Modality
Vaccine
MOA
PCSK9i
Target
MALT1
Pathway
Hedgehog
Hemophilia AOvarian CaTTR Amyloidosis
Development Pipeline
Preclinical
~Apr 2011
→ ~Jul 2012
Phase 1
~Oct 2012
→ ~Jan 2014
Phase 2
~Apr 2014
→ ~Jul 2015
Phase 3
~Oct 2015
→ ~Jan 2017
NDA/BLA
Apr 2017
→ Dec 2025
NDA/BLACurrent
NCT07574638
55 pts·Hemophilia A
2017-04→2025-12·Recruiting
NCT06073177
337 pts·Ovarian Ca
2022-09→TBD·Terminated
392 total pts2 indications
Approved
CompletedCurrentUpcoming
Catalysts (1)
2025-12-223mo agoPh3 Readout· Hemophilia A
Trial Timeline
Q2Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q4
NDA/BLA
Recruit…
NDA/BLA
Termina…
Catalysts
Ph3 Readout
2025-12-22 · 3mo ago
Hemophilia A
RecruitingTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07574638 | NDA/BLA | Hemophilia A | Recruiting | 55 | ORR |
| NCT06073177 | NDA/BLA | Ovarian Ca | Terminated | 337 | HAM-D |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| JNJ-8232 | Johnson & Johnson | NDA/BLA | MALT1 | |
| MRK-8368 | Merck & Co | Phase 3 | ALK | |
| Ribofutibatinib | AstraZeneca | Phase 2/3 | SOS1 | |
| AZN-4015 | AstraZeneca | Preclinical | BCMA | |
| BMY-7975 | Bristol-Myers Squibb | Phase 1 | MALT1 | |
| GSK-6516 | GSK | Phase 1/2 | MALT1 | |
| Polazasiran | Amgen | Phase 2 | LAG-3 | |
| Cevitinib | Regeneron | Phase 3 | FGFR | |
| BII-5240 | Biogen | Phase 2/3 | MALT1 | |
| ALN-3958 | Alnylam | Phase 2 | MALT1 |