Table 2. Complement therapeutics in clinical trials.
| Target | Drug candidate (Company) | Entity | Clinical trial(s) | Indication(s) | Refs | |
|---|---|---|---|---|---|---|
| Phase | Trial number | |||||
| C1s | BIVV009 (Bioverativ) | Antibody | I | NCT02502903 | Cold agglutinin disease | 110 |
| MASP2 | OMS721 (Omeros) | Antibody | II | NCT02222545 | Thrombotic microangiopathies | 213 |
| NCT02682407 | IgA nephropathy, LN, MN, C3G | 116 | ||||
| III | NCT03205995 | aHUS | 117 | |||
| Properdin | CLG561 (Novartis) | Antibody | II | NCT02515942* | AMD and/or GA | 78 |
| C3 | AMY-101 (Amyndas) | Peptide | I | NCT03316521 | C3G | 163 |
| II | NA | C3G, PNH, ABOi kidney transplantation, peridontitis | 214 | |||
| APL-1 (Apellis) | Peptide | I | NA | COPD | 215 | |
| APL-2 (Apellis) | Peptide | I | NCT02588833 | PNH | 156 | |
| NCT02264639 | PNH (add-on therapy) | 155 | ||||
| NCT02461771‡ | AMD (CNV) | 216 | ||||
| II | NCT02503332 | AMD and/or GA | 217 | |||
| NCT03226678 | wAIHA, CAD | 159 | ||||
| APL-9 (Apellis) | Peptide | I | ACTRN12616000862448 | PNH | 160 | |
| FB | IONIS-FB-LRx (Ionis, GSK) | Oligonucleotide | I | 2015-001837-25 | AMD and/or GA | 141 |
| FD | Lampalizumab (Genentech) | Antibody | II | NCT02288559 | AMD and/or GA | 130 |
| III | NCT02745119 | AMD and/or GA | 218 | |||
| NCT02247531 | AMD and/or GA | 132 | ||||
| NCT02247479 | AMD and/or GA | 133 | ||||
| ACH-4471 (Achillion) | Small molecule | II | NCT03053102 | PNH | 125 | |
| Convertases | Mirococept (MRC) | Protein | III | ISRCTN49958194 | Transplantation | 147,148 |
| C5 | Ravulizumab; also known as ALXN1210 (Alexion) | Antibody | I/II | NCT02598583 | PNH | 219 |
| II | NCT02605993 | PNH | 220 | |||
| III | NCT02946463 | PNH (naive) | 103 | |||
| NCT03056040 | PNH (treated) | 104 | ||||
| NCT02949128 | aHUS (naive) | 102 | ||||
| Tesidolumab; also known as LFG316 (Novartis and MorphoSys) | Antibody | I | NCT02878616‡ | Transplantation | 221 | |
| II | NCT02763644 | Transplant-associated microangiopathy | 76 | |||
| NCT01527500‡ | AMD and/or GA | 222 | ||||
| NCT02515942* | AMD | 78 | ||||
| NCT02534909 | PNH | 75 | ||||
| NCT01526889 | Uveitis and/or panuveitis | 77 | ||||
| SKY59; also known as RG6107 and RO7112689 (Chugai and Roche) | Antibody | I/II | NCT03157635 | PNH | 223 | |
| REGN3918 (Regeneron) | Antibody | I | NCT03115996 | PNH | 82 | |
| ABP959 (Amgen) | Antibody§ | I | ACTRN12616000509460 | PNH, aHUS | 71 | |
| GNR-045 (Generium) | Antibody§ | I | ECU-PNH-I | PNH | 224 | |
| Coversin (Akari) | Protein | II | NCT02591862 | PNH | 89 | |
| RA101495 (Ra Pharma) | Peptide | II | NCT03030183 | PNH (poor responders) | 96 | |
| NCT03078582 | PNH | 95 | ||||
| Zimura (Ophthotech) | Oligonucleotide | II | NCT02397954‡ | IPCV | 85 | |
| II/III | NCT02686658 | AMD | 86 | |||
| Cemdisiran (Alnylam) | Oligonucleotide | I/II | NCT02352493 | PNH | 98 | |
| II | NA | aHUS | 101 | |||
| C5a | ALXN1007 (Alexion) | Antibody | II | NCT02245412‖ | GVHD | 225 |
| NCT02128269‖ | APS | 226 | ||||
| IFX-1 (InflaRx) | Antibody | II | NCT02246595‡ | Sepsis | 227 | |
| NCT02866825‡ | SIRS, complex cardiac surgery | 228 | ||||
| NCT03001622 | Hidradenitis suppurativa | 229 | ||||
| C5aR1 | Avacopan; also known as CCX168 (ChemoCentryx) | Small molecule | II | NCT02464891‖ | aHUS | 172 |
| NCT02384317‡ | IgA nephropathy | 173 | ||||
| III | NCT02994927 | A AV | 169 | |||
The table includes ongoing, completed and terminated trials from 2012 onwards. Completed trials are not listed when the development has progressed to the next phase for the same indication. AAV, anti-neutrophil cytoplasmic-antibody-associated vasculitis; aHUS, atypical haemolytic uraemic syndrome; ABOi, ABO incompatible; AMD, age-related macular degeneration; APS, antiphospholipid syndrome; C1s, complement C1s; C3, complement C3; C3G, C3 glomerulopathy; C5, complement C5; C5a, anaphylatoxin formed from the degradation of complement C5; C5aR1, C5a receptor 1; FB, factor B; FD, factor D; CNV, choroidal neovascularization; COPD, chronic obstructive pulmonary disease; GA, geographic atrophy; GVHD, graft versus host disease; IPCV, idiopathic polypoidal choroidal vasculopathy; LN, lupus nephritis; MASP2, mannose-binding lectin-associated serine protease 2; MN, membranous nephropathy; NA, not available; PNH, paroxysmal nocturnal haemoglobinuria; SIRS, systemic inflammatory response syndrome; wAIHA, warm autoimmune haemolytic anaemia.
CLG561 and tesidolumab were used as a combination therapy in the same trial.
Completed trial.
Biosimilar antibody of eculizumab.
Terminated trial.