Modality
ERT
MOA
JAK1i
Target
CFTR
Pathway
RAS/MAPK
EoEMCC
Development Pipeline
Preclinical
~Jun 2016
→ ~Sep 2017
Phase 1
~Dec 2017
→ ~Mar 2019
Phase 2
Jun 2019
→ Sep 2028
Phase 2Current
NCT03608020
2,854 pts·EoE
2020-06→2026-07·Not yet recruiting
NCT06771969
2,198 pts·MCC
2019-06→2028-09·Completed
5,052 total pts2 indications
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-07-274mo awayPh3 Readout· EoE
2028-09-102.4y awayPh3 Readout· MCC
Trial Timeline
Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q4
P2/3
Complet…
P2/3
Not yet…
Catalysts
Ph3 Readout
2026-07-27 · 4mo away
EoE
Ph3 Readout
2028-09-10 · 2.4y away
MCC
CompletedNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03608020 | Phase 2/3 | EoE | Not yet recr... | 2854 | MRD |
| NCT06771969 | Phase 2/3 | MCC | Completed | 2198 | SeizFreq |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| JNJ-7458 | Johnson & Johnson | Phase 1/2 | PD-1 | |
| Nidasacituzumab | Eli Lilly | Approved | CFTR | |
| Datoglumide | AbbVie | Approved | CFTR | |
| SNY-7254 | Sanofi | Phase 2 | CFTR | |
| Geliderotide | Sanofi | Phase 1/2 | PRMT5 | |
| SNY-5894 | Sanofi | Approved | CFTR | |
| Pexaosocimab | Daiichi Sankyo | Phase 2 | PD-1 | |
| Adagratapinarof | Vertex Pharma | Preclinical | BCMA | |
| ARG-1924 | Argenx | Preclinical | DLL3 | |
| Sovarelsin | BioMarin | Phase 2 | CFTR |