Modality
Small Molecule
MOA
IL-23i
Target
MDM2
Pathway
Wnt
SchizophreniaRett
Development Pipeline
Preclinical
~Feb 2013
→ ~May 2014
Phase 1
~Aug 2014
→ ~Nov 2015
Phase 2
~Feb 2016
→ ~May 2017
Phase 3
~Aug 2017
→ ~Nov 2018
NDA/BLA
Feb 2019
→ Apr 2028
NDA/BLACurrent
NCT06733075
1,083 pts·Rett
2025-04→2025-02·Terminated
NCT03546131
2,684 pts·Rett
2019-02→2028-04·Completed
3,767 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-02-221.1y agoPh3 Readout· Rett
2028-04-032.0y awayPh3 Readout· Rett
Trial Timeline
2019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2
NDA/BLA
Complet…
NDA/BLA
Termina…
Catalysts
Ph3 Readout
2025-02-22 · 1.1y ago
Rett
Ph3 Readout
2028-04-03 · 2.0y away
Rett
CompletedTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT06733075 | NDA/BLA | Rett | Terminated | 1083 | OS |
| NCT03546131 | NDA/BLA | Rett | Completed | 2684 | Biomarker |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| LLY-6079 | Eli Lilly | Phase 2 | MDM2 | |
| LLY-3251 | Eli Lilly | Phase 2 | MDM2 | |
| GSK-6983 | GSK | Phase 2 | ALK | |
| Motarapivir | GSK | Preclinical | AuroraA | |
| GSK-7987 | GSK | Phase 3 | MDM2 | |
| AMG-2752 | Amgen | Preclinical | MDM2 | |
| REG-3155 | Regeneron | Phase 1/2 | MDM2 | |
| Zenotapinarof | Vertex Pharma | Preclinical | CD38 | |
| SRP-1135 | Sarepta | Phase 2/3 | MDM2 | |
| Bemasotorasib | Exelixis | Phase 2 | CD38 |