Novel nonfactor agents for the treatment of hemophilia
| Novel therapeutic . | Mechanism of action . | Intended application . | Status of clinical development . | ClinicalTrials.gov . |
|---|---|---|---|---|
| Emicizumab | Bispecific antibody to FX and FIXa | HA with and without inhibitors | FDA approved | NCT02847637 |
| NCT03361137 | ||||
| NCT02795767 | ||||
| NCT02622321 | ||||
| NCT03191799 | ||||
| NCT03315455 | ||||
| NCT03020160 | ||||
| BS027125 | Bispecific antibody to FX and FIXa | Hemophilia A with and without inhibitors | Preclinical | N/A |
| Fitusiran | siRNA knockdown of AT | Cross segment | Phase 3 | NCT03549871 |
| NCT03417102 | ||||
| NCT03417245 | ||||
| Concizumab | Monoclonal antibody against TFPI | Cross segment | Phase 2 | NCT03196297 |
| NCT03196284 | ||||
| PF-06741086 | Monoclonal antibody against TFPI | Cross segment | Phase 2 | NCT02974855 |
| NCT03363321 | ||||
| BAY1093884 | Monoclonal antibody against TFPI | Cross segment | Phase 1 | NCT02571569 |
| NCT03597022 | ||||
| NCT03481946 | ||||
| SerpinPC | Serine protease inhibitor of APC | Cross segment | Preclinical | N/A |
| HAPC1573 | Monoclonal antibody against APC | Cross segment | Preclinical | N/A |
| PS siRNA | siRNA knockdown of PS | Cross segment | Preclinical | N/A |
| Novel therapeutic . | Mechanism of action . | Intended application . | Status of clinical development . | ClinicalTrials.gov . |
|---|---|---|---|---|
| Emicizumab | Bispecific antibody to FX and FIXa | HA with and without inhibitors | FDA approved | NCT02847637 |
| NCT03361137 | ||||
| NCT02795767 | ||||
| NCT02622321 | ||||
| NCT03191799 | ||||
| NCT03315455 | ||||
| NCT03020160 | ||||
| BS027125 | Bispecific antibody to FX and FIXa | Hemophilia A with and without inhibitors | Preclinical | N/A |
| Fitusiran | siRNA knockdown of AT | Cross segment | Phase 3 | NCT03549871 |
| NCT03417102 | ||||
| NCT03417245 | ||||
| Concizumab | Monoclonal antibody against TFPI | Cross segment | Phase 2 | NCT03196297 |
| NCT03196284 | ||||
| PF-06741086 | Monoclonal antibody against TFPI | Cross segment | Phase 2 | NCT02974855 |
| NCT03363321 | ||||
| BAY1093884 | Monoclonal antibody against TFPI | Cross segment | Phase 1 | NCT02571569 |
| NCT03597022 | ||||
| NCT03481946 | ||||
| SerpinPC | Serine protease inhibitor of APC | Cross segment | Preclinical | N/A |
| HAPC1573 | Monoclonal antibody against APC | Cross segment | Preclinical | N/A |
| PS siRNA | siRNA knockdown of PS | Cross segment | Preclinical | N/A |
Cross segment: HA and HB, with and without inhibitors.
APC, activated protein C; FDA, US Food and Drug Administration; FIXa, activated FIX; N/A, not applicable; PS, protein S; TFPI, tissue factor pathway inhibitor.