Modality
Gene Therapy
MOA
CGRPant
Target
B7-H3
Pathway
NF-κB
AngelmanUrothelial CaRett
Development Pipeline
Preclinical
~Aug 2019
→ ~Nov 2020
Phase 1
~Feb 2021
→ ~May 2022
Phase 2
~Aug 2022
→ ~Nov 2023
Phase 3
Feb 2024
Phase 3Current
NCT07051515
1,061 pts·Rett
2024-02→TBD·Terminated
1,061 total pts1 indication
NDA/BLA
Approved
CompletedCurrentUpcoming
Trial Timeline
P3
Termina…
Terminated|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07051515 | Phase 3 | Rett | Terminated | 1061 | EFS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Terafutibatinib | Pfizer | Phase 1/2 | CD38 | |
| Sotovorutinib | Pfizer | Approved | Aβ | |
| LLY-4358 | Eli Lilly | Phase 3 | B7-H3 | |
| Ceviglumide | Roche | NDA/BLA | B7-H3 | |
| ABB-3060 | AbbVie | Phase 2 | LAG-3 | |
| SNY-9600 | Sanofi | NDA/BLA | B7-H3 | |
| SNY-8628 | Sanofi | NDA/BLA | B7-H3 | |
| SNY-9073 | Sanofi | Phase 2 | B7-H3 | |
| NVO-7840 | Novo Nordisk | Phase 2/3 | B7-H3 | |
| NVO-7872 | Novo Nordisk | NDA/BLA | B7-H3 |