Modality
Gene Editing
MOA
HPK1i
Target
AuroraA
Pathway
Hedgehog
PNH
Development Pipeline
Preclinical
~Jan 2019
→ ~Apr 2020
Phase 1
~Jul 2020
→ ~Oct 2021
Phase 2
~Jan 2022
→ ~Apr 2023
Phase 3
~Jul 2023
→ ~Oct 2024
NDA/BLA
Jan 2025
→ May 2030
NDA/BLACurrent
NCT06190756
2,608 pts·PNH
2025-01→2030-05·Recruiting
NCT05474443
2,983 pts·PNH
2025-10→2028-05·Active
5,591 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (2)
2028-05-122.1y awayPh3 Readout· PNH
2030-05-044.1y awayPh3 Readout· PNH
Trial Timeline
2025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2
NDA/BLA
Recruit…
NDA/BLA
Active
Catalysts
Ph3 Readout
2028-05-12 · 2.1y away
PNH
Ph3 Readout
2030-05-04 · 4.1y away
PNH
RecruitingActive|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT06190756 | NDA/BLA | PNH | Recruiting | 2608 | Safety |
| NCT05474443 | NDA/BLA | PNH | Active | 2983 | FEV1 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-1944 | Pfizer | NDA/BLA | APOC3 | |
| NVS-6974 | Novartis | Preclinical | CD19 | |
| ABB-7516 | AbbVie | Phase 3 | PD-L1 | |
| Motarapivir | GSK | Preclinical | AuroraA | |
| BAY-8733 | Bayer | Preclinical | AuroraA | |
| BNT-8090 | BioNTech | Phase 2 | AuroraA | |
| Bemanesiran | BioNTech | Phase 3 | PLK4 | |
| BII-5240 | Biogen | Phase 2/3 | MALT1 | |
| ARG-6988 | Argenx | Preclinical | AuroraA | |
| Semasacituzumab | Illumina | Phase 3 | Nectin-4 |