Modality
Cell Therapy
MOA
CFTRmod
Target
PSMA
Pathway
JAK/STAT
Dravet
Development Pipeline
Preclinical
~Nov 2014
→ ~Feb 2016
Phase 1
~May 2016
→ ~Aug 2017
Phase 2
Nov 2017
→ Apr 2030
Phase 2Current
NCT07767237
2,308 pts·Dravet
2017-11→2030-04·Active
NCT03267530
1,936 pts·Dravet
2022-12→2028-08·Not yet recruiting
4,244 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2028-08-042.3y awayPh2 Data· Dravet
2030-04-034.0y awayPh2 Data· Dravet
Trial Timeline
Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3
P2
Active
P2
Not yet…
Catalysts
Ph2 Data
2028-08-04 · 2.3y away
Dravet
Ph2 Data
2030-04-03 · 4.0y away
Dravet
ActiveNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07767237 | Phase 2 | Dravet | Active | 2308 | BodyWt |
| NCT03267530 | Phase 2 | Dravet | Not yet recr... | 1936 | FEV1 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| LLY-4358 | Eli Lilly | Phase 3 | B7-H3 | |
| Gelinaritide | AbbVie | Preclinical | FcRn | |
| AZN-3724 | AstraZeneca | Preclinical | PSMA | |
| Doxacagene | Sanofi | Approved | PSMA | |
| GSK-2051 | GSK | Phase 1 | PSMA | |
| GIL-2445 | Gilead Sciences | Phase 2/3 | PSMA | |
| Darafutibatinib | BioNTech | Preclinical | PRMT5 | |
| BII-1564 | Biogen | Phase 2 | PSMA | |
| ALN-8757 | Alnylam | Preclinical | TIGIT | |
| BGN-3859 | BeiGene | Approved | PSMA |