Hemophilia B - Pipeline Review, H1 2015

Published: May, 2015 | Pages: 106 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Hemophilia B - Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Hemophilia B - Pipeline Review, H1 2015', provides an overview of the Hemophilia B's therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Hemophilia B, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Hemophilia B and special features on late-stage 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

- The report provides a snapshot of the global therapeutic landscape of Hemophilia B
- The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities 
- The report reviews key players involved in the therapeutics development for Hemophilia B and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects 
- A review of the Hemophilia B products under development by companies and universities/research institutes based on information derived from company and industry-specific sources
- Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- A detailed assessment of monotherapy and combination therapy pipeline projects
- Coverage of the Hemophilia B pipeline on the basis of target, MoA, route of administration and molecule type
- Latest news and deals relating related to pipeline products

Reasons to buy

- Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Develop strategic initiatives by understanding the focus areas of leading companies
- Identify and understand important and diverse types of therapeutics under development for Hemophilia B
- Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
- 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 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 20
Hemophilia B - Companies Involved in Therapeutics Development 21
Alnylam Pharmaceuticals, Inc. 21
Amarna Therapeutics B.V. 22
AstraZeneca PLC 23
Bayer AG 24
Biogen, Inc. 25
Catalyst Biosciences, Inc. 26
CSL Limited 27
Dimension Therapeutics, Inc. 28
Emergent BioSolutions Inc. 29
Novo Nordisk A/S 30
OPKO Health, Inc. 31
Pharming Group N.V. 32
rEVO Biologics 33
Sangamo BioSciences, Inc. 34
Spark Therapeutics, Inc. 35
The International Biotechnology Center (IBC) "Generium" 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
albutrepenonacog alfa - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
ALN-AT3 - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
AMT-060 - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
AZ-10047130 - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
BAX-335 - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
BAY-1093884 - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
CB-2679d - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
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 VIIa (recombinant) - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
concizumab - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
CSL-689 - Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
DTX-101 - Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
Gene Therapy for Hemophilia B - Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
Gene Therapy to Activate Factor IX for Hemophilia B - Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
LR-769 - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
MOD-5014 - Drug Profile 68
Product Description 68
Mechanism of Action 68
R&D Progress 68
nonacog alfa biosimilar - Drug Profile 70
Product Description 70
Mechanism of Action 70
R&D Progress 70
nonacog beta pegol - Drug Profile 71
Product Description 71
Mechanism of Action 71
R&D Progress 71
PF-05280602 - Drug Profile 73
Product Description 73
Mechanism of Action 73
R&D Progress 73
Recombinant Enzyme to Replace Factor IX for Hemophilia B - Drug Profile 74
Product Description 74
Mechanism of Action 74
R&D Progress 74
SHP-624 - Drug Profile 75
Product Description 75
Mechanism of Action 75
R&D Progress 75
SPK-FIX - Drug Profile 76
Product Description 76
Mechanism of Action 76
R&D Progress 76
SVF-VIIa - Drug Profile 77
Product Description 77
Mechanism of Action 77
R&D Progress 77
Hemophilia B - Recent Pipeline Updates 78
Hemophilia B - Dormant Projects 96
Hemophilia B - Discontinued Products 97
Hemophilia B - Product Development Milestones 98
Featured News & Press Releases 98
Mar 30, 2015: European Medicines Agency Commences Review of CSL Behring's Regulatory Submission for rIX-FP for Hemophilia B Patients 98
Mar 24, 2015: LFB Receives Go Ahead From Data Monitoring Committee to Expand The Global PERSEPT Phase 3 Program of LR769 in Hemophilia A or B Patients with Inhibitors 98
Mar 13, 2015: Baxter BioScience Announces Positive Phase III Results for BAX 817, Investigational Recombinant Treatment for Hemophilia A and B Patients with Inhibitors 99
Feb 04, 2015: U.S. FDA Accepts for Review CSL Behring's Biologics License Application for rIX-FP for Hemophilia B Patients 100
Dec 17, 2014: LFB Announces Achievement of Patient Enrollment Target for PerSept 1 Program for the Evaluation of Recombinant Factor Seven Efficacy by Prospective Clinical Trials 100
Dec 16, 2014: CSL Behring Submits Biologics License Application for FDA Approval of Recombinant Fusion Protein Linking Coagulation Factor IX with Recombinant Albumin (rIX-FP) for Hemophilia B Patients 100
May 14, 2014: LFB SA Announces That First Patient Has Begun Treatment with New Coagulation Factor VIIa (Recombinant) in Global Phase 3 Clinical Trial in Hemophilia A and B with Inhibitors 101
May 11, 2014: New Findings Support Less Frequent Dosing in Hemophilia B Patients Treated with Coagulation Factor IX with Recombinant Albumin 101
Mar 03, 2014: LFB Initiates Global Phase 3 Clinical Trial of New Coagulation Factor VIIa (Recombinant) in Patients with Hemophilia A and B 102
Oct 29, 2013: Alnylam Files Clinical Trial Application to Initiate a Phase I Study for ALN-AT3, a Subcutaneously Administered RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia A and B 103
Appendix 105
Methodology 105
Coverage 105
Secondary Research 105
Primary Research 105
Expert Panel Validation 105
Contact Us 105
Disclaimer 106

List of Tables

Number of Products under Development for Hemophilia B, H1 2015 10
Number of Products under Development for Hemophilia B - Comparative Analysis, H1 2015 11
Number of Products under Development by Companies, H1 2015 13
Number of Products under Investigation by Universities/Institutes, H1 2015 14
Comparative Analysis by Late Stage Development, H1 2015 15
Comparative Analysis by Clinical Stage Development, H1 2015 16
Comparative Analysis by Early Stage Development, H1 2015 17
Products under Development by Companies, H1 2015 18
Products under Development by Companies, H1 2015 (Contd..1) 19
Products under Investigation by Universities/Institutes, H1 2015 20
Hemophilia B - Pipeline by Alnylam Pharmaceuticals, Inc., H1 2015 21
Hemophilia B - Pipeline by Amarna Therapeutics B.V., H1 2015 22
Hemophilia B - Pipeline by AstraZeneca PLC, H1 2015 23
Hemophilia B - Pipeline by Bayer AG, H1 2015 24
Hemophilia B - Pipeline by Biogen, Inc., H1 2015 25
Hemophilia B - Pipeline by Catalyst Biosciences, Inc., H1 2015 26
Hemophilia B - Pipeline by CSL Limited, H1 2015 27
Hemophilia B - Pipeline by Dimension Therapeutics, Inc., H1 2015 28
Hemophilia B - Pipeline by Emergent BioSolutions Inc., H1 2015 29
Hemophilia B - Pipeline by Novo Nordisk A/S, H1 2015 30
Hemophilia B - Pipeline by OPKO Health, Inc., H1 2015 31
Hemophilia B - Pipeline by Pharming Group N.V., H1 2015 32
Hemophilia B - Pipeline by rEVO Biologics, H1 2015 33
Hemophilia B - Pipeline by Sangamo BioSciences, Inc., H1 2015 34
Hemophilia B - Pipeline by Spark Therapeutics, Inc., H1 2015 35
Hemophilia B - Pipeline by The International Biotechnology Center (IBC) "Generium", H1 2015 36
Hemophilia B - Pipeline by uniQure N.V., H1 2015 37
Assessment by Monotherapy Products, H1 2015 38
Number of Products by Stage and Target, H1 2015 40
Number of Products by Stage and Mechanism of Action, H1 2015 42
Number of Products by Stage and Route of Administration, H1 2015 44
Number of Products by Stage and Molecule Type, H1 2015 46
Hemophilia B Therapeutics - Recent Pipeline Updates, H1 2015 78
Hemophilia B - Dormant Projects, H1 2015 96
Hemophilia B - Discontinued Products, H1 2015 97