Modality
Gene Editing
MOA
Anti-Aβ
Target
Tau
Pathway
Hedgehog
Dravet
Development Pipeline
Preclinical
~Mar 2014
→ ~Jun 2015
Phase 1
~Sep 2015
→ ~Dec 2016
Phase 2
~Mar 2017
→ ~Jun 2018
Phase 3
~Sep 2018
→ ~Dec 2019
NDA/BLA
Mar 2020
→ Sep 2029
NDA/BLACurrent
NCT04154912
1,626 pts·Dravet
2020-03→TBD·Active
NCT08003020
2,473 pts·Dravet
2025-04→2029-09·Recruiting
4,099 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (1)
2029-09-253.5y awayPh3 Readout· Dravet
Trial Timeline
2020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q4
NDA/BLA
Active
NDA/BLA
Recruit…
Catalysts
Ph3 Readout
2029-09-25 · 3.5y away
Dravet
RecruitingActive|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04154912 | NDA/BLA | Dravet | Active | 1626 | SeizFreq |
| NCT08003020 | NDA/BLA | Dravet | Recruiting | 2473 | OS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| LLY-8369 | Eli Lilly | Approved | Menin | |
| ABB-8696 | AbbVie | Phase 3 | BCMA | |
| NVO-9087 | Novo Nordisk | Phase 1/2 | Tau | |
| Tezecilimab | Takeda | NDA/BLA | Tau | |
| TAK-8262 | Takeda | NDA/BLA | Tau | |
| BAY-6035 | Bayer | Phase 1 | C5 | |
| Zenotinib | BioNTech | Phase 1 | CGRP | |
| RLA-6817 | Relay Therapeutics | Phase 3 | TIGIT | |
| Lisoosocimab | Zealand Pharma | Approved | KIF18A | |
| PRA-419 | Praxis Precision | Approved | Cl18.2 |