Table 2.
Developmental treatments for hereditary angioedema with C1-inhibitor deficiency
| Drug (trade name) | Manufacturer | Mechanism of action | Planned indication | Administration | Regulatory status |
|---|---|---|---|---|---|
| ALN-F12® | Alnylam Pharmaceuticals | RNA interference targeted at FXII | LTP | Subcutaneous | Preclinical development |
| ARC-F12® | Arrowhead Pharmaceuticals | RNA interference targeted at FXII | LTP | Subcutaneous | Preclinical development |
| ATN-249® | Attune Pharmaceuticals | Kallikrein inhibitor | LTP | Oral | Phase 1 trial is complete |
| Berotralstat (BCX7353) | BioCryst Pharmaceuticals | Kallikrein inhibitor | ODT | Oral | Phase 2 trial is complete |
| BMN 331® | BioMarin | Adeno-associated virus-mediated antibody delivery gene therapy | LTP | Intravenous | Preclinical development |
| Conestat alfa (Ruconest®) | Pharming Group NV | Recombinant C1-INH concentrate | LTP | Intravenous | Phase 2 trial is complete |
| Garadacimab® | CSL Behring | Humanised anti-FXIIa monoclonal antibody | LTP | Subcutaneous | Phase 2 trial is recruiting |
| IONIS-PKK-LRx® | IONIS Pharmaceuticals | Antisense oligonucleotide targeted at prekallikrein | LTP | Subcutaneous | Phase 2 results are expected Q2 2021 |
| KVD824® | KalVista Pharmaceuticals | Kallikrein inhibitor | LTP | Oral | Phase 2 trial is expected to start in 2021 |
| KVD900® | KalVista Pharmaceuticals | Kallikrein inhibitor | ODT | Oral | Phase 2 trial data are expected in Q1 2021 |
| NTLA-2002® | Intellia Therapeutics | CRISPR/Cas9 editing of KLKB1 | LTP | Intravenous | Preclinical development |
| PHA022121® | Pharvaris | BKRB2 antagonist | LTP, ODT | Oral | Phase 2 trial is in progress |
BKRB2 bradykinin receptor B2, C1-INH C1 esterase inhibitor, LTP long-term prophylaxis, ODT on demand treatment