VivoSeq
Neurology

Dravet Syndrome

14

Pipeline Programs↗

10

Active Trials↗

13

Companies Involved↗

Dravet syndrome is an emerging landscape with 14 programs across 13 companies. The most active players are Encoded Therapeutics, Val Adams, PTC Therapeutics. 7 programs are in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

45/100

Pipeline Velocity

6/100

Mech. Concentration

25/100

Completion Rate

69/100

Attrition Rate

16/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

E

Encoded Therapeutics

1 program

Phase 2V

Val Adams

1 program

Phase 2P

PTC Therapeutics

1 program

Phase 2Eisai logo

Eisai

1 program

Phase 3Lundbeck logo

Lundbeck

1 program

Phase 3L

Longboard Pharmaceuticals

1 program

Phase 3

Pipeline

Programs by Phase

Analysis

Modality Breakdown

93%

Small molecule

14 of 15 classified programs — the dominant modality here.

7%ALL OTHERSRNA therapy17%

Small molecule leads; the ring shows all 15 classified programs, each share of the total. Modality classified for 88% of 17 pipeline programs.

Analysis

Trial Status

32

clinical trials

Completed11 · 34%
Recruiting6 · 19%
Active, not recruiting4 · 13%
No longer available3 · 9%
Terminated3 · 9%
Withdrawn2 · 6%
Other3 · 9%

Clinical Trials

Trial Activity

32 trials

A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)
Recruiting·LLongboard Pharmaceuticals·Phase 3·NCT06660394·160 enrolled
Assessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under 24 Months of Age
Recruiting·UUniversity of Colorado, Denver·Phase 4·NCT06598449·12 enrolled
ENDEAVOR: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Infants and Children With SCN1A-Positive Dravet Syndrome
Recruiting·EEncoded Therapeutics·Preclinical·NCT05419492·47 enrolled
EMPEROR: A Multicenter, Randomized, Double-blind, Sham-controlled, Parallel Group, Phase 3 Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen (STK-001) in Patients With Dravet Syndrome
Recruiting·SStoke Therapeutics, Inc·Phase 3·NCT06872125·170 enrolled
A 20-Week Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of EPX-100 (Clemizole Hydrochloride) as Adjunctive Therapy in Children and Adult Participants With Dravet Syndrome (ARGUS Trial)
Recruiting·EEpygenix·Phase 3·NCT04462770·150 enrolled
Phase 1-2, Open-Label, Single and Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION337 in Patients With Dravet Syndrome
Recruiting·Ionis Pharmaceuticals logoIonis Pharmaceuticals·Preclinical·NCT07531745·32 enrolled
An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome
Active, not recruiting·SStoke Therapeutics, Inc·Phase 2·NCT04740476·60 enrolled
A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome
Active, not recruiting·UCB logoUCB·Phase 3·NCT06118255·25 enrolled
A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (Australia Only)
Active, not recruiting·EEncoded Therapeutics·Phase 1/2·NCT06112275·4 enrolled
A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome
Active, not recruiting·EEncoded Therapeutics·Phase 1/2·NCT06283212·5 enrolled
Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
Completed·NNYU Langone Health·Phase 2·NCT02758626·15 enrolled
An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome
Completed·ZZogenix, Inc.·Phase 3·NCT02823145·375 enrolled
A Dose-ranging Pharmacokinetics and Safety Study of GWP42003-P in Children With Dravet Syndrome (GWPCARE1)
Completed·Jazz Pharmaceuticals logoJazz Pharmaceuticals·Phase 2·NCT02091206·34 enrolled
Antiepileptic Efficacy Study of GWP42003-P in Children and Young Adults With Dravet Syndrome (GWPCARE1)
Completed·Jazz Pharmaceuticals logoJazz Pharmaceuticals·Phase 3·NCT02091375·120 enrolled
A Study to Assess the Safety and Tolerability of ZX008 in Children and Young Adults With Dravet Syndrome or Lennox Gastaut Syndrome Currently Taking Cannabidiol
Completed·ZZogenix, Inc.·Phase 1·NCT03467113·9 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)