profile 149
Factor VIII Gene Therapy Program - Drug Profile 150
TheraPEG-FVIII - Drug Profile 151
Recombinant Factor IX - Drug Profile 152
Recombinant Factor Xa - Drug Profile 153
MT-026 - Drug Profile 154
MT-033 - Drug Profile 155
eptacog alfa biosimilar - Drug Profile 156
moroctocog alfa Biosimilar - Drug Profile 157
TheraPEG Factor IX - Drug Profile 158
TheraPEG Factor VIIa - Drug Profile 159
Peptide Mimetic - Drug Profile 160
rFVIII - Drug Profile 161
MT-039 - Drug Profile 162
Lentiviral Based Gene Therapy - Drug Profile 163
AZ-10047130 - Drug Profile 164
AZ-10776241 - Drug Profile 165
Hemophilia Therapeutics - Drug Profile Updates 166
Hemophilia Therapeutics - Discontinued Products 201
Hemophilia Therapeutics - Dormant Products 202
Hemophilia - Product Development Milestones 205
Featured News and Press Releases 205
Appendix 213
Methodology 213
Coverage 213
Secondary Research 213
Primary Research 213
Expert Panel Validation 213
Contact Us 214
Disclaimer 214



List of Tables

Number of Products Under Development for Hemophilia, H2 2013 16
Products under Development for Hemophilia - Comparative Analysis, H2 2013 17
Number of Products under Development by Companies, H2 2013 19
Number of Products under Development by Companies, H2 2013 (Contd..1) 20
Number of Products under Development by Companies, H2 2013 (Contd..2) 21
Number of Products under Investigation by Universities/Institutes, H2 2013 22
Comparative Analysis by Late Stage Development, H2 2013 23
Comparative Analysis by Mid Clinical Stage Development, H2 2013 24
Comparative Analysis by Early Clinical Stage Development, H2 2013 25
Comparative Analysis by Discovery and Pre-Clinical Stage Development, H2 2013 26
Products under Development by Companies, H2 2013 27
Products under Development by Companies, H2 2013 (Contd..1) 28
Products under Development by Companies, H2 2013 (Contd..2) 29
Products under Development by Companies, H2 2013 (Contd..3) 30
Products under Development by Companies, H2 2013 (Contd..4) 31
Products under Investigation by Universities/Institutes, H2 2013 32
Baxter International Inc., H2 2013 33
Biogen Idec Inc., H2 2013 34
AstraZeneca PLC, H2 2013 35
Nektar Therapeutics, H2 2013 36
Cangene Corporation, H2 2013 37
Lentigen Corporation, H2 2013 38
Sangamo BioSciences, Inc., H2 2013 39
Pharming Group N.V., H2 2013 40
Novo Nordisk A/S, H2 2013 41
BioMarin Pharmaceutical Inc., H2 2013 42
Chugai Pharmaceutical Co. Ltd, H2 2013 43
Dong-A Pharmaceutical Co., Ltd., H2 2013 44
GTC Biotherapeutics, Inc., H2 2013 45
Octapharma AG, H2 2013 46
Pain Therapeutics, Inc., H2 2013 47
Pfizer Inc., H2 2013 48
Bayer AG, H2 2013 49
Living Cell Technologies Limited, H2 2013 50
PROLOR Biotech, Inc., H2 2013 51
ReGenX Biosciences, LLC, H2 2013 52
Alnylam Pharmaceuticals, Inc, H2 2013 53
CSL Limited, H2 2013 54
Green Cross Holdings Corporation, H2 2013 55
Catalyst Biosciences, Inc., H2 2013 56
NanoCarrier Co., Ltd., H2 2013 57
Amarna Therapeutics B.V., H2 2013 59
EpiVax, Inc., H2 2013 60
Expression Therapeutics LLC, H2 2013 61
Asklepios BioPharmaceutical, Inc., H2 2013 62
REGiMMUNE Corporation, H2 2013 63
China Biologic Products, Inc., H2 2013 64
Amunix, Inc., H2 2013 65
LFB Biotechnologies, S.A.S.U., H2 2013 66
Ascendis Pharma A/S, H2 2013 67
PolyTherics Limited, H2 2013 68
Celtic Pharmaceutical Holdings L.P., H2 2013 69
Assessment by Monotherapy Products, H2 2013 70
Assessment by Stage and Route of Administration, H2 2013 72
Assessment by Stage and Molecule Type, H2 2013 74
Hemophilia Therapeutics - Drug Profile Updates 172
Hemophilia Therapeutics - Discontinued Products 207
Hemophilia Therapeutics - Dormant Products 208
Hemophilia Therapeutics - Dormant Products (Contd..1) 209
Hemophilia Therapeutics - Dormant Products (Contd..2) 210



List of Figures

Number of Products under Development for Hemophilia, H2 2013 16
Products under Development for Hemophilia - Comparative Analysis, H2 2013 17
Products under Development by Companies, H2 2013 18
Products under Investigation by Universities/Institutes, H2 2013 22
Late Stage Products, H2 2013 23
Mid Clinical Stage Products, H2 2013 24
Early Clinical Stage Products, H2 2013 25
Discovery and Pre-Clinical Stage Products, H2 2013 26
Assessment by Monotherapy Products, H2 2013 70
Assessment by Route of Administration, H2 2013 71
Assessment by Stage and Route of Administration, H2 2013 72
Assessment by Molecule Type, H2 2013 73
Assessment by Stage and Molecule Type, H2 2013 74



Companies Mentioned

Baxter International Inc.
Biogen Idec Inc.
AstraZeneca PLC
Nektar Therapeutics
Cangene Corporation
Lentigen Corporation
Sangamo BioSciences, Inc.
Pharming Group N.V.
Novo Nordisk A/S
BioMarin Pharmaceutical Inc.
Chugai Pharmaceutical Co. Ltd
Dong-A Pharmaceutical Co., Ltd.
GTC Biotherapeutics, Inc.
Octapharma AG
Pain Therapeutics, Inc.
Pfizer Inc.
Bayer AG
Living Cell Technologies Limited
PROLOR Biotech, Inc.
ReGenX Biosciences, LLC
Alnylam Pharmaceuticals, Inc
CSL Limited
Green Cross Holdings Corporation
Catalyst Biosciences, Inc.
NanoCarrier Co., Ltd.
Recoly N.V.
Amarna Therapeutics B.V.
EpiVax, Inc.
Expression Therapeutics LLC
Asklepios BioPharmaceutical, Inc.
REGiMMUNE Corporation
China Biologic Products, Inc.
Amunix, Inc.
LFB Biotechnologies, S.A.S.U.
Ascendis Pharma A/S
PolyTherics Limited
Celtic Pharmaceutical Holdings L.P.

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