Modality
siRNA
MOA
FXIai
Target
GIP-R
Pathway
JAK/STAT
SMAHemophilia A
Development Pipeline
Preclinical
~Jul 2020
→ ~Oct 2021
Phase 1
~Jan 2022
→ ~Apr 2023
Phase 2
Jul 2023
→ May 2028
Phase 2Current
NCT05761744
1,182 pts·SMA
2024-03→2026-06·Completed
NCT04759861
2,502 pts·Hemophilia A
2023-07→2028-05·Not yet recruiting
3,684 total pts2 indications
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-06-012mo awayPh2 Data· SMA
2028-05-212.1y awayPh2 Data· Hemophilia A
Trial Timeline
Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2
P2
Not yet…
P2
Complet…
Catalysts
Ph2 Data
2026-06-01 · 2mo away
SMA
Ph2 Data
2028-05-21 · 2.1y away
Hemophilia A
CompletedNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05761744 | Phase 2 | SMA | Completed | 1182 | OS |
| NCT04759861 | Phase 2 | Hemophilia A | Not yet recr... | 2502 | ORR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| JNJ-5094 | Johnson & Johnson | Phase 2/3 | GIP-R | |
| RHH-3592 | Roche | Phase 1/2 | GIP-R | |
| NVS-6360 | Novartis | Approved | CD38 | |
| MRK-3745 | Merck & Co | Phase 2 | WRN | |
| AZN-1715 | AstraZeneca | Phase 3 | AHR | |
| AZN-5978 | AstraZeneca | Phase 2/3 | GIP-R | |
| BMY-9931 | Bristol-Myers Squibb | Phase 1 | GPRC5D | |
| NVO-7877 | Novo Nordisk | Preclinical | GPRC5D | |
| Ivotuximab | GSK | NDA/BLA | GIP-R | |
| TAK-8730 | Takeda | Phase 2 | VEGF |