Modality
ASO
MOA
FXIai
Target
BET
Pathway
Autophagy
MDSHemophilia AHCC
Development Pipeline
Preclinical
~Sep 2012
→ ~Dec 2013
Phase 1
~Mar 2014
→ ~Jun 2015
Phase 2
~Sep 2015
→ ~Dec 2016
Phase 3
Mar 2017
→ Aug 2030
Phase 3Current
NCT08982743
1,386 pts·Hemophilia A
2017-03→2030-04·Not yet recruiting
NCT08926070
2,428 pts·HCC
2021-12→2025-09·Active
NCT08396136
1,787 pts·Hemophilia A
2020-09→2030-08·Recruiting
5,601 total pts2 indications
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (3)
2025-09-186mo agoPh3 Readout· HCC
2030-04-074.0y awayPh3 Readout· Hemophilia A
2030-08-044.3y awayPh3 Readout· Hemophilia A
Trial Timeline
2017Q2Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q4
P3
Not yet…
P3
Recruit…
P3
Active
Catalysts
Ph3 Readout
2025-09-18 · 6mo ago
HCC
Ph3 Readout
2030-04-07 · 4.0y away
Hemophilia A
Ph3 Readout
2030-08-04 · 4.3y away
Hemophilia A
RecruitingActiveNot yet recruiting|StartCompletionToday
Trials (3)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08982743 | Phase 3 | Hemophilia A | Not yet recr... | 1386 | ORR |
| NCT08926070 | Phase 3 | HCC | Active | 2428 | UPCR |
| NCT08396136 | Phase 3 | Hemophilia A | Recruiting | 1787 | ORR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-5767 | Pfizer | Phase 1/2 | BET | |
| RHH-1969 | Roche | Approved | BET | |
| NVS-5439 | Novartis | Preclinical | BET | |
| NVS-6360 | Novartis | Approved | CD38 | |
| MRK-3745 | Merck & Co | Phase 2 | WRN | |
| AZN-1715 | AstraZeneca | Phase 3 | AHR | |
| Ivocapivasertib | Bristol-Myers Squibb | Phase 1 | BET | |
| BMY-9931 | Bristol-Myers Squibb | Phase 1 | GPRC5D | |
| NVO-7877 | Novo Nordisk | Preclinical | GPRC5D | |
| TAK-8730 | Takeda | Phase 2 | VEGF |