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Table 4 Complement inhibitors under clinical investigation for treatment of HSCT-TMA

From: Role of the lectin pathway of complement in hematopoietic stem cell transplantation-associated endothelial injury and thrombotic microangiopathy

Drug Target/mechanism of action Class Company Status
Eculizumab C5 inhibition mAb Alexion Pharmaceuticals Phase 2 ongoing; off-label use in clinic [161] NCT03518203 (pediatric + adult) [162]
Ravulizumab (ALXN1210) C5 inhibition mAb Alexion Pharmaceuticals Phase 3 ongoing NCT04543591 (adolescent + adult) [141]
NCT04557735 (pediatric) [142]
Nomacopan (Coversin) C5 and LTB4 inhibition Recombinant protein Akari Therapeutics Phase 3 ongoing NCT04784455 (pediatric) [145]
Narsoplimab (OMS721) MASP-2 inhibition mAb Omeros Corporation Phase 2 complete NCT02222545 (adult) [147]
  1. HSCT-TMA Hematopoietic stem cell transplantation-associated thrombotic microangiopathy, LTB4 Leukotriene B4, mAb Monoclonal antibody, MASP-2 Mannan-binding lectin-associated serine protease 2