Modality
Gene Editing
MOA
CFTRmod
Target
MET
Pathway
Apoptosis
HSNBGIST
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
→ Sep 2027
NDA/BLACurrent
NCT06096581
461 pts·HS
2025-01→2027-09·Terminated
461 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (1)
2027-09-031.4y awayPh3 Readout· HS
Trial Timeline
2025Q2Q3Q42026Q2Q3Q42027Q2Q3
NDA/BLA
Termina…
Catalysts
Ph3 Readout
2027-09-03 · 1.4y away
HS
Terminated|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT06096581 | NDA/BLA | HS | Terminated | 461 | NT-proBNP |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| LLY-4358 | Eli Lilly | Phase 3 | B7-H3 | |
| Gelinaritide | AbbVie | Preclinical | FcRn | |
| SNY-5783 | Sanofi | Phase 1/2 | MET | |
| Sotosacituzumab | Vertex Pharma | Phase 1 | MET | |
| Darafutibatinib | BioNTech | Preclinical | PRMT5 | |
| ALN-8757 | Alnylam | Preclinical | TIGIT | |
| RVM-274 | Revolution Medicines | Phase 3 | MET | |
| Ivotuximab | Madrigal Pharma | Phase 2 | CD123 | |
| DAW-8159 | Day One Bio | Phase 2 | PSMA | |
| RAP-2478 | Rapport Ther | Phase 3 | MET |