Modality
ASO
MOA
ALKi
Target
PARP
Pathway
Incretin
DravetBreast CaTTR Amyloidosis
Development Pipeline
Preclinical
~Jun 2017
→ ~Sep 2018
Phase 1
Dec 2018
→ Aug 2030
Phase 1Current
NCT08051536
2,758 pts·Dravet
2020-08→2026-07·Terminated
NCT05134858
2,787 pts·Dravet
2018-12→2030-08·Recruiting
5,545 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-07-224mo awayInterim· Dravet
2030-08-044.3y awayInterim· Dravet
Trial Timeline
Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q4
P1
Recruit…
P1
Termina…
Catalysts
Interim
2026-07-22 · 4mo away
Dravet
Interim
2030-08-04 · 4.3y away
Dravet
RecruitingTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08051536 | Phase 1 | Dravet | Terminated | 2758 | Biomarker |
| NCT05134858 | Phase 1 | Dravet | Recruiting | 2787 | CR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| MRK-7739 | Merck & Co | Preclinical | IL-23 | |
| BMY-2495 | Bristol-Myers Squibb | Phase 3 | KRASG12D | |
| NVO-2974 | Novo Nordisk | NDA/BLA | PARP | |
| DSN-5254 | Daiichi Sankyo | Phase 1 | B7-H3 | |
| Sovacapivasertib | Amgen | Phase 3 | WEE1 | |
| Zanuratamab | Gilead Sciences | Phase 1/2 | CDK2 | |
| GIL-2445 | Gilead Sciences | Phase 2/3 | PSMA | |
| Semainavolisib | Gilead Sciences | Phase 2 | PARP | |
| REG-861 | Regeneron | Phase 2 | PARP | |
| ALN-3284 | Alnylam | Phase 2/3 | CDK2 |