Modality
ASO
MOA
Menini
Target
JAK1
Pathway
RNA Splicing
NarcolepsyDravet
Development Pipeline
Preclinical
~Oct 2014
→ ~Jan 2016
Phase 1
~Apr 2016
→ ~Jul 2017
Phase 2
Oct 2017
→ Apr 2030
Phase 2Current
NCT05031248
1,003 pts·Dravet
2025-07→2030-04·Active
NCT04791119
820 pts·Narcolepsy
2017-10→2025-09·Recruiting
1,823 total pts2 indications
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-09-027mo agoPh2 Data· Narcolepsy
2030-04-054.0y awayPh2 Data· Dravet
Trial Timeline
Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3
P2
Recruit…
P2
Active
Catalysts
Ph2 Data
2025-09-02 · 7mo ago
Narcolepsy
Ph2 Data
2030-04-05 · 4.0y away
Dravet
RecruitingActive|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05031248 | Phase 2 | Dravet | Active | 1003 | BodyWt |
| NCT04791119 | Phase 2 | Narcolepsy | Recruiting | 820 | UPCR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Mavutenlimab | Johnson & Johnson | Phase 3 | Cl18.2 | |
| Ivorelsin | Eli Lilly | Approved | JAK1 | |
| RHH-974 | Roche | Preclinical | JAK1 | |
| NVO-2974 | Novo Nordisk | NDA/BLA | PARP | |
| Fixatenlimab | GSK | Phase 1/2 | JAK1 | |
| GIL-8529 | Gilead Sciences | Phase 1/2 | JAK1 | |
| Olpafutibatinib | Immunocore | Approved | SMN2 | |
| MLY-3531 | Mineralys | NDA/BLA | JAK1 | |
| Kemanesiran | Sage | Approved | JAK1 | |
| VYG-2970 | Voyager | Phase 3 | JAK1 |