Modality
mRNA
MOA
MDM2i
Target
APOC3
Pathway
Autophagy
Alzheimer'sDravet
Development Pipeline
Preclinical
~Nov 2017
→ ~Feb 2019
Phase 1
~May 2019
→ ~Aug 2020
Phase 2
~Nov 2020
→ ~Feb 2022
Phase 3
May 2022
→ Oct 2028
Phase 3Current
NCT07715421
930 pts·Dravet
2022-05→2028-10·Completed
930 total pts1 indication
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2028-10-192.6y awayPh3 Readout· Dravet
Trial Timeline
Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q4
P3
Complet…
Catalysts
Ph3 Readout
2028-10-19 · 2.6y away
Dravet
Completed|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07715421 | Phase 3 | Dravet | Completed | 930 | UPCR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-1944 | Pfizer | NDA/BLA | APOC3 | |
| AZN-6294 | AstraZeneca | Phase 2/3 | APOC3 | |
| BMY-8678 | Bristol-Myers Squibb | Approved | RET | |
| Zanutinib | Bristol-Myers Squibb | Phase 2/3 | APOC3 | |
| AMG-415 | Amgen | Phase 1 | CDK2 | |
| GIL-2037 | Gilead Sciences | Approved | KRASG12D | |
| Capiglumide | BioNTech | Preclinical | CGRP | |
| ALN-5628 | Alnylam | Phase 1/2 | APOC3 | |
| INC-1582 | Incyte | Phase 1/2 | APOC3 | |
| Rimasertib | Exelixis | Phase 3 | B7-H3 |