Modality
ERT
MOA
FGFRi
Target
PARP
Pathway
Wnt
NMOSDSMA
Development Pipeline
Preclinical
~Jan 2016
→ ~Apr 2017
Phase 1
Jul 2017
→ Jun 2027
Phase 1Current
NCT04758743
934 pts·SMA
2017-07→TBD·Active
NCT07559964
2,182 pts·NMOSD
2025-07→2027-06·Recruiting
3,116 total pts2 indications
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2027-06-151.2y awayInterim· NMOSD
Trial Timeline
Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3
P1
Active
P1
Recruit…
Catalysts
Interim
2027-06-15 · 1.2y away
NMOSD
RecruitingActive|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04758743 | Phase 1 | SMA | Active | 934 | UPCR |
| NCT07559964 | Phase 1 | NMOSD | Recruiting | 2182 | DAS28 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Miriosocimab | Pfizer | Phase 1 | PARP | |
| LLY-9749 | Eli Lilly | Phase 3 | C5 | |
| LLY-1956 | Eli Lilly | Phase 1/2 | BCMA | |
| NVO-2974 | Novo Nordisk | NDA/BLA | PARP | |
| Semainavolisib | Gilead Sciences | Phase 2 | PARP | |
| REG-861 | Regeneron | Phase 2 | PARP | |
| SRP-9822 | Sarepta | Phase 3 | SOS1 | |
| Rimainavolisib | Illumina | Phase 2 | PD-1 | |
| Elrainavolisib | Roivant Sciences | Preclinical | Nectin-4 | |
| CRS-337 | CRISPR Therapeutics | Phase 2/3 | PARP |