Modality
ERT
MOA
Anti-Tau
Target
CD19
Pathway
Wnt
FabryADPKD
Development Pipeline
Preclinical
~Oct 2015
→ ~Jan 2017
Phase 1
~Apr 2017
→ ~Jul 2018
Phase 2
~Oct 2018
→ ~Jan 2020
Phase 3
Apr 2020
→ Feb 2030
Phase 3Current
NCT07885693
127 pts·ADPKD
2020-04→2028-08·Recruiting
NCT03381759
1,250 pts·Fabry
2025-10→2028-09·Not yet recruiting
NCT07358575
762 pts·Fabry
2020-10→2030-02·Active
+1 more trial
3,115 total pts2 indications
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (4)
2025-09-017mo agoPh3 Readout· Fabry
2028-08-122.4y awayPh3 Readout· ADPKD
2028-09-062.4y awayPh3 Readout· Fabry
2030-02-273.9y awayPh3 Readout· Fabry
Trial Timeline
Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2
P3
Recruit…
P3
Active
P3
Not yet…
P3
Termina…
Catalysts
Ph3 Readout
2025-09-01 · 7mo ago
Fabry
Ph3 Readout
2028-08-12 · 2.4y away
ADPKD
Ph3 Readout
2028-09-06 · 2.4y away
Fabry
Ph3 Readout
2030-02-27 · 3.9y away
Fabry
RecruitingActiveTerminatedNot yet recruiting|StartCompletionToday
Trials (4)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07885693 | Phase 3 | ADPKD | Recruiting | 127 | HAM-D |
| NCT03381759 | Phase 3 | Fabry | Not yet recr... | 1250 | EASI-75 |
| NCT07358575 | Phase 3 | Fabry | Active | 762 | VA |
| NCT05425939 | Phase 3 | Fabry | Terminated | 976 | DOR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-8662 | Pfizer | NDA/BLA | CD19 | |
| RHH-7558 | Roche | Phase 1 | SGLT2 | |
| NVS-5439 | Novartis | Preclinical | BET | |
| NVS-6974 | Novartis | Preclinical | CD19 | |
| Voxarasimod | Merck & Co | Phase 1/2 | CD19 | |
| NVO-9630 | Novo Nordisk | Phase 2 | CD3 | |
| REG-2172 | Regeneron | Phase 1/2 | PCSK9 | |
| BGN-3305 | BeiGene | Phase 2 | BCMA | |
| Sovarasimod | Halozyme | Phase 1/2 | WEE1 | |
| Voxamavacamten | Corcept | Approved | BET |