Modality
Gene Editing
MOA
PCSK9i
Target
KIF18A
Pathway
T-cell
Endometrial CaPsoriasisHemophilia A
Development Pipeline
Preclinical
~Apr 2013
→ ~Jul 2014
Phase 1
~Oct 2014
→ ~Jan 2016
Phase 2
~Apr 2016
→ ~Jul 2017
Phase 3
~Oct 2017
→ ~Jan 2019
NDA/BLA
Apr 2019
→ Aug 2027
NDA/BLACurrent
NCT06397924
918 pts·Endometrial Ca
2019-07→TBD·Not yet recruiting
NCT03406222
680 pts·Hemophilia A
2019-04→2027-08·Not yet recruiting
1,598 total pts2 indications
Approved
CompletedCurrentUpcoming
Catalysts (1)
2027-08-191.4y awayPh3 Readout· Hemophilia A
Trial Timeline
Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q4
NDA/BLA
Not yet…
NDA/BLA
Not yet…
Catalysts
Ph3 Readout
2027-08-19 · 1.4y away
Hemophilia A
Not yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT06397924 | NDA/BLA | Endometrial Ca | Not yet recr... | 918 | EFS |
| NCT03406222 | NDA/BLA | Hemophilia A | Not yet recr... | 680 | CR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Niranesiran | Johnson & Johnson | Phase 2 | KIF18A | |
| MRK-8368 | Merck & Co | Phase 3 | ALK | |
| Ribofutibatinib | AstraZeneca | Phase 2/3 | SOS1 | |
| AZN-4015 | AstraZeneca | Preclinical | BCMA | |
| Polazasiran | Amgen | Phase 2 | LAG-3 | |
| Cevitinib | Regeneron | Phase 3 | FGFR | |
| Riluinavolisib | Moderna | NDA/BLA | KIF18A | |
| INC-1261 | Incyte | Phase 1/2 | PRMT5 | |
| Mavutuximab | Hansoh Pharma | Phase 2/3 | PI3Kα | |
| NUV-2032 | Nuvalent | Preclinical | KIF18A |