Modality
ASO
MOA
JAK1i
Target
CFTR
Pathway
Apoptosis
NMOSDASADHD
Development Pipeline
Preclinical
~Oct 2013
→ ~Jan 2015
Phase 1
~Apr 2015
→ ~Jul 2016
Phase 2
~Oct 2016
→ ~Jan 2018
Phase 3
Apr 2018
→ Oct 2031
Phase 3Current
NCT08045638
1,336 pts·ADHD
2018-04→2028-08·Recruiting
NCT03232991
1,778 pts·AS
2018-07→2031-10·Recruiting
NCT04547912
124 pts·ADHD
2021-09→2031-02·Completed
+1 more trial
3,773 total pts3 indications
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (4)
2025-11-075mo agoPDUFA· NMOSD
2028-08-042.3y awayPh3 Readout· ADHD
2031-02-114.9y awayPh3 Readout· ADHD
2031-10-105.5y awayPh3 Readout· AS
Trial Timeline
2018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
P3
Recruit…
P3
Recruit…
P3
Not yet…
P3
Complet…
Catalysts
PDUFA
2025-11-07 · 5mo ago
NMOSD
Ph3 Readout
2028-08-04 · 2.3y away
ADHD
Ph3 Readout
2031-02-11 · 4.9y away
ADHD
Ph3 Readout
2031-10-10 · 5.5y away
AS
RecruitingCompletedNot yet recruiting|StartCompletionToday
Trials (4)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08045638 | Phase 3 | ADHD | Recruiting | 1336 | BodyWt |
| NCT03232991 | Phase 3 | AS | Recruiting | 1778 | MRD |
| NCT04547912 | Phase 3 | ADHD | Completed | 124 | HbA1c |
| NCT04145683 | Phase 3 | NMOSD | Not yet recr... | 535 | PFS |
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 |