Modality
Gene Therapy
MOA
PLK4i
Target
GLP-1R
Pathway
DDR
FabryMSPV
Development Pipeline
Preclinical
~Jan 2019
→ ~Apr 2020
Phase 1
Jul 2020
→ Jul 2030
Phase 1Current
NCT04325810
2,313 pts·PV
2020-07→2030-07·Recruiting
NCT04386316
2,150 pts·Fabry
2025-05→TBD·Not yet recruiting
4,463 total pts2 indications
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-11-045mo agoNDA· Fabry
2030-07-054.3y awayInterim· PV
Trial Timeline
Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3
P1
Recruit…
P1
Not yet…
Catalysts
NDA
2025-11-04 · 5mo ago
Fabry
Interim
2030-07-05 · 4.3y away
PV
RecruitingNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04325810 | Phase 1 | PV | Recruiting | 2313 | VA |
| NCT04386316 | Phase 1 | Fabry | Not yet recr... | 2150 | LiverFat |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-2191 | Pfizer | NDA/BLA | GLP-1R | |
| Riboinavolisib | Johnson & Johnson | Phase 2/3 | GLP-1R | |
| Tirzepatide | Eli Lilly | Approved | GLP-1R | |
| MRK-5413 | Merck & Co | Phase 1 | GLP-1R | |
| Voxacilimab | AstraZeneca | Phase 1/2 | GLP-1R | |
| AZN-5171 | AstraZeneca | Approved | GLP-1R | |
| Mavuglumide | Bayer | Approved | GLP-1R | |
| Bemarelsin | Daiichi Sankyo | Preclinical | GLP-1R | |
| AMG-2597 | Amgen | Phase 2/3 | CD38 | |
| VRT-5441 | Vertex Pharma | Phase 3 | GLP-1R |