Paroxysmal Nocturnal Hemoglobinuria – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Paroxysmal Nocturnal Hemoglobinuria – Pipeline Review, H2 2017’, 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

Companies mentioned

Achillion Pharmaceuticals Inc

Akari Therapeutics Plc

Alexion Pharmaceuticals Inc

Alnylam Pharmaceuticals Inc

Amgen Inc

Apellis Pharmaceuticals Inc

F. Hoffmann-La Roche Ltd

ISU ABXIS Co Ltd

Novartis AG

NovelMed Therapeutics Inc

Omeros Corp

Ra Pharmaceuticals Inc

Regeneron Pharmaceuticals Inc

Regenesance BV

Table of Contents

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

Paroxysmal Nocturnal Hemoglobinuria - Therapeutics Development 7

Pipeline Overview 7

Pipeline by Companies 8

Products under Development by Companies 10

Paroxysmal Nocturnal Hemoglobinuria - Therapeutics Assessment 12

Assessment by Target 12

Assessment by Mechanism of Action 14

Assessment by Route of Administration 16

Assessment by Molecule Type 18

Paroxysmal Nocturnal Hemoglobinuria - Companies Involved in Therapeutics Development 20

Achillion Pharmaceuticals Inc 20

Akari Therapeutics Plc 20

Alexion Pharmaceuticals Inc 21

Alnylam Pharmaceuticals Inc 21

Amgen Inc 22

Apellis Pharmaceuticals Inc 22

F. Hoffmann-La Roche Ltd 22

ISU ABXIS Co Ltd 23

Novartis AG 23

NovelMed Therapeutics Inc 24

Omeros Corp 24

Ra Pharmaceuticals Inc 25

Regeneron Pharmaceuticals Inc 25

Regenesance BV 26

Paroxysmal Nocturnal Hemoglobinuria - Drug Profiles 27

ACH-4471 - Drug Profile 27

ACH-5228 - Drug Profile 30

ALN-CC5 - Drug Profile 31

AMY-101 - Drug Profile 35

APL-2 - Drug Profile 37

APL-9 - Drug Profile 40

Coversin - Drug Profile 41

eculizumab biosimilar - Drug Profile 46

eculizumab biosimilar - Drug Profile 47

Gene Therapy for Paroxysmal Nocturnal Hemoglobinuria and Atypical Hemolytic Uremic Syndrome - Drug Profile 48

GNR-045 - Drug Profile 49

Monoclonal Antibody for Paroxysmal Nocturnal Hemoglobinuria - Drug Profile 50

NM-9405 - Drug Profile 51

OMS-721 - Drug Profile 52

OMS-906 - Drug Profile 62

RA-101348 - Drug Profile 63

RA-101495 - Drug Profile 64

ravulizumab - Drug Profile 67

Regenemab - Drug Profile 69

REGN-3918 - Drug Profile 70

RG-6107 - Drug Profile 71

Small Molecule to Inhibit Factor D for C3 glomerulopathy and Paroxysmal Nocturnal Hemoglobinuria - Drug Profile 72

Synthetic Peptide to Inhibit Complement C5 for Autoimmune Disorders, Atypical Hemolytic Uremic Syndrome, Central Nervous System and Paroxysmal Nocturnal Hemoglobinuria - Drug Profile 73

tesidolumab - Drug Profile 74

Paroxysmal Nocturnal Hemoglobinuria - Dormant Projects 76

Paroxysmal Nocturnal Hemoglobinuria - Discontinued Products 77

Paroxysmal Nocturnal Hemoglobinuria - Product Development Milestones 78

Featured News & Press Releases 78

Appendix 88

Methodology 88

Coverage 88

Secondary Research 88

Primary Research 88

Expert Panel Validation 88

Contact Us 88

Disclaimer 89

List of Tables

List of Tables

Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, H2 2017

Number of Products under Development by Companies, H2 2017

Products under Development by Companies, H2 2017

Products under Development by Companies, H2 2017 (Contd..1), H2 2017

Number of Products by Stage and Target, H2 2017

Number of Products by Stage and Mechanism of Action, H2 2017

Number of Products by Stage and Route of Administration, H2 2017

Number of Products by Stage and Molecule Type, H2 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Achillion Pharmaceuticals Inc, H2 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Akari Therapeutics Plc, H2 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Alexion Pharmaceuticals Inc, H2 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Alnylam Pharmaceuticals Inc, H2 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Amgen Inc, H2 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Apellis Pharmaceuticals Inc, H2 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by F. Hoffmann-La Roche Ltd, H2 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by ISU ABXIS Co Ltd, H2 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Novartis AG, H2 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by NovelMed Therapeutics Inc, H2 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Omeros Corp, H2 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Ra Pharmaceuticals Inc, H2 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Regeneron Pharmaceuticals Inc, H2 2017

Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Regenesance BV, H2 2017

Paroxysmal Nocturnal Hemoglobinuria – Dormant Projects, H2 2017

Paroxysmal Nocturnal Hemoglobinuria – Discontinued Products, H2 2017

List of Figures

List of Figures

Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products by Targets, H2 2017

Number of Products by Stage and Targets, H2 2017

Number of Products by Mechanism of Actions, H2 2017

Number of Products by Stage and Mechanism of Actions, H2 2017

Number of Products by Routes of Administration, H2 2017

Number of Products by Stage and Routes of Administration, H2 2017

Number of Products by Molecule Types, H2 2017

Number of Products by Stage and Molecule Types, H2 2017

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