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Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Pipeline Review, H1 2015

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Pipeline Review, H1 2015


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Executive Summary

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Pipeline Review, H1 2015', provides an overview of the Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)'s therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome), 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 Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome) 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 Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)

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 Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome) and enlists all their major and minor projects

The report summarizes all the dormant and discontinued pipeline projects

A review of the Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome) 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 Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome) 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 Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)

Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline

Devise corrective measures for pipeline projects by understanding Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome) 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

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome) Overview 6

Therapeutics Development 7

Pipeline Products for Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Overview 7

Pipeline Products for Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Comparative Analysis 8

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Therapeutics under Development by Companies 9

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Pipeline Products Glance 10

Clinical Stage Products 10

Early Stage Products 11

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Products under Development by Companies 12

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Companies Involved in Therapeutics Development 13

Adienne Pharma & Biotech 13

Alnylam Pharmaceuticals, Inc. 14

ChemoCentryx, Inc. 15

Omeros Corporation 16

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Therapeutics Assessment 17

Assessment by Monotherapy Products 17

Assessment by Target 18

Assessment by Mechanism of Action 20

Assessment by Route of Administration 22

Assessment by Molecule Type 24

Drug Profiles 26

ALN-CC5-Drug Profile 26

Product Description 26

Mechanism of Action 26

R&D Progress 26

CCX-168-Drug Profile 27

Product Description 27

Mechanism of Action 27

R&D Progress 27

ET-006-Drug Profile 29

Product Description 29

Mechanism of Action 29

R&D Progress 29

ETR-001-Drug Profile 30

Product Description 30

Mechanism of Action 30

R&D Progress 30

Mubodina-Drug Profile 31

Product Description 31

Mechanism of Action 31

R&D Progress 31

OMS-721-Drug Profile 32

Product Description 32

Mechanism of Action 32

R&D Progress 32

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Recent Pipeline Updates 34

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Dormant Projects 41

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Product Development Milestones 42

Featured News & Press Releases 42

Nov 20, 2014: ChemoCentryx's CCX168, an Orally Administered Complement C5a Receptor Inhibitor, Granted Orphan-Drug Designation in Atypical Hemolytic Uremic Syndrome Treatment by the FDA 42

Nov 19, 2014: OMS721 Inhibits Thrombus Formation in Sera from aHUS Patients 42

Apr 03, 2014: Investigational New Drug Application Cleared by FDA for OMS721 in Thrombotic Microangiopathies 43

Mar 10, 2014: Omeros Announces Positive OMS721 Data in Serum from Patients with aHUS 44

Mar 04, 2014: Omeros Submits IND to Advance OMS721 Phase 2 Clinical Program 45

Feb 03, 2014: Omeros Announces Positive Data after Completion of Dosing in Phase 1 Clinical Trial with OMS721 47

Dec 18, 2013: FDA Grants Orphan Drug Designation to Omeros' OMS721 for Complement-Mediated Thrombotic Microangiopathies 47

Jun 20, 2013: Alnylam Scientists Present Preclinical Data With ALN-CC5 For Treatment Of Complement-mediated Diseases 48

Jun 13, 2013: Omeros Obtains Regulatory Approval To Initiate Phase I Clinical Trial For Lead Antibody In MASP-2 Program 50

May 28, 2013: Omeros Files Clinical Trial Application For Lead Antibody In MASP-2 Program 51

Appendix 53

Methodology 53

Coverage 53

Secondary Research 53

Primary Research 53

Expert Panel Validation 53

Contact Us 54

Disclaimer 54

List of Tables

Number of Products under Development for Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome), H1 2015 7

Number of Products under Development for Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-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

Products under Development by Companies, H1 2015 12

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Pipeline by Adienne Pharma & Biotech, H1 2015 13

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Pipeline by Alnylam Pharmaceuticals, Inc., H1 2015 14

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Pipeline by ChemoCentryx, Inc., H1 2015 15

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Pipeline by Omeros Corporation, H1 2015 16

Assessment by Monotherapy Products, H1 2015 17

Number of Products by Stage and Target, H1 2015 19

Number of Products by Stage and Mechanism of Action, H1 2015 21

Number of Products by Stage and Route of Administration, H1 2015 23

Number of Products by Stage and Molecule Type, H1 2015 25

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome) Therapeutics-Recent Pipeline Updates, H1 2015 34

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Dormant Projects, H1 2015 41

List of Figures

Number of Products under Development for Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome), H1 2015 7

Number of Products under Development for Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome)-Comparative Analysis, H1 2015 8

Number of Products under Development by Companies, H1 2015 9

Comparative Analysis by Early Stage Products, H1 2015 11

Assessment by Monotherapy Products, H1 2015 17

Number of Products by Top 10 Targets, H1 2015 18

Number of Products by Stage and Top 10 Targets, H1 2015 19

Number of Products by Top 10 Mechanism of Actions, H1 2015 20

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

Number of Products by Top 10 Routes of Administration, H1 2015 22

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

Number of Products by Top 10 Molecule Types, H1 2015 24

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

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Adienne Pharma & Biotech

Alnylam Pharmaceuticals, Inc.

ChemoCentryx, Inc.

Omeros Corporation

Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome) Therapeutic Products under Development, Key Players in Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome) Therapeutics, Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome) Pipeline Overview, Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome) Pipeline, Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome) Pipeline Assessment


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