Modality
Gene Therapy
MOA
TYK2i
Target
Menin
Pathway
Autophagy
WilmsPSP
Development Pipeline
Preclinical
~Dec 2009
→ ~Mar 2011
Phase 1
~Jun 2011
→ ~Sep 2012
Phase 2
~Dec 2012
→ ~Mar 2014
Phase 3
~Jun 2014
→ ~Sep 2015
NDA/BLA
~Dec 2015
→ ~Mar 2017
Approved
Jun 2017
→ Aug 2025
ApprovedCurrent
NCT05687884
1,867 pts·Wilms
2020-05→TBD·Not yet recruiting
NCT05733038
1,932 pts·PSP
2017-06→TBD·Active
NCT08932075
1,048 pts·PSP
2019-01→2025-08·Recruiting
4,847 total pts2 indications
CompletedCurrentUpcoming
Catalysts (1)
2025-08-078mo agoPh3 Readout· PSP
Trial Timeline
Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q4
Approved
Active
Approved
Recruit…
Approved
Not yet…
Catalysts
Ph3 Readout
2025-08-07 · 8mo ago
PSP
RecruitingActiveNot yet recruiting|StartCompletionToday
Trials (3)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05687884 | Approved | Wilms | Not yet recr... | 1867 | HbA1c |
| NCT05733038 | Approved | PSP | Active | 1932 | PASI75 |
| NCT08932075 | Approved | PSP | Recruiting | 1048 | HbA1c |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Nidasacituzumab | Eli Lilly | Approved | CFTR | |
| LLY-8369 | Eli Lilly | Approved | Menin | |
| Lisonaritide | Eli Lilly | Phase 3 | IL-13 | |
| NVS-1475 | Novartis | Phase 2 | Menin | |
| MRK-2473 | Merck & Co | Phase 1/2 | Nectin-4 | |
| Zorisotorasib | AstraZeneca | Phase 1 | WRN | |
| SNY-8628 | Sanofi | NDA/BLA | B7-H3 | |
| Voxacagene | Gilead Sciences | Phase 2/3 | SOS1 | |
| Sotosacituzumab | Vertex Pharma | Phase 1 | MET | |
| VRT-9481 | Vertex Pharma | Phase 1/2 | Menin |