Modality
ERT
MOA
KRASG12Ci
Target
WEE1
Pathway
JAK/STAT
MSRATTR Amyloidosis
Development Pipeline
Preclinical
~Mar 2023
→ ~Jun 2024
Phase 1
Sep 2024
→ Apr 2028
Phase 1Current
NCT08076429
504 pts·TTR Amyloidosis
2025-06→2028-02·Completed
NCT04020867
1,682 pts·RA
2024-09→2028-04·Terminated
2,186 total pts2 indications
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2028-02-041.8y awayInterim· TTR Amyloidosis
2028-04-042.0y awayInterim· RA
Trial Timeline
Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2
P1
Termina…
P1
Complet…
Catalysts
Interim
2028-02-04 · 1.8y away
TTR Amyloidosis
Interim
2028-04-04 · 2.0y away
RA
CompletedTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08076429 | Phase 1 | TTR Amyloidosis | Completed | 504 | OS |
| NCT04020867 | Phase 1 | RA | Terminated | 1682 | PFS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Lisolucimab | Novartis | Approved | CD20 | |
| Gozelemzoparlimab | Merck & Co | Phase 2 | FXIa | |
| AZN-8478 | AstraZeneca | NDA/BLA | SHP2 | |
| Pexazasiran | Sanofi | Preclinical | FXIa | |
| Terasacituzumab | Novo Nordisk | NDA/BLA | IL-23 | |
| TAK-2403 | Takeda | Phase 2 | WEE1 | |
| Sovacapivasertib | Amgen | Phase 3 | WEE1 | |
| ALN-6288 | Alnylam | Approved | LAG-3 | |
| Fixaglumide | Genmab | Phase 2/3 | WEE1 | |
| BMR-9762 | BioMarin | Phase 3 | Aβ |