Modality
siRNA
MOA
TYK2i
Target
FLT3
Pathway
JAK/STAT
PNH
Development Pipeline
Preclinical
~Mar 2016
→ ~Jun 2017
Phase 1
~Sep 2017
→ ~Dec 2018
Phase 2
~Mar 2019
→ ~Jun 2020
Phase 3
~Sep 2020
→ ~Dec 2021
NDA/BLA
~Mar 2022
→ ~Jun 2023
Approved
Sep 2023
→ Feb 2031
ApprovedCurrent
NCT04156931
1,445 pts·PNH
2023-09→2031-02·Active
1,445 total pts1 indication
CompletedCurrentUpcoming
Catalysts (1)
2031-02-114.9y awayPh3 Readout· PNH
Trial Timeline
Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2
Approved
Active
Catalysts
Ph3 Readout
2031-02-11 · 4.9y away
PNH
Active|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04156931 | Approved | PNH | Active | 1445 | UPDRS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| JNJ-8168 | Johnson & Johnson | Phase 2/3 | FLT3 | |
| Nidasacituzumab | Eli Lilly | Approved | CFTR | |
| Lisonaritide | Eli Lilly | Phase 3 | IL-13 | |
| MRK-2473 | Merck & Co | Phase 1/2 | Nectin-4 | |
| Zorisotorasib | AstraZeneca | Phase 1 | WRN | |
| SNY-8628 | Sanofi | NDA/BLA | B7-H3 | |
| Voxacagene | Gilead Sciences | Phase 2/3 | SOS1 | |
| Sotosacituzumab | Vertex Pharma | Phase 1 | MET | |
| Olpainavolisib | Moderna | Phase 1 | Menin | |
| ARG-4339 | Argenx | Phase 2 | BCL-2 |