Modality
Gene Editing
MOA
TNFi
Target
RET
Pathway
Amyloid
FTD
Development Pipeline
Preclinical
~May 2016
→ ~Aug 2017
Phase 1
~Nov 2017
→ ~Feb 2019
Phase 2
~May 2019
→ ~Aug 2020
Phase 3
~Nov 2020
→ ~Feb 2022
NDA/BLA
~May 2022
→ ~Aug 2023
Approved
Nov 2023
→ May 2025
ApprovedCurrent
NCT03340265
297 pts·FTD
2023-11→2025-05·Recruiting
297 total pts1 indication
CompletedCurrentUpcoming
Catalysts (1)
2025-05-1011mo agoPh3 Readout· FTD
Trial Timeline
2024Q2Q3Q42025Q2
Approved
Recruit…
Catalysts
Ph3 Readout
2025-05-10 · 11mo ago
FTD
Recruiting|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03340265 | Approved | FTD | Recruiting | 297 | DAS28 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| RHH-5389 | Roche | Preclinical | RET | |
| Fixanesiran | AbbVie | Preclinical | RET | |
| AZN-7403 | AstraZeneca | Phase 2/3 | FGFR | |
| BMY-8678 | Bristol-Myers Squibb | Approved | RET | |
| TAK-8262 | Takeda | NDA/BLA | Tau | |
| Talalemzoparlimab | Gilead Sciences | Phase 1 | FcRn | |
| VRT-1576 | Vertex Pharma | Phase 2/3 | RET | |
| ARG-3458 | Argenx | Preclinical | RET | |
| Ribozanubrutinib | Genmab | Approved | GPRC5D | |
| 369-789 | Hansoh Pharma | Phase 3 | PI3Kα |