Modality
siRNA
MOA
CFTRmod
Target
Tau
Pathway
DDR
MSGISTAngelman
Development Pipeline
Preclinical
~May 2015
→ ~Aug 2016
Phase 1
~Nov 2016
→ ~Feb 2018
Phase 2
~May 2018
→ ~Aug 2019
Phase 3
~Nov 2019
→ ~Feb 2021
NDA/BLA
~May 2021
→ ~Aug 2022
Approved
Nov 2022
→ Sep 2026
ApprovedCurrent
NCT05269372
2,996 pts·Angelman
2022-11→2026-09·Completed
2,996 total pts1 indication
CompletedCurrentUpcoming
Catalysts (1)
2026-09-025mo awayPh3 Readout· Angelman
Trial Timeline
2023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3
Approved
Complet…
Catalysts
Ph3 Readout
2026-09-02 · 5mo away
Angelman
Completed|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05269372 | Approved | Angelman | Completed | 2996 | Safety |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| LLY-4358 | Eli Lilly | Phase 3 | B7-H3 | |
| Gelinaritide | AbbVie | Preclinical | FcRn | |
| NVO-9087 | Novo Nordisk | Phase 1/2 | Tau | |
| Tezecilimab | Takeda | NDA/BLA | Tau | |
| TAK-8262 | Takeda | NDA/BLA | Tau | |
| Darafutibatinib | BioNTech | Preclinical | PRMT5 | |
| ALN-8757 | Alnylam | Preclinical | TIGIT | |
| Ivotuximab | Madrigal Pharma | Phase 2 | CD123 | |
| Talazasiran | Kymera | NDA/BLA | MET | |
| DAW-8159 | Day One Bio | Phase 2 | PSMA |