Modality
ERT
MOA
BiTE
Target
MDM2
Pathway
Cell Cycle
SCDNASH
Development Pipeline
Preclinical
~May 2015
→ ~Aug 2016
Phase 1
~Nov 2016
→ ~Feb 2018
Phase 2
May 2018
→ Mar 2031
Phase 2Current
NCT03203219
2,029 pts·SCD
2019-06→2031-03·Terminated
NCT05132136
2,444 pts·SCD
2018-05→2029-11·Not yet recruiting
4,473 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (3)
2026-11-198mo awayConference· SCD
2029-11-083.6y awayPh2 Data· SCD
2031-03-114.9y awayPh2 Data· SCD
Trial Timeline
Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2
P2
Not yet…
P2
Termina…
Catalysts
Conference
2026-11-19 · 8mo away
SCD
Ph2 Data
2029-11-08 · 3.6y away
SCD
Ph2 Data
2031-03-11 · 4.9y away
SCD
TerminatedNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03203219 | Phase 2 | SCD | Terminated | 2029 | Safety |
| NCT05132136 | Phase 2 | SCD | Not yet recr... | 2444 | HAM-D |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| LLY-6079 | Eli Lilly | Phase 2 | MDM2 | |
| Zanutinib | Bristol-Myers Squibb | Phase 2/3 | APOC3 | |
| GSK-7987 | GSK | Phase 3 | MDM2 | |
| Bemarelsin | Daiichi Sankyo | Preclinical | GLP-1R | |
| DSN-7360 | Daiichi Sankyo | Approved | SGLT2 | |
| AMG-2752 | Amgen | Preclinical | MDM2 | |
| REG-3155 | Regeneron | Phase 1/2 | MDM2 | |
| BII-8315 | Biogen | NDA/BLA | CDK2 | |
| SRP-1135 | Sarepta | Phase 2/3 | MDM2 | |
| Geliglumide | Samsung Biologics | Phase 1 | Aβ |