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Acquired (Autoimmune) Hemolytic Anemia-Pipeline Review, H1 2015

Acquired (Autoimmune) Hemolytic Anemia-Pipeline Review, H1 2015


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Executive Summary

Acquired (Autoimmune) Hemolytic Anemia-Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Acquired (Autoimmune) Hemolytic Anemia-Pipeline Review, H1 2015', 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

Agios Pharmaceuticals, Inc. 14

Alexion Pharmaceuticals, Inc. 15

Hansa Medical AB 16

Shire Plc 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

AG-348-Drug Profile 27

Product Description 27

Mechanism of Action 27

R&D Progress 27

C1 esterase inhibitor (human)-Drug Profile 28

Product Description 28

Mechanism of Action 28

R&D Progress 28

eculizumab-Drug Profile 30

Product Description 30

Mechanism of Action 30

R&D Progress 30

Endoglycosidase of Streptococcus pyogenes-Drug Profile 33

Product Description 33

Mechanism of Action 33

R&D Progress 33

TNT-003-Drug Profile 34

Product Description 34

Mechanism of Action 34

R&D Progress 34

TNT-009-Drug Profile 35

Product Description 35

Mechanism of Action 35

R&D Progress 35

Acquired (Autoimmune) Hemolytic Anemia-Recent Pipeline Updates 36

Acquired (Autoimmune) Hemolytic Anemia-Dormant Projects 46

Acquired (Autoimmune) Hemolytic Anemia-Product Development Milestones 47

Featured News & Press Releases 47

Dec 08, 2014: Agios Reports Positive Phase 1 Data in Healthy Volunteers for AG-348, a First-in-Class Investigational Medicine That Targets the Underlying Cause of Pyruvate Kinase (PK) Deficiency 47

Nov 06, 2014: Agios Pharmaceuticals To Present Phase 1 Data On AG-348 At The 2014 American Society Of Hematology Annual Meeting 48

Oct 06, 2014: Agios Pharmaceuticals to Provides Update on AG-348 Webcast R&D Day on October 15, 2014 49

Jun 09, 2014: Agios Pharmaceuticals Initiates Multiple Ascending Dose Trial in Healthy Volunteers of AG-348 for the Potential Treatment of PK Deficiency, a Rare, Hemolytic Anemia 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 51

Dec 16, 2013: Soliris Concentrated Solution for Intravenous Infusion by Alexion: Recall-Visible Particles 52

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 53

Jun 11, 2011: Alexion Presents Additional Data At EHA Congress Describing Long-Term Efficacy And Survival Data With Sustained Soliris Therapy 54

Appendix 56

Methodology 56

Coverage 56

Secondary Research 56

Primary Research 56

Expert Panel Validation 56

Contact Us 56

Disclaimer 57

List of Tables

Number of Products under Development for Acquired (Autoimmune) Hemolytic Anemia, H1 2015 7

Number of Products under Development for Acquired (Autoimmune) Hemolytic Anemia-Comparative Analysis, H1 2015 8

Number of Products under Development by Companies, H1 2015 9

Comparative Analysis by Clinical Stage Development, H1 2015 10

Comparative Analysis by Early Stage Development, H1 2015 11

Comparative Analysis by Unknown Stage Development, H1 2015 12

Products under Development by Companies, H1 2015 13

Acquired (Autoimmune) Hemolytic Anemia-Pipeline by Agios Pharmaceuticals, Inc., H1 2015 14

Acquired (Autoimmune) Hemolytic Anemia-Pipeline by Alexion Pharmaceuticals, Inc., H1 2015 15

Acquired (Autoimmune) Hemolytic Anemia-Pipeline by Hansa Medical AB, H1 2015 16

Acquired (Autoimmune) Hemolytic Anemia-Pipeline by Shire Plc, H1 2015 17

Assessment by Monotherapy Products, H1 2015 18

Number of Products by Stage and Target, H1 2015 20

Number of Products by Stage and Mechanism of Action, H1 2015 22

Number of Products by Stage and Route of Administration, H1 2015 24

Number of Products by Stage and Molecule Type, H1 2015 26

Acquired (Autoimmune) Hemolytic Anemia Therapeutics-Recent Pipeline Updates, H1 2015 36

Acquired (Autoimmune) Hemolytic Anemia-Dormant Projects, H1 2015 46

List of Figures

Number of Products under Development for Acquired (Autoimmune) Hemolytic Anemia, H1 2015 7

Number of Products under Development for Acquired (Autoimmune) Hemolytic Anemia-Comparative Analysis, H1 2015 8

Number of Products under Development by Companies, H1 2015 9

Comparative Analysis by Clinical Stage Development, H1 2015 10

Comparative Analysis by Early Stage Products, H1 2015 11

Assessment by Monotherapy Products, H1 2015 18

Number of Products by Top 10 Targets, H1 2015 19

Number of Products by Stage and Top 10 Targets, H1 2015 20

Number of Products by Top 10 Mechanism of Actions, H1 2015 21

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015 22

Number of Products by Top 10 Routes of Administration, H1 2015 23

Number of Products by Stage and Top 10 Routes of Administration, H1 2015 24

Number of Products by Top 10 Molecule Types, H1 2015 25

Number of Products by Stage and Top 10 Molecule Types, H1 2015 26

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Agios Pharmaceuticals, Inc.

Alexion Pharmaceuticals, Inc.

Hansa Medical AB

Shire Plc

Acquired (Autoimmune) Hemolytic Anemia Therapeutic Products under Development, Key Players in Acquired (Autoimmune) Hemolytic Anemia Therapeutics, Acquired (Autoimmune) Hemolytic Anemia Pipeline Overview, Acquired (Autoimmune) Hemolytic Anemia Pipeline, Acquired (Autoimmune) Hemolytic Anemia Pipeline Assessment


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