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Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H1 2016

Published: Mar 30, 2016 | Pages: 78 | Publisher: Global Markets Direct | Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H1 2016

Summary

Global Markets Direct's, 'Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H1 2016', provides an overview of the Paroxysmal Nocturnal Hemoglobinuria pipeline landscape. 

The report provides comprehensive information on the therapeutics under development for Paroxysmal Nocturnal Hemoglobinuria, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Paroxysmal Nocturnal Hemoglobinuria and features dormant 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, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

- The report provides a snapshot of the global therapeutic landscape of Paroxysmal Nocturnal Hemoglobinuria
- The report reviews pipeline therapeutics for Paroxysmal Nocturnal Hemoglobinuria by companies and universities/research institutes based on information derived from company and industry-specific sources 
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages 
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities 
- The report reviews key players involved Paroxysmal Nocturnal Hemoglobinuria therapeutics and enlists all their major and minor projects
- The report assesses Paroxysmal Nocturnal Hemoglobinuria therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type 
- The report summarizes all the dormant and discontinued pipeline projects 
- The report reviews latest news related to pipeline therapeutics for Paroxysmal Nocturnal Hemoglobinuria

Reasons to Buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage 
- Identify and understand important and diverse types of therapeutics under development for Paroxysmal Nocturnal Hemoglobinuria
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies 
- Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics
- 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 5 List of Figures 5 Introduction 6 Global Markets Direct Report Coverage 6 Paroxysmal Nocturnal Hemoglobinuria Overview 7 Therapeutics Development 8 Pipeline Products for Paroxysmal Nocturnal Hemoglobinuria - Overview 8 Paroxysmal Nocturnal Hemoglobinuria - Therapeutics under Development by Companies 9 Paroxysmal Nocturnal Hemoglobinuria - Pipeline Products Glance 10 Clinical Stage Products 10 Early Stage Products 11 Paroxysmal Nocturnal Hemoglobinuria - Products under Development by Companies 12 Paroxysmal Nocturnal Hemoglobinuria - Companies Involved in Therapeutics Development 13 Achillion Pharmaceuticals, Inc. 13 Akari Therapeutics, Plc 14 Alexion Pharmaceuticals, Inc. 15 Alnylam Pharmaceuticals, Inc. 16 Amyndas Pharmaceuticals LLC 17 Apellis Pharmaceuticals, Inc. 18 Epirus Biopharmaceuticals, Inc. 19 Novartis AG 20 Omeros Corporation 21 Ra Pharmaceuticals, Inc. 22 Regenesance BV 23 The International Biotechnology Center (IBC) Generium 24 Paroxysmal Nocturnal Hemoglobinuria - Therapeutics Assessment 25 Assessment by Monotherapy Products 25 Assessment by Target 26 Assessment by Mechanism of Action 28 Assessment by Route of Administration 30 Assessment by Molecule Type 32 Drug Profiles 34 ACH-4471 - Drug Profile 34 Product Description 34 Mechanism of Action 34 R&D Progress 34 ALN-CC5 - Drug Profile 35 Product Description 35 Mechanism of Action 35 R&D Progress 35 ALXN-1210 - Drug Profile 36 Product Description 36 Mechanism of Action 36 R&D Progress 36 AMY-101 - Drug Profile 37 Product Description 37 Mechanism of Action 37 R&D Progress 37 APL-2 - Drug Profile 39 Product Description 39 Mechanism of Action 39 R&D Progress 39 Coversin - Drug Profile 41 Product Description 41 Mechanism of Action 41 R&D Progress 41 eculizumab biosimilar - Drug Profile 43 Product Description 43 Mechanism of Action 43 R&D Progress 43 GNR-045 - Drug Profile 44 Product Description 44 Mechanism of Action 44 R&D Progress 44 Monoclonal Antibody for Paroxysmal Nocturnal Hemoglobinuria - Drug Profile 45 Product Description 45 Mechanism of Action 45 R&D Progress 45 NM-9405 - Drug Profile 46 Product Description 46 Mechanism of Action 46 R&D Progress 46 OMS-721 - Drug Profile 47 Product Description 47 Mechanism of Action 47 R&D Progress 47 OMS-906 - Drug Profile 49 Product Description 49 Mechanism of Action 49 R&D Progress 49 RA-101348 - Drug Profile 50 Product Description 50 Mechanism of Action 50 R&D Progress 50 RA-101495 - Drug Profile 51 Product Description 51 Mechanism of Action 51 R&D Progress 51 regenemab - Drug Profile 52 Product Description 52 Mechanism of Action 52 R&D Progress 52 tesidolumab - Drug Profile 53 Product Description 53 Mechanism of Action 53 R&D Progress 53 Paroxysmal Nocturnal Hemoglobinuria - Recent Pipeline Updates 55 Paroxysmal Nocturnal Hemoglobinuria - Dormant Projects 65 Paroxysmal Nocturnal Hemoglobinuria - Discontinued Products 66 Paroxysmal Nocturnal Hemoglobinuria - Product Development Milestones 67 Featured News & Press Releases 67 Feb 11, 2016: Achillion Initiates Phase 1 Study of ACH-4471, First Orally-Administered Small Molecule Complement Factor D Inhibitor 67 Jan 29, 2016: Akari Therapeutics Receives Approval From the UK Medicines & Healthcare Products Regulatory Agency for Phase IB Multiple Ascending Dose Trial 68 Jan 05, 2016: Akari Therapeutics Announces Additional Data from Non-Human Primate Safety Study Demonstrating Equivalent Coversin Efficacy in Both Elisa CH50 and Hemolytic SRBC Assays 68 Dec 07, 2015: Akari Therapeutics Announces Update From Non-Human Primate Safety Study at International PNH Interest Group 10th Annual Scientific Meeting 69 Dec 05, 2015: Achillion Announces Upcoming Oral Presentation at the 57th Annual Meeting of the American Society of Hematology and Nomination of ACH-4471, a Novel Small Molecule Factor D Inhibitor, for Clinical Development 70 Dec 02, 2015: Ra Pharmaceuticals Announces Initiation of First-in-Human Clinical Trial of Novel Complement C5 Inhibitor 71 Nov 05, 2015: Akari Therapeutics Announces Upcoming Data Presentations at the 57th American Society of Hematology Annual Meeting 72 Dec 04, 2014: Ra Pharmaceuticals Announces Presentation of Data Supporting Clinical Development of Novel Complement C5 Inhibitor at 2014 ASH Annual Meeting 72 Mar 28, 2013: Omeros Announces Toxicology Data That Support Advancing MASP-2 Inhibitor Into Clinical Trials 74 Feb 07, 2013: Omeros Announces Toxicology Study Data From MASP-2 Inhibitor 75 Appendix 77 Methodology 77 Coverage 77 Secondary Research 77 Primary Research 77 Expert Panel Validation 77 Contact Us 77 Disclaimer 78
List of Tables
Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, H1 2016 8 Number of Products under Development by Companies, H1 2016 9 Comparative Analysis by Clinical Stage Development, H1 2016 10 Comparative Analysis by Early Stage Development, H1 2016 11 Products under Development by Companies, H1 2016 12 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Achillion Pharmaceuticals, Inc., H1 2016 13 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Akari Therapeutics, Plc, H1 2016 14 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Alexion Pharmaceuticals, Inc., H1 2016 15 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Alnylam Pharmaceuticals, Inc., H1 2016 16 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Amyndas Pharmaceuticals LLC, H1 2016 17 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Apellis Pharmaceuticals, Inc., H1 2016 18 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Epirus Biopharmaceuticals, Inc., H1 2016 19 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Novartis AG, H1 2016 20 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Omeros Corporation, H1 2016 21 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Ra Pharmaceuticals, Inc., H1 2016 22 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Regenesance BV, H1 2016 23 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by The International Biotechnology Center (IBC) Generium, H1 2016 24 Assessment by Monotherapy Products, H1 2016 25 Number of Products by Stage and Target, H1 2016 27 Number of Products by Stage and Mechanism of Action, H1 2016 29 Number of Products by Stage and Route of Administration, H1 2016 31 Number of Products by Stage and Molecule Type, H1 2016 33 Paroxysmal Nocturnal Hemoglobinuria Therapeutics - Recent Pipeline Updates, H1 2016 55 Paroxysmal Nocturnal Hemoglobinuria - Dormant Projects, H1 2016 65 Paroxysmal Nocturnal Hemoglobinuria - Discontinued Products, H1 2016 66


List of Figures
Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, H1 2016 8 Number of Products under Development by Companies, H1 2016 9 Comparative Analysis by Clinical Stage Development, H1 2016 10 Assessment by Monotherapy Products, H1 2016 25 Number of Products by Targets, H1 2016 26 Number of Products by Stage and Targets, H1 2016 26 Number of Products by Mechanism of Actions, H1 2016 28 Number of Products by Stage and Mechanism of Actions, H1 2016 28 Number of Products by Routes of Administration, H1 2016 30 Number of Products by Stage and Routes of Administration, H1 2016 30 Number of Products by Molecule Types, H1 2016 32 Number of Products by Stage and Molecule Types, H1 2016 32

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