Modality
Gene Therapy
MOA
RAS(ON)i
Target
BTK
Pathway
RAS/MAPK
SCDHSALL
Development Pipeline
Preclinical
~Aug 2012
→ ~Nov 2013
Phase 1
~Feb 2014
→ ~May 2015
Phase 2
~Aug 2015
→ ~Nov 2016
Phase 3
Feb 2017
→ Jul 2028
Phase 3Current
NCT04785217
117 pts·ALL
2017-02→2027-06·Active
NCT08500928
39 pts·SCD
2020-12→TBD·Not yet recruiting
NCT08359551
2,182 pts·ALL
2019-06→2028-07·Active
2,338 total pts2 indications
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2027-06-161.2y awayPh3 Readout· ALL
2028-07-082.3y awayPh3 Readout· ALL
Trial Timeline
2017Q2Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3
P3
Active
P3
Active
P3
Not yet…
Catalysts
Ph3 Readout
2027-06-16 · 1.2y away
ALL
Ph3 Readout
2028-07-08 · 2.3y away
ALL
ActiveNot yet recruiting|StartCompletionToday
Trials (3)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04785217 | Phase 3 | ALL | Active | 117 | Safety |
| NCT08500928 | Phase 3 | SCD | Not yet recr... | 39 | HAM-D |
| NCT08359551 | Phase 3 | ALL | Active | 2182 | CR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-5501 | Pfizer | Phase 1 | BTK | |
| RHH-8482 | Roche | Phase 1 | BTK | |
| MRK-6781 | Merck & Co | Approved | TIGIT | |
| SNY-9073 | Sanofi | Phase 2 | B7-H3 | |
| Zorizanubrutinib | Novo Nordisk | Phase 1 | BTK | |
| Tixarelsin | Vertex Pharma | Phase 1 | ALK | |
| Fixatuximab | Legend Biotech | Preclinical | CFTR | |
| ION-1378 | Ionis | NDA/BLA | TROP-2 | |
| 180-3597 | Innovent Bio | Preclinical | SHP2 | |
| 369-8021 | Hansoh Pharma | Approved | BTK |