Modality
Peptide
MOA
CD47i
Target
MALT1
Pathway
Checkpoint
PompeDravet
Development Pipeline
Preclinical
~May 2012
→ ~Aug 2013
Phase 1
~Nov 2013
→ ~Feb 2015
Phase 2
~May 2015
→ ~Aug 2016
Phase 3
~Nov 2016
→ ~Feb 2018
NDA/BLA
~May 2018
→ ~Aug 2019
Approved
Nov 2019
→ Dec 2031
ApprovedCurrent
NCT05931828
2,260 pts·Dravet
2020-12→2027-10·Not yet recruiting
NCT08513987
960 pts·Dravet
2020-09→2031-12·Terminated
NCT03055788
2,200 pts·Pompe
2019-11→2030-09·Completed
5,420 total pts2 indications
CompletedCurrentUpcoming
Catalysts (3)
2027-10-201.6y awayPh3 Readout· Dravet
2030-09-194.5y awayPh3 Readout· Pompe
2031-12-255.7y awayPh3 Readout· Dravet
Trial Timeline
Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
Approved
Complet…
Approved
Termina…
Approved
Not yet…
Catalysts
Ph3 Readout
2027-10-20 · 1.6y away
Dravet
Ph3 Readout
2030-09-19 · 4.5y away
Pompe
Ph3 Readout
2031-12-25 · 5.7y away
Dravet
CompletedTerminatedNot yet recruiting|StartCompletionToday
Trials (3)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05931828 | Approved | Dravet | Not yet recr... | 2260 | VA |
| NCT08513987 | Approved | Dravet | Terminated | 960 | PFS |
| NCT03055788 | Approved | Pompe | Completed | 2200 | HAM-D |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Nirarelsin | Johnson & Johnson | Phase 2 | PRMT5 | |
| Cevizanubrutinib | Johnson & Johnson | Preclinical | FcRn | |
| JNJ-8232 | Johnson & Johnson | NDA/BLA | MALT1 | |
| Ceviglumide | Roche | NDA/BLA | B7-H3 | |
| NVS-1475 | Novartis | Phase 2 | Menin | |
| AZN-6294 | AstraZeneca | Phase 2/3 | APOC3 | |
| BMY-7975 | Bristol-Myers Squibb | Phase 1 | MALT1 | |
| Zenonesiran | GSK | Preclinical | DLL3 | |
| GSK-6516 | GSK | Phase 1/2 | MALT1 | |
| AMG-7379 | Amgen | Preclinical | DLL3 |