Modality
siRNA
MOA
BCL-2i
Target
MALT1
Pathway
Sphingolipid
PNH
Development Pipeline
Preclinical
~Nov 2012
→ ~Feb 2014
Phase 1
~May 2014
→ ~Aug 2015
Phase 2
~Nov 2015
→ ~Feb 2017
Phase 3
~May 2017
→ ~Aug 2018
NDA/BLA
Nov 2018
→ Dec 2028
NDA/BLACurrent
NCT04437869
1,624 pts·PNH
2018-11→2028-06·Completed
NCT03469690
2,815 pts·PNH
2025-05→2028-12·Completed
4,439 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (2)
2028-06-032.2y awayPh3 Readout· PNH
2028-12-272.7y awayPh3 Readout· PNH
Trial Timeline
Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q4
NDA/BLA
Complet…
NDA/BLA
Complet…
Catalysts
Ph3 Readout
2028-06-03 · 2.2y away
PNH
Ph3 Readout
2028-12-27 · 2.7y away
PNH
Completed|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04437869 | NDA/BLA | PNH | Completed | 1624 | UPCR |
| NCT03469690 | NDA/BLA | PNH | Completed | 2815 | HAM-D |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-5501 | Pfizer | Phase 1 | BTK | |
| JNJ-8232 | Johnson & Johnson | NDA/BLA | MALT1 | |
| RHH-3592 | Roche | Phase 1/2 | GIP-R | |
| Geliderotide | Novartis | NDA/BLA | IL-17A | |
| BMY-7975 | Bristol-Myers Squibb | Phase 1 | MALT1 | |
| GSK-6516 | GSK | Phase 1/2 | MALT1 | |
| Tezecilimab | Takeda | NDA/BLA | Tau | |
| Rimaosocimab | Amgen | Preclinical | FXIa | |
| BII-5240 | Biogen | Phase 2/3 | MALT1 | |
| ALN-3958 | Alnylam | Phase 2 | MALT1 |