Modality
mRNA
MOA
CD3xCD20
Target
BTK
Pathway
Amyloid
Pompe
Development Pipeline
Preclinical
~Sep 2015
→ ~Dec 2016
Phase 1
Mar 2017
→ Sep 2030
Phase 1Current
NCT06026756
1,490 pts·Pompe
2020-04→2029-11·Completed
NCT08753632
1,988 pts·Pompe
2017-03→2030-09·Terminated
3,478 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2029-11-213.6y awayInterim· Pompe
2030-09-064.4y awayInterim· Pompe
Trial Timeline
2017Q2Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q4
P1
Termina…
P1
Complet…
Catalysts
Interim
2029-11-21 · 3.6y away
Pompe
Interim
2030-09-06 · 4.4y away
Pompe
CompletedTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT06026756 | Phase 1 | Pompe | Completed | 1490 | ORR |
| NCT08753632 | Phase 1 | Pompe | Terminated | 1988 | ORR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-5501 | Pfizer | Phase 1 | BTK | |
| LLY-1184 | Eli Lilly | NDA/BLA | C5 | |
| RHH-8482 | Roche | Phase 1 | BTK | |
| Zanuderotide | Novartis | Phase 1/2 | VEGF | |
| NVO-1361 | Novo Nordisk | Phase 3 | CD38 | |
| Pexamavacamten | Daiichi Sankyo | NDA/BLA | TIGIT | |
| ITC-1028 | Intra-Cellular | Phase 2 | BCMA | |
| 369-8021 | Hansoh Pharma | Approved | BTK | |
| Cevizasiran | Acadia Pharma | Phase 1/2 | GPRC5D | |
| Pemitinib | Xenon Pharma | Preclinical | BTK |