Modality
Gene Therapy
MOA
JAK1i
Target
BTK
Pathway
Hedgehog
ACCPsARett
Development Pipeline
Preclinical
~Apr 2016
→ ~Jul 2017
Phase 1
Oct 2017
→ Jan 2030
Phase 1Current
NCT05985784
2,271 pts·Rett
2017-10→2030-01·Active
NCT03758696
398 pts·ACC
2021-02→2027-07·Not yet recruiting
2,669 total pts2 indications
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2027-07-181.3y awayPh2 Data· ACC
2030-01-173.8y awayPh2 Data· Rett
Trial Timeline
Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2
P1/2
Active
P1/2
Not yet…
Catalysts
Ph2 Data
2027-07-18 · 1.3y away
ACC
Ph2 Data
2030-01-17 · 3.8y away
Rett
ActiveNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05985784 | Phase 1/2 | Rett | Active | 2271 | 6MWD |
| NCT03758696 | Phase 1/2 | ACC | Not yet recr... | 398 | Biomarker |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-5501 | Pfizer | Phase 1 | BTK | |
| JNJ-7458 | Johnson & Johnson | Phase 1/2 | PD-1 | |
| RHH-8482 | Roche | Phase 1 | BTK | |
| Geliderotide | Sanofi | Phase 1/2 | PRMT5 | |
| Zorizanubrutinib | Novo Nordisk | Phase 1 | BTK | |
| Pexaosocimab | Daiichi Sankyo | Phase 2 | PD-1 | |
| Adagratapinarof | Vertex Pharma | Preclinical | BCMA | |
| ARG-1924 | Argenx | Preclinical | DLL3 | |
| 369-8021 | Hansoh Pharma | Approved | BTK | |
| ROI-1081 | Roivant Sciences | Phase 1/2 | PI3Kα |