Hemophilia B - Pipeline Review, H2 2016

Published: Sep, 2016 | Pages: 112 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Hemophilia B - Pipeline Review, H2 2016, provides an overview of the Hemophilia B (Hematological Disorders) pipeline landscape.

Hemophilia B is a hereditary bleeding disorder caused by a lack of blood clotting factor IX. Symptoms include bleeding into joints and associated pain and swelling, bruising, excessive bleeding following circumcision, gastrointestinal tract and urinary tract hemorrhage and nosebleeds. Treatment includes replacing the defective clotting factor.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Hemophilia B - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Hemophilia B (Hematological Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Hemophilia B (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Hemophilia B and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, IND/CTA Filed, Preclinical and Discovery stages are 2, 3, 6, 6, 1, 6 and 1 respectively for Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 1 and 1 molecules, respectively for Hemophilia B.

Hemophilia B (Hematological Disorders) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Hemophilia B (Hematological Disorders).
- The pipeline guide reviews pipeline therapeutics for Hemophilia B (Hematological Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Hemophilia B (Hematological Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Hemophilia B (Hematological Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Hemophilia B (Hematological Disorders)

Reasons To Buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Hemophilia B (Hematological Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Hemophilia B (Hematological Disorders) 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what 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 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

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