Modality
Fusion Protein
MOA
C5i
Target
BTK
Pathway
mTOR
DMD
Development Pipeline
Preclinical
Jun 2017
→ Apr 2026
PreclinicalCurrent
NCT04829540
2,175 pts·DMD
2021-05→2025-07·Terminated
NCT08393372
1,395 pts·DMD
2017-06→2026-04·Active
3,570 total pts1 indication
Phase 1
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-07-248mo agoInterim· DMD
2026-04-264w awayInterim· DMD
Trial Timeline
Q2Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2
Preclinical
Active
Preclinical
Termina…
Catalysts
Interim
2025-07-24 · 8mo ago
DMD
Interim
2026-04-26 · 4w away
DMD
ActiveTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04829540 | Preclinical | DMD | Terminated | 2175 | MRD |
| NCT08393372 | Preclinical | DMD | Active | 1395 | FEV1 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-5501 | Pfizer | Phase 1 | BTK | |
| RHH-8482 | Roche | Phase 1 | BTK | |
| Zorizanubrutinib | Novo Nordisk | Phase 1 | BTK | |
| DSN-2247 | Daiichi Sankyo | NDA/BLA | EZH2 | |
| 369-8021 | Hansoh Pharma | Approved | BTK | |
| Rimabrutinib | Madrigal Pharma | Phase 1/2 | GPRC5D | |
| XEN-577 | Xenon Pharma | Phase 2/3 | C5 | |
| Pemitinib | Xenon Pharma | Preclinical | BTK | |
| Terazumab | Rapport Ther | Phase 2 | BTK | |
| EXA-4696 | Exscientia | Phase 2/3 | KRASG12C |