Hemophilia A – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Hemophilia A – Pipeline Review, H2 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

Companies mentioned

Alnylam Pharmaceuticals Inc

Amarna Therapeutics BV

Apitope International NV

Bayer AG

Biogen Inc

BioMarin Pharmaceutical Inc

Catalyst Biosciences Inc

Chugai Pharmaceutical Co Ltd

CSL Ltd

DBV Technologies SA

Dimension Therapeutics Inc

Emergent BioSolutions Inc

EpiVax Inc

Expression Therapeutics LLC

Green Cross Corp

Idogen AB

Lentigen Technology Inc

mAbxience SA

Novo Nordisk A/S

Octapharma AG

OPKO Biologics Ltd

Pfizer Inc

Pharming Group NV

rEVO Biologics Inc

Sangamo BioSciences Inc

Shire Plc

SK Chemicals Co Ltd

Spark Therapeutics Inc

UniQure NV

XL-protein GmbH

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 5

List of Figures 6

Introduction 7

Global Markets Direct Report Coverage 7

Hemophilia A Overview 8

Therapeutics Development 9

Pipeline Products for Hemophilia A - Overview 9

Pipeline Products for Hemophilia A - Comparative Analysis 10

Hemophilia A - Therapeutics under Development by Companies 11

Hemophilia A - Therapeutics under Investigation by Universities/Institutes 13

Hemophilia A - Pipeline Products Glance 14

Late Stage Products 14

Clinical Stage Products 15

Early Stage Products 16

Unknown Stage Products 17

Hemophilia A - Products under Development by Companies 18

Hemophilia A - Products under Investigation by Universities/Institutes 21

Hemophilia A - Companies Involved in Therapeutics Development 22

Alnylam Pharmaceuticals Inc 22

Amarna Therapeutics BV 23

Apitope International NV 24

Bayer AG 25

Biogen Inc 26

BioMarin Pharmaceutical Inc 27

Catalyst Biosciences Inc 28

Chugai Pharmaceutical Co Ltd 29

CSL Ltd 30

DBV Technologies SA 31

Dimension Therapeutics Inc 32

Emergent BioSolutions Inc 33

EpiVax Inc 34

Expression Therapeutics LLC 35

Green Cross Corp 36

Idogen AB 37

Lentigen Technology Inc 38

mAbxience SA 39

Novo Nordisk A/S 40

Octapharma AG 41

OPKO Biologics Ltd 42

Pfizer Inc 43

Pharming Group NV 44

rEVO Biologics Inc 45

Sangamo BioSciences Inc 46

Shire Plc 47

SK Chemicals Co Ltd 48

Spark Therapeutics Inc 49

UniQure NV 50

XL-protein GmbH 51

Hemophilia A - Therapeutics Assessment 52

Assessment by Monotherapy Products 52

Assessment by Target 53

Assessment by Mechanism of Action 55

Assessment by Route of Administration 57

Assessment by Molecule Type 59

Drug Profiles 61

antihemophilic factor - Drug Profile 61

antihemophilic factor (human) - Drug Profile 65

antihemophilic factor (recombinant) - Drug Profile 69

antihemophilic factor (recombinant) - Drug Profile 70

antihemophilic factor (recombinant) - Drug Profile 71

antihemophilic factor (recombinant) - Drug Profile 72

antihemophilic factor (recombinant) - Drug Profile 73

antihemophilic factor (recombinant) - Drug Profile 74

antihemophilic factor (recombinant) biosimilar - Drug Profile 75

antihemophilic factor (recombinant) biosimilar - Drug Profile 76

antihemophilic factor (recombinant), pegylated - Drug Profile 77

antihemophilic factor (recombinant), single chain - Drug Profile 79

ATXF-8117 - Drug Profile 82

BAX-888 - Drug Profile 83

BAY-1093884 - Drug Profile 84

BMN-270 - Drug Profile 85

Cell Therapy to Target Coagulation Factor VIII for Hemophilia A - Drug Profile 87

coagulation factor VIIa (recombinant) - Drug Profile 88

coagulation factor VIII (human) - Drug Profile 90

concizumab - Drug Profile 91

CSL-689 - Drug Profile 92

damoctocog alfa pegol - Drug Profile 94

Deimmunized FVIII - Drug Profile 96

DTX-201 - Drug Profile 97

emicizumab - Drug Profile 98

ET-3 - Drug Profile 101

fitusiran - Drug Profile 102

Gene Therapy for Hematological Disorders - Drug Profile 108

Gene Therapy for Hemophilia A - Drug Profile 109

Gene Therapy to Activate Factor VIII for Hemophilia A - Drug Profile 110

IN-3012 - Drug Profile 111

Kovaltry - Drug Profile 112

LG-889 - Drug Profile 114

LR-769 - Drug Profile 115

marzeptacog alfa - Drug Profile 117

MG-1113A - Drug Profile 119

MOD-5014 - Drug Profile 120

PBB-8-IN - Drug Profile 123

PF-06741086 - Drug Profile 124

Recombinant Factor VIII Replacement for Hemophilia A - Drug Profile 125

Recombinant Factor VIII Replacement for Hemophilia A - Drug Profile 127

Recombinant Protein for Hemophilia A - Drug Profile 128

Recombinant Proteins for Hemophilia A - Drug Profile 129

SB-525 - Drug Profile 130

SB-FVIII - Drug Profile 131

SHP-656 - Drug Profile 133

SPK-8011 - Drug Profile 135

SVF-VIIa - Drug Profile 136

Hemophilia A - Dormant Projects 137

Hemophilia A - Discontinued Products 139

Hemophilia A - Product Development Milestones 140

Featured News & Press Releases 140

Appendix 154

Methodology 154

Coverage 154

Secondary Research 154

Primary Research 154

Expert Panel Validation 154

Contact Us 154

Disclaimer 155

List of Tables

List of Tables

Number of Products under Development for Hemophilia A, H2 2016 13

Number of Products under Development for Hemophilia A – Comparative Analysis, H2 2016 14

Number of Products under Development by Companies, H2 2016 15

Number of Products under Development by Companies, H2 2016 (Contd..1) 16

Number of Products under Investigation by Universities/Institutes, H2 2016 17

Comparative Analysis by Late Stage Development, H2 2016 18

Comparative Analysis by Clinical Stage Development, H2 2016 19

Comparative Analysis by Early Stage Development, H2 2016 20

Comparative Analysis by Unknown Stage Development, H2 2016 21

Products under Development by Companies, H2 2016 22

Products under Development by Companies, H2 2016 (Contd..1) 23

Products under Development by Companies, H2 2016 (Contd..2) 24

Products under Investigation by Universities/Institutes, H2 2016 25

Hemophilia A – Pipeline by Alnylam Pharmaceuticals Inc, H2 2016 26

Hemophilia A – Pipeline by Amarna Therapeutics BV, H2 2016 27

Hemophilia A – Pipeline by Apitope International NV, H2 2016 28

Hemophilia A – Pipeline by Bayer AG, H2 2016 29

Hemophilia A – Pipeline by Biogen Inc, H2 2016 30

Hemophilia A – Pipeline by BioMarin Pharmaceutical Inc, H2 2016 31

Hemophilia A – Pipeline by Catalyst Biosciences Inc, H2 2016 32

Hemophilia A – Pipeline by Chugai Pharmaceutical Co Ltd, H2 2016 33

Hemophilia A – Pipeline by CSL Ltd, H2 2016 34

Hemophilia A – Pipeline by DBV Technologies SA, H2 2016 35

Hemophilia A – Pipeline by Dimension Therapeutics Inc, H2 2016 36

Hemophilia A – Pipeline by Emergent BioSolutions Inc, H2 2016 37

Hemophilia A – Pipeline by EpiVax Inc, H2 2016 38

Hemophilia A – Pipeline by Expression Therapeutics LLC, H2 2016 39

Hemophilia A – Pipeline by Green Cross Corp, H2 2016 40

Hemophilia A – Pipeline by Idogen AB, H2 2016 41

Hemophilia A – Pipeline by Lentigen Technology Inc, H2 2016 42

Hemophilia A – Pipeline by mAbxience SA, H2 2016 43

Hemophilia A – Pipeline by Novo Nordisk A/S, H2 2016 44

Hemophilia A – Pipeline by Octapharma AG, H2 2016 45

Hemophilia A – Pipeline by OPKO Biologics Ltd, H2 2016 46

Hemophilia A – Pipeline by Pfizer Inc, H2 2016 47

Hemophilia A – Pipeline by Pharming Group NV, H2 2016 48

Hemophilia A – Pipeline by rEVO Biologics Inc, H2 2016 49

Hemophilia A – Pipeline by Sangamo BioSciences Inc, H2 2016 50

Hemophilia A – Pipeline by Shire Plc, H2 2016 51

Hemophilia A – Pipeline by SK Chemicals Co Ltd, H2 2016 52

Hemophilia A – Pipeline by Spark Therapeutics Inc, H2 2016 53

Hemophilia A – Pipeline by UniQure NV, H2 2016 54

Hemophilia A – Pipeline by XL-protein GmbH, H2 2016 55

Assessment by Monotherapy Products, H2 2016 56

Number of Products by Stage and Target, H2 2016 58

Number of Products by Stage and Mechanism of Actions H2 2016 60

Number of Products by Stage and Route of Administration, H2 2016 62

Number of Products by Stage and Molecule Type, H2 2016 64

Hemophilia A – Dormant Projects, H2 2016 141

Hemophilia A – Dormant Projects (Contd..1), H2 2016 142

Hemophilia A – Discontinued Products, H2 2016 143

List of Figures

List of Figures

Number of Products under Development for Hemophilia A, H2 2016 13

Number of Products under Development for Hemophilia A – Comparative Analysis, H2 2016 14

Number of Products under Development by Companies, H2 2016 15

Comparative Analysis by Late Stage Development, H2 2016 18

Comparative Analysis by Clinical Stage Development, H2 2016 19

Comparative Analysis by Early Stage Products, H2 2016 20

Assessment by Monotherapy Products, H2 2016 56

Number of Products by Targets, H2 2016 57

Number of Products by Stage and Targets, H2 2016 57

Number of Products by Mechanism of Actions, H2 2016 59

Number of Products by Stage and Mechanism of Actions, H2 2016 59

Number of Products by Routes of Administration, H2 2016 61

Number of Products by Stage and Routes of Administration, H2 2016 61

Number of Products by Top 10 Molecule Types, H2 2016 63

Number of Products by Stage and Top 10 Molecule Types, H2 2016 63

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