Modality
Gene Therapy
MOA
RAS(ON)i
Target
DLL3
Pathway
Notch
CMLPAH
Development Pipeline
Preclinical
~Aug 2019
→ ~Nov 2020
Phase 1
~Feb 2021
→ ~May 2022
Phase 2
~Aug 2022
→ ~Nov 2023
Phase 3
Feb 2024
→ Mar 2031
Phase 3Current
NCT06509023
2,105 pts·CML
2024-02→2031-03·Completed
NCT08066305
1,084 pts·PAH
2025-07→2027-04·Active
3,189 total pts2 indications
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2027-04-081.0y awayPh3 Readout· PAH
2031-03-074.9y awayPh3 Readout· CML
Trial Timeline
2024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2
P3
Complet…
P3
Active
Catalysts
Ph3 Readout
2027-04-08 · 1.0y away
PAH
Ph3 Readout
2031-03-07 · 4.9y away
CML
ActiveCompleted|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT06509023 | Phase 3 | CML | Completed | 2105 | PFS |
| NCT08066305 | Phase 3 | PAH | Active | 1084 | ACR20 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-7809 | Pfizer | Phase 1 | DLL3 | |
| MRK-6781 | Merck & Co | Approved | TIGIT | |
| Pexarelsin | AstraZeneca | Phase 1 | DLL3 | |
| Zorivorutinib | Bristol-Myers Squibb | Approved | DLL3 | |
| SNY-9073 | Sanofi | Phase 2 | B7-H3 | |
| Zenonesiran | GSK | Preclinical | DLL3 | |
| AMG-7379 | Amgen | Preclinical | DLL3 | |
| Tixarelsin | Vertex Pharma | Phase 1 | ALK | |
| ARG-1924 | Argenx | Preclinical | DLL3 | |
| Cevinaritide | Genmab | Phase 2/3 | DLL3 |