Modality
ERT
MOA
CFTRmod
Target
MDM2
Pathway
Angiogenesis
Heart FailurePompe
Development Pipeline
Preclinical
~Aug 2016
→ ~Nov 2017
Phase 1
Feb 2018
→ Aug 2025
Phase 1Current
NCT07960257
2,677 pts·Pompe
2018-02→2025-08·Terminated
2,677 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2025-08-177mo agoInterim· Pompe
Trial Timeline
2018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q4
P1
Termina…
Catalysts
Interim
2025-08-17 · 7mo ago
Pompe
Terminated|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07960257 | Phase 1 | Pompe | Terminated | 2677 | SeizFreq |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| LLY-6079 | Eli Lilly | Phase 2 | MDM2 | |
| LLY-4358 | Eli Lilly | Phase 3 | B7-H3 | |
| LLY-3251 | Eli Lilly | Phase 2 | MDM2 | |
| Gelinaritide | AbbVie | Preclinical | FcRn | |
| GSK-7987 | GSK | Phase 3 | MDM2 | |
| AMG-2752 | Amgen | Preclinical | MDM2 | |
| REG-3155 | Regeneron | Phase 1/2 | MDM2 | |
| Darafutibatinib | BioNTech | Preclinical | PRMT5 | |
| ALN-8757 | Alnylam | Preclinical | TIGIT | |
| SRP-1135 | Sarepta | Phase 2/3 | MDM2 |