Modality
Gene Editing
MOA
C5i
Target
GPRC5D
Pathway
Autophagy
PNHEpilepsy
Development Pipeline
Preclinical
~Dec 2016
→ ~Mar 2018
Phase 1
Jun 2018
→ May 2031
Phase 1Current
NCT07840002
2,204 pts·Epilepsy
2022-09→TBD·Terminated
NCT07481208
544 pts·Epilepsy
2018-06→2031-05·Recruiting
2,748 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2031-05-285.2y awayPh2 Data· Epilepsy
Trial Timeline
Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3
P1/2
Recruit…
P1/2
Termina…
Catalysts
Ph2 Data
2031-05-28 · 5.2y away
Epilepsy
RecruitingTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07840002 | Phase 1/2 | Epilepsy | Terminated | 2204 | FEV1 |
| NCT07481208 | Phase 1/2 | Epilepsy | Recruiting | 544 | HAM-D |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Pexatenlimab | Novartis | Phase 1/2 | GPRC5D | |
| Ribotuximab | Merck & Co | Phase 2 | GPRC5D | |
| BMY-9931 | Bristol-Myers Squibb | Phase 1 | GPRC5D | |
| NVO-7877 | Novo Nordisk | Preclinical | GPRC5D | |
| DSN-2247 | Daiichi Sankyo | NDA/BLA | EZH2 | |
| AMG-9654 | Amgen | Phase 2 | GPRC5D | |
| GIL-9227 | Gilead Sciences | Phase 2/3 | GPRC5D | |
| Ribozanubrutinib | Genmab | Approved | GPRC5D | |
| ASN-7408 | Ascendis Pharma | NDA/BLA | GPRC5D | |
| RVM-3746 | Revolution Medicines | Phase 1/2 | GPRC5D |