Hemophilia A - Pipeline Review, H1 2016

Published: May, 2016 | Pages: 159 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Hemophilia A - Pipeline Review, H1 2016

Summary

Global Markets Direct's, 'Hemophilia A - Pipeline Review, H1 2016', provides an overview of the Hemophilia A pipeline landscape. 

The report provides comprehensive information on the therapeutics under development for Hemophilia A, 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 Hemophilia A 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 Hemophilia A
- The report reviews pipeline therapeutics for Hemophilia A 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 Hemophilia A therapeutics and enlists all their major and minor projects
- The report assesses Hemophilia A 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 Hemophilia A

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 Hemophilia A
- 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 Hemophilia A 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 8
List of Figures 10
Introduction 11
Global Markets Direct Report Coverage 11
Hemophilia A Overview 12
Therapeutics Development 13
Pipeline Products for Hemophilia A - Overview 13
Pipeline Products for Hemophilia A - Comparative Analysis 14
Hemophilia A - Therapeutics under Development by Companies 15
Hemophilia A - Therapeutics under Investigation by Universities/Institutes 17
Hemophilia A - Pipeline Products Glance 18
Late Stage Products 18
Clinical Stage Products 19
Early Stage Products 20
Unknown Stage Products 21
Hemophilia A - Products under Development by Companies 22
Hemophilia A - Products under Investigation by Universities/Institutes 25
Hemophilia A - Companies Involved in Therapeutics Development 26
Alnylam Pharmaceuticals, Inc. 26
Amarna Therapeutics B.V. 27
Apitope International NV 28
Baxalta Incorporated 29
Bayer AG 30
Biogen, Inc. 31
BioMarin Pharmaceutical Inc. 32
Catalyst Biosciences, Inc. 33
CSL Limited 34
DBV Technologies S.A. 35
Dimension Therapeutics, Inc. 36
Emergent BioSolutions Inc. 37
EpiVax, Inc. 38
Expression Therapeutics LLC 39
F. Hoffmann-La Roche Ltd. 40
Green Cross Corporation 41
Idogen AB 42
Lentigen Technology, Inc. 43
mAbxience S.A. 44
Novo Nordisk A/S 45
Octapharma AG 46
OPKO Health, Inc. 47
Pfizer Inc. 48
Pharming Group N.V. 49
rEVO Biologics, Inc. 50
Sangamo BioSciences, Inc. 51
SK Chemicals Co., Ltd. 52
Spark Therapeutics, Inc. 53
The International Biotechnology Center (IBC) Generium 54
UniQure N.V. 55
Hemophilia A - Therapeutics Assessment 56
Assessment by Monotherapy Products 56
Assessment by Target 57
Assessment by Mechanism of Action 59
Assessment by Route of Administration 61
Assessment by Molecule Type 63
Drug Profiles 65
antihemophilic factor - Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
antihemophilic factor (human) - Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
antihemophilic factor (recombinant) - Drug Profile 69
Product Description 69
Mechanism of Action 69
R&D Progress 69
antihemophilic factor (recombinant) - Drug Profile 70
Product Description 70
Mechanism of Action 70
R&D Progress 70
antihemophilic factor (recombinant) - Drug Profile 72
Product Description 72
Mechanism of Action 72
R&D Progress 72
antihemophilic factor (recombinant) - Drug Profile 73
Product Description 73
Mechanism of Action 73
R&D Progress 73
antihemophilic factor (recombinant) - Drug Profile 74
Product Description 74
Mechanism of Action 74
R&D Progress 74
antihemophilic factor (recombinant) biosimilar - Drug Profile 75
Product Description 75
Mechanism of Action 75
R&D Progress 75
ATXF-8117 - Drug Profile 76
Product Description 76
Mechanism of Action 76
R&D Progress 76
BAX-826 - Drug Profile 77
Product Description 77
Mechanism of Action 77
R&D Progress 77
BAX-888 - Drug Profile 78
Product Description 78
Mechanism of Action 78
R&D Progress 78
BAY-1093884 - Drug Profile 79
Product Description 79
Mechanism of Action 79
R&D Progress 79
BMN-270 - Drug Profile 80
Product Description 80
Mechanism of Action 80
R&D Progress 80
CB-813d - Drug Profile 81
Product Description 81
Mechanism of Action 81
R&D Progress 81
Cell Therapy to Target Coagulation Factor VIII for Hemophilia A - Drug Profile 83
Product Description 83
Mechanism of Action 83
R&D Progress 83
coagulation factor VIIa (recombinant) - Drug Profile 84
Product Description 84
Mechanism of Action 84
R&D Progress 84
coagulation factor VIII (human) - Drug Profile 86
Product Description 86
Mechanism of Action 86
R&D Progress 86
concizumab - Drug Profile 87
Product Description 87
Mechanism of Action 87
R&D Progress 87
CSL-689 - Drug Profile 88
Product Description 88
Mechanism of Action 88
R&D Progress 88
damoctocog alfa pegol - Drug Profile 90
Product Description 90
Mechanism of Action 90
R&D Progress 90
Deimmunized FVIII - Drug Profile 91
Product Description 91
Mechanism of Action 91
R&D Progress 91
DTX-201 - Drug Profile 93
Product Description 93
Mechanism of Action 93
R&D Progress 93
emicizumab - Drug Profile 94
Product Description 94
Mechanism of Action 94
R&D Progress 94
ET-3 - Drug Profile 95
Product Description 95
Mechanism of Action 95
R&D Progress 95
factor VIII (recombinant) biosimilar - Drug Profile 96
Product Description 96
Mechanism of Action 96
R&D Progress 96
fitusiran - Drug Profile 97
Product Description 97
Mechanism of Action 97
R&D Progress 97
Gene Therapy for Bleeding and Clotting Disorders - Drug Profile 98
Product Description 98
Mechanism of Action 98
R&D Progress 98
Gene Therapy for Hemophilia A - Drug Profile 99
Product Description 99
Mechanism of Action 99
R&D Progress 99
Gene Therapy to Activate Factor VIII for Hemophilia A - Drug Profile 100
Product Description 100
Mechanism of Action 100
R&D Progress 100
IN-3012 - Drug Profile 101
Product Description 101
Mechanism of Action 101
R&D Progress 101
Kovaltry - Drug Profile 102
Product Description 102
Mechanism of Action 102
R&D Progress 102
LG-889 - Drug Profile 104
Product Description 104
Mechanism of Action 104
R&D Progress 104
LR-769 - Drug Profile 105
Product Description 105
Mechanism of Action 105
R&D Progress 105
MOD-5014 - Drug Profile 107
Product Description 107
Mechanism of Action 107
R&D Progress 107
PF-06741086 - Drug Profile 108
Product Description 108
Mechanism of Action 108
R&D Progress 108
Recombinant Factor VIII Replacement for Hemophilia A - Drug Profile 109
Product Description 109
Mechanism of Action 109
R&D Progress 109
Recombinant Factor VIII Replacement for Hemophilia A - Drug Profile 110
Product Description 110
Mechanism of Action 110
R&D Progress 110
Recombinant Protein for Hemophilia A - Drug Profile 111
Product Description 111
Mechanism of Action 111
R&D Progress 111
Recombinant Proteins for Hemophilia A - Drug Profile 112
Product Description 112
Mechanism of Action 112
R&D Progress 112
SB-FVIII - Drug Profile 113
Product Description 113
Mechanism of Action 113
R&D Progress 113
SPK-FVIII - Drug Profile 114
Product Description 114
Mechanism of Action 114
R&D Progress 114
SVF-VIIa - Drug Profile 115
Product Description 115
Mechanism of Action 115
R&D Progress 115
turoctocog alfa pegol - Drug Profile 116
Product Description 116
Mechanism of Action 116
R&D Progress 116
Hemophilia A - Recent Pipeline Updates 118
Hemophilia A - Dormant Projects 147
Hemophilia A - Discontinued Products 149
Hemophilia A - Product Development Milestones 150
Featured News & Press Releases 150
May 07, 2016: Dimension Therapeutics Announces Preclinical Data From Bayer-Partnered Hemophilia A Program At American Society For Gene And Cell Therapy (ASGCT) Annual Meeting 150
Apr 18, 2016: Dimension Therapeutics announces preclinical data on hemophilia A program at upcoming American Society For Gene And Cell Therapy (ASGCT) annual meeting 150
Apr 04, 2016: Baxalta Expands Global Reach of ADYNOVATE for Hemophilia A Patients with Approval in Japan 152
Mar 17, 2016: Bayer Receives FDA Approval for Kovaltry for the Treatment of Children and Adults with Hemophilia A 152
Mar 04, 2016: Baxalta Commences Phase 1 Clinical Trial of BAX 826, the Company's Second Extended Half-Life Factor VIII Treatment for Hemophilia A 153
Mar 02, 2016: Baxalta Submits Marketing Authorization Application to the European Medicines Agency for ADYNOVI for Treatment of Hemophilia A Patients 154
Feb 25, 2016: Baxalta Announces Submission of Supplemental BLAs to Expand Use of ADYNOVATE to Pediatric Patients and Surgical Settings 154
Feb 23, 2016: OPKO Health Announces Dosing of First Patient in Phase 2a Clinical Study of a Long-Acting Factor VIIa for the Treatment of Hemophilia 155
Feb 22, 2016: Bayer Receives EU Approval of Kovaltry for Treatment of Hemophilia A 156
Feb 03, 2016: Health Canada Approves New Treatment Option For Canadian Adults and Children Living with Hemophilia A 156
Appendix 158
Methodology 158
Coverage 158
Secondary Research 158
Primary Research 158
Expert Panel Validation 158
Contact Us 158
Disclaimer 159

List of Tables

Number of Products under Development for Hemophilia A, H1 2016 13
Number of Products under Development for Hemophilia A - Comparative Analysis, H1 2016 14
Number of Products under Development by Companies, H1 2016 15
Number of Products under Development by Companies, H1 2016 (Contd..1) 16
Number of Products under Investigation by Universities/Institutes, H1 2016 17
Comparative Analysis by Late Stage Development, H1 2016 18
Comparative Analysis by Clinical Stage Development, H1 2016 19
Comparative Analysis by Early Stage Development, H1 2016 20
Comparative Analysis by Unknown Stage Development, H1 2016 21
Products under Development by Companies, H1 2016 22
Products under Development by Companies, H1 2016 (Contd..1) 23
Products under Development by Companies, H1 2016 (Contd..2) 24
Products under Investigation by Universities/Institutes, H1 2016 25
Hemophilia A - Pipeline by Alnylam Pharmaceuticals, Inc., H1 2016 26
Hemophilia A - Pipeline by Amarna Therapeutics B.V., H1 2016 27
Hemophilia A - Pipeline by Apitope International NV, H1 2016 28
Hemophilia A - Pipeline by Baxalta Incorporated, H1 2016 29
Hemophilia A - Pipeline by Bayer AG, H1 2016 30
Hemophilia A - Pipeline by Biogen, Inc., H1 2016 31
Hemophilia A - Pipeline by BioMarin Pharmaceutical Inc., H1 2016 32
Hemophilia A - Pipeline by Catalyst Biosciences, Inc., H1 2016 33
Hemophilia A - Pipeline by CSL Limited, H1 2016 34
Hemophilia A - Pipeline by DBV Technologies S.A., H1 2016 35
Hemophilia A - Pipeline by Dimension Therapeutics, Inc., H1 2016 36
Hemophilia A - Pipeline by Emergent BioSolutions Inc., H1 2016 37
Hemophilia A - Pipeline by EpiVax, Inc., H1 2016 38
Hemophilia A - Pipeline by Expression Therapeutics LLC, H1 2016 39
Hemophilia A - Pipeline by F. Hoffmann-La Roche Ltd., H1 2016 40
Hemophilia A - Pipeline by Green Cross Corporation, H1 2016 41
Hemophilia A - Pipeline by Idogen AB, H1 2016 42
Hemophilia A - Pipeline by Lentigen Technology, Inc., H1 2016 43
Hemophilia A - Pipeline by mAbxience S.A., H1 2016 44
Hemophilia A - Pipeline by Novo Nordisk A/S, H1 2016 45
Hemophilia A - Pipeline by Octapharma AG, H1 2016 46
Hemophilia A - Pipeline by OPKO Health, Inc., H1 2016 47
Hemophilia A - Pipeline by Pfizer Inc., H1 2016 48
Hemophilia A - Pipeline by Pharming Group N.V., H1 2016 49
Hemophilia A - Pipeline by rEVO Biologics, Inc., H1 2016 50
Hemophilia A - Pipeline by Sangamo BioSciences, Inc., H1 2016 51
Hemophilia A - Pipeline by SK Chemicals Co., Ltd., H1 2016 52
Hemophilia A - Pipeline by Spark Therapeutics, Inc., H1 2016 53
Hemophilia A - Pipeline by The International Biotechnology Center (IBC) Generium, H1 2016 54
Hemophilia A - Pipeline by UniQure N.V., H1 2016 55
Assessment by Monotherapy Products, H1 2016 56
Number of Products by Stage and Target, H1 2016 58
Number of Products by Stage and Mechanism of Action, H1 2016 60
Number of Products by Stage and Route of Administration, H1 2016 62
Number of Products by Stage and Molecule Type, H1 2016 64
Hemophilia A Therapeutics - Recent Pipeline Updates, H1 2016 118
Hemophilia A - Dormant Projects, H1 2016 147
Hemophilia A - Dormant Projects (Contd..1), H1 2016 148
Hemophilia A - Discontinued Products, H1 2016 149