Modality
siRNA
MOA
FcRni
Target
PRMT5
Pathway
Cell Cycle
CholangiocarcinomaPompe
Development Pipeline
Preclinical
~Feb 2014
→ ~May 2015
Phase 1
~Aug 2015
→ ~Nov 2016
Phase 2
~Feb 2017
→ ~May 2018
Phase 3
Aug 2018
→ Sep 2025
Phase 3Current
NCT05887547
2,525 pts·Pompe
2024-06→2025-07·Completed
NCT06642700
1,493 pts·Pompe
2018-08→2025-09·Completed
4,018 total pts1 indication
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-07-139mo agoPh3 Readout· Pompe
2025-09-127mo agoPh3 Readout· Pompe
Trial Timeline
Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q4
P3
Complet…
P3
Complet…
Catalysts
Ph3 Readout
2025-07-13 · 9mo ago
Pompe
Ph3 Readout
2025-09-12 · 7mo ago
Pompe
Completed|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05887547 | Phase 3 | Pompe | Completed | 2525 | DAS28 |
| NCT06642700 | Phase 3 | Pompe | Completed | 1493 | Mayo |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Nirarelsin | Johnson & Johnson | Phase 2 | PRMT5 | |
| MRK-853 | Merck & Co | Phase 1 | PRMT5 | |
| ABB-2476 | AbbVie | Phase 1/2 | USP1 | |
| Geliderotide | Sanofi | Phase 1/2 | PRMT5 | |
| Capifutibatinib | Sanofi | Approved | PRMT5 | |
| NVO-9615 | Novo Nordisk | Phase 2/3 | PRMT5 | |
| REG-6699 | Regeneron | Phase 2/3 | TIM-3 | |
| Elrarapivir | Vertex Pharma | NDA/BLA | PRMT5 | |
| Darafutibatinib | BioNTech | Preclinical | PRMT5 | |
| Daratuximab | BioMarin | Phase 1 | PRMT5 |