Modality
Peptide
MOA
STINGag
Target
KIF18A
Pathway
Lipid Met
AsthmaRettSCD
Development Pipeline
Preclinical
~Dec 2017
→ ~Mar 2019
Phase 1
~Jun 2019
→ ~Sep 2020
Phase 2
Dec 2020
→ Nov 2031
Phase 2Current
NCT07755130
800 pts·SCD
2025-11→2027-01·Active
NCT03200111
119 pts·Rett
2020-12→2031-11·Recruiting
919 total pts2 indications
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2027-01-129mo awayPh2 Data· SCD
2031-11-075.6y awayPh2 Data· Rett
Trial Timeline
Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
P2
Recruit…
P2
Active
Catalysts
Ph2 Data
2027-01-12 · 9mo away
SCD
Ph2 Data
2031-11-07 · 5.6y away
Rett
RecruitingActive|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07755130 | Phase 2 | SCD | Active | 800 | Mayo |
| NCT03200111 | Phase 2 | Rett | Recruiting | 119 | LiverFat |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Niranesiran | Johnson & Johnson | Phase 2 | KIF18A | |
| RHH-5389 | Roche | Preclinical | RET | |
| SNY-5894 | Sanofi | Approved | CFTR | |
| SNY-4496 | Sanofi | Phase 2/3 | CD3 | |
| SNY-5783 | Sanofi | Phase 1/2 | MET | |
| DSN-1421 | Daiichi Sankyo | Phase 3 | Nectin-4 | |
| GIL-4361 | Gilead Sciences | Preclinical | PCSK9 | |
| Riluinavolisib | Moderna | NDA/BLA | KIF18A | |
| INC-5849 | Incyte | Phase 2 | PCSK9 | |
| Miritinib | Neurocrine | Phase 3 | TIGIT |