Modality
Radioligand
MOA
ALKi
Target
CFTR
Pathway
Wnt
AngelmanALL
Development Pipeline
Preclinical
~Jan 2019
→ ~Apr 2020
Phase 1
~Jul 2020
→ ~Oct 2021
Phase 2
Jan 2022
→ Sep 2031
Phase 2Current
NCT04453613
2,512 pts·ALL
2025-01→2031-09·Recruiting
NCT05976040
1,698 pts·Angelman
2022-01→2026-05·Not yet recruiting
4,210 total pts2 indications
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-05-101mo awayPh3 Readout· Angelman
2031-09-095.4y awayPh3 Readout· ALL
Trial Timeline
2022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
P2/3
Not yet…
P2/3
Recruit…
Catalysts
Ph3 Readout
2026-05-10 · 1mo away
Angelman
Ph3 Readout
2031-09-09 · 5.4y away
ALL
RecruitingNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04453613 | Phase 2/3 | ALL | Recruiting | 2512 | UPDRS |
| NCT05976040 | Phase 2/3 | Angelman | Not yet recr... | 1698 | Safety |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Miriosocimab | Pfizer | Phase 1 | PARP | |
| Nidasacituzumab | Eli Lilly | Approved | CFTR | |
| MRK-7739 | Merck & Co | Preclinical | IL-23 | |
| Datoglumide | AbbVie | Approved | CFTR | |
| BMY-2495 | Bristol-Myers Squibb | Phase 3 | KRASG12D | |
| SNY-7254 | Sanofi | Phase 2 | CFTR | |
| SNY-5894 | Sanofi | Approved | CFTR | |
| DSN-5254 | Daiichi Sankyo | Phase 1 | B7-H3 | |
| Sovacapivasertib | Amgen | Phase 3 | WEE1 | |
| Zanuratamab | Gilead Sciences | Phase 1/2 | CDK2 |