Modality
Gene Therapy
MOA
SHP2i
Target
WRN
Pathway
Neuroinflam
Ewing SarcomaADPKD
Development Pipeline
Preclinical
~Mar 2023
→ ~Jun 2024
Phase 1
Sep 2024
→ May 2031
Phase 1Current
NCT07869694
1,608 pts·ADPKD
2024-09→2031-05·Completed
NCT06938147
2,601 pts·Ewing Sarcoma
2024-10→2029-04·Terminated
4,209 total pts2 indications
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2029-04-263.1y awayPh2 Data· Ewing Sarcoma
2031-05-275.2y awayPh2 Data· ADPKD
Trial Timeline
Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3
P1/2
Complet…
P1/2
Termina…
Catalysts
Ph2 Data
2029-04-26 · 3.1y away
Ewing Sarcoma
Ph2 Data
2031-05-27 · 5.2y away
ADPKD
CompletedTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07869694 | Phase 1/2 | ADPKD | Completed | 1608 | HAM-D |
| NCT06938147 | Phase 1/2 | Ewing Sarcoma | Terminated | 2601 | VA |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-2191 | Pfizer | NDA/BLA | GLP-1R | |
| MRK-3745 | Merck & Co | Phase 2 | WRN | |
| Zorisotorasib | AstraZeneca | Phase 1 | WRN | |
| BMY-3371 | Bristol-Myers Squibb | Phase 3 | WRN | |
| Doxacagene | Sanofi | Approved | PSMA | |
| Lisorapivir | Takeda | Preclinical | WRN | |
| Nirafutibatinib | Bayer | Phase 2 | WRN | |
| VRT-1576 | Vertex Pharma | Phase 2/3 | RET | |
| Sovafutibatinib | Moderna | Approved | FcRn | |
| Daratuximab | BioMarin | Phase 1 | PRMT5 |