Modality
Gene Editing
MOA
AHRant
Target
RET
Pathway
Hedgehog
UC
Development Pipeline
Preclinical
~Nov 2013
→ ~Feb 2015
Phase 1
~May 2015
→ ~Aug 2016
Phase 2
~Nov 2016
→ ~Feb 2018
Phase 3
~May 2018
→ ~Aug 2019
NDA/BLA
~Nov 2019
→ ~Feb 2021
Approved
May 2021
→ Jun 2027
ApprovedCurrent
NCT06651615
55 pts·UC
2021-05→2027-06·Completed
55 total pts1 indication
CompletedCurrentUpcoming
Catalysts (2)
2025-11-154mo agoAdCom· UC
2027-06-041.2y awayPh3 Readout· UC
Trial Timeline
Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3
Approved
Complet…
Catalysts
AdCom
2025-11-15 · 4mo ago
UC
Ph3 Readout
2027-06-04 · 1.2y away
UC
Completed|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT06651615 | Approved | UC | Completed | 55 | ACR20 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| RHH-5389 | Roche | Preclinical | RET | |
| Fixanesiran | AbbVie | Preclinical | RET | |
| AZN-5978 | AstraZeneca | Phase 2/3 | GIP-R | |
| BMY-8678 | Bristol-Myers Squibb | Approved | RET | |
| VRT-1576 | Vertex Pharma | Phase 2/3 | RET | |
| BII-5449 | Biogen | Phase 3 | FXIa | |
| ARG-3458 | Argenx | Preclinical | RET | |
| Elratapinarof | Halozyme | Phase 2 | C5 | |
| Nidaratamab | Exelixis | Phase 3 | FLT3 | |
| Nidaratamab | Krystal Biotech | Preclinical | RET |