Modality
Gene Therapy
MOA
WRNi
Target
CD38
Pathway
NF-κB
CSUDMD
Development Pipeline
Preclinical
~Feb 2014
→ ~May 2015
Phase 1
~Aug 2015
→ ~Nov 2016
Phase 2
~Feb 2017
→ ~May 2018
Phase 3
~Aug 2018
→ ~Nov 2019
NDA/BLA
~Feb 2020
→ ~May 2021
Approved
Aug 2021
→ Mar 2028
ApprovedCurrent
NCT08236037
2,631 pts·CSU
2025-06→2028-03·Terminated
NCT07579894
2,135 pts·CSU
2022-12→2025-11·Completed
NCT05335249
1,102 pts·CSU
2021-08→2025-07·Completed
5,868 total pts1 indication
CompletedCurrentUpcoming
Catalysts (3)
2025-07-288mo agoPh3 Readout· CSU
2025-11-095mo agoPh3 Readout· CSU
2028-03-081.9y awayPh3 Readout· CSU
Trial Timeline
Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2
Approved
Complet…
Approved
Complet…
Approved
Termina…
Catalysts
Ph3 Readout
2025-07-28 · 8mo ago
CSU
Ph3 Readout
2025-11-09 · 5mo ago
CSU
Ph3 Readout
2028-03-08 · 1.9y away
CSU
CompletedTerminated|StartCompletionToday
Trials (3)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08236037 | Approved | CSU | Terminated | 2631 | FEV1 |
| NCT07579894 | Approved | CSU | Completed | 2135 | CfB |
| NCT05335249 | Approved | CSU | Completed | 1102 | LiverFat |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Terafutibatinib | Pfizer | Phase 1/2 | CD38 | |
| Tirafotisoran | Roche | Phase 2 | CD38 | |
| NVS-6360 | Novartis | Approved | CD38 | |
| Doxasotorasib | AbbVie | Phase 2 | CD38 | |
| ABB-1817 | AbbVie | Phase 3 | SHP2 | |
| BMY-7975 | Bristol-Myers Squibb | Phase 1 | MALT1 | |
| SNY-2289 | Sanofi | Phase 2/3 | CD38 | |
| NVO-1361 | Novo Nordisk | Phase 3 | CD38 | |
| GSK-4334 | GSK | Preclinical | CD47 | |
| AMG-2752 | Amgen | Preclinical | MDM2 |