Non-exhaustive list of disclosed C5 therapeutics
| Drug name | Company | Modality | Highest clinical stage achieved | Target indications |
|---|---|---|---|---|
| Eculizumab | Alexion/AZ | Humanized mAb | Marketed | aHUS (market), gMG (market), PNH (market), NMOSD (market), gMG pediatric (filed), NMOSD pediatric: phase 3 |
| Ravulizumab | Alexion/AZ | Humanized long acting mAb | Marketed | PNH (market), aHUS (market), PNH pediatrics (market), aHUS pediatrics (market), gMG (market), NMOSD (filed), HSCT-TMA (phase 3), CSA-AKI (phase 3), Renal Basket (phase 2), Dermatomyositis (phase 2) |
| Avacopan | ChemoCentryx/Amgen | C5aR antagonist small molecule | Marketed | AAV: approved (United States, Japan, and European Union)C3G: phase 2HS: phase 2 |
| Zimura (Avacincaptad pegol) | Iveric Bio | Pegylated RNA aptamer | Marketed | GA: marketed (in the United States)Stargardt disease: phase 2 |
| Zilucoplan | UCB | 15-amino acid macrocyclic peptide | Marketed | gMG (market) |
| Vilobelimab | InflaRx | Anti-C5a mAb | Marketed | COVID-19: emergency authorization in the United States, Pyoderma Gangrenosum: phase 3 |
| Pozelimab | Regeneron | mAb | Approved (CHAPLE) | PNH: phase 3gMG: phase 3GA: phase 3 (planned) |
| Eculizumab biosimilars | Multiple | mAb | Multiple phases including marketed | PNH (marketed) |
| Nomacopan (Coversin, AK576) | Akari | Small protein | Phase 3 | HSCT-TMA: phase 3GA: preclinical |
| Cemdisiran | Alnylam | GalNAc conjugated siRNA | Phase 3 | PNH: phase 3gMG: Phase 3GA: phase 3 (planned) |
| Crovalimab | Chigai/Roche | Humanized long acting mAb | Phase 3 | PNH: filed (Japan, China, United States, and European Union)aHUS: phase 3LN: phase 1GBS: phase 3Scleroderma: phase 2 |
| CAN106 | CanBridge | Humanized long acting mAb | Phase1/2 | PNH: phase 1b/2 (China) |
| RLYB-114, RLYB, 116 | Rallybio | Antibody mimetic fusion protein + HSA binder, pegylated mAb | Phase 1 | Rare disease, ophthalmology |
| KP-104 | Kira Bio | Bi-functional mAb (include FH1-5 tail) | Phase 1 | IgAN, C3G, SLE-TMA, PNH: all indications phase 2 |
| CB-301, CB-401 | Cascade Biotech | Not disclosed | Preclinical | Not disclosed |
| PBP1603 | Prestige | Eculizumab biosimilar | Preclinical | Not disclosed |
| HMI-104 | Homology Medicine | rAAV gene therapy | Preclinical | Not disclosed |
| STP145G | Sirnaomics | siRNA | Preclinical | Not disclosed |
| IAB-101 | ImmunAbs | mAb | Preclinical | Not disclosed |
GalNAc: N-acetylgalactosamine; aHUS: atypical haemolytic uremic syndrome; AAV: antineutrophilic cytoplasmic antibody-associated vasculitis; C3G: C3 glomerulopathy; CSA-AKI: cardiac surgery-associated acute kidney injury; COVID-19: coronavirus disease 2019; FH: factor H; gMG: generalized myasthenia gravis; GA: geographic atrophy; GBS: Guillain-Barre syndrome; HSCT-TMA: hematopoietic stem cell transplantation-associated thrombotic microangiopathy; HS: hidradenitis suppurativa; HAS: human serum albumin; IgAN: IgA nephropathy; mAb: monoclonal antibody; NMO: Neuromyelitis Optica; PNH: paroxysmal nocturnal haemoglobinuria; rAAV: recombinant adeno-associated virus; siRNA: silencing ribonucleic acid; SLE-TMA: systemic lupus erythematosus-associated thrombotic microangiopathy; LN: lupus nephritis