Modality
siRNA
MOA
BETi
Target
FGFR
Pathway
Apoptosis
T2DSMAEwing Sarcoma
Development Pipeline
Preclinical
~May 2019
→ ~Aug 2020
Phase 1
~Nov 2020
→ ~Feb 2022
Phase 2
~May 2022
→ ~Aug 2023
Phase 3
~Nov 2023
→ ~Feb 2025
NDA/BLA
May 2025
→ Oct 2028
NDA/BLACurrent
NCT04524281
2,706 pts·Ewing Sarcoma
2025-08→TBD·Terminated
NCT08711746
2,889 pts·SMA
2025-05→2028-10·Terminated
5,595 total pts2 indications
Approved
CompletedCurrentUpcoming
Catalysts (1)
2028-10-262.6y awayPh3 Readout· SMA
Trial Timeline
Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q4
NDA/BLA
Termina…
NDA/BLA
Termina…
Catalysts
Ph3 Readout
2028-10-26 · 2.6y away
SMA
Terminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04524281 | NDA/BLA | Ewing Sarcoma | Terminated | 2706 | VA |
| NCT08711746 | NDA/BLA | SMA | Terminated | 2889 | ACR20 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| JNJ-179 | Johnson & Johnson | Approved | KRASG12C | |
| Doxarasimod | Merck & Co | NDA/BLA | FGFR | |
| Sovarapivir | AbbVie | Phase 2/3 | IL-13 | |
| AZN-7403 | AstraZeneca | Phase 2/3 | FGFR | |
| BAY-8733 | Bayer | Preclinical | AuroraA | |
| Cevitinib | Regeneron | Phase 3 | FGFR | |
| BII-1564 | Biogen | Phase 2 | PSMA | |
| Sovanaritide | Argenx | Phase 3 | TNFα | |
| 369-8021 | Hansoh Pharma | Approved | BTK | |
| COR-9456 | Corcept | Preclinical | TIGIT |