Modality
ADC
MOA
CAR-T CD19
Target
SGLT2
Pathway
Hedgehog
Pompe
Development Pipeline
Preclinical
~Sep 2021
→ ~Dec 2022
Phase 1
~Mar 2023
→ ~Jun 2024
Phase 2
Sep 2024
→ Nov 2028
Phase 2Current
NCT04163624
1,024 pts·Pompe
2024-09→2028-11·Recruiting
1,024 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2028-11-102.6y awayPh3 Readout· Pompe
Trial Timeline
Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q4
P2/3
Recruit…
Catalysts
Ph3 Readout
2028-11-10 · 2.6y away
Pompe
Recruiting|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04163624 | Phase 2/3 | Pompe | Recruiting | 1024 | FEV1 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| RHH-7558 | Roche | Phase 1 | SGLT2 | |
| RHH-1546 | Roche | Phase 2/3 | C5 | |
| Tiramavacamten | Roche | Preclinical | SGLT2 | |
| Datoglumide | AbbVie | Approved | CFTR | |
| Rimatenlimab | Novo Nordisk | Phase 2/3 | SGLT2 | |
| DSN-7360 | Daiichi Sankyo | Approved | SGLT2 | |
| GIL-2259 | Gilead Sciences | Approved | SMN2 | |
| BII-4342 | Biogen | Phase 2/3 | CGRP | |
| SRP-3270 | Sarepta | Phase 1/2 | SGLT2 | |
| Tirarapivir | United Therapeutics | Approved | SGLT2 |