Modality
Gene Editing
MOA
CFTRmod
Target
PRMT5
Pathway
Epigenetic
HCC
Development Pipeline
Preclinical
~Sep 2012
→ ~Dec 2013
Phase 1
~Mar 2014
→ ~Jun 2015
Phase 2
~Sep 2015
→ ~Dec 2016
Phase 3
~Mar 2017
→ ~Jun 2018
NDA/BLA
~Sep 2018
→ ~Dec 2019
Approved
Mar 2020
→ Dec 2031
ApprovedCurrent
NCT04445047
1,888 pts·HCC
2020-03→2031-12·Terminated
1,888 total pts1 indication
CompletedCurrentUpcoming
Catalysts (1)
2031-12-025.7y awayPh3 Readout· HCC
Trial Timeline
2020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
Approved
Termina…
Catalysts
Ph3 Readout
2031-12-02 · 5.7y away
HCC
Terminated|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04445047 | Approved | HCC | Terminated | 1888 | ACR20 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Nirarelsin | Johnson & Johnson | Phase 2 | PRMT5 | |
| LLY-4358 | Eli Lilly | Phase 3 | B7-H3 | |
| MRK-853 | Merck & Co | Phase 1 | PRMT5 | |
| Gelinaritide | AbbVie | Preclinical | FcRn | |
| Geliderotide | Sanofi | Phase 1/2 | PRMT5 | |
| Capifutibatinib | Sanofi | Approved | PRMT5 | |
| NVO-9615 | Novo Nordisk | Phase 2/3 | PRMT5 | |
| Suracagene | GSK | Phase 3 | PRMT5 | |
| Elrarapivir | Vertex Pharma | NDA/BLA | PRMT5 | |
| Darafutibatinib | BioNTech | Preclinical | PRMT5 |