Modality
Cell Therapy
MOA
STINGag
Target
BTK
Pathway
Innate Imm
CFFTD
Development Pipeline
Preclinical
~Apr 2012
→ ~Jul 2013
Phase 1
~Oct 2013
→ ~Jan 2015
Phase 2
~Apr 2015
→ ~Jul 2016
Phase 3
~Oct 2016
→ ~Jan 2018
NDA/BLA
Apr 2018
→ Feb 2031
NDA/BLACurrent
NCT03151925
93 pts·FTD
2025-05→2029-03·Completed
NCT03223131
782 pts·CF
2018-04→2031-02·Terminated
875 total pts2 indications
Approved
CompletedCurrentUpcoming
Catalysts (2)
2029-03-102.9y awayPh3 Readout· FTD
2031-02-224.9y awayPh3 Readout· CF
Trial Timeline
2018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2
NDA/BLA
Termina…
NDA/BLA
Complet…
Catalysts
Ph3 Readout
2029-03-10 · 2.9y away
FTD
Ph3 Readout
2031-02-22 · 4.9y away
CF
CompletedTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03151925 | NDA/BLA | FTD | Completed | 93 | Mayo |
| NCT03223131 | NDA/BLA | CF | Terminated | 782 | SeizFreq |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-5501 | Pfizer | Phase 1 | BTK | |
| RHH-5389 | Roche | Preclinical | RET | |
| RHH-8482 | Roche | Phase 1 | BTK | |
| SNY-5894 | Sanofi | Approved | CFTR | |
| SNY-4496 | Sanofi | Phase 2/3 | CD3 | |
| SNY-5783 | Sanofi | Phase 1/2 | MET | |
| Zorizanubrutinib | Novo Nordisk | Phase 1 | BTK | |
| DSN-1421 | Daiichi Sankyo | Phase 3 | Nectin-4 | |
| GIL-4361 | Gilead Sciences | Preclinical | PCSK9 | |
| INC-5849 | Incyte | Phase 2 | PCSK9 |