Modality
Gene Therapy
MOA
MALT1i
Target
PI3Kα
Pathway
Ferroptosis
NASHWilms
Development Pipeline
Preclinical
~Aug 2011
→ ~Nov 2012
Phase 1
~Feb 2013
→ ~May 2014
Phase 2
~Aug 2014
→ ~Nov 2015
Phase 3
~Feb 2016
→ ~May 2017
NDA/BLA
Aug 2017
→ Jan 2027
NDA/BLACurrent
NCT06326452
2,719 pts·Wilms
2017-08→2026-11·Recruiting
NCT03815197
1,289 pts·Wilms
2020-02→2027-01·Active
4,008 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-11-228mo awayPh3 Readout· Wilms
2027-01-1610mo awayPh3 Readout· Wilms
Trial Timeline
Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027
NDA/BLA
Recruit…
NDA/BLA
Active
Catalysts
Ph3 Readout
2026-11-22 · 8mo away
Wilms
Ph3 Readout
2027-01-16 · 10mo away
Wilms
RecruitingActive|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT06326452 | NDA/BLA | Wilms | Recruiting | 2719 | CR |
| NCT03815197 | NDA/BLA | Wilms | Active | 1289 | DAS28 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Voxaderotide | Johnson & Johnson | NDA/BLA | PI3Kα | |
| JNJ-2735 | Johnson & Johnson | Phase 2/3 | Cl18.2 | |
| Riboinavolisib | Johnson & Johnson | Phase 2/3 | GLP-1R | |
| RHH-974 | Roche | Preclinical | JAK1 | |
| NVS-3297 | Novartis | Preclinical | CDK2 | |
| Rimamavacamten | Merck & Co | Phase 1 | PI3Kα | |
| ABB-8985 | AbbVie | Phase 2 | CD20 | |
| Fixazanubrutinib | Amgen | Phase 2 | PI3Kα | |
| Sovarelsin | Alnylam | Phase 2/3 | LAG-3 | |
| GMA-1468 | Genmab | NDA/BLA | PI3Kα |