Modality
ERT
MOA
CD47i
Target
DLL3
Pathway
STING
MGDravetPBC
Development Pipeline
Preclinical
Jan 2022
→ Dec 2025
PreclinicalCurrent
NCT07937775
1,020 pts·Dravet
2022-01→2025-12·Recruiting
NCT06834863
950 pts·MG
2025-08→TBD·Recruiting
1,970 total pts2 indications
Phase 1
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2025-12-114mo agoInterim· Dravet
Trial Timeline
2022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q4
Preclinical
Recruit…
Preclinical
Recruit…
Catalysts
Interim
2025-12-11 · 4mo ago
Dravet
Recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07937775 | Preclinical | Dravet | Recruiting | 1020 | 6MWD |
| NCT06834863 | Preclinical | MG | Recruiting | 950 | UPCR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-7809 | Pfizer | Phase 1 | DLL3 | |
| Nirarelsin | Johnson & Johnson | Phase 2 | PRMT5 | |
| Cevizanubrutinib | Johnson & Johnson | Preclinical | FcRn | |
| Ceviglumide | Roche | NDA/BLA | B7-H3 | |
| NVS-1475 | Novartis | Phase 2 | Menin | |
| Pexarelsin | AstraZeneca | Phase 1 | DLL3 | |
| AZN-6294 | AstraZeneca | Phase 2/3 | APOC3 | |
| Zorivorutinib | Bristol-Myers Squibb | Approved | DLL3 | |
| Zenonesiran | GSK | Preclinical | DLL3 | |
| ARG-1924 | Argenx | Preclinical | DLL3 |