Modality
Nanobody
MOA
FXIai
Target
SGLT2
Pathway
Wnt
ALLWilmsPompe
Development Pipeline
Preclinical
~Jan 2012
→ ~Apr 2013
Phase 1
~Jul 2013
→ ~Oct 2014
Phase 2
~Jan 2015
→ ~Apr 2016
Phase 3
~Jul 2016
→ ~Oct 2017
NDA/BLA
Jan 2018
→ Nov 2031
NDA/BLACurrent
NCT04238338
2,822 pts·Pompe
2021-02→2030-10·Active
NCT03455015
969 pts·Wilms
2018-01→2031-11·Recruiting
3,791 total pts2 indications
Approved
CompletedCurrentUpcoming
Catalysts (2)
2030-10-224.6y awayPh3 Readout· Pompe
2031-11-235.6y awayPh3 Readout· Wilms
Trial Timeline
2018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
NDA/BLA
Recruit…
NDA/BLA
Active
Catalysts
Ph3 Readout
2030-10-22 · 4.6y away
Pompe
Ph3 Readout
2031-11-23 · 5.6y away
Wilms
RecruitingActive|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04238338 | NDA/BLA | Pompe | Active | 2822 | EDSS |
| NCT03455015 | NDA/BLA | Wilms | Recruiting | 969 | SeizFreq |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| RHH-7558 | Roche | Phase 1 | SGLT2 | |
| Tiramavacamten | Roche | Preclinical | SGLT2 | |
| NVS-6360 | Novartis | Approved | CD38 | |
| MRK-3745 | Merck & Co | Phase 2 | WRN | |
| AZN-1715 | AstraZeneca | Phase 3 | AHR | |
| BMY-9931 | Bristol-Myers Squibb | Phase 1 | GPRC5D | |
| NVO-7877 | Novo Nordisk | Preclinical | GPRC5D | |
| Rimatenlimab | Novo Nordisk | Phase 2/3 | SGLT2 | |
| TAK-8730 | Takeda | Phase 2 | VEGF | |
| DSN-7360 | Daiichi Sankyo | Approved | SGLT2 |