Modality
ASO
MOA
JAK1/2i
Target
CDK2
Pathway
Wnt
PompeADPKDDravet
Development Pipeline
Preclinical
~Sep 2015
→ ~Dec 2016
Phase 1
Mar 2017
→ Oct 2028
Phase 1Current
NCT06915514
1,013 pts·Pompe
2022-04→2028-10·Terminated
NCT05819854
1,989 pts·Dravet
2017-03→2025-07·Active
3,002 total pts2 indications
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (3)
2025-07-049mo agoPh2 Data· Dravet
2026-05-151mo awayPDUFA· Dravet
2028-10-162.5y awayPh2 Data· Pompe
Trial Timeline
2017Q2Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q4
P1/2
Active
P1/2
Termina…
Catalysts
Ph2 Data
2025-07-04 · 9mo ago
Dravet
PDUFA
2026-05-15 · 1mo away
Dravet
Ph2 Data
2028-10-16 · 2.5y away
Pompe
ActiveTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT06915514 | Phase 1/2 | Pompe | Terminated | 1013 | OS |
| NCT05819854 | Phase 1/2 | Dravet | Active | 1989 | ORR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-3703 | Pfizer | Phase 1/2 | CDK2 | |
| NVS-3297 | Novartis | Preclinical | CDK2 | |
| Fixanesiran | AbbVie | Preclinical | RET | |
| Fixainavolisib | Takeda | Preclinical | CDK2 | |
| AMG-1919 | Amgen | Phase 2/3 | CDK2 | |
| AMG-415 | Amgen | Phase 1 | CDK2 | |
| AMG-9052 | Amgen | Phase 2/3 | CDK2 | |
| Motavorutinib | Amgen | Phase 3 | CDK4/6 | |
| Zanuratamab | Gilead Sciences | Phase 1/2 | CDK2 | |
| VRT-9481 | Vertex Pharma | Phase 1/2 | Menin |