Modality
ASO
MOA
PD-1i
Target
CFTR
Pathway
RAS/MAPK
CKDCMLFTD
Development Pipeline
Preclinical
Sep 2019
→ Jul 2025
PreclinicalCurrent
NCT03297822
98 pts·CKD
2019-09→TBD·Active
NCT08641548
1,027 pts·FTD
2023-06→2025-07·Recruiting
1,125 total pts2 indications
Phase 1
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2025-07-059mo agoInterim· FTD
Trial Timeline
Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3
Preclinical
Active
Preclinical
Recruit…
Catalysts
Interim
2025-07-05 · 9mo ago
FTD
RecruitingActive|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03297822 | Preclinical | CKD | Active | 98 | PASI75 |
| NCT08641548 | Preclinical | FTD | Recruiting | 1027 | FEV1 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Nidasacituzumab | Eli Lilly | Approved | CFTR | |
| Datoglumide | AbbVie | Approved | CFTR | |
| SNY-7254 | Sanofi | Phase 2 | CFTR | |
| SNY-5894 | Sanofi | Approved | CFTR | |
| GSK-7987 | GSK | Phase 3 | MDM2 | |
| Nirabrutinib | BioNTech | Phase 2/3 | CDK2 | |
| Cevinaritide | Genmab | Phase 2/3 | DLL3 | |
| Sovarelsin | BioMarin | Phase 2 | CFTR | |
| Fixatuximab | Legend Biotech | Preclinical | CFTR | |
| Doxasotorasib | Samsung Biologics | Phase 2/3 | CFTR |