Modality
ERT
MOA
IL-13i
Target
APOC3
Pathway
Tau
PompeHNSCC
Development Pipeline
Preclinical
~Aug 2020
→ ~Nov 2021
Phase 1
~Feb 2022
→ ~May 2023
Phase 2
~Aug 2023
→ ~Nov 2024
Phase 3
Feb 2025
→ Aug 2030
Phase 3Current
NCT04775477
1,272 pts·Pompe
2025-02→2030-08·Active
1,272 total pts1 indication
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2030-08-094.4y awayPh3 Readout· Pompe
Trial Timeline
2025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3
P3
Active
Catalysts
Ph3 Readout
2030-08-09 · 4.4y away
Pompe
Active|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04775477 | Phase 3 | Pompe | Active | 1272 | LiverFat |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-1944 | Pfizer | NDA/BLA | APOC3 | |
| Voxaderotide | Johnson & Johnson | NDA/BLA | PI3Kα | |
| Doxacapivasertib | Eli Lilly | Phase 3 | APOC3 | |
| RHH-7975 | Roche | Phase 3 | FXIa | |
| NVS-8902 | Novartis | Preclinical | TROP-2 | |
| AZN-6294 | AstraZeneca | Phase 2/3 | APOC3 | |
| Gozesotorasib | Bristol-Myers Squibb | NDA/BLA | HER2 | |
| Zanutinib | Bristol-Myers Squibb | Phase 2/3 | APOC3 | |
| BMY-4586 | Bristol-Myers Squibb | Preclinical | KRASG12C | |
| AMG-1919 | Amgen | Phase 2/3 | CDK2 |