Modality
siRNA
MOA
IL-17i
Target
PI3Kα
Pathway
Complement
ALSSchizophrenia
Development Pipeline
Preclinical
~Dec 2021
→ ~Mar 2023
Phase 1
Jun 2023
Phase 1Current
NCT04441971
1,035 pts·ALS
2023-06→TBD·Completed
1,035 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2025-07-278mo agoNDA· ALS
Trial Timeline
Q3Q42024Q2Q3Q42025Q2Q3
P1/2
Complet…
Catalysts
NDA
2025-07-27 · 8mo ago
ALS
Completed|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04441971 | Phase 1/2 | ALS | Completed | 1035 | CfB |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Voxaderotide | Johnson & Johnson | NDA/BLA | PI3Kα | |
| Rimamavacamten | Merck & Co | Phase 1 | PI3Kα | |
| SNY-2289 | Sanofi | Phase 2/3 | CD38 | |
| Fixainavolisib | Takeda | Preclinical | CDK2 | |
| Fixazanubrutinib | Amgen | Phase 2 | PI3Kα | |
| Elrarapivir | Vertex Pharma | NDA/BLA | PRMT5 | |
| Rimaglumide | Biogen | Phase 1/2 | BCL-2 | |
| ARG-3458 | Argenx | Preclinical | RET | |
| GMA-1468 | Genmab | NDA/BLA | PI3Kα | |
| 369-789 | Hansoh Pharma | Phase 3 | PI3Kα |