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Hereditary Angioedema (HAE) - Pipeline Review, H2 2014

  • November 2014
  • -
  • Global Markets Direct
  • -
  • 77 pages

Hereditary Angioedema (HAE) - Pipeline Review, H2 2014

Summary

Global Markets Direct’s, ‘Hereditary Angioedema (HAE) - Pipeline Review, H2 2014’, provides an overview of the Hereditary Angioedema (HAE)’s therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Hereditary Angioedema (HAE), 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 Hereditary Angioedema (HAE) 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 Hereditary Angioedema (HAE)
- 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 Hereditary Angioedema (HAE) and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects
- A review of the Hereditary Angioedema (HAE) 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 Hereditary Angioedema (HAE) 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 Hereditary Angioedema (HAE)
- Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
- Devise corrective measures for pipeline projects by understanding Hereditary Angioedema (HAE) 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

Hereditary Angioedema (HAE) - Pipeline Review, H2 2014
Table of Contents

Introduction 6
Global Markets Direct Report Coverage 6
Hereditary Angioedema (HAE) Overview 7
Therapeutics Development 8
Pipeline Products for Hereditary Angioedema (HAE) - Overview 8
Pipeline Products for Hereditary Angioedema (HAE) - Comparative Analysis 9
Hereditary Angioedema (HAE) - Therapeutics under Development by Companies 10
Hereditary Angioedema (HAE) - Pipeline Products Glance 12
Late Stage Products 12
Clinical Stage Products 13
Early Stage Products 14
Hereditary Angioedema (HAE) - Products under Development by Companies 15
Hereditary Angioedema (HAE) - Companies Involved in Therapeutics Development 16
BioCryst Pharmaceuticals, Inc. 16
Cevec Pharmaceuticals GmbH 17
CSL Limited 18
Dyax Corp. 19
Global Blood Therapeutics, Inc. 20
iBio, Inc. 21
Isis Pharmaceuticals, Inc. 22
Pharming Group N.V. 23
Ra Pharmaceuticals, Inc. 24
Shire Plc 25
Hereditary Angioedema (HAE) - Therapeutics Assessment 26
Assessment by Monotherapy Products 26
Assessment by Target 27
Assessment by Mechanism of Action 29
Assessment by Route of Administration 31
Assessment by Molecule Type 33
Drug Profiles 35
BCX-4161 - Drug Profile 35
Product Description 35
Mechanism of Action 35
RandD Progress 35
BEL-0215 - Drug Profile 36
Product Description 36
Mechanism of Action 36
RandD Progress 36
C1 esterase inhibitor (human) - Drug Profile 37
Product Description 37
Mechanism of Action 37
RandD Progress 37
C1 esterase inhibitor (human) - Drug Profile 39
Product Description 39
Mechanism of Action 39
RandD Progress 39
C1 esterase inhibitor (human) - Drug Profile 41
Product Description 41
Mechanism of Action 41
RandD Progress 41
C1 esterase inhibitor (recombinant) - Drug Profile 42
Product Description 42
Mechanism of Action 42
RandD Progress 42
CSL-3F7 - Drug Profile 47
Product Description 47
Mechanism of Action 47
RandD Progress 47
Cyclomimetic - Drug Profile 48
Product Description 48
Mechanism of Action 48
RandD Progress 48
DX-2930 - Drug Profile 49
Product Description 49
Mechanism of Action 49
RandD Progress 49
icatibant acetate - Drug Profile 50
Product Description 50
Mechanism of Action 50
RandD Progress 50
ISIS-PKKRx - Drug Profile 52
Product Description 52
Mechanism of Action 52
RandD Progress 52
Peptide to Inhibit Kallikrein for Hereditary Angioedema - Drug Profile 53
Product Description 53
Mechanism of Action 53
RandD Progress 53
Recombinant Protein to Inhibit C1-esterase for Hereditary Angioedema - Drug Profile 54
Product Description 54
Mechanism of Action 54
RandD Progress 54
Small Molecule 1 to Inhibit Plasma Kallikrein for Hereditary Angioedema - Drug Profile 55
Product Description 55
Mechanism of Action 55
RandD Progress 55
Small Molecule 2 to Inhibit Plasma Kallikrein for Hereditary Angioedema - Drug Profile 56
Product Description 56
Mechanism of Action 56
RandD Progress 56
Small Molecule for Hereditary Angioedema - Drug Profile 57
Product Description 57
Mechanism of Action 57
RandD Progress 57
Hereditary Angioedema (HAE) - Recent Pipeline Updates 58
Hereditary Angioedema (HAE) - Dormant Projects 68
Hereditary Angioedema (HAE) - Product Development Milestones 69
Featured News and Press Releases 69
Nov 04, 2014: Pharming Confirms Receipt Of US$20 Million Milestone Payment From Salix Pharmaceuticals 69
Nov 03, 2014: Salix and Pharming Announce the Launch of RUCONEST in the U.S. for the Treatment of Acute Angioedema Attacks in Patients with Hereditary Angioedema (HAE) 69
Nov 03, 2014: Dyax Announces Expansion of Phase 1b Clinical Trial for DX-2930 70
Oct 13, 2014: Pharming and Swedish Orphan Biovitrum Amend and Extend Ruconest Distribution Agreement 71
Oct 07, 2014: BioCryst Announces Late Breaker Presentation of OPuS-1 Phase 2 Trial Results at the 23rd EADV Congress 71
Sep 15, 2014: Dyax Announces Issuance of Two Key U.S. Patents for DX-2930 72
Aug 28, 2014: Pharming and Salix Announce Initiation of Clinical Study of RUCONEST for Prophylaxis of Hereditary Angioedema 72
Jul 30, 2014: Study Suggests C1-INH May Aid in Prevention of Antibody-Mediated Rejection Following Kidney Transplant 73
Jul 23, 2014: Dyax Corp. Announces Publication of Scientific Data for DX-2930 in Peer-Reviewed Journals 74
Jul 17, 2014: Salix and Pharming Announce FDA Approval of RUCONEST for the Treatment of Acute Angioedema Attacks in Patients with Hereditary Angioedema 74
Appendix 76
Methodology 76
Coverage 76
Secondary Research 76
Primary Research 76
Expert Panel Validation 76
Contact Us 77
Disclaimer 77

List of Tables

Number of Products under Development for Hereditary Angioedema (HAE), H2 2014 8
Number of Products under Development for Hereditary Angioedema (HAE) - Comparative Analysis, H2 2014 9
Number of Products under Development by Companies, H2 2014 11
Comparative Analysis by Late Stage Development, H2 2014 12
Comparative Analysis by Clinical Stage Development, H2 2014 13
Comparative Analysis by Early Stage Development, H2 2014 14
Products under Development by Companies, H2 2014 15
Hereditary Angioedema (HAE) - Pipeline by BioCryst Pharmaceuticals, Inc., H2 2014 16
Hereditary Angioedema (HAE) - Pipeline by Cevec Pharmaceuticals GmbH, H2 2014 17
Hereditary Angioedema (HAE) - Pipeline by CSL Limited, H2 2014 18
Hereditary Angioedema (HAE) - Pipeline by Dyax Corp., H2 2014 19
Hereditary Angioedema (HAE) - Pipeline by Global Blood Therapeutics, Inc., H2 2014 20
Hereditary Angioedema (HAE) - Pipeline by iBio, Inc., H2 2014 21
Hereditary Angioedema (HAE) - Pipeline by Isis Pharmaceuticals, Inc., H2 2014 22
Hereditary Angioedema (HAE) - Pipeline by Pharming Group N.V., H2 2014 23
Hereditary Angioedema (HAE) - Pipeline by Ra Pharmaceuticals, Inc., H2 2014 24
Hereditary Angioedema (HAE) - Pipeline by Shire Plc, H2 2014 25
Assessment by Monotherapy Products, H2 2014 26
Number of Products by Stage and Target, H2 2014 28
Number of Products by Stage and Mechanism of Action, H2 2014 30
Number of Products by Stage and Route of Administration, H2 2014 32
Number of Products by Stage and Molecule Type, H2 2014 34
Hereditary Angioedema (HAE) Therapeutics - Recent Pipeline Updates, H2 2014 58
Hereditary Angioedema (HAE) - Dormant Projects, H2 2014 68

List of Figures

Number of Products under Development for Hereditary Angioedema (HAE), H2 2014 8
Number of Products under Development for Hereditary Angioedema (HAE) - Comparative Analysis, H2 2014 9
Number of Products under Development by Companies, H2 2014 10
Comparative Analysis by Clinical Stage Development, H2 2014 13
Comparative Analysis by Early Stage Products, H2 2014 14
Assessment by Monotherapy Products, H2 2014 26
Number of Products by Top 10 Targets, H2 2014 27
Number of Products by Stage and Top 10 Targets, H2 2014 28
Number of Products by Top 10 Mechanism of Actions, H2 2014 29
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2014 30
Number of Products by Top 10 Routes of Administration, H2 2014 31
Number of Products by Stage and Top 10 Routes of Administration, H2 2014 32
Number of Products by Top 10 Molecule Types, H2 2014 33
Number of Products by Stage and Top 10 Molecule Types, H2 2014 34

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