Modality
Vaccine
MOA
PCSK9i
Target
RET
Pathway
JAK/STAT
DravetMDS
Development Pipeline
Preclinical
~Oct 2016
→ ~Jan 2018
Phase 1
Apr 2018
→ Mar 2029
Phase 1Current
NCT06426455
1,430 pts·Dravet
2018-04→2029-02·Active
NCT03457682
128 pts·Dravet
2019-12→2029-03·Terminated
1,558 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2029-02-092.9y awayPh2 Data· Dravet
2029-03-283.0y awayPh2 Data· Dravet
Trial Timeline
Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2
P1/2
Active
P1/2
Termina…
Catalysts
Ph2 Data
2029-02-09 · 2.9y away
Dravet
Ph2 Data
2029-03-28 · 3.0y away
Dravet
ActiveTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT06426455 | Phase 1/2 | Dravet | Active | 1430 | PFS |
| NCT03457682 | Phase 1/2 | Dravet | Terminated | 128 | BodyWt |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| RHH-5389 | Roche | Preclinical | RET | |
| MRK-8368 | Merck & Co | Phase 3 | ALK | |
| Fixanesiran | AbbVie | Preclinical | RET | |
| Ribofutibatinib | AstraZeneca | Phase 2/3 | SOS1 | |
| AZN-4015 | AstraZeneca | Preclinical | BCMA | |
| BMY-8678 | Bristol-Myers Squibb | Approved | RET | |
| Polazasiran | Amgen | Phase 2 | LAG-3 | |
| Cevitinib | Regeneron | Phase 3 | FGFR | |
| VRT-1576 | Vertex Pharma | Phase 2/3 | RET | |
| ARG-3458 | Argenx | Preclinical | RET |