Modality
Degrader
MOA
BiTE
Target
PI3Kα
Pathway
PD-1/PD-L1
Angelman
Development Pipeline
Preclinical
~Jan 2016
→ ~Apr 2017
Phase 1
~Jul 2017
→ ~Oct 2018
Phase 2
~Jan 2019
→ ~Apr 2020
Phase 3
~Jul 2020
→ ~Oct 2021
NDA/BLA
Jan 2022
→ Feb 2030
NDA/BLACurrent
NCT03064068
1,572 pts·Angelman
2022-01→2030-02·Active
NCT07235357
819 pts·Angelman
2023-06→2026-03·Completed
2,391 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (3)
2025-10-146mo agoPh1 Dose Esc· Angelman
2026-03-034w agoPh3 Readout· Angelman
2030-02-243.9y awayPh3 Readout· Angelman
Trial Timeline
2022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2
NDA/BLA
Active
NDA/BLA
Complet…
Catalysts
Ph1 Dose Esc
2025-10-14 · 6mo ago
Angelman
Ph3 Readout
2026-03-03 · 4w ago
Angelman
Ph3 Readout
2030-02-24 · 3.9y away
Angelman
ActiveCompleted|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03064068 | NDA/BLA | Angelman | Active | 1572 | CR |
| NCT07235357 | NDA/BLA | Angelman | Completed | 819 | HAM-D |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Voxaderotide | Johnson & Johnson | NDA/BLA | PI3Kα | |
| LLY-3251 | Eli Lilly | Phase 2 | MDM2 | |
| Rimamavacamten | Merck & Co | Phase 1 | PI3Kα | |
| Zanutinib | Bristol-Myers Squibb | Phase 2/3 | APOC3 | |
| Bemarelsin | Daiichi Sankyo | Preclinical | GLP-1R | |
| DSN-7360 | Daiichi Sankyo | Approved | SGLT2 | |
| Fixazanubrutinib | Amgen | Phase 2 | PI3Kα | |
| BII-8315 | Biogen | NDA/BLA | CDK2 | |
| GMA-1468 | Genmab | NDA/BLA | PI3Kα | |
| 369-789 | Hansoh Pharma | Phase 3 | PI3Kα |