Modality
Bispecific Ab
MOA
PCSK9i
Target
PI3Kα
Pathway
Sphingolipid
ACCSCLCHemophilia A
Development Pipeline
Preclinical
~Sep 2018
→ ~Dec 2019
Phase 1
~Mar 2020
→ ~Jun 2021
Phase 2
Sep 2021
→ May 2031
Phase 2Current
NCT03453657
1,148 pts·Hemophilia A
2021-09→2026-06·Recruiting
NCT08533319
2,076 pts·Hemophilia A
2023-09→2031-05·Not yet recruiting
3,224 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-06-233mo awayPh3 Readout· Hemophilia A
2031-05-285.2y awayPh3 Readout· Hemophilia A
Trial Timeline
Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3
P2/3
Recruit…
P2/3
Not yet…
Catalysts
Ph3 Readout
2026-06-23 · 3mo away
Hemophilia A
Ph3 Readout
2031-05-28 · 5.2y away
Hemophilia A
RecruitingNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03453657 | Phase 2/3 | Hemophilia A | Recruiting | 1148 | NT-proBNP |
| NCT08533319 | Phase 2/3 | Hemophilia A | Not yet recr... | 2076 | EASI-75 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Voxaderotide | Johnson & Johnson | NDA/BLA | PI3Kα | |
| MRK-8368 | Merck & Co | Phase 3 | ALK | |
| Rimamavacamten | Merck & Co | Phase 1 | PI3Kα | |
| Ribofutibatinib | AstraZeneca | Phase 2/3 | SOS1 | |
| AZN-4015 | AstraZeneca | Preclinical | BCMA | |
| Polazasiran | Amgen | Phase 2 | LAG-3 | |
| Fixazanubrutinib | Amgen | Phase 2 | PI3Kα | |
| Cevitinib | Regeneron | Phase 3 | FGFR | |
| GMA-1468 | Genmab | NDA/BLA | PI3Kα | |
| INC-1261 | Incyte | Phase 1/2 | PRMT5 |