Modality
ASO
MOA
SGLT2i
Target
WEE1
Pathway
Sphingolipid
Hemophilia ADravet
Development Pipeline
Preclinical
Feb 2021
→ Aug 2031
PreclinicalCurrent
NCT07376135
2,653 pts·Hemophilia A
2021-02→2031-08·Not yet recruiting
NCT07720359
1,857 pts·Dravet
2022-09→2025-07·Completed
4,510 total pts2 indications
Phase 1
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-07-238mo agoInterim· Dravet
2031-08-025.3y awayInterim· Hemophilia A
Trial Timeline
2021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
Preclinical
Not yet…
Preclinical
Complet…
Catalysts
Interim
2025-07-23 · 8mo ago
Dravet
Interim
2031-08-02 · 5.3y away
Hemophilia A
CompletedNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07376135 | Preclinical | Hemophilia A | Not yet recr... | 2653 | 6MWD |
| NCT07720359 | Preclinical | Dravet | Completed | 1857 | Mayo |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| JNJ-8232 | Johnson & Johnson | NDA/BLA | MALT1 | |
| Bemalucimab | Bristol-Myers Squibb | Phase 1/2 | Aβ | |
| TAK-2403 | Takeda | Phase 2 | WEE1 | |
| BAY-3308 | Bayer | Phase 1 | ALK | |
| Sovacapivasertib | Amgen | Phase 3 | WEE1 | |
| MRN-8225 | Moderna | Preclinical | TNFα | |
| Motazanubrutinib | Biogen | Preclinical | CD38 | |
| ALN-5628 | Alnylam | Phase 1/2 | APOC3 | |
| Fixaglumide | Genmab | Phase 2/3 | WEE1 | |
| Sovarasimod | Halozyme | Phase 1/2 | WEE1 |