Modality
ERT
MOA
FXIai
Target
Tau
Pathway
STING
DravetHSFabry
Development Pipeline
Preclinical
~May 2023
→ ~Aug 2024
Phase 1
Nov 2024
→ Jan 2030
Phase 1Current
NCT07414430
2,219 pts·HS
2024-11→2030-01·Active
2,219 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2030-01-063.8y awayInterim· HS
Trial Timeline
Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030
P1
Active
Catalysts
Interim
2030-01-06 · 3.8y away
HS
Active|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07414430 | Phase 1 | HS | Active | 2219 | DOR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| NVS-6360 | Novartis | Approved | CD38 | |
| MRK-3745 | Merck & Co | Phase 2 | WRN | |
| AZN-1715 | AstraZeneca | Phase 3 | AHR | |
| BMY-9931 | Bristol-Myers Squibb | Phase 1 | GPRC5D | |
| NVO-7877 | Novo Nordisk | Preclinical | GPRC5D | |
| NVO-9087 | Novo Nordisk | Phase 1/2 | Tau | |
| TAK-8730 | Takeda | Phase 2 | VEGF | |
| Tezecilimab | Takeda | NDA/BLA | Tau | |
| TAK-8262 | Takeda | NDA/BLA | Tau | |
| Semazasiran | BeiGene | Phase 1 | PD-1 |