Modality
Gene Editing
MOA
KRASG12Di
Target
SMN2
Pathway
DDR
PAH
Development Pipeline
Preclinical
~Nov 2017
→ ~Feb 2019
Phase 1
~May 2019
→ ~Aug 2020
Phase 2
~Nov 2020
→ ~Feb 2022
Phase 3
~May 2022
→ ~Aug 2023
NDA/BLA
~Nov 2023
→ ~Feb 2025
Approved
May 2025
→ Oct 2029
ApprovedCurrent
NCT03871162
558 pts·PAH
2025-05→2029-10·Terminated
558 total pts1 indication
CompletedCurrentUpcoming
Catalysts (1)
2029-10-163.5y awayPh3 Readout· PAH
Trial Timeline
Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q4
Approved
Termina…
Catalysts
Ph3 Readout
2029-10-16 · 3.5y away
PAH
Terminated|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03871162 | Approved | PAH | Terminated | 558 | OS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Doxarasimod | Merck & Co | NDA/BLA | FGFR | |
| Zanusertib | AbbVie | Approved | PCSK9 | |
| Pexarelsin | AstraZeneca | Phase 1 | DLL3 | |
| NVO-6275 | Novo Nordisk | Phase 1 | SMN2 | |
| Ivotuximab | GSK | NDA/BLA | GIP-R | |
| TAK-5300 | Takeda | Phase 1 | SMN2 | |
| GIL-2259 | Gilead Sciences | Approved | SMN2 | |
| REG-647 | Regeneron | Preclinical | SMN2 | |
| Terarasimod | Krystal Biotech | Phase 1/2 | PI3Kα | |
| Olpafutibatinib | Immunocore | Approved | SMN2 |