Modality
siRNA
MOA
PD-1i
Target
KIF18A
Pathway
Checkpoint
Rett
Development Pipeline
Preclinical
~Feb 2019
→ ~May 2020
Phase 1
~Aug 2020
→ ~Nov 2021
Phase 2
~Feb 2022
→ ~May 2023
Phase 3
Aug 2023
→ Dec 2031
Phase 3Current
NCT07287921
2,463 pts·Rett
2024-08→2031-12·Terminated
NCT08351632
936 pts·Rett
2023-08→2026-09·Active
NCT04412409
1,105 pts·Rett
2025-10→2029-04·Completed
4,504 total pts1 indication
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (3)
2026-09-186mo awayPh3 Readout· Rett
2029-04-193.1y awayPh3 Readout· Rett
2031-12-095.7y awayPh3 Readout· Rett
Trial Timeline
Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
P3
Active
P3
Termina…
P3
Complet…
Catalysts
Ph3 Readout
2026-09-18 · 6mo away
Rett
Ph3 Readout
2029-04-19 · 3.1y away
Rett
Ph3 Readout
2031-12-09 · 5.7y away
Rett
ActiveCompletedTerminated|StartCompletionToday
Trials (3)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07287921 | Phase 3 | Rett | Terminated | 2463 | DAS28 |
| NCT08351632 | Phase 3 | Rett | Active | 936 | UPDRS |
| NCT04412409 | Phase 3 | Rett | Completed | 1105 | 6MWD |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Niranesiran | Johnson & Johnson | Phase 2 | KIF18A | |
| GSK-7987 | GSK | Phase 3 | MDM2 | |
| Riluinavolisib | Moderna | NDA/BLA | KIF18A | |
| Nirabrutinib | BioNTech | Phase 2/3 | CDK2 | |
| Cevinaritide | Genmab | Phase 2/3 | DLL3 | |
| NUV-2032 | Nuvalent | Preclinical | KIF18A | |
| DNL-9555 | Denali | Phase 1/2 | KIF18A | |
| Lisoosocimab | Zealand Pharma | Approved | KIF18A | |
| Talatenlimab | Praxis Precision | Phase 1/2 | KIF18A | |
| ZYM-2450 | Zymeworks | Phase 2 | GLP-1R |