Hemophilia B – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Hemophilia B – Pipeline Review, H2 2016’, provides an overview of the Hemophilia B pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Hemophilia B, 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 B 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 B

The report reviews pipeline therapeutics for Hemophilia B 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 B therapeutics and enlists all their major and minor projects

The report assesses Hemophilia B 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 B

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 B

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 B 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 B.V.

Bayer AG

Biogen Inc

Catalyst Biosciences, Inc.

China Biologic Products, Inc.

CSL Limited

Dimension Therapeutics, Inc.

Green Cross Corporation

MolMed S.p.A.

Novo Nordisk A/S

OPKO Biologics Ltd

Pfizer Inc.

Pharming Group N.V.

RegenxBio Inc.

rEVO Biologics, Inc.

Sangamo BioSciences, Inc.

Shire Plc

Spark Therapeutics, Inc.

UniQure N.V.

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 6

List of Figures 7

Introduction 8

Global Markets Direct Report Coverage 8

Hemophilia B Overview 9

Therapeutics Development 10

Pipeline Products for Hemophilia B - Overview 10

Pipeline Products for Hemophilia B - Comparative Analysis 11

Hemophilia B - Therapeutics under Development by Companies 12

Hemophilia B - Therapeutics under Investigation by Universities/Institutes 14

Hemophilia B - Pipeline Products Glance 15

Late Stage Products 15

Clinical Stage Products 16

Early Stage Products 17

Hemophilia B - Products under Development by Companies 18

Hemophilia B - Products under Investigation by Universities/Institutes 19

Hemophilia B - Companies Involved in Therapeutics Development 20

Alnylam Pharmaceuticals, Inc. 20

Amarna Therapeutics B.V. 21

Bayer AG 22

Biogen Inc 23

Catalyst Biosciences, Inc. 24

China Biologic Products, Inc. 25

CSL Limited 26

Dimension Therapeutics, Inc. 27

Green Cross Corporation 28

MolMed S.p.A. 29

Novo Nordisk A/S 30

OPKO Biologics Ltd 31

Pfizer Inc. 32

Pharming Group N.V. 33

RegenxBio Inc. 34

rEVO Biologics, Inc. 35

Sangamo BioSciences, Inc. 36

Shire Plc 37

Spark Therapeutics, Inc. 38

UniQure N.V. 39

Hemophilia B - Therapeutics Assessment 40

Assessment by Monotherapy Products 40

Assessment by Target 41

Assessment by Route of Administration 43

Assessment by Molecule Type 45

Drug Profiles 47

AMT-060 - Drug Profile 47

Product Description 47

Mechanism Of Action 47

R&D Progress 47

BAX-335 - Drug Profile 50

Product Description 50

Mechanism Of Action 50

R&D Progress 50

BAY-1093884 - Drug Profile 52

Product Description 52

Mechanism Of Action 52

R&D Progress 52

CB-2679d - Drug Profile 53

Product Description 53

Mechanism Of Action 53

R&D Progress 53

Cell Therapy for Hemophilia B - Drug Profile 54

Product Description 54

Mechanism Of Action 54

R&D Progress 54

coagulation factor IX (human) - Drug Profile 55

Product Description 55

Mechanism Of Action 55

R&D Progress 55

coagulation factor IX (recombinant) - Drug Profile 56

Product Description 56

Mechanism Of Action 56

R&D Progress 56

coagulation factor IX (recombinant), albumin fusion protein - Drug Profile 57

Product Description 57

Mechanism Of Action 57

R&D Progress 57

coagulation factor IX (recombinant), pegylated - Drug Profile 62

Product Description 62

Mechanism Of Action 62

R&D Progress 62

coagulation factor VIIa (recombinant) - Drug Profile 64

Product Description 64

Mechanism Of Action 64

R&D Progress 64

concizumab - Drug Profile 66

Product Description 66

Mechanism Of Action 66

R&D Progress 66

CSL-689 - Drug Profile 67

Product Description 67

Mechanism Of Action 67

R&D Progress 67

DTX-101 - Drug Profile 69

Product Description 69

Mechanism Of Action 69

R&D Progress 69

fitusiran - Drug Profile 71

Product Description 71

Mechanism Of Action 71

R&D Progress 71

Gene Therapy for Hemophilia B - Drug Profile 77

Product Description 77

Mechanism Of Action 77

R&D Progress 77

Gene Therapy for Hemophilia B - Drug Profile 78

Product Description 78

Mechanism Of Action 78

R&D Progress 78

Gene Therapy to Activate Factor IX for Hemophilia B - Drug Profile 79

Product Description 79

Mechanism Of Action 79

R&D Progress 79

Gene Therapy to Activate Factor IX for Hemophilia B - Drug Profile 80

Product Description 80

Mechanism Of Action 80

R&D Progress 80

LR-769 - Drug Profile 82

Product Description 82

Mechanism Of Action 82

R&D Progress 82

marzeptacog alfa - Drug Profile 84

Product Description 84

Mechanism Of Action 84

R&D Progress 84

MG-1113A - Drug Profile 86

Product Description 86

Mechanism Of Action 86

R&D Progress 86

MOD-5014 - Drug Profile 87

Product Description 87

Mechanism Of Action 87

R&D Progress 87

PF-06741086 - Drug Profile 90

Product Description 90

Mechanism Of Action 90

R&D Progress 90

Recombinant Factor IX Replacement for Hemophilia B - Drug Profile 91

Product Description 91

Mechanism Of Action 91

R&D Progress 91

SB-FIX - Drug Profile 92

Product Description 92

Mechanism Of Action 92

R&D Progress 92

SPK-9001 - Drug Profile 94

Product Description 94

Mechanism Of Action 94

R&D Progress 94

SVF-VIIa - Drug Profile 96

Product Description 96

Mechanism Of Action 96

R&D Progress 96

Hemophilia B - Dormant Projects 97

Hemophilia B - Discontinued Products 98

Hemophilia B - Product Development Milestones 99

Featured News & Press Releases 99

Sep 26, 2016: Alprolix obtains reimbursement across the United Kingdom 99

Sep 07, 2016: Catalyst Biosciences to Focus on Improved Factor IX Clinical Hemostasis Program 99

Sep 06, 2016: Sangamo BioSciences Receives Orphan Drug Designation From The FDA For SB-FIX, The First Application Of Therapeutic In Vivo Genome Editing 100

Jul 26, 2016: CSL Behring Presents Phase III Data for IDELVION for Hemophilia B at the World Federation of Hemophilia 2016 World Congress 101

Jul 25, 2016: Alnylam Announces New Positive Interim Phase 1 Study Results for Fitusiran, a Once-Monthly, Subcutaneous, Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia and Rare Bleeding Disorders 102

Jul 25, 2016: Alnylam to Report New Clinical Results with Fitusiran at the World Federation of Hemophilia 2016 World Congress 105

Jul 19, 2016: CSL Behring to Present New Data for IDELVION at the World Federation of Hemophilia 2016 World Congress 105

Jul 18, 2016: Biogen and Sobi to Showcase Long-Term Efficacy and Safety Data on ALPROLIX at World Federation of Hemophilia 2016 World Congress 106

Jun 06, 2016: Sobi: First Alprolix sales in the EU 107

May 24, 2016: Catalyst Biosciences and CMC Biologics Announce Manufacturing Agreement for Catalyst Next-Generation Factor VIIa Product CB 813d 107

May 16, 2016: Novo Nordisk files for regulatory approval in the US of long-acting factor IX for the treatment of hemophilia B 107

May 13, 2016: Alprolix (rFIXFc) approved in the EU for the treatment of hemophilia B 108

May 12, 2016: IDELVION (rFIX-FP) - European Commission Approval 109

May 11, 2016: CSL Behring's Innovative Therapy for Hemophilia B Patients, IDELVION, Receives 7 Years of Marketing Exclusivity from the U.S. Food and Drug Administration 109

May 11, 2016: European Commission Approves IDELVION --CSL Behring Novel Hemophilia B Treatment with Up to 14-day Dosing Intervals 110

Appendix 111

Methodology 111

Coverage 111

Secondary Research 111

Primary Research 111

Expert Panel Validation 111

Contact Us 111

Disclaimer 112

List of Tables

List of Tables

Number of Products under Development for Hemophilia B, H2 2016 10

Number of Products under Development for Hemophilia B – Comparative Analysis, H2 2016 11

Number of Products under Development by Companies, H2 2016 13

Number of Products under Investigation by Universities/Institutes, H2 2016 14

Comparative Analysis by Late Stage Development, H2 2016 15

Comparative Analysis by Clinical Stage Development, H2 2016 16

Comparative Analysis by Early Stage Development, H2 2016 17

Products under Development by Companies, H2 2016 18

Products under Investigation by Universities/Institutes, H2 2016 19

Hemophilia B – Pipeline by Alnylam Pharmaceuticals, Inc., H2 2016 20

Hemophilia B – Pipeline by Amarna Therapeutics B.V., H2 2016 21

Hemophilia B – Pipeline by Bayer AG, H2 2016 22

Hemophilia B – Pipeline by Biogen Inc, H2 2016 23

Hemophilia B – Pipeline by Catalyst Biosciences, Inc., H2 2016 24

Hemophilia B – Pipeline by China Biologic Products, Inc., H2 2016 25

Hemophilia B – Pipeline by CSL Limited, H2 2016 26

Hemophilia B – Pipeline by Dimension Therapeutics, Inc., H2 2016 27

Hemophilia B – Pipeline by Green Cross Corporation, H2 2016 28

Hemophilia B – Pipeline by MolMed S.p.A., H2 2016 29

Hemophilia B – Pipeline by Novo Nordisk A/S, H2 2016 30

Hemophilia B – Pipeline by OPKO Biologics Ltd, H2 2016 31

Hemophilia B – Pipeline by Pfizer Inc., H2 2016 32

Hemophilia B – Pipeline by Pharming Group N.V., H2 2016 33

Hemophilia B – Pipeline by RegenxBio Inc., H2 2016 34

Hemophilia B – Pipeline by rEVO Biologics, Inc., H2 2016 35

Hemophilia B – Pipeline by Sangamo BioSciences, Inc., H2 2016 36

Hemophilia B – Pipeline by Shire Plc, H2 2016 37

Hemophilia B – Pipeline by Spark Therapeutics, Inc., H2 2016 38

Hemophilia B – Pipeline by UniQure N.V., H2 2016 39

Assessment by Monotherapy Products, H2 2016 40

Number of Products by Stage and Target, H2 2016 42

Number of Products by Stage and Route of Administration, H2 2016 44

Number of Products by Stage and Molecule Type, H2 2016 46

Hemophilia B – Dormant Projects, H2 2016 97

Hemophilia B – Discontinued Products, H2 2016 98

List of Figures

List of Figures

Number of Products under Development for Hemophilia B, H2 2016 10

Number of Products under Development for Hemophilia B – Comparative Analysis, H2 2016 11

Number of Products under Development by Companies, H2 2016 12

Number of Products under Investigation by Universities/Institutes, H2 2016 14

Comparative Analysis by Late Stage Development, H2 2016 15

Comparative Analysis by Clinical Stage Development, H2 2016 16

Comparative Analysis by Early Stage Products, H2 2016 17

Assessment by Monotherapy Products, H2 2016 40

Number of Products by Targets, H2 2016 41

Number of Products by Stage and Targets, H2 2016 41

Number of Products by Routes of Administration, H2 2016 43

Number of Products by Stage and Routes of Administration, H2 2016 43

Number of Products by Molecule Types, H2 2016 45

Number of Products by Stage and Molecule Types, H2 2016 45

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