Modality
Gene Therapy
MOA
JAK1/2i
Target
HER2
Pathway
Epigenetic
CeliacALSPBC
Development Pipeline
Preclinical
~Dec 2020
→ ~Mar 2022
Phase 1
Jun 2022
→ Apr 2028
Phase 1Current
NCT03415865
810 pts·PBC
2022-06→2028-04·Not yet recruiting
810 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2028-04-032.0y awayInterim· PBC
Trial Timeline
Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2
P1
Not yet…
Catalysts
Interim
2028-04-03 · 2.0y away
PBC
Not yet recruiting|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03415865 | Phase 1 | PBC | Not yet recr... | 810 | Mayo |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Fixanesiran | AbbVie | Preclinical | RET | |
| Gozesotorasib | Bristol-Myers Squibb | NDA/BLA | HER2 | |
| Motavorutinib | Amgen | Phase 3 | CDK4/6 | |
| VRT-4938 | Vertex Pharma | Preclinical | HER2 | |
| VRT-9481 | Vertex Pharma | Phase 1/2 | Menin | |
| Sovarelsin | BioMarin | Phase 2 | CFTR | |
| Peminaritide | Sarepta | NDA/BLA | PLK4 | |
| Niramavacamten | Hansoh Pharma | Phase 2 | SGLT2 | |
| ROI-9675 | Roivant Sciences | NDA/BLA | HER2 | |
| Talazumab | Zealand Pharma | Phase 1/2 | WEE1 |