Modality
ERT
MOA
KRASG12Ci
Target
JAK2
Pathway
RAS/MAPK
Narcolepsy
Development Pipeline
Preclinical
~Nov 2017
→ ~Feb 2019
Phase 1
~May 2019
→ ~Aug 2020
Phase 2
Nov 2020
→ Feb 2026
Phase 2Current
NCT03969468
1,662 pts·Narcolepsy
2020-11→2025-12·Completed
NCT08558565
584 pts·Narcolepsy
2022-04→2026-02·Terminated
2,246 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-12-263mo agoPh3 Readout· Narcolepsy
2026-02-042mo agoPh3 Readout· Narcolepsy
Trial Timeline
Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026
P2/3
Complet…
P2/3
Termina…
Catalysts
Ph3 Readout
2025-12-26 · 3mo ago
Narcolepsy
Ph3 Readout
2026-02-04 · 2mo ago
Narcolepsy
CompletedTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03969468 | Phase 2/3 | Narcolepsy | Completed | 1662 | EDSS |
| NCT08558565 | Phase 2/3 | Narcolepsy | Terminated | 584 | VA |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Lisolucimab | Novartis | Approved | CD20 | |
| Gozelemzoparlimab | Merck & Co | Phase 2 | FXIa | |
| AZN-8478 | AstraZeneca | NDA/BLA | SHP2 | |
| Pexazasiran | Sanofi | Preclinical | FXIa | |
| Terasacituzumab | Novo Nordisk | NDA/BLA | IL-23 | |
| ALN-6288 | Alnylam | Approved | LAG-3 | |
| GMA-5010 | Genmab | Phase 3 | JAK2 | |
| BMR-9762 | BioMarin | Phase 3 | Aβ | |
| Niralucimab | Ionis | Phase 1 | ALK | |
| Pexaosocimab | United Therapeutics | NDA/BLA | PLK4 |