Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H2 2016

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Date: 14-Sep-2016
No. of pages: 87
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Global Markets Direct's, ‘Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H2 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

Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H2 2016

Table of Contents

Table of Contents 2
List of Tables 6
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Paroxysmal Nocturnal Hemoglobinuria Overview 8
Therapeutics Development 9
Pipeline Products for Paroxysmal Nocturnal Hemoglobinuria - Overview 9
Paroxysmal Nocturnal Hemoglobinuria - Therapeutics under Development by Companies 10
Paroxysmal Nocturnal Hemoglobinuria - Pipeline Products Glance 11
Clinical Stage Products 11
Early Stage Products 12
Paroxysmal Nocturnal Hemoglobinuria - Products under Development by Companies 13
Paroxysmal Nocturnal Hemoglobinuria - Companies Involved in Therapeutics Development 14
Achillion Pharmaceuticals, Inc. 14
Akari Therapeutics, Plc 15
Alexion Pharmaceuticals Inc 16
Alnylam Pharmaceuticals, Inc. 17
Amgen Inc. 18
Amyndas Pharmaceuticals LLC 19
Apellis Pharmaceuticals Inc 20
Epirus Biopharmaceuticals, Inc. 21
Novartis AG 22
Omeros Corporation 23
Ra Pharmaceuticals, Inc. 24
Regenesance BV 25
The International Biotechnology Center (IBC) Generium 26
Paroxysmal Nocturnal Hemoglobinuria - Therapeutics Assessment 27
Assessment by Monotherapy Products 27
Assessment by Target 28
Assessment by Mechanism of Action 30
Assessment by Route of Administration 32
Assessment by Molecule Type 34
Drug Profiles 36
ACH-4471 - Drug Profile 36
Product Description 36
Mechanism Of Action 36
R&D Progress 36
ALN-CC5 - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
ALXN-1210 - Drug Profile 42
Product Description 42
Mechanism Of Action 42
R&D Progress 42
AMY-101 - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
APL-2 - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
APL-9 - Drug Profile 48
Product Description 48
Mechanism Of Action 48
R&D Progress 48
Coversin - Drug Profile 49
Product Description 49
Mechanism Of Action 49
R&D Progress 49
eculizumab biosimilar - Drug Profile 52
Product Description 52
Mechanism Of Action 52
R&D Progress 52
eculizumab biosimilar - Drug Profile 53
Product Description 53
Mechanism Of Action 53
R&D Progress 53
GNR-045 - Drug Profile 54
Product Description 54
Mechanism Of Action 54
R&D Progress 54
Monoclonal Antibody for Paroxysmal Nocturnal Hemoglobinuria - Drug Profile 55
Product Description 55
Mechanism Of Action 55
R&D Progress 55
NM-9405 - Drug Profile 56
Product Description 56
Mechanism Of Action 56
R&D Progress 56
OMS-721 - Drug Profile 57
Product Description 57
Mechanism Of Action 57
R&D Progress 57
OMS-906 - Drug Profile 63
Product Description 63
Mechanism Of Action 63
R&D Progress 63
RA-101348 - Drug Profile 64
Product Description 64
Mechanism Of Action 64
R&D Progress 64
RA-101495 - Drug Profile 65
Product Description 65
Mechanism Of Action 65
R&D Progress 65
regenemab - Drug Profile 67
Product Description 67
Mechanism Of Action 67
R&D Progress 67
Synthetic Peptide to Inhibit Complement C5 for Paroxysmal Nocturnal Hemoglobinuria and Atypical Hemolytic Uremic Syndrome - Drug Profile 68
Product Description 68
Mechanism Of Action 68
R&D Progress 68
Synthetic Peptide to Target Complement C3 for Immunology, PNH and CNS Disorders - Drug Profile 69
Product Description 69
Mechanism Of Action 69
R&D Progress 69
Synthetic Peptides to Inhibit Factor D for Age-Related Macular Degeneration, Paroxysmal Nocturnal Hemoglobinuria and Renal Disease - Drug Profile 70
Product Description 70
Mechanism Of Action 70
R&D Progress 70
tesidolumab - Drug Profile 71
Product Description 71
Mechanism Of Action 71
R&D Progress 71
Paroxysmal Nocturnal Hemoglobinuria - Dormant Projects 73
Paroxysmal Nocturnal Hemoglobinuria - Discontinued Products 74
Paroxysmal Nocturnal Hemoglobinuria - Product Development Milestones 75
Featured News & Press Releases 75
Aug 10, 2016: Omeros' Lead MASP-3 Inhibitor OMS906 Demonstrates Efficacy in Paroxysmal Nocturnal Hemoglobinuria Model 75
Aug 04, 2016: Omeros' Lead MASP-3 Inhibitor OMS906 Demonstrates Blockade of the Alternative Pathway of Complement System 75
Jul 13, 2016: Akari Therapeutics Receives Approval from the UK Medicines & Healthcare Products Regulatory Agency to Conduct Phase 2 Trial in Paroxysmal Nocturnal Hemoglobinuria 76
Jul 12, 2016: Akari Therapeutics Receives Positive Opinion for Orphan Drug Designation for Coversin in the European Union for Treatment of Paroxysmal Nocturnal Hemoglobinuria 76
Jul 06, 2016: Akari Therapeutics Announces Positive Interim Update from Phase Ib Trial Demonstrating Sustained Complement Inhibition Using Once-Daily Subcutaneous Maintenance Dosing with Coversin 77
Jun 23, 2016: Apellis Announces Positive Results from Phase 1 Clinical Trials of APL-2, a C3 Complement Inhibitor 77
Jun 10, 2016: Achillion Presents Interim Phase 1 Results for ACH-4471, a Novel Orally-Administered Factor D Inhibitor, at the 21st Congress of the European Hematology Association 78
Jun 10, 2016: Late-Breaking Data Presented at EHA: All Patients with PNH Treated with Once-Monthly Dosing of ALXN1210 in Phase 1/2 Study Exhibit Rapid and Sustained Reductions in LDH 79
Jun 10, 2016: Ra Pharmaceuticals Presents Clinical Data on Complement C5 Inhibitor RA101495 at the 21st Congress of the European Hematology Association 81
Jun 01, 2016: European Commission Grants Orphan Drug Designation to ALXN1210 for the Treatment of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) 82
May 19, 2016: Achillion Announces Acceptance of Late Breaking Abstract for ACH-4471 at the 21st Congress of the European Hematology Association 83
May 19, 2016: Researchers to Present Late-Breaking Data on ALXN1210 in Patients with PNH at EHA Annual Congress 84
May 19, 2016: Akari Therapeutics Announces Upcoming Data Presentation at the 21st European Hematology Association Annual Congress and Update on First PNH Patient Treated 84
May 19, 2016: Ra Pharmaceuticals to Present at the 21st Congress of the European Hematology Association 85
May 09, 2016: EPIRUS Announces Reprioritization of Pipeline, to focus on biosimilar product BOW080 86
Appendix 87
Methodology 87
Coverage 87
Secondary Research 87
Primary Research 87
Expert Panel Validation 87
Contact Us 87
Disclaimer 88

List of Tables

Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, H2 2016 9
Number of Products under Development by Companies, H2 2016 10
Comparative Analysis by Clinical Stage Development, H2 2016 11
Comparative Analysis by Early Stage Development, H2 2016 12
Products under Development by Companies, H2 2016 13
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Achillion Pharmaceuticals, Inc., H2 2016 14
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Akari Therapeutics, Plc, H2 2016 15
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Alexion Pharmaceuticals Inc, H2 2016 16
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Alnylam Pharmaceuticals, Inc., H2 2016 17
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Amgen Inc., H2 2016 18
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Amyndas Pharmaceuticals LLC, H2 2016 19
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Apellis Pharmaceuticals Inc, H2 2016 20
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Epirus Biopharmaceuticals, Inc., H2 2016 21
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Novartis AG, H2 2016 22
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Omeros Corporation, H2 2016 23
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Ra Pharmaceuticals, Inc., H2 2016 24
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Regenesance BV, H2 2016 25
Paroxysmal Nocturnal Hemoglobinuria - Pipeline by The International Biotechnology Center (IBC) Generium, H2 2016 26
Assessment by Monotherapy Products, H2 2016 27
Number of Products by Stage and Target, H2 2016 29
Number of Products by Stage and Mechanism of Action, H2 2016 31
Number of Products by Stage and Route of Administration, H2 2016 33
Number of Products by Stage and Molecule Type, H2 2016 35
Paroxysmal Nocturnal Hemoglobinuria - Dormant Projects, H2 2016 73
Paroxysmal Nocturnal Hemoglobinuria - Discontinued Products, H2 2016 74

List of Figures

Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, H2 2016 9
Number of Products under Development by Companies, H2 2016 10
Comparative Analysis by Clinical Stage Development, H2 2016 11
Comparative Analysis by Early Stage Products, H2 2016 12
Assessment by Monotherapy Products, H2 2016 27
Number of Products by Targets, H2 2016 28
Number of Products by Stage and Targets, H2 2016 28
Number of Products by Mechanism of Actions, H2 2016 30
Number of Products by Stage and Mechanism of Actions, H2 2016 30
Number of Products by Routes of Administration, H2 2016 32
Number of Products by Stage and Routes of Administration, H2 2016 32
Number of Products by Molecule Types, H2 2016 34
Number of Products by Stage and Top 10 Molecule Types, H2 2016 34
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