Modality
Gene Editing
MOA
PD-L1i
Target
EZH2
Pathway
Lipid Met
Dravet
Development Pipeline
Preclinical
~Feb 2015
→ ~May 2016
Phase 1
~Aug 2016
→ ~Nov 2017
Phase 2
~Feb 2018
→ ~May 2019
Phase 3
~Aug 2019
→ ~Nov 2020
NDA/BLA
Feb 2021
→ Oct 2031
NDA/BLACurrent
NCT04160458
2,245 pts·Dravet
2021-02→2031-10·Completed
NCT07612301
527 pts·Dravet
2021-02→TBD·Completed
2,772 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (1)
2031-10-215.6y awayPh3 Readout· Dravet
Trial Timeline
2021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
NDA/BLA
Complet…
NDA/BLA
Complet…
Catalysts
Ph3 Readout
2031-10-21 · 5.6y away
Dravet
Completed|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04160458 | NDA/BLA | Dravet | Completed | 2245 | PANSS |
| NCT07612301 | NDA/BLA | Dravet | Completed | 527 | ORR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-1085 | Pfizer | Preclinical | FXIa | |
| Rilulemzoparlimab | Novartis | Phase 2/3 | KRASG12D | |
| SNY-9600 | Sanofi | NDA/BLA | B7-H3 | |
| Nirafutibatinib | Bayer | Phase 2 | WRN | |
| DSN-2247 | Daiichi Sankyo | NDA/BLA | EZH2 | |
| Motainavolisib | Amgen | NDA/BLA | CD47 | |
| BNT-8090 | BioNTech | Phase 2 | AuroraA | |
| Miriglumide | Alnylam | Phase 3 | CD3 | |
| SRP-1135 | Sarepta | Phase 2/3 | MDM2 | |
| HAL-9635 | Halozyme | Phase 2/3 | EZH2 |