Modality
Gene Therapy
MOA
FGFRi
Target
PD-1
Pathway
Sphingolipid
PNH
Development Pipeline
Preclinical
~Dec 2017
→ ~Mar 2019
Phase 1
~Jun 2019
→ ~Sep 2020
Phase 2
Dec 2020
→ Apr 2028
Phase 2Current
NCT05199170
1,226 pts·PNH
2020-12→2028-04·Recruiting
1,226 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2028-04-142.0y awayPh2 Data· PNH
Trial Timeline
2021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2
P2
Recruit…
Catalysts
Ph2 Data
2028-04-14 · 2.0y away
PNH
Recruiting|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05199170 | Phase 2 | PNH | Recruiting | 1226 | PASI75 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| JNJ-7458 | Johnson & Johnson | Phase 1/2 | PD-1 | |
| LLY-9749 | Eli Lilly | Phase 3 | C5 | |
| LLY-1956 | Eli Lilly | Phase 1/2 | BCMA | |
| Pexaosocimab | Daiichi Sankyo | Phase 2 | PD-1 | |
| MRN-8133 | Moderna | Phase 2 | PD-1 | |
| Semazasiran | BeiGene | Phase 1 | PD-1 | |
| SRP-9822 | Sarepta | Phase 3 | SOS1 | |
| Elrainavolisib | Roivant Sciences | Preclinical | Nectin-4 | |
| Motanaritide | Intellia | Approved | PD-1 | |
| VYG-2970 | Voyager | Phase 3 | JAK1 |