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Published: Sep, 2016 | Pages:
87 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H2 2016 Summary 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
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
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