Hemophilia B - Pipeline Review, H1 2016

Published: Apr, 2016 | Pages: 122 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Hemophilia B - Pipeline Review, H1 2016

Summary

Global Markets Direct's, 'Hemophilia B - Pipeline Review, H1 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

 Table of Contents

Table of Content 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
Baxalta Incorporated 22
Bayer AG 23
Biogen, Inc. 24
Catalyst Biosciences, Inc. 25
CSL Limited 26
Dimension Therapeutics, Inc. 27
Emergent BioSolutions Inc. 28
Novo Nordisk A/S 29
OPKO Health, Inc. 30
Pfizer Inc. 31
Pharming Group N.V. 32
RegenxBio Inc. 33
rEVO Biologics, Inc. 34
Sangamo BioSciences, Inc. 35
Spark Therapeutics, Inc. 36
UniQure N.V. 37
Hemophilia B - Therapeutics Assessment 38
Assessment by Monotherapy Products 38
Assessment by Target 39
Assessment by Mechanism of Action 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 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
BAY-1093884 - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
CB-2679d - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
CB-813d - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
Cell Therapy for Hemophilia B - 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) - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
coagulation factor IX (recombinant), albumin fusion protein - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
coagulation factor IX (recombinant), Fc fusion protein - Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
coagulation factor VIIa (recombinant) - Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
concizumab - Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
CSL-689 - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
DTX-101 - Drug Profile 68
Product Description 68
Mechanism of Action 68
R&D Progress 68
fitusiran - Drug Profile 69
Product Description 69
Mechanism of Action 69
R&D Progress 69
Gene Therapy for Hemophilia B - Drug Profile 70
Product Description 70
Mechanism of Action 70
R&D Progress 70
Gene Therapy to Activate Factor IX for Hemophilia B - Drug Profile 71
Product Description 71
Mechanism of Action 71
R&D Progress 71
Gene Therapy to Activate Factor IX for Hemophilia B - Drug Profile 73
Product Description 73
Mechanism of Action 73
R&D Progress 73
LR-769 - Drug Profile 75
Product Description 75
Mechanism of Action 75
R&D Progress 75
MOD-5014 - Drug Profile 77
Product Description 77
Mechanism of Action 77
R&D Progress 77
MOD-9017 - Drug Profile 79
Product Description 79
Mechanism of Action 79
R&D Progress 79
nonacog beta pegol - Drug Profile 80
Product Description 80
Mechanism of Action 80
R&D Progress 80
PF-06741086 - Drug Profile 82
Product Description 82
Mechanism of Action 82
R&D Progress 82
SB-FIX - Drug Profile 83
Product Description 83
Mechanism of Action 83
R&D Progress 83
SPK-9001 - Drug Profile 85
Product Description 85
Mechanism of Action 85
R&D Progress 85
SVF-VIIa - Drug Profile 86
Product Description 86
Mechanism of Action 86
R&D Progress 86
Hemophilia B - Recent Pipeline Updates 87
Hemophilia B - Dormant Projects 113
Hemophilia B - Discontinued Products 114
Hemophilia B - Product Development Milestones 115
Featured News & Press Releases 115
Mar 21, 2016: IDELVION for Hemophilia B Now Available in the U.S. 115
Mar 04, 2016: FDA approves first coagulation factor-albumin fusion protein to treat patients with hemophilia B 115
Feb 29, 2016: IDELVION (rFIX-FP) - CHMP recommendation 116
Feb 26, 2016: Sobi and Biogen Receive Positive Opinion from CHMP for Alprolix (rFIXFc) for the Treatment of Hemophilia B 116
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 117
Feb 03, 2016: Biogen Announces The Availability Of ALPROLIX To Canadians With Hemophilia B 117
Jan 07, 2016: Novo Nordisk files for regulatory approval of long-acting factor IX in the EU for the treatment of hemophilia B 118
Dec 22, 2015: Sobi receives orphan drug designation for Alprolix in Switzerland 119
Dec 21, 2015: CSL Behring Submits New Drug Application to Japan's Pharmaceuticals and Medical Devices Agency for rIX-FP for Hemophilia B Patients 119
Dec 08, 2015: New Data Show ALPROLIX May Help Control Target Joint Bleeds in People with Hemophilia B 119
Appendix 121
Methodology 121
Coverage 121
Secondary Research 121
Primary Research 121
Expert Panel Validation 121
Contact Us 121
Disclaimer 122

List of Tables

Number of Products under Development for Hemophilia B, H1 2016 10
Number of Products under Development for Hemophilia B - Comparative Analysis, H1 2016 11
Number of Products under Development by Companies, H1 2016 13
Number of Products under Investigation by Universities/Institutes, H1 2016 14
Comparative Analysis by Late Stage Development, H1 2016 15
Comparative Analysis by Clinical Stage Development, H1 2016 16
Comparative Analysis by Early Stage Development, H1 2016 17
Products under Development by Companies, H1 2016 18
Products under Investigation by Universities/Institutes, H1 2016 19
Hemophilia B - Pipeline by Alnylam Pharmaceuticals, Inc., H1 2016 20
Hemophilia B - Pipeline by Amarna Therapeutics B.V., H1 2016 21
Hemophilia B - Pipeline by Baxalta Incorporated, H1 2016 22
Hemophilia B - Pipeline by Bayer AG, H1 2016 23
Hemophilia B - Pipeline by Biogen, Inc., H1 2016 24
Hemophilia B - Pipeline by Catalyst Biosciences, Inc., H1 2016 25
Hemophilia B - Pipeline by CSL Limited, H1 2016 26
Hemophilia B - Pipeline by Dimension Therapeutics, Inc., H1 2016 27
Hemophilia B - Pipeline by Emergent BioSolutions Inc., H1 2016 28
Hemophilia B - Pipeline by Novo Nordisk A/S, H1 2016 29
Hemophilia B - Pipeline by OPKO Health, Inc., H1 2016 30
Hemophilia B - Pipeline by Pfizer Inc., H1 2016 31
Hemophilia B - Pipeline by Pharming Group N.V., H1 2016 32
Hemophilia B - Pipeline by RegenxBio Inc., H1 2016 33
Hemophilia B - Pipeline by rEVO Biologics, Inc., H1 2016 34
Hemophilia B - Pipeline by Sangamo BioSciences, Inc., H1 2016 35
Hemophilia B - Pipeline by Spark Therapeutics, Inc., H1 2016 36
Hemophilia B - Pipeline by UniQure N.V., H1 2016 37
Assessment by Monotherapy Products, H1 2016 38
Number of Products by Stage and Target, H1 2016 40
Number of Products by Stage and Mechanism of Action, H1 2016 42
Number of Products by Stage and Route of Administration, H1 2016 44
Number of Products by Stage and Molecule Type, H1 2016 46
Hemophilia B Therapeutics - Recent Pipeline Updates, H1 2016 87
Hemophilia B - Dormant Projects, H1 2016 113
Hemophilia B - Discontinued Products, H1 2016 114