Modality
siRNA
MOA
VEGFi
Target
Nectin-4
Pathway
Amyloid
SMAGIST
Development Pipeline
Preclinical
~Dec 2011
→ ~Mar 2013
Phase 1
~Jun 2013
→ ~Sep 2014
Phase 2
~Dec 2014
→ ~Mar 2016
Phase 3
~Jun 2016
→ ~Sep 2017
NDA/BLA
Dec 2017
→ May 2028
NDA/BLACurrent
NCT05626764
975 pts·SMA
2023-10→2025-03·Recruiting
NCT08752770
1,224 pts·SMA
2017-12→2028-05·Completed
2,199 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-03-081.1y agoPh3 Readout· SMA
2028-05-202.1y awayPh3 Readout· SMA
Trial Timeline
Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3
NDA/BLA
Complet…
NDA/BLA
Recruit…
Catalysts
Ph3 Readout
2025-03-08 · 1.1y ago
SMA
Ph3 Readout
2028-05-20 · 2.1y away
SMA
RecruitingCompleted|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05626764 | NDA/BLA | SMA | Recruiting | 975 | DAS28 |
| NCT08752770 | NDA/BLA | SMA | Completed | 1224 | EASI-75 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| MRK-2473 | Merck & Co | Phase 1/2 | Nectin-4 | |
| MRK-5413 | Merck & Co | Phase 1 | GLP-1R | |
| Doxasotorasib | AbbVie | Phase 2 | CD38 | |
| Ceviderotide | Novo Nordisk | Preclinical | IL-13 | |
| DSN-1421 | Daiichi Sankyo | Phase 3 | Nectin-4 | |
| VRT-6833 | Vertex Pharma | Preclinical | ALK | |
| BGN-8936 | BeiGene | Phase 1/2 | FXIa | |
| ION-3857 | Ionis | Preclinical | Nectin-4 | |
| Capifutibatinib | Intra-Cellular | Phase 3 | GIP-R | |
| Gozeosocimab | Illumina | Phase 2 | BCMA |