Modality
Small Molecule
MOA
CFTRmod
Target
BCL-2
Pathway
JAK/STAT
RBDMDDLBCL
Development Pipeline
Preclinical
~Jun 2017
→ ~Sep 2018
Phase 1
~Dec 2018
→ ~Mar 2020
Phase 2
Jun 2020
→ Apr 2030
Phase 2Current
NCT08952725
517 pts·DMD
2020-06→2030-04·Completed
517 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2030-04-044.0y awayPh2 Data· DMD
Trial Timeline
Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2
P2
Complet…
Catalysts
Ph2 Data
2030-04-04 · 4.0y away
DMD
Completed|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08952725 | Phase 2 | DMD | Completed | 517 | EDSS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| LLY-4358 | Eli Lilly | Phase 3 | B7-H3 | |
| RHH-8550 | Roche | Approved | BCL-2 | |
| Gelinaritide | AbbVie | Preclinical | FcRn | |
| Darafutibatinib | BioNTech | Preclinical | PRMT5 | |
| Rimaglumide | Biogen | Phase 1/2 | BCL-2 | |
| ALN-8757 | Alnylam | Preclinical | TIGIT | |
| ARG-4339 | Argenx | Phase 2 | BCL-2 | |
| GMA-729 | Genmab | Phase 2 | BCL-2 | |
| HAL-996 | Halozyme | Phase 1/2 | BCL-2 | |
| Bemaderotide | Revolution Medicines | Phase 3 | BCL-2 |