Modality
Gene Editing
MOA
ALKi
Target
B7-H3
Pathway
PD-1/PD-L1
CKDHemophilia A
Development Pipeline
Preclinical
~Nov 2018
→ ~Feb 2020
Phase 1
May 2020
→ Oct 2029
Phase 1Current
NCT05255097
2,730 pts·Hemophilia A
2020-05→2029-10·Not yet recruiting
NCT04124487
1,359 pts·Hemophilia A
2021-09→2027-08·Completed
4,089 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2027-08-141.4y awayInterim· Hemophilia A
2029-10-083.5y awayInterim· Hemophilia A
Trial Timeline
Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q4
P1
Not yet…
P1
Complet…
Catalysts
Interim
2027-08-14 · 1.4y away
Hemophilia A
Interim
2029-10-08 · 3.5y away
Hemophilia A
CompletedNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05255097 | Phase 1 | Hemophilia A | Not yet recr... | 2730 | SeizFreq |
| NCT04124487 | Phase 1 | Hemophilia A | Completed | 1359 | PFS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Miriosocimab | Pfizer | Phase 1 | PARP | |
| LLY-4358 | Eli Lilly | Phase 3 | B7-H3 | |
| Ceviglumide | Roche | NDA/BLA | B7-H3 | |
| MRK-7739 | Merck & Co | Preclinical | IL-23 | |
| BMY-2495 | Bristol-Myers Squibb | Phase 3 | KRASG12D | |
| SNY-9600 | Sanofi | NDA/BLA | B7-H3 | |
| SNY-8628 | Sanofi | NDA/BLA | B7-H3 | |
| SNY-9073 | Sanofi | Phase 2 | B7-H3 | |
| NVO-7840 | Novo Nordisk | Phase 2/3 | B7-H3 | |
| NVO-7872 | Novo Nordisk | NDA/BLA | B7-H3 |