Modality
ERT
MOA
AHRant
Target
BCL-2
Pathway
Notch
PompeACCNB
Development Pipeline
Preclinical
~Jun 2019
→ ~Sep 2020
Phase 1
Dec 2020
→ Nov 2029
Phase 1Current
NCT04833988
2,981 pts·Pompe
2020-12→2029-11·Recruiting
2,981 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2029-11-233.6y awayInterim· Pompe
Trial Timeline
Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q4
P1
Recruit…
Catalysts
Interim
2029-11-23 · 3.6y away
Pompe
Recruiting|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04833988 | Phase 1 | Pompe | Recruiting | 2981 | MRD |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| RHH-8550 | Roche | Approved | BCL-2 | |
| AZN-5978 | AstraZeneca | Phase 2/3 | GIP-R | |
| BII-5449 | Biogen | Phase 3 | FXIa | |
| Rimaglumide | Biogen | Phase 1/2 | BCL-2 | |
| ARG-4339 | Argenx | Phase 2 | BCL-2 | |
| GMA-729 | Genmab | Phase 2 | BCL-2 | |
| Elratapinarof | Halozyme | Phase 2 | C5 | |
| HAL-996 | Halozyme | Phase 1/2 | BCL-2 | |
| Nidaratamab | Exelixis | Phase 3 | FLT3 | |
| Bemaderotide | Revolution Medicines | Phase 3 | BCL-2 |