Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H2 2014

Published: Sep, 2014 | Pages: 65 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)

Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H2 2014

Summary

Global Markets Direct's, 'Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H2 2014', provides an overview of the Paroxysmal Nocturnal Hemoglobinuria's therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Paroxysmal Nocturnal Hemoglobinuria, 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 Paroxysmal Nocturnal Hemoglobinuria 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 Paroxysmal Nocturnal Hemoglobinuria
- 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 Paroxysmal Nocturnal Hemoglobinuria and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects 
- A review of the Paroxysmal Nocturnal Hemoglobinuria 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 Paroxysmal Nocturnal Hemoglobinuria 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 Paroxysmal Nocturnal Hemoglobinuria
- Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
- Devise corrective measures for pipeline projects by understanding Paroxysmal Nocturnal Hemoglobinuria 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
Paroxysmal Nocturnal Hemoglobinuria Overview 6
Therapeutics Development 7
Pipeline Products for Paroxysmal Nocturnal Hemoglobinuria - Overview 7
Pipeline Products for Paroxysmal Nocturnal Hemoglobinuria - Comparative Analysis 8
Paroxysmal Nocturnal Hemoglobinuria - Therapeutics under Development by Companies 9
Paroxysmal Nocturnal Hemoglobinuria - Pipeline Products Glance 11
Clinical Stage Products 11
Early Stage Products 12
Unknown Stage Products 13
Paroxysmal Nocturnal Hemoglobinuria - Products under Development by Companies 14
Paroxysmal Nocturnal Hemoglobinuria - Companies Involved in Therapeutics Development 15
Shire Plc 15
Alnylam Pharmaceuticals, Inc. 16
MABLife S.A.S 17
Omeros Corporation 18
Apellis Pharmaceuticals, Inc. 19
Paroxysmal Nocturnal Hemoglobinuria - Therapeutics Assessment 20
Assessment by Monotherapy Products 20
Assessment by Target 21
Assessment by Mechanism of Action 23
Assessment by Route of Administration 25
Assessment by Molecule Type 27
Drug Profiles 29
Recombinant Coversin - Drug Profile 29
Product Description 29
Mechanism of Action 29
R&D Progress 29
APL-2 - 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
Long Acting APL-1 - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
Cp-40 - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
ALN-CC5 - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
Monoclonal Antibody for Paroxysmal Nocturnal Hemoglobinuria - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
MAT-303 - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
C1 esterase inhibitor (human) - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
Paroxysmal Nocturnal Hemoglobinuria - Recent Pipeline Updates 42
Paroxysmal Nocturnal Hemoglobinuria - Dormant Projects 49
Paroxysmal Nocturnal Hemoglobinuria - Product Development Milestones 50
Featured News & Press Releases 50
Dec 13, 2013: Alexion Provides Update on Previously Communicated November 2013 Voluntary Nationwide Recall of Two Lots of Soliris Concentrated Solution for Intravenous Infusion 50
Dec 08, 2013: New Data Presented at ASH Annual Meeting Enhance Understanding of PNH and aHUS to Provide Optimal Care for Patients with These Life-threatening Disorders 50
Jun 20, 2013: Alnylam Scientists Present Preclinical Data With ALN-CC5 For Treatment Of Complement-mediated Diseases 54
Mar 28, 2013: Alexion Pharma Receives FDA Warning Letter For Manufacturing Facility In Rhode Island, US 55
Mar 28, 2013: Omeros Announces Toxicology Data That Support Advancing MASP-2 Inhibitor Into Clinical Trials 56
Mar 22, 2013: Alexion Receives CHMP Positive Opinion For Soliris 57
Feb 07, 2013: Omeros Announces Toxicology Study Data From MASP-2 Inhibitor 58
Dec 10, 2012: Alexion Pharma Presents New Data Highlighting Long-Term Efficacy And Safety Outcomes Of Soliris In Paroxysmal Nocturnal Hemoglobinuria Patients At ASH Annual Meeting 59
Nov 07, 2012: Alexion Pharma Announces Presentation Of Additional Data On Soliris As Treatment For Patients With PNH And aHUS At ASH Annual Meeting 61
Jun 17, 2012: Alexion's Soliris Demonstrates Efficacy In Pediatric Patients With Atypical Hemolytic Uremic Syndrome 62
Appendix 64
Methodology 64
Coverage 64
Secondary Research 64
Primary Research 64
Expert Panel Validation 64
Contact Us 65
Disclaimer 65

List of Tables
Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, H2 2014 7
Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria - Comparative Analysis, H2 2014 8
Number of Products under Development by Companies, H2 2014 10
Comparative Analysis by Clinical Stage Development, H2 2014 11
Comparative Analysis by Early Stage Development, H2 2014 12
Comparative Analysis by Unknown Stage Development, H2 2014 13
Products under Development by Companies, H2 2014 14
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Shire Plc, H2 2014 15
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Alnylam Pharmaceuticals, Inc., H2 2014 16
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by MABLife S.A.S, H2 2014 17
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Omeros Corporation, H2 2014 18
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Apellis Pharmaceuticals, Inc., H2 2014 19
Assessment by Monotherapy Products, H2 2014 20
Number of Products by Stage and Target, H2 2014 22
Number of Products by Stage and Mechanism of Action, H2 2014 24
Number of Products by Stage and Route of Administration, H2 2014 26
Number of Products by Stage and Molecule Type, H2 2014 28
Paroxysmal Nocturnal Hemoglobinuria Therapeutics - Recent Pipeline Updates, H2 2014 42
Paroxysmal Nocturnal Hemoglobinuria - Dormant Projects, H2 2014 49