Sections
| Title | Starting Page | Number of Pages |
|---|
| Table of Contents | 2 | 5 |
| List of Tables | 6 | 1 |
| List of Figures | 6 | 1 |
| Introduction | 7 | 1 |
| Global Markets Direct Report Coverage | 7 | 1 |
| Paroxysmal Nocturnal Hemoglobinuria Overview | 8 | 1 |
| Therapeutics Development | 9 | 1 |
| Pipeline Products for Paroxysmal Nocturnal Hemoglobinuria Overview | 9 | 1 |
| Paroxysmal Nocturnal Hemoglobinuria Therapeutics under Development by Companies | 10 | 1 |
| Paroxysmal Nocturnal Hemoglobinuria Pipeline Products Glance | 11 | 2 |
| Clinical Stage Products | 11 | 1 |
| Early Stage Products | 12 | 1 |
| Paroxysmal Nocturnal Hemoglobinuria Products under Development by Companies | 13 | 1 |
| Paroxysmal Nocturnal Hemoglobinuria Companies Involved in Therapeutics Development | 14 | 13 |
| Achillion Pharmaceuticals, Inc. | 14 | 1 |
| Akari Therapeutics, Plc | 15 | 1 |
| Alexion Pharmaceuticals Inc | 16 | 1 |
| Alnylam Pharmaceuticals, Inc. | 17 | 1 |
| Amgen Inc. | 18 | 1 |
| Amyndas Pharmaceuticals LLC | 19 | 1 |
| Apellis Pharmaceuticals Inc | 20 | 1 |
| Epirus Biopharmaceuticals, Inc. | 21 | 1 |
| Novartis AG | 22 | 1 |
| Omeros Corporation | 23 | 1 |
| Ra Pharmaceuticals, Inc. | 24 | 1 |
| Regenesance BV | 25 | 1 |
| The International Biotechnology Center (IBC) Generium | 26 | 1 |
| Paroxysmal Nocturnal Hemoglobinuria Therapeutics Assessment | 27 | 9 |
| Assessment by Monotherapy Products | 27 | 1 |
| Assessment by Target | 28 | 2 |
| Assessment by Mechanism of Action | 30 | 2 |
| Assessment by Route of Administration | 32 | 2 |
| Assessment by Molecule Type | 34 | 2 |
| Drug Profiles | 36 | 36 |
| ACH-4471 Drug Profile | 36 | 2 |
| ALN-CC5 Drug Profile | 38 | 4 |
| ALXN-1210 Drug Profile | 42 | 2 |
| AMY-101 Drug Profile | 44 | 2 |
| APL-2 Drug Profile | 46 | 2 |
| APL-9 Drug Profile | 48 | 1 |
| Coversin Drug Profile | 49 | 3 |
| eculizumab biosimilar Drug Profile | 52 | 1 |
| eculizumab biosimilar Drug Profile | 53 | 1 |
| GNR-045 Drug Profile | 54 | 1 |
| Monoclonal Antibody for Paroxysmal Nocturnal Hemoglobinuria Drug Profile | 55 | 1 |
| NM-9405 Drug Profile | 56 | 1 |
| OMS-721 Drug Profile | 57 | 5 |
| OMS-906 Drug Profile | 62 | 1 |
| RA-101348 Drug Profile | 63 | 1 |
| RA-101495 Drug Profile | 64 | 2 |
| regenemab Drug Profile | 66 | 1 |
| Synthetic Peptide to Inhibit Complement C5 for Paroxysmal Nocturnal Hemoglobinuria and Atypical Hemolytic Uremic Syndrome Drug Profile | 67 | 1 |
| Synthetic Peptide to Target Complement C3 for Immunology, PNH and CNS Disorders Drug Profile | 68 | 1 |
| Synthetic Peptides to Inhibit Factor D for Age-Related Macular Degeneration, Paroxysmal Nocturnal Hemoglobinuria and Renal Disease Drug Profile | 69 | 1 |
| tesidolumab Drug Profile | 70 | 2 |
| Paroxysmal Nocturnal Hemoglobinuria Dormant Projects | 72 | 1 |
| Paroxysmal Nocturnal Hemoglobinuria Discontinued Products | 73 | 1 |
| Paroxysmal Nocturnal Hemoglobinuria Product Development Milestones | 74 | 12 |
| Featured News &Press Releases | 74 | 1 |
| Aug 10, 2016: Omeros Lead MASP-3 Inhibitor OMS906 Demonstrates Efficacy in Paroxysmal Nocturnal Hemoglobinuria Model | 74 | 1 |
| Aug 04, 2016: Omeros Lead MASP-3 Inhibitor OMS906 Demonstrates Blockade of the Alternative Pathway of Complement System | 74 | 1 |
| Jul 13, 2016: Akari Therapeutics Receives Approval from the UK Medicines &Healthcare Products Regulatory Agency to Conduct Phase 2 Trial in Paroxysmal Nocturnal Hemoglobinuria | 75 | 1 |
| Jul 12, 2016: Akari Therapeutics Receives Positive Opinion for Orphan Drug Designation for Coversin in the European Union for Treatment of Paroxysmal Nocturnal Hemoglobinuria | 75 | 1 |
| 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 | 76 | 1 |
| Jun 23, 2016: Apellis Announces Positive Results from Phase 1 Clinical Trials of APL-2, a C3 Complement Inhibitor | 76 | 1 |
| 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 | 77 | 1 |
| 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 | 78 | 2 |
| Jun 10, 2016: Ra Pharmaceuticals Presents Clinical Data on Complement C5 Inhibitor RA101495 at the 21st Congress of the European Hematology Association | 80 | 1 |
| Jun 01, 2016: European Commission Grants Orphan Drug Designation to ALXN1210 for the Treatment of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) | 81 | 1 |
| May 19, 2016: Achillion Announces Acceptance of Late Breaking Abstract for ACH-4471 at the 21st Congress of the European Hematology Association | 82 | 1 |
| May 19, 2016: Researchers to Present Late-Breaking Data on ALXN1210 in Patients with PNH at EHA Annual Congress | 83 | 1 |
| May 19, 2016: Akari Therapeutics Announces Upcoming Data Presentation at the 21st European Hematology Association Annual Congress and Update on First PNH Patient Treated | 83 | 1 |
| May 19, 2016: Ra Pharmaceuticals to Present at the 21st Congress of the European Hematology Association | 84 | 1 |
| May 09, 2016: EPIRUS Announces Reprioritization of Pipeline, to focus on biosimilar product BOW080 | 85 | 1 |
| Appendix | 86 | 2 |
| Methodology | 86 | 1 |
| Coverage | 86 | 1 |
| Secondary Research | 86 | 1 |
| Primary Research | 86 | 1 |
| Expert Panel Validation | 86 | 1 |
| Contact Us | 86 | 1 |
| Disclaimer | 87 | 1 |
