Modality
ERT
MOA
FcRni
Target
DLL3
Pathway
Tau
Pompe
Development Pipeline
Preclinical
~Dec 2009
→ ~Mar 2011
Phase 1
~Jun 2011
→ ~Sep 2012
Phase 2
~Dec 2012
→ ~Mar 2014
Phase 3
~Jun 2014
→ ~Sep 2015
NDA/BLA
~Dec 2015
→ ~Mar 2017
Approved
Jun 2017
→ Aug 2026
ApprovedCurrent
NCT07719258
1,394 pts·Pompe
2017-06→TBD·Active
NCT04231586
2,826 pts·Pompe
2024-11→2026-04·Recruiting
NCT04071388
2,015 pts·Pompe
2025-01→2025-09·Recruiting
+1 more trial
6,889 total pts1 indication
CompletedCurrentUpcoming
Catalysts (3)
2025-09-226mo agoPh3 Readout· Pompe
2026-04-122w awayPh3 Readout· Pompe
2026-08-195mo awayPh3 Readout· Pompe
Trial Timeline
Q2Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q4
Approved
Active
Approved
Recruit…
Approved
Recruit…
Approved
Recruit…
Catalysts
Ph3 Readout
2025-09-22 · 6mo ago
Pompe
Ph3 Readout
2026-04-12 · 2w away
Pompe
Ph3 Readout
2026-08-19 · 5mo away
Pompe
RecruitingActive|StartCompletionToday
Trials (4)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07719258 | Approved | Pompe | Active | 1394 | NT-proBNP |
| NCT04231586 | Approved | Pompe | Recruiting | 2826 | EDSS |
| NCT04071388 | Approved | Pompe | Recruiting | 2015 | CR |
| NCT07274048 | Approved | Pompe | Recruiting | 654 | LiverFat |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-7809 | Pfizer | Phase 1 | DLL3 | |
| ABB-2476 | AbbVie | Phase 1/2 | USP1 | |
| Pexarelsin | AstraZeneca | Phase 1 | DLL3 | |
| Zorivorutinib | Bristol-Myers Squibb | Approved | DLL3 | |
| Zenonesiran | GSK | Preclinical | DLL3 | |
| Suracagene | GSK | Phase 3 | PRMT5 | |
| AMG-7379 | Amgen | Preclinical | DLL3 | |
| REG-6699 | Regeneron | Phase 2/3 | TIM-3 | |
| ARG-1924 | Argenx | Preclinical | DLL3 | |
| Cevinaritide | Genmab | Phase 2/3 | DLL3 |