Hemophilia B - Pipeline Review, H2 2014

Published: Aug, 2014 | Pages: 96 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)

Hemophilia B - Pipeline Review, H2 2014

Summary

Global Markets Direct's, 'Hemophilia B - Pipeline Review, H2 2014', 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 - Pipeline Products Glance 14
Late Stage Products 14
Clinical Stage Products 15
Early Stage Products 16
Hemophilia B - Products under Development by Companies 17
Hemophilia B - Companies Involved in Therapeutics Development 18
Baxter International Inc. 18
AstraZeneca PLC 19
Emergent BioSolutions Inc. 20
Sangamo BioSciences, Inc. 21
Novo Nordisk A/S 22
rEVO Biologics 23
Pfizer Inc. 24
OPKO Health, Inc. 25
ReGenX Biosciences, LLC 26
Alnylam Pharmaceuticals, Inc. 27
CSL Limited 28
Catalyst Biosciences, Inc. 29
Amarna Therapeutics B.V. 30
AlphaMab Co., Ltd 31
Hemophilia B - Therapeutics Assessment 32
Assessment by Monotherapy Products 32
Assessment by Target 33
Assessment by Mechanism of Action 35
Assessment by Route of Administration 37
Assessment by Molecule Type 39
Drug Profiles 41
coagulation factor IX (recombinant) - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
trenonacog alfa - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
CSL-654 - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
nonacog beta pegol - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
LR-769 - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
coagulation factor VIIa (recombinant) - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
CSL-689 - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
nonacog alfa biosimilar - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
AMT-060 - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
AskBio-009 - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
Gene Therapy to Activate Factor IX for Hemophilia B - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
PF-05280602 - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
concizumab - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
Gene Therapy to Activate Factor IX for Hemophilia B - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
ALN-AT3 - Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
eptacog alfa - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
ZFP Transcription Factors for Hemophilia - Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
MOD-5014 - Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
CB-2679d - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
Gene Therapy for Hemophilia B - Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
SVF-VIIa - Drug Profile 68
Product Description 68
Mechanism of Action 68
R&D Progress 68
AZ-10047130 - Drug Profile 69
Product Description 69
Mechanism of Action 69
R&D Progress 69
AZ-10776241 - Drug Profile 70
Product Description 70
Mechanism of Action 70
R&D Progress 70
Hemophilia B - Recent Pipeline Updates 71
Hemophilia B - Dormant Projects 85
Hemophilia B - Discontinued Products 86
Hemophilia B - Product Development Milestones 87
Featured News & Press Releases 87
Jul 16, 2014: Pfizer Announces Positive Top-Line Results From Phase 3 Study Of NONACOG ALFA (BeneFIX) Once-Weekly Prophylaxis For Hemophilia B 87
Jul 09, 2014: OPKO's Long-Acting Clotting Factor VIIa-CTP Receives Positive Opinion For Three Orphan Drug Designations in Europe 88
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 88
May 11, 2014: New Findings Support Less Frequent Dosing in Hemophilia B Patients Treated with Coagulation Factor IX with Recombinant Albumin 89
Mar 03, 2014: LFB Initiates Global Phase 3 Clinical Trial of New Coagulation Factor VIIa (Recombinant) in Patients with Hemophilia A and B 90
Feb 28, 2014: OPKO's Longer-Acting Clotting Factor VIIa-CTP Receives Orphan Drug Designation in the U.S. 90
Dec 09, 2013: Baxter Files for Pediatric Indication of RIXUBIS [Coagulation Factor IX (Recombinant)] Treatment for Hemophilia B in the United States 91
Oct 31, 2013: Baxter Submits Application for European Approval of RIXUBIS, Recombinant Factor IX (nonacog gamma) for Hemophilia B 91
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 92
Aug 20, 2013: Alnylam Receives Additional Orphan Drug Designation from U.S. Food & Drug Administration for ALN-AT3, an RNAi Therapeutic for the Treatment of Hemophilia 93
Appendix 95
Methodology 95
Coverage 95
Secondary Research 95
Primary Research 95
Expert Panel Validation 95
Contact Us 96
Disclaimer 96

List of Tables
Number of Products under Development for Hemophilia B, H2 2014 10
Number of Products under Development for Hemophilia B - Comparative Analysis, H2 2014 11
Number of Products under Development by Companies, H2 2014 13
Comparative Analysis by Late Stage Development, H2 2014 14
Comparative Analysis by Clinical Stage Development, H2 2014 15
Comparative Analysis by Early Stage Development, H2 2014 16
Products under Development by Companies, H2 2014 17
Hemophilia B - Pipeline by Baxter International Inc., H2 2014 18
Hemophilia B - Pipeline by AstraZeneca PLC, H2 2014 19
Hemophilia B - Pipeline by Emergent BioSolutions Inc., H2 2014 20
Hemophilia B - Pipeline by Sangamo BioSciences, Inc., H2 2014 21
Hemophilia B - Pipeline by Novo Nordisk A/S, H2 2014 22
Hemophilia B - Pipeline by rEVO Biologics, H2 2014 23
Hemophilia B - Pipeline by Pfizer Inc., H2 2014 24
Hemophilia B - Pipeline by OPKO Health, Inc., H2 2014 25
Hemophilia B - Pipeline by ReGenX Biosciences, LLC, H2 2014 26
Hemophilia B - Pipeline by Alnylam Pharmaceuticals, Inc., H2 2014 27
Hemophilia B - Pipeline by CSL Limited, H2 2014 28
Hemophilia B - Pipeline by Catalyst Biosciences, Inc., H2 2014 29
Hemophilia B - Pipeline by Amarna Therapeutics B.V., H2 2014 30
Hemophilia B - Pipeline by AlphaMab Co., Ltd, H2 2014 31
Assessment by Monotherapy Products, H2 2014 32
Number of Products by Stage and Target, H2 2014 34
Number of Products by Stage and Mechanism of Action, H2 2014 36
Number of Products by Stage and Route of Administration, H2 2014 38
Number of Products by Stage and Molecule Type, H2 2014 40
Hemophilia B Therapeutics - Recent Pipeline Updates, H2 2014 71
Hemophilia B - Dormant Projects, H2 2014 85
Hemophilia B - Discontinued Products, H2 2014 86