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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


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.


- 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

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