Modality
ASO
MOA
JAK1/2i
Target
Aβ
Pathway
DDR
MyelofibrosisDMD
Development Pipeline
Preclinical
~Jun 2017
→ ~Sep 2018
Phase 1
Dec 2018
→ Aug 2027
Phase 1Current
NCT05887751
1,104 pts·Myelofibrosis
2018-12→2026-09·Recruiting
NCT05101884
2,211 pts·DMD
2022-07→2027-08·Active
3,315 total pts2 indications
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-09-145mo awayInterim· Myelofibrosis
2027-08-181.4y awayInterim· DMD
Trial Timeline
Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q4
P1
Recruit…
P1
Active
Catalysts
Interim
2026-09-14 · 5mo away
Myelofibrosis
Interim
2027-08-18 · 1.4y away
DMD
RecruitingActive|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05887751 | Phase 1 | Myelofibrosis | Recruiting | 1104 | EFS |
| NCT05101884 | Phase 1 | DMD | Active | 2211 | LiverFat |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-7550 | Pfizer | Phase 3 | Aβ | |
| Sotovorutinib | Pfizer | Approved | Aβ | |
| Fixanesiran | AbbVie | Preclinical | RET | |
| Bemalucimab | Bristol-Myers Squibb | Phase 1/2 | Aβ | |
| Datobrutinib | Sanofi | Phase 3 | Aβ | |
| DSN-791 | Daiichi Sankyo | NDA/BLA | Aβ | |
| Motavorutinib | Amgen | Phase 3 | CDK4/6 | |
| VRT-5853 | Vertex Pharma | Phase 3 | Aβ | |
| VRT-9481 | Vertex Pharma | Phase 1/2 | Menin | |
| ARG-314 | Argenx | Approved | Aβ |