Modality
Small Molecule
MOA
AuroraAi
Target
CD123
Pathway
Hedgehog
PVAngelman
Development Pipeline
Preclinical
~Dec 2016
→ ~Mar 2018
Phase 1
~Jun 2018
→ ~Sep 2019
Phase 2
Dec 2019
→ Dec 2031
Phase 2Current
NCT07046151
2,625 pts·Angelman
2024-07→TBD·Recruiting
NCT08887453
1,413 pts·Angelman
2019-12→2031-12·Completed
4,038 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2031-12-215.7y awayPh3 Readout· Angelman
Trial Timeline
Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
P2/3
Complet…
P2/3
Recruit…
Catalysts
Ph3 Readout
2031-12-21 · 5.7y away
Angelman
RecruitingCompleted|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07046151 | Phase 2/3 | Angelman | Recruiting | 2625 | ORR |
| NCT08887453 | Phase 2/3 | Angelman | Completed | 1413 | eGFR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Voxarasimod | Merck & Co | Phase 1/2 | CD19 | |
| TAK-5300 | Takeda | Phase 1 | SMN2 | |
| DSN-791 | Daiichi Sankyo | NDA/BLA | Aβ | |
| REG-7737 | Regeneron | Preclinical | CD123 | |
| REG-861 | Regeneron | Phase 2 | PARP | |
| MRN-5715 | Moderna | NDA/BLA | CDK4/6 | |
| BGN-6990 | BeiGene | Phase 2/3 | BCMA | |
| Semasacituzumab | Jazz Pharma | Phase 2 | AHR | |
| ILM-2412 | Illumina | Phase 3 | PLK4 | |
| Ivotuximab | Madrigal Pharma | Phase 2 | CD123 |