Table 1.
Complement therapeutics on the market or in clinical trials
| Product (company) | Activity | Stage of development |
|---|---|---|
| Protease inhibitors (A) | ||
| C1-INH (Cetor/Sanquin, BerinertP/CSL Behring, Lev Pharma) | Purified plasma protein; inhibition of C1r/C1s, kallikrein and other proteases | Marketed (HAE)a; clinical phase 3 (HAE)a; preclinical/phase 1 for other indications (AMI, CABG) |
| Rhucin/rhC1INH (Pharming Group N.V.) | Recombinant human C1-INH from transgenic rabbits | Clinical phase 3 (HAE) |
| Soluble complement regulators (B) | ||
| sCR1/TP10 (Avant Immunotherapeutics) | Extracellular part of CR1; decay accelerator, factor I cofactor | Clinical phase 2 (CABG)b |
| CAB-2/MLN-2222 (Millenium Pharmaceuticals) | Chimera of DAF and MCP; decay accelerator, factor I cofactor | Clinical phase 1 (CABG)b |
| Therapeutic antibodies (C) | ||
| Eculizumab/Soliris (Alexion Pharmaceuticals) | Humanized long-acting mAb against C5 | Marketed (PNH), preclinical for other indications |
| Pexelizumab (Alexion Pharmaceuticals) | Humanized short-acting mAb against C5 | Clinical phase 3 (AMI, CABG)b |
| Ofatumumab (Genmab A/S) | Humanized anti-CD20 mAb | Clinical phase 2 |
| Complement component inhibitors (D) | ||
| Compstatin/POT-4 (Potentia Pharmaceuticals) | Peptidic C3 inhibitor; no generation of C3a/C3b | Clinical phase 1 (AMD) |
| Receptor antagonists (E) | ||
| PMX-53 (Peptech Ltd.) | Peptidic C5aR antagonist | Clinical phase 2 (RA, psoriasis) |
| Other | ||
| rhMBL (Enzon Pharmaceuticals) | Recombinant human MBL as substitution therapy | Clinical phase 1b (MBL deficiency) |
a
C1-INH preparation are only approved in some countries, whereas they are in clinical phase 3 in the USA.
b
Clinical trials have recently been performed but no product development has been undertaken since then, or the development was officially discontinued. AMD, age-related macular degeneration; AMI, acute myocardial infarction; CABG, coronary artery bypass grafting; HAE, hereditary angioedema; PNH, paroxysmal nocturnal hemoglobinuria; RA, rheumatoid arthritis.