Modality
ASO
MOA
Cl18.2
Target
WEE1
Pathway
Notch
Pompe
Development Pipeline
Preclinical
~Oct 2014
→ ~Jan 2016
Phase 1
~Apr 2016
→ ~Jul 2017
Phase 2
Oct 2017
→ Aug 2025
Phase 2Current
NCT07985379
1,540 pts·Pompe
2023-07→2025-08·Active
NCT04584381
2,642 pts·Pompe
2017-10→TBD·Terminated
4,182 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-08-167mo agoPh2 Data· Pompe
2026-04-081w awayEnrollment Complete· Pompe
Trial Timeline
Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2
P2
Termina…
P2
Active
Catalysts
Ph2 Data
2025-08-16 · 7mo ago
Pompe
Enrollment Complete
2026-04-08 · 1w away
Pompe
ActiveTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07985379 | Phase 2 | Pompe | Active | 1540 | 6MWD |
| NCT04584381 | Phase 2 | Pompe | Terminated | 2642 | CR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Zorivorutinib | Bristol-Myers Squibb | Approved | DLL3 | |
| TAK-2403 | Takeda | Phase 2 | WEE1 | |
| Sovacapivasertib | Amgen | Phase 3 | WEE1 | |
| Elrarapivir | Regeneron | Phase 1 | GIP-R | |
| Fixaglumide | Genmab | Phase 2/3 | WEE1 | |
| HAL-9635 | Halozyme | Phase 2/3 | EZH2 | |
| Sovarasimod | Halozyme | Phase 1/2 | WEE1 | |
| VKT-6548 | Viking Therapeutics | Phase 2/3 | PD-L1 | |
| Tiratapinarof | Intellia | NDA/BLA | APOC3 | |
| Bemavorutinib | Acadia Pharma | Phase 1/2 | WEE1 |