Table 1.
Complement therapeutics with known active development programs
| Compound (Company) | Main Target | Class | Clinical Phase (Trial No.)1 | Indications2 |
|---|---|---|---|---|
| Initiation Pathways | ||||
| Cinryze (Shire) | C1r/s, MASP | Protein | Clinic P3 (NCT02547220) | HAE Transplantation |
| Berinert (CSL Behring) | C1r/s, MASP | Protein | Clinic P1/2 (NCT02134314) | HAE Transplantation |
| Cetor (Sanquin) | C1r/s, MASP | Protein | Clinic | HAE |
| Ruconest (Pharming) | C1r/s, MASP | Protein | Clinic | HAE |
| ANX005 (Annexon) | C1q | Antibody | PC | Neurodegenerative |
| TNT009 (True North) | C1s | Antibody | P1 (NCT02502903) | CAD & others |
| N/A (Prothix) | C2 | Antibody | PC | N/A |
| OMS721 (Omeros) | MASP-2 | Antibody | P2 (NCT02222545) P2 (NCT02682407) |
TMA Glomerulopathies |
| OMS906 (Omeros) | MASP-3 | Antibody | PC | PNH & others |
| CLG561 (Novartis) | Properdin | Antibody | P2 (NCT02515942) | AMD |
| NM9401 (Novelmed) | Properdin | Antibody | PC | N/A |
| Activation & Amplification | ||||
| AMY-101 (Amyndas) | C3 | Peptide | PC | Transplantation,C3G, Periodontitis, PNH |
| APL-1 (Apellis) | C3 | Peptide | P1 | COPD |
| APL-2 (Apellis) | C3 | Peptide (PEGylated) | P1 (NCT02588833) P2 (NCT02503332) |
PNH AMD |
| CB 2782 (Catalyst) | C3 | Enzyme | PC | IRI |
| AMY-201 (Amyndas) | C3b,convertases | Protein | PC | PNH, AMD |
| Mirococept (MRC) | C3b,convertases | Protein | P2 (EMPIRIKAL) | Transplantation |
| Bikaciomab (Novelmed) | FB | Antibody | PC | AMD |
| N/A (Novartis) | FB | Small Molecule | ||
| Lampalizumab (Genentech) | FD | Antibody | P3 (NCT02247531) P3 (NCT02247479) |
AMD AMD |
| ACH-4471 (Achillion) | FD | Small Molecule | P1 (ACTRN12616000082404p) | PNH |
| ‘Compound 6’ (Novartis) | FD | Small Molecule | PC | AMD |
| Terminal Pathway & Effectors | ||||
| Soliris (Alexion) | C5 | Antibody | Clinic P2-P3 |
PNH, aHUS Various3 |
| ALXN1210 (Alexion) | C5 | Antibody | P2 (NCT02605993) | PNH |
| ALXN5500 (Alexion) | C5 | Antibody | P1 | N/A |
| LFG316 (Novartis) | C5 | Antibody | P2 (NCT02763644) P2 (NCT02515942) |
TMA AMD |
| Coversin (Akari) | C5 | Protein | P2 (NCT02591862) | PNH |
| RA101495 (Ra Pharma) | C5 | Peptide | P1 (ACTRN12615001143516) | PNH |
| Zimura (Ophthotech) | C5 | Aptamer | P2/3 (NCT02686658) | AMD |
| ALN-CC5 (Alnylam) | C5 | RNAi | P1/2 (NCT02352493) | PNH |
| Regenemab(Regenesance) | C6 | Antibody | PC | PNH, ALS, others |
| IFX-1 (InflaRx) | C5a | Antibody | P2 (NCT02246595) | Sepsis |
| ALXN-1007 (Alexion) | C5a | Antibody | P2 (NCT02245412) P2 (NCT02128269) |
GVHD APS |
| NOX-D21 (Noxxon) | C5a | Spiegelmer | PC | N/A |
| CCX168 (Chemocentryx) | C5aR1 | Small Molecule | P2 (NCT02222155) P2 (NCT02464891) |
ANCA Vasculitis aHUS |
1
Only major/select clinical trials shown; PC, preclinical, P1-3, clinical phase 1–3; ClinicalTrials.gov ID shown in parentheses (anzctr.org.au for ACH-4471 and RA101495).
2
Abbeviations: aHUS, atypical hemolytic uremic syndrome; ALS, amyotrophic lateral sclerosis; AMD, age-related macular degeneration; APS, antiphospholipid syndrome; C3G, C3 glomerulopathy; CAD, cold agglutinin disease; COPD, chronic obstructive pulmonary disease; GVHD, graft versus host disease; HAE, hereditary angioedema; IRI, ischemia-reperfusion injury; PNH, paroxysmal nocturnal hemoglobinuria; TMA, thrombotic microangiopathies. 3For a recent list of trials we refer to Ref. [6].