Paroxysmal Nocturnal Hemoglobinuria-Pipeline Review, H1 2016

Paroxysmal Nocturnal Hemoglobinuria-Pipeline Review, H1 2016

  • Products Id :- GMDHC7736IDB
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  • Pages: 78
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Executive 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.


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

Read More

Contact Us:
Ken Research
Ankur Gupta, Head Marketing & Communications


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 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

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

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Achillion Pharmaceuticals, Inc. Akari Therapeutics, Plc Alexion Pharmaceuticals, Inc. Alnylam Pharmaceuticals, Inc. Amyndas Pharmaceuticals LLC Apellis Pharmaceuticals, Inc. Epirus Biopharmaceuticals, Inc. Novartis AG Omeros Corporation Ra Pharmaceuticals, Inc. Regenesance BV The International Biotechnology Center (IBC) Generium

Paroxysmal Nocturnal Hemoglobinuria Therapeutic Products under Development, Key Players in Paroxysmal Nocturnal Hemoglobinuria Therapeutics, Paroxysmal Nocturnal Hemoglobinuria Pipeline Overview, Paroxysmal Nocturnal Hemoglobinuria Pipeline, Paroxysmal Nocturnal Hemoglobinuria Pipeline Assessment

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