“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″
