Modality
ERT
MOA
TNFi
Target
CD123
Pathway
Autophagy
NB
Development Pipeline
Preclinical
~Feb 2013
→ ~May 2014
Phase 1
~Aug 2014
→ ~Nov 2015
Phase 2
~Feb 2016
→ ~May 2017
Phase 3
~Aug 2017
→ ~Nov 2018
NDA/BLA
~Feb 2019
→ ~May 2020
Approved
Aug 2020
→ Nov 2028
ApprovedCurrent
NCT07477129
526 pts·NB
2025-02→TBD·Recruiting
NCT04192829
1,370 pts·NB
2020-08→TBD·Recruiting
NCT07839121
1,908 pts·NB
2022-01→2026-05·Completed
+1 more trial
6,399 total pts1 indication
CompletedCurrentUpcoming
Catalysts (2)
2026-05-202mo awayPh3 Readout· NB
2028-11-142.6y awayPh3 Readout· NB
Trial Timeline
Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q4
Approved
Recruit…
Approved
Active
Approved
Complet…
Approved
Recruit…
Catalysts
Ph3 Readout
2026-05-20 · 2mo away
NB
Ph3 Readout
2028-11-14 · 2.6y away
NB
RecruitingActiveCompleted|StartCompletionToday
Trials (4)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07477129 | Approved | NB | Recruiting | 526 | 6MWD |
| NCT04192829 | Approved | NB | Recruiting | 1370 | EDSS |
| NCT07839121 | Approved | NB | Completed | 1908 | UPCR |
| NCT05117691 | Approved | NB | Active | 2595 | EDSS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| AZN-7403 | AstraZeneca | Phase 2/3 | FGFR | |
| TAK-8262 | Takeda | NDA/BLA | Tau | |
| Talalemzoparlimab | Gilead Sciences | Phase 1 | FcRn | |
| REG-7737 | Regeneron | Preclinical | CD123 | |
| Ribozanubrutinib | Genmab | Approved | GPRC5D | |
| 369-789 | Hansoh Pharma | Phase 3 | PI3Kα | |
| Ivotuximab | Madrigal Pharma | Phase 2 | CD123 | |
| Zorizumab | Beam | Approved | CD123 | |
| Rimatinib | Ventyx Bio | Phase 1 | CD20 | |
| Talatuximab | Protagonist | Phase 2 | B7-H3 |