Modality
Nanobody
MOA
IL-17i
Target
RET
Pathway
Amyloid
Hemophilia A
Development Pipeline
Preclinical
Dec 2021
→ Oct 2030
PreclinicalCurrent
NCT03973894
603 pts·Hemophilia A
2023-06→2030-04·Recruiting
NCT05862647
2,532 pts·Hemophilia A
2021-12→2030-10·Terminated
3,135 total pts1 indication
Phase 1
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2030-04-264.1y awayInterim· Hemophilia A
2030-10-164.5y awayInterim· Hemophilia A
Trial Timeline
Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q4
Preclinical
Termina…
Preclinical
Recruit…
Catalysts
Interim
2030-04-26 · 4.1y away
Hemophilia A
Interim
2030-10-16 · 4.5y away
Hemophilia A
RecruitingTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03973894 | Preclinical | Hemophilia A | Recruiting | 603 | OS |
| NCT05862647 | Preclinical | Hemophilia A | Terminated | 2532 | Safety |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| RHH-5389 | Roche | Preclinical | RET | |
| Fixanesiran | AbbVie | Preclinical | RET | |
| BMY-8678 | Bristol-Myers Squibb | Approved | RET | |
| SNY-2289 | Sanofi | Phase 2/3 | CD38 | |
| Fixainavolisib | Takeda | Preclinical | CDK2 | |
| Elrarapivir | Vertex Pharma | NDA/BLA | PRMT5 | |
| VRT-1576 | Vertex Pharma | Phase 2/3 | RET | |
| Rimaglumide | Biogen | Phase 1/2 | BCL-2 | |
| ARG-3458 | Argenx | Preclinical | RET | |
| Doxazasiran | Axsome | Phase 1 | TIM-3 |