Hemophilia A – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Hemophilia A – Pipeline Review, H2 2017’, 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

Amarna Therapeutics BV

Apitope International NV

Bayer AG

BioMarin Pharmaceutical Inc

Bioverativ Inc

Catalyst Biosciences Inc

Chugai Pharmaceutical Co Ltd

CSL Ltd

DBV Technologies SA

Dimension Therapeutics Inc

EpiVax Inc

Expression Therapeutics LLC

Green Cross Corp

Idogen AB

Immusoft Corp

LFB SA

mAbxience SA

Novo Nordisk AS

OPKO Biologics Ltd

Pangen Biotech Inc.

Pfizer Inc

Pharming Group NV

Promethera Biosciences SA

Sangamo Therapeutics 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

Hemophilia A - Therapeutics Development 9

Pipeline Overview 9

Pipeline by Companies 10

Pipeline by Universities/Institutes 13

Products under Development by Companies 14

Products under Development by Universities/Institutes 17

Hemophilia A - Therapeutics Assessment 18

Assessment by Target 18

Assessment by Mechanism of Action 20

Assessment by Route of Administration 22

Assessment by Molecule Type 24

Hemophilia A - Companies Involved in Therapeutics Development 26

Amarna Therapeutics BV 26

Apitope International NV 26

Bayer AG 26

BioMarin Pharmaceutical Inc 27

Bioverativ Inc 27

Catalyst Biosciences Inc 28

Chugai Pharmaceutical Co Ltd 29

CSL Ltd 29

DBV Technologies SA 30

Dimension Therapeutics Inc 30

EpiVax Inc 31

Expression Therapeutics LLC 31

Green Cross Corp 31

Idogen AB 32

Immusoft Corp 32

LFB SA 33

mAbxience SA 33

Novo Nordisk AS 33

OPKO Biologics Ltd 34

Pangen Biotech Inc. 34

Pfizer Inc 34

Pharming Group NV 35

Promethera Biosciences SA 35

Sangamo Therapeutics Inc 36

Shire Plc 36

SK Chemicals Co Ltd 37

Spark Therapeutics Inc 37

UniQure NV 38

XL-protein GmbH 38

Hemophilia A - Drug Profiles 40

antihemophilic factor (recombinant) - Drug Profile 40

antihemophilic factor (recombinant) - Drug Profile 41

antihemophilic factor (recombinant) - Drug Profile 42

antihemophilic factor (recombinant) - Drug Profile 43

antihemophilic factor (recombinant) - Drug Profile 44

antihemophilic factor (recombinant) biosimilar - Drug Profile 45

antihemophilic factor (recombinant) biosimilar - Drug Profile 46

antihemophilic factor (recombinant) biosimilar - Drug Profile 47

antihemophilic factor (recombinant), PEGylated - Drug Profile 48

antihemophilic factor (recombinant), pegylated - Drug Profile 53

antihemophilic factor (recombinant), porcine sequence - Drug Profile 56

antihemophilic factor (recombinant), single chain - Drug Profile 60

ATXF-8117 - Drug Profile 64

BAY-1093884 - Drug Profile 65

BIVV-001 - Drug Profile 66

BS-027125 - Drug Profile 69

Cell Therapy to Target Coagulation Factor VIII for Hemophilia A - Drug Profile 70

concizumab - Drug Profile 71

CSL-689 - Drug Profile 73

damoctocog alfa pegol - Drug Profile 76

Deimmunized FVIII - Drug Profile 79

DTX-201 - Drug Profile 80

emicizumab - Drug Profile 82

ET-3 - Drug Profile 87

Gene Therapy for Hematological Disorders - Drug Profile 88

Gene Therapy to Activate Factor VIII for Hemophilia A - Drug Profile 89

Gene Therapy to Activate Factor VIII for Hemophilia A - Drug Profile 90

Gene Therapy to Activate Factor VIII for Hemophilia A - Drug Profile 91

HepaStem - Drug Profile 93

IN-3012 - Drug Profile 96

LR-769 - Drug Profile 98

marzeptacog alfa - Drug Profile 101

MG-1113A - Drug Profile 104

MOD-5014 - Drug Profile 105

NIBX-2101 - Drug Profile 108

PBB-8-IN - Drug Profile 109

PF-06741086 - Drug Profile 110

Recombinant Factor VIII Replacement for Hemophilia A - Drug Profile 111

Recombinant Protein to Inhibit Coagulation Factor XIV for Hemophilia A and Hemophilia B - Drug Profile 112

Recombinant Protein to Replace Coagulation Factor VIII for Hemophilia A - Drug Profile 113

Recombinant Proteins for Hemophilia A - Drug Profile 114

SB-525 - Drug Profile 115

SEL-201 - Drug Profile 117

SHP-654 - Drug Profile 118

SPK-8011 - Drug Profile 120

Stem Cell Therapy to Activate Coagulation Factor VIII for Hemophilia A - Drug Profile 122

SVF-VIIa - Drug Profile 123

valoctocogene roxaparvovec - Drug Profile 124

Hemophilia A - Dormant Projects 128

Hemophilia A - Discontinued Products 130

Hemophilia A - Product Development Milestones 131

Featured News & Press Releases 131

Appendix 142

Methodology 142

Coverage 142

Secondary Research 142

Primary Research 142

Expert Panel Validation 142

Contact Us 142

Disclaimer 143

List of Tables

List of Tables

Number of Products under Development for Hemophilia A, H2 2017 12

Number of Products under Development by Companies, H2 2017 14

Number of Products under Development by Companies, H2 2017 (Contd..1), H2 2017 15

Number of Products under Development by Universities/Institutes, H2 2017 16

Products under Development by Companies, H2 2017 17

Products under Development by Companies, H2 2017 (Contd..1), H2 2017 18

Products under Development by Companies, H2 2017 (Contd..2), H2 2017 19

Products under Development by Universities/Institutes, H2 2017 20

Number of Products by Stage and Target, H2 2017 22

Number of Products by Stage and Mechanism of Action, H2 2017 24

Number of Products by Stage and Route of Administration, H2 2017 26

Number of Products by Stage and Molecule Type, H2 2017 28

Hemophilia A – Pipeline by Amarna Therapeutics BV, H2 2017 29

Hemophilia A – Pipeline by Apitope International NV, H2 2017 29

Hemophilia A – Pipeline by Bayer AG, H2 2017 30

Hemophilia A – Pipeline by BioMarin Pharmaceutical Inc, H2 2017 30

Hemophilia A – Pipeline by Bioverativ Inc, H2 2017 31

Hemophilia A – Pipeline by Catalyst Biosciences Inc, H2 2017 32

Hemophilia A – Pipeline by Chugai Pharmaceutical Co Ltd, H2 2017 32

Hemophilia A – Pipeline by CSL Ltd, H2 2017 33

Hemophilia A – Pipeline by DBV Technologies SA, H2 2017 33

Hemophilia A – Pipeline by Dimension Therapeutics Inc, H2 2017 34

Hemophilia A – Pipeline by EpiVax Inc, H2 2017 34

Hemophilia A – Pipeline by Expression Therapeutics LLC, H2 2017 34

Hemophilia A – Pipeline by Green Cross Corp, H2 2017 35

Hemophilia A – Pipeline by Idogen AB, H2 2017 35

Hemophilia A – Pipeline by Immusoft Corp, H2 2017 35

Hemophilia A – Pipeline by LFB SA, H2 2017 36

Hemophilia A – Pipeline by mAbxience SA, H2 2017 36

Hemophilia A – Pipeline by Novo Nordisk AS, H2 2017 37

Hemophilia A – Pipeline by OPKO Biologics Ltd, H2 2017 37

Hemophilia A – Pipeline by Pangen Biotech Inc., H2 2017 37

Hemophilia A – Pipeline by Pfizer Inc, H2 2017 38

Hemophilia A – Pipeline by Pharming Group NV, H2 2017 38

Hemophilia A – Pipeline by Promethera Biosciences SA, H2 2017 39

Hemophilia A – Pipeline by Sangamo Therapeutics Inc, H2 2017 39

Hemophilia A – Pipeline by Shire Plc, H2 2017 40

Hemophilia A – Pipeline by SK Chemicals Co Ltd, H2 2017 40

Hemophilia A – Pipeline by Spark Therapeutics Inc, H2 2017 41

Hemophilia A – Pipeline by UniQure NV, H2 2017 41

Hemophilia A – Pipeline by XL-protein GmbH, H2 2017 42

Hemophilia A – Dormant Projects, H2 2017 131

Hemophilia A – Dormant Projects, H2 2017 (Contd..1), H2 2017 132

Hemophilia A – Discontinued Products, H2 2017 133

List of Figures

List of Figures

Number of Products under Development for Hemophilia A, H2 2017 12

Number of Products under Development by Companies, H2 2017 13

Number of Products under Development by Universities/Institutes, H2 2017 16

Number of Products by Targets, H2 2017 21

Number of Products by Stage and Targets, H2 2017 21

Number of Products by Mechanism of Actions, H2 2017 23

Number of Products by Stage and Mechanism of Actions, H2 2017 23

Number of Products by Routes of Administration, H2 2017 25

Number of Products by Stage and Routes of Administration, H2 2017 25

Number of Products by Top 10 Molecule Types, H2 2017 27

Number of Products by Stage and Top 10 Molecule Types, H2 2017 27

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