Table 2.
Recently approved or in clinical trial novel hemophilia A therapeutics
| Name | Description | Clinical development | Ref |
|---|---|---|---|
| Efraloctocog alfa (Eloctate) | rBDD FVIII bound to Fc | Phase I–III: 1.5× increase in t1/2 | 35–39 |
| N8-GP | PEG rBDD FVIII | Phase I–III complete: preliminary results report 1.6× increase in t1/2 | 53 |
| BAX 855 | PEG rFVIII | Preclinical: 1.5–2× increase in t1/2 Ongoing Phase II/III clinical trial |
54 |
| Bay 94-9027 | PEG rBDD-rFVIII | Phase I study: 1.5× increase in t1/2 | 55 |
| rFVIII single-chain | rBDD-FVIII single chain with greater vWF affinity | Phase I/II study 1.5–2× increase in t1/2 | 56,57 |
| ACE910 | Humanize antifactor IX/X bispecific antibody, SC delivery | Phase I preliminary results reported safety and efficacy at weekly dosing | 58–60 |
| Concizumab | Monoclonal humanized IgG4 antibody targeting TFPI, IV or SC delivery | Phase I study reported favorable safety profile after single IV or SC administration | 61 |
| ALN-AT3 | RNAi that knocks down hepatocyte AT synthesis | Phase I preliminary results reported safety and demonstrated effect up to 70 days | 62 |
Abbreviations: rBDD, recombinant B-domain; Fc, dimeric constant region; FVIII, factor VIII; rFVIII, recombinant factor VIII; PEG, polyethylene glycol; vWF, von Willebrand factor; TFPI, tissue factor pathway inhibitor; SC, subcutaneous; IV, intravenous; RNAi, RNA interference; AT, antithrombin; Ref, references.