Modality
Gene Therapy
MOA
AuroraAi
Target
Tau
Pathway
Lipid Met
MyelofibrosisPompe
Development Pipeline
Preclinical
~Apr 2019
→ ~Jul 2020
Phase 1
~Oct 2020
→ ~Jan 2022
Phase 2
Apr 2022
→ Aug 2025
Phase 2Current
NCT07121748
2,442 pts·Pompe
2022-04→2025-08·Terminated
2,442 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2025-08-277mo agoPh3 Readout· Pompe
Trial Timeline
Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3
P2/3
Termina…
Catalysts
Ph3 Readout
2025-08-27 · 7mo ago
Pompe
Terminated|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07121748 | Phase 2/3 | Pompe | Terminated | 2442 | UPCR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Voxarasimod | Merck & Co | Phase 1/2 | CD19 | |
| NVO-9087 | Novo Nordisk | Phase 1/2 | Tau | |
| Tezecilimab | Takeda | NDA/BLA | Tau | |
| TAK-8262 | Takeda | NDA/BLA | Tau | |
| TAK-5300 | Takeda | Phase 1 | SMN2 | |
| DSN-791 | Daiichi Sankyo | NDA/BLA | Aβ | |
| REG-861 | Regeneron | Phase 2 | PARP | |
| MRN-5715 | Moderna | NDA/BLA | CDK4/6 | |
| BGN-6990 | BeiGene | Phase 2/3 | BCMA | |
| Semasacituzumab | Jazz Pharma | Phase 2 | AHR |