Modality
Gene Editing
MOA
KRASG12Di
Target
BTK
Pathway
NF-κB
HSMCC
Development Pipeline
Preclinical
~Apr 2014
→ ~Jul 2015
Phase 1
~Oct 2015
→ ~Jan 2017
Phase 2
Apr 2017
→ Dec 2030
Phase 2Current
NCT03540597
869 pts·HS
2025-10→2028-05·Not yet recruiting
NCT03190933
2,081 pts·MCC
2017-04→2030-12·Terminated
2,950 total pts2 indications
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2028-05-112.1y awayPh3 Readout· HS
2030-12-274.7y awayPh3 Readout· MCC
Trial Timeline
2017Q2Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q4
P2/3
Termina…
P2/3
Not yet…
Catalysts
Ph3 Readout
2028-05-11 · 2.1y away
HS
Ph3 Readout
2030-12-27 · 4.7y away
MCC
TerminatedNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03540597 | Phase 2/3 | HS | Not yet recr... | 869 | CfB |
| NCT03190933 | Phase 2/3 | MCC | Terminated | 2081 | ORR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-5501 | Pfizer | Phase 1 | BTK | |
| RHH-8482 | Roche | Phase 1 | BTK | |
| Doxarasimod | Merck & Co | NDA/BLA | FGFR | |
| Zanusertib | AbbVie | Approved | PCSK9 | |
| Pexarelsin | AstraZeneca | Phase 1 | DLL3 | |
| Zorizanubrutinib | Novo Nordisk | Phase 1 | BTK | |
| Ivotuximab | GSK | NDA/BLA | GIP-R | |
| 369-8021 | Hansoh Pharma | Approved | BTK | |
| Terarasimod | Krystal Biotech | Phase 1/2 | PI3Kα | |
| Pemitinib | Xenon Pharma | Preclinical | BTK |