Modality
ERT
MOA
CGRPant
Target
PCSK9
Pathway
Amyloid
NMOSDMyelofibrosis
Development Pipeline
Preclinical
~Jan 2017
→ ~Apr 2018
Phase 1
~Jul 2018
→ ~Oct 2019
Phase 2
Jan 2020
→ Mar 2025
Phase 2Current
NCT04248967
1,153 pts·Myelofibrosis
2020-01→TBD·Recruiting
NCT03686554
2,510 pts·NMOSD
2024-04→2025-03·Completed
3,663 total pts2 indications
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2025-03-111.1y agoPh3 Readout· NMOSD
Trial Timeline
2020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2
P2/3
Recruit…
P2/3
Complet…
Catalysts
Ph3 Readout
2025-03-11 · 1.1y ago
NMOSD
RecruitingCompleted|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04248967 | Phase 2/3 | Myelofibrosis | Recruiting | 1153 | Biomarker |
| NCT03686554 | Phase 2/3 | NMOSD | Completed | 2510 | PASI75 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Terafutibatinib | Pfizer | Phase 1/2 | CD38 | |
| Sotovorutinib | Pfizer | Approved | Aβ | |
| ABB-3060 | AbbVie | Phase 2 | LAG-3 | |
| Zanusertib | AbbVie | Approved | PCSK9 | |
| GIL-6239 | Gilead Sciences | Phase 2 | TNFα | |
| GIL-4361 | Gilead Sciences | Preclinical | PCSK9 | |
| Tezecilimab | Regeneron | Approved | IL-23 | |
| REG-2172 | Regeneron | Phase 1/2 | PCSK9 | |
| BMR-8377 | BioMarin | Phase 1/2 | PCSK9 | |
| INC-2432 | Incyte | Phase 2 | CD47 |