Modality
Small Molecule
MOA
CAR-T BCMA
Target
APOC3
Pathway
Checkpoint
DravetFSGSMCC
Development Pipeline
Preclinical
~Nov 2011
→ ~Feb 2013
Phase 1
~May 2013
→ ~Aug 2014
Phase 2
~Nov 2014
→ ~Feb 2016
Phase 3
~May 2016
→ ~Aug 2017
NDA/BLA
Nov 2017
→ Jul 2031
NDA/BLACurrent
NCT08227388
2,280 pts·MCC
2017-11→2031-07·Active
NCT03575276
90 pts·FSGS
2022-05→2031-01·Not yet recruiting
2,370 total pts2 indications
Approved
CompletedCurrentUpcoming
Catalysts (2)
2031-01-164.8y awayPh3 Readout· FSGS
2031-07-145.3y awayPh3 Readout· MCC
Trial Timeline
Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
NDA/BLA
Active
NDA/BLA
Not yet…
Catalysts
Ph3 Readout
2031-01-16 · 4.8y away
FSGS
Ph3 Readout
2031-07-14 · 5.3y away
MCC
ActiveNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08227388 | NDA/BLA | MCC | Active | 2280 | UPDRS |
| NCT03575276 | NDA/BLA | FSGS | Not yet recr... | 90 | eGFR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-1944 | Pfizer | NDA/BLA | APOC3 | |
| Voxacilimab | Pfizer | Phase 2 | TYK2 | |
| Doxacapivasertib | Eli Lilly | Phase 3 | APOC3 | |
| AZN-6294 | AstraZeneca | Phase 2/3 | APOC3 | |
| Zanutinib | Bristol-Myers Squibb | Phase 2/3 | APOC3 | |
| NVO-9087 | Novo Nordisk | Phase 1/2 | Tau | |
| Fixazanubrutinib | Amgen | Phase 2 | PI3Kα | |
| REG-8074 | Regeneron | Phase 2/3 | KRASG12C | |
| ALN-5628 | Alnylam | Phase 1/2 | APOC3 | |
| INC-1582 | Incyte | Phase 1/2 | APOC3 |