Modality
Radioligand
MOA
PD-L1i
Target
CD47
Pathway
Incretin
PompeAngelman
Development Pipeline
Preclinical
~Feb 2012
→ ~May 2013
Phase 1
~Aug 2013
→ ~Nov 2014
Phase 2
~Feb 2015
→ ~May 2016
Phase 3
~Aug 2016
→ ~Nov 2017
NDA/BLA
Feb 2018
→ Nov 2026
NDA/BLACurrent
NCT04164054
2,435 pts·Angelman
2022-06→2026-08·Active
NCT05616115
125 pts·Pompe
2018-02→2026-11·Completed
2,560 total pts2 indications
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-08-074mo awayPh3 Readout· Angelman
2026-11-047mo awayPh3 Readout· Pompe
Trial Timeline
2018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q4
NDA/BLA
Complet…
NDA/BLA
Active
Catalysts
Ph3 Readout
2026-08-07 · 4mo away
Angelman
Ph3 Readout
2026-11-04 · 7mo away
Pompe
ActiveCompleted|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04164054 | NDA/BLA | Angelman | Active | 2435 | ACR20 |
| NCT05616115 | NDA/BLA | Pompe | Completed | 125 | Biomarker |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-1085 | Pfizer | Preclinical | FXIa | |
| LLY-8903 | Eli Lilly | Preclinical | CD47 | |
| Rilulemzoparlimab | Novartis | Phase 2/3 | KRASG12D | |
| SNY-9600 | Sanofi | NDA/BLA | B7-H3 | |
| GSK-4334 | GSK | Preclinical | CD47 | |
| Nirafutibatinib | Bayer | Phase 2 | WRN | |
| BNT-8090 | BioNTech | Phase 2 | AuroraA | |
| Miriglumide | Alnylam | Phase 3 | CD3 | |
| BMR-1165 | BioMarin | Phase 2 | CD47 | |
| INC-2432 | Incyte | Phase 2 | CD47 |