Modality
mRNA
MOA
IL-13i
Target
HER2
Pathway
Amyloid
Hemophilia AHeart FailureLGS
Development Pipeline
Preclinical
~Nov 2015
→ ~Feb 2017
Phase 1
~May 2017
→ ~Aug 2018
Phase 2
~Nov 2018
→ ~Feb 2020
Phase 3
~May 2020
→ ~Aug 2021
NDA/BLA
Nov 2021
→ Jul 2025
NDA/BLACurrent
NCT05368713
1,411 pts·Hemophilia A
2024-10→TBD·Not yet recruiting
NCT06285794
446 pts·Hemophilia A
2021-11→2025-07·Recruiting
1,857 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (1)
2025-07-168mo agoPh3 Readout· Hemophilia A
Trial Timeline
2022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3
NDA/BLA
Recruit…
NDA/BLA
Not yet…
Catalysts
Ph3 Readout
2025-07-16 · 8mo ago
Hemophilia A
RecruitingNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05368713 | NDA/BLA | Hemophilia A | Not yet recr... | 1411 | HAM-D |
| NCT06285794 | NDA/BLA | Hemophilia A | Recruiting | 446 | PANSS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Voxaderotide | Johnson & Johnson | NDA/BLA | PI3Kα | |
| RHH-7975 | Roche | Phase 3 | FXIa | |
| NVS-8902 | Novartis | Preclinical | TROP-2 | |
| BMY-4586 | Bristol-Myers Squibb | Preclinical | KRASG12C | |
| AMG-1919 | Amgen | Phase 2/3 | CDK2 | |
| Ribozanubrutinib | Regeneron | Phase 1 | CDK4/6 | |
| VRT-4938 | Vertex Pharma | Preclinical | HER2 | |
| ROI-9675 | Roivant Sciences | NDA/BLA | HER2 | |
| ROI-3353 | Roivant Sciences | Phase 1/2 | PLK4 | |
| CRS-337 | CRISPR Therapeutics | Phase 2/3 | PARP |