Modality
mAb
MOA
GLP-1ag
Target
WRN
Pathway
RAS/MAPK
Hemophilia A
Development Pipeline
Preclinical
~Aug 2009
→ ~Nov 2010
Phase 1
~Feb 2011
→ ~May 2012
Phase 2
~Aug 2012
→ ~Nov 2013
Phase 3
~Feb 2014
→ ~May 2015
NDA/BLA
~Aug 2015
→ ~Nov 2016
Approved
Feb 2017
→ Dec 2028
ApprovedCurrent
NCT05372738
2,453 pts·Hemophilia A
2024-03→2028-12·Not yet recruiting
NCT03949695
1,275 pts·Hemophilia A
2020-06→2026-07·Recruiting
NCT03589659
1,533 pts·Hemophilia A
2017-02→2027-11·Active
5,261 total pts1 indication
CompletedCurrentUpcoming
Catalysts (3)
2026-07-274mo awayPh3 Readout· Hemophilia A
2027-11-271.7y awayPh3 Readout· Hemophilia A
2028-12-012.7y awayPh3 Readout· Hemophilia A
Trial Timeline
2017Q2Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q4
Approved
Active
Approved
Recruit…
Approved
Not yet…
Catalysts
Ph3 Readout
2026-07-27 · 4mo away
Hemophilia A
Ph3 Readout
2027-11-27 · 1.7y away
Hemophilia A
Ph3 Readout
2028-12-01 · 2.7y away
Hemophilia A
RecruitingActiveNot yet recruiting|StartCompletionToday
Trials (3)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05372738 | Approved | Hemophilia A | Not yet recr... | 2453 | PASI75 |
| NCT03949695 | Approved | Hemophilia A | Recruiting | 1275 | UPDRS |
| NCT03589659 | Approved | Hemophilia A | Active | 1533 | Mayo |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-8662 | Pfizer | NDA/BLA | CD19 | |
| MRK-3745 | Merck & Co | Phase 2 | WRN | |
| Zorisotorasib | AstraZeneca | Phase 1 | WRN | |
| BMY-3371 | Bristol-Myers Squibb | Phase 3 | WRN | |
| NVO-7840 | Novo Nordisk | Phase 2/3 | B7-H3 | |
| Lisorapivir | Takeda | Preclinical | WRN | |
| Nirafutibatinib | Bayer | Phase 2 | WRN | |
| MRN-7601 | Moderna | Phase 2 | IL-13 | |
| ARG-1250 | Argenx | Phase 2 | C5 | |
| ILM-5680 | Illumina | Phase 2 | BET |