Acquired (Autoimmune) Hemolytic Anemia - Pipeline Review, H1 2014

Acquired (Autoimmune) Hemolytic Anemia - Pipeline Review, H1 2014

Summary

Global Markets Direct’s, ‘Acquired (Autoimmune) Hemolytic Anemia - Pipeline Review, H1 2014’, provides an overview of the Acquired (Autoimmune) Hemolytic Anemia’s therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Acquired (Autoimmune) Hemolytic Anemia, 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 Acquired (Autoimmune) Hemolytic Anemia 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 Acquired (Autoimmune) Hemolytic Anemia
- 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 Acquired (Autoimmune) Hemolytic Anemia and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects
- A review of the Acquired (Autoimmune) Hemolytic Anemia 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 Acquired (Autoimmune) Hemolytic Anemia 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 Acquired (Autoimmune) Hemolytic Anemia
- Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
- Devise corrective measures for pipeline projects by understanding Acquired (Autoimmune) Hemolytic Anemia 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 4
List of Figures 4
Introduction 5
Global Markets Direct Report Coverage 5
Acquired (Autoimmune) Hemolytic Anemia Overview 6
Therapeutics Development 7
Pipeline Products for Acquired (Autoimmune) Hemolytic Anemia - Overview 7
Pipeline Products for Acquired (Autoimmune) Hemolytic Anemia - Comparative Analysis 8
Acquired (Autoimmune) Hemolytic Anemia - Therapeutics under Development by Companies 9
Acquired (Autoimmune) Hemolytic Anemia - Pipeline Products Glance 10
Clinical Stage Products 10
Early Stage Products 11
Unknown Stage Products 12
Acquired (Autoimmune) Hemolytic Anemia - Products under Development by Companies 13
Acquired (Autoimmune) Hemolytic Anemia - Companies Involved in Therapeutics Development 14
Shire Plc 14
Accentia Biopharmaceuticals, Inc. 15
Alexion Pharmaceuticals, Inc. 16
Agios Pharmaceuticals, Inc. 17
Acquired (Autoimmune) Hemolytic Anemia - Therapeutics Assessment 18
Assessment by Monotherapy Products 18
Assessment by Target 19
Assessment by Mechanism of Action 21
Assessment by Route of Administration 23
Assessment by Molecule Type 25
Drug Profiles 27
eculizumab - Drug Profile 27
Product Description 27
Mechanism of Action 27
R&D Progress 27
AG-348 - Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
C1 esterase inhibitor (human) - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
TNT-009 - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
cyclophosphamide - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
Acquired (Autoimmune) Hemolytic Anemia - Recent Pipeline Updates 37
Acquired (Autoimmune) Hemolytic Anemia - Product Development Milestones 50
Featured News & Press Releases 50
Apr 17, 2014: Agios Initiates Phase 1 Study of AG-348, a First-in-class PKR Activator, for Pyruvate Kinase Deficiency 50
Apr 14, 2014: True North Therapeutics Publishes Study in Blood Demonstrating Ex Vivo Efficacy of a C1s Antibody in Cold Agglutinin Disease Patient Samples 50
Dec 16, 2013: Soliris Concentrated Solution for Intravenous Infusion by Alexion: Recall - Visible Particles 51
Dec 13, 2013: Alexion Provides Update on Previously Communicated November 2013 Voluntary Nationwide Recall of Two Lots of Soliris Concentrated Solution for Intravenous Infusion 52
Dec 09, 2013: True North Therapeutics Presents Preclinical Results Showing Potential of TNT009 to Treat Complement-Mediated Rare Diseases 52
Jun 23, 2011: FDA Grants Orphan Drug Status For Accentia's Revimmune Therapy In Systemic Sclerosis And Hemolytic Anemia 53
Jun 11, 2011: Alexion Presents Additional Data At EHA Congress Describing Long-Term Efficacy And Survival Data With Sustained Soliris Therapy 54
May 23, 2011: Researchers To Present Nine Studies On Alexion's Soliris In Patients With PNH And aHUS At EHA Congress 55
Appendix 57
Methodology 57
Coverage 57
Secondary Research 57
Primary Research 57
Expert Panel Validation 57
Contact Us 58
Disclaimer 58

List of Tables
Number of Products under Development for Acquired (Autoimmune) Hemolytic Anemia, H1 2014 7
Number of Products under Development for Acquired (Autoimmune) Hemolytic Anemia - Comparative Analysis, H1 2014 8
Number of Products under Development by Companies, H1 2014 9
Comparative Analysis by Clinical Stage Development, H1 2014 10
Comparative Analysis by Early Stage Development, H1 2014 11
Comparative Analysis by Unknown Stage Development, H1 2014 12
Products under Development by Companies, H1 2014 13
Acquired (Autoimmune) Hemolytic Anemia - Pipeline by Shire Plc, H1 2014 14
Acquired (Autoimmune) Hemolytic Anemia - Pipeline by Accentia Biopharmaceuticals, Inc., H1 2014 15
Acquired (Autoimmune) Hemolytic Anemia - Pipeline by Alexion Pharmaceuticals, Inc., H1 2014 16
Acquired (Autoimmune) Hemolytic Anemia - Pipeline by Agios Pharmaceuticals, Inc., H1 2014 17
Assessment by Monotherapy Products, H1 2014 18
Number of Products by Stage and Target, H1 2014 20
Number of Products by Stage and Mechanism of Action, H1 2014 22
Number of Products by Stage and Route of Administration, H1 2014 24
Number of Products by Stage and Molecule Type, H1 2014 26
Acquired (Autoimmune) Hemolytic Anemia Therapeutics - Recent Pipeline Updates, H1 2014 37

List of Figures
Number of Products under Development for Acquired (Autoimmune) Hemolytic Anemia, H1 2014 7
Number of Products under Development for Acquired (Autoimmune) Hemolytic Anemia - Comparative Analysis, H1 2014 8
Number of Products under Development by Companies, H1 2014 9
Comparative Analysis by Clinical Stage Development, H1 2014 10
Assessment by Monotherapy Products, H1 2014 18
Number of Products by Top 10 Target, H1 2014 19
Number of Products by Stage and Top 10 Target, H1 2014 20
Number of Products by Top 10 Mechanism of Action, H1 2014 21
Number of Products by Stage and Top 10 Mechanism of Action, H1 2014 22
Number of Products by Top 10 Route of Administration, H1 2014 23
Number of Products by Stage and Top 10 Route of Administration, H1 2014 24
Number of Products by Top 10 Molecule Type, H1 2014 25
Number of Products by Stage and Top 10 Molecule Type, H1 2014 26