Modality
Gene Editing
MOA
RAS(ON)i
Target
PI3Kα
Pathway
JAK/STAT
PBC
Development Pipeline
Preclinical
Mar 2018
PreclinicalCurrent
NCT03460222
338 pts·PBC
2018-03→TBD·Terminated
338 total pts1 indication
Phase 1
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2026-07-043mo awayPh1 Dose Esc· PBC
Trial Timeline
2018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3
Preclinical
Termina…
Catalysts
Ph1 Dose Esc
2026-07-04 · 3mo away
PBC
Terminated|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03460222 | Preclinical | PBC | Terminated | 338 | EFS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Voxaderotide | Johnson & Johnson | NDA/BLA | PI3Kα | |
| MRK-6781 | Merck & Co | Approved | TIGIT | |
| Rimamavacamten | Merck & Co | Phase 1 | PI3Kα | |
| SNY-9073 | Sanofi | Phase 2 | B7-H3 | |
| Fixazanubrutinib | Amgen | Phase 2 | PI3Kα | |
| Tixarelsin | Vertex Pharma | Phase 1 | ALK | |
| GMA-1468 | Genmab | NDA/BLA | PI3Kα | |
| Fixatuximab | Legend Biotech | Preclinical | CFTR | |
| ION-1378 | Ionis | NDA/BLA | TROP-2 | |
| 180-3597 | Innovent Bio | Preclinical | SHP2 |