Modality
mRNA
MOA
TYK2i
Target
CD123
Pathway
PI3K/AKT
Angelman
Development Pipeline
Preclinical
~Jul 2020
→ ~Oct 2021
Phase 1
Jan 2022
→ Oct 2031
Phase 1Current
NCT06657660
1,433 pts·Angelman
2022-01→2031-10·Recruiting
1,433 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-08-165mo awayPDUFA· Angelman
2031-10-165.5y awayPh2 Data· Angelman
Trial Timeline
2022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
P1/2
Recruit…
Catalysts
PDUFA
2026-08-16 · 5mo away
Angelman
Ph2 Data
2031-10-16 · 5.5y away
Angelman
Recruiting|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT06657660 | Phase 1/2 | Angelman | Recruiting | 1433 | PASI75 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Nidasacituzumab | Eli Lilly | Approved | CFTR | |
| Lisonaritide | Eli Lilly | Phase 3 | IL-13 | |
| MRK-2473 | Merck & Co | Phase 1/2 | Nectin-4 | |
| Zorisotorasib | AstraZeneca | Phase 1 | WRN | |
| SNY-8628 | Sanofi | NDA/BLA | B7-H3 | |
| Voxacagene | Gilead Sciences | Phase 2/3 | SOS1 | |
| REG-7737 | Regeneron | Preclinical | CD123 | |
| Sotosacituzumab | Vertex Pharma | Phase 1 | MET | |
| Sotorapivir | Moderna | Approved | FLT3 | |
| Olpainavolisib | Moderna | Phase 1 | Menin |