Modality
ERT
MOA
PARPi
Target
ALK
Pathway
Neuroinflam
NASHFabryAML
Development Pipeline
Preclinical
~Apr 2011
→ ~Jul 2012
Phase 1
~Oct 2012
→ ~Jan 2014
Phase 2
~Apr 2014
→ ~Jul 2015
Phase 3
~Oct 2015
→ ~Jan 2017
NDA/BLA
Apr 2017
→ Mar 2029
NDA/BLACurrent
NCT08984918
1,424 pts·AML
2025-12→2026-06·Not yet recruiting
NCT06335635
2,979 pts·AML
2017-04→2029-03·Completed
4,403 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-06-253mo awayPh3 Readout· AML
2029-03-253.0y awayPh3 Readout· AML
Trial Timeline
Q2Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2
NDA/BLA
Complet…
NDA/BLA
Not yet…
Catalysts
Ph3 Readout
2026-06-25 · 3mo away
AML
Ph3 Readout
2029-03-25 · 3.0y away
AML
CompletedNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08984918 | NDA/BLA | AML | Not yet recr... | 1424 | PASI75 |
| NCT06335635 | NDA/BLA | AML | Completed | 2979 | Safety |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| MRK-3732 | Merck & Co | Phase 1 | TYK2 | |
| MRK-8368 | Merck & Co | Phase 3 | ALK | |
| GSK-6983 | GSK | Phase 2 | ALK | |
| GSK-6516 | GSK | Phase 1/2 | MALT1 | |
| BAY-3308 | Bayer | Phase 1 | ALK | |
| BAY-3684 | Bayer | Approved | PD-L1 | |
| Tixarelsin | Vertex Pharma | Phase 1 | ALK | |
| VRT-6833 | Vertex Pharma | Preclinical | ALK | |
| BNT-5232 | BioNTech | NDA/BLA | TIGIT | |
| ALN-3958 | Alnylam | Phase 2 | MALT1 |