Modality
Gene Therapy
MOA
CDK4/6i
Target
Cl18.2
Pathway
Lipid Met
Urothelial CaSMA
Development Pipeline
Preclinical
Aug 2022
→ Apr 2026
PreclinicalCurrent
NCT04648075
1,165 pts·SMA
2022-08→2026-04·Completed
1,165 total pts1 indication
Phase 1
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2026-04-213w awayInterim· SMA
Trial Timeline
Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2
Preclinical
Complet…
Catalysts
Interim
2026-04-21 · 3w away
SMA
Completed|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04648075 | Preclinical | SMA | Completed | 1165 | FEV1 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-5767 | Pfizer | Phase 1/2 | BET | |
| Mavutenlimab | Johnson & Johnson | Phase 3 | Cl18.2 | |
| JNJ-2735 | Johnson & Johnson | Phase 2/3 | Cl18.2 | |
| RHH-1969 | Roche | Approved | BET | |
| Ribosacituzumab | Roche | Approved | Cl18.2 | |
| Adagrazumab | Novartis | Phase 2/3 | Cl18.2 | |
| Ivocapivasertib | Bristol-Myers Squibb | Phase 1 | BET | |
| Fixatenlimab | GSK | Phase 1/2 | JAK1 | |
| Doxafotisoran | Daiichi Sankyo | Phase 1 | Cl18.2 | |
| VRT-4938 | Vertex Pharma | Preclinical | HER2 |