Modality
siRNA
MOA
PCSK9i
Target
CGRP
Pathway
Innate Imm
Hemophilia A
Development Pipeline
Preclinical
~May 2012
→ ~Aug 2013
Phase 1
~Nov 2013
→ ~Feb 2015
Phase 2
~May 2015
→ ~Aug 2016
Phase 3
~Nov 2016
→ ~Feb 2018
NDA/BLA
May 2018
→ Sep 2029
NDA/BLACurrent
NCT06719690
2,304 pts·Hemophilia A
2018-05→2029-09·Terminated
2,304 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (1)
2029-09-163.5y awayPh3 Readout· Hemophilia A
Trial Timeline
Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q4
NDA/BLA
Termina…
Catalysts
Ph3 Readout
2029-09-16 · 3.5y away
Hemophilia A
Terminated|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT06719690 | NDA/BLA | Hemophilia A | Terminated | 2304 | eGFR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| MRK-8368 | Merck & Co | Phase 3 | ALK | |
| Ribofutibatinib | AstraZeneca | Phase 2/3 | SOS1 | |
| AZN-4015 | AstraZeneca | Preclinical | BCMA | |
| TAK-9344 | Takeda | Phase 3 | CGRP | |
| Polazasiran | Amgen | Phase 2 | LAG-3 | |
| Cevitinib | Regeneron | Phase 3 | FGFR | |
| Zenotinib | BioNTech | Phase 1 | CGRP | |
| Capiglumide | BioNTech | Preclinical | CGRP | |
| BII-4342 | Biogen | Phase 2/3 | CGRP | |
| INC-1261 | Incyte | Phase 1/2 | PRMT5 |