Modality
ASO
MOA
PCSK9i
Target
IL-17A
Pathway
RAS/MAPK
Hemophilia AMM
Development Pipeline
Preclinical
~Jun 2014
→ ~Sep 2015
Phase 1
~Dec 2015
→ ~Mar 2017
Phase 2
Jun 2017
→ Jun 2026
Phase 2Current
NCT03223955
1,481 pts·Hemophilia A
2019-06→2025-01·Recruiting
NCT03735320
1,987 pts·MM
2017-06→2026-06·Active
3,468 total pts2 indications
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-01-231.2y agoPh3 Readout· Hemophilia A
2026-06-032mo awayPh3 Readout· MM
Trial Timeline
Q2Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3
P2/3
Active
P2/3
Recruit…
Catalysts
Ph3 Readout
2025-01-23 · 1.2y ago
Hemophilia A
Ph3 Readout
2026-06-03 · 2mo away
MM
RecruitingActive|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03223955 | Phase 2/3 | Hemophilia A | Recruiting | 1481 | Biomarker |
| NCT03735320 | Phase 2/3 | MM | Active | 1987 | EDSS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Geliderotide | Novartis | NDA/BLA | IL-17A | |
| MRK-8368 | Merck & Co | Phase 3 | ALK | |
| Ribofutibatinib | AstraZeneca | Phase 2/3 | SOS1 | |
| AZN-4015 | AstraZeneca | Preclinical | BCMA | |
| Tirasotorasib | Takeda | NDA/BLA | IL-17A | |
| Polazasiran | Amgen | Phase 2 | LAG-3 | |
| GIL-9779 | Gilead Sciences | Phase 1 | IL-17A | |
| Cevitinib | Regeneron | Phase 3 | FGFR | |
| INC-1261 | Incyte | Phase 1/2 | PRMT5 | |
| Mavutuximab | Hansoh Pharma | Phase 2/3 | PI3Kα |