Modality
Nanobody
MOA
PLK4i
Target
WRN
Pathway
DDR
SLEET
Development Pipeline
Preclinical
~Jul 2014
→ ~Oct 2015
Phase 1
~Jan 2016
→ ~Apr 2017
Phase 2
Jul 2017
→ Jan 2026
Phase 2Current
NCT05644642
488 pts·SLE
2017-07→2026-01·Not yet recruiting
NCT03483990
1,470 pts·ET
2025-06→2025-08·Terminated
1,958 total pts2 indications
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-08-237mo agoPh3 Readout· ET
2026-01-023mo agoPh3 Readout· SLE
Trial Timeline
Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026
P2/3
Not yet…
P2/3
Termina…
Catalysts
Ph3 Readout
2025-08-23 · 7mo ago
ET
Ph3 Readout
2026-01-02 · 3mo ago
SLE
TerminatedNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05644642 | Phase 2/3 | SLE | Not yet recr... | 488 | UPDRS |
| NCT03483990 | Phase 2/3 | ET | Terminated | 1470 | OS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| MRK-3745 | Merck & Co | Phase 2 | WRN | |
| Zorisotorasib | AstraZeneca | Phase 1 | WRN | |
| BMY-3371 | Bristol-Myers Squibb | Phase 3 | WRN | |
| Lisorapivir | Takeda | Preclinical | WRN | |
| Nirafutibatinib | Bayer | Phase 2 | WRN | |
| AMG-2597 | Amgen | Phase 2/3 | CD38 | |
| Riluinavolisib | Moderna | NDA/BLA | KIF18A | |
| Liravorutinib | Biomea Fusion | Phase 1/2 | GPRC5D | |
| TSH-7567 | Taysha Gene | Phase 1/2 | WRN | |
| BLU-5648 | Bluebird Bio | Approved | WRN |