Modality
mRNA
MOA
EGFRi
Target
SOS1
Pathway
Epigenetic
DMDCrohn'sSchizophrenia
Development Pipeline
Preclinical
~Aug 2019
→ ~Nov 2020
Phase 1
~Feb 2021
→ ~May 2022
Phase 2
~Aug 2022
→ ~Nov 2023
Phase 3
~Feb 2024
→ ~May 2025
NDA/BLA
Aug 2025
→ Aug 2030
NDA/BLACurrent
NCT08160030
750 pts·DMD
2025-08→2030-08·Recruiting
750 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (1)
2030-08-014.3y awayPh3 Readout· DMD
Trial Timeline
Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3
NDA/BLA
Recruit…
Catalysts
Ph3 Readout
2030-08-01 · 4.3y away
DMD
Recruiting|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08160030 | NDA/BLA | DMD | Recruiting | 750 | HbA1c |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Niranesiran | Johnson & Johnson | Phase 2 | KIF18A | |
| JNJ-5094 | Johnson & Johnson | Phase 2/3 | GIP-R | |
| Terarelsin | AbbVie | Preclinical | SOS1 | |
| Ribofutibatinib | AstraZeneca | Phase 2/3 | SOS1 | |
| GSK-2051 | GSK | Phase 1 | PSMA | |
| Voxacagene | Gilead Sciences | Phase 2/3 | SOS1 | |
| GIL-8529 | Gilead Sciences | Phase 1/2 | JAK1 | |
| SRP-9822 | Sarepta | Phase 3 | SOS1 | |
| 207-2754 | Samsung Biologics | Phase 1/2 | KRASG12D | |
| Mavuratamab | Blueprint Medicines | NDA/BLA | BET |