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Disease
Genetic developmental and epileptic encephalopathy
Late-stage therapeutic development
Also known as hereditary developmental and epileptic encephalopathy, developmental and epileptic encephalopathy.
20
Clinical trials
12
Associated genes
12
Related proteins
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
Executive briefingUpdating summary…Momentum: Moderate
Key developments
- 6 clinical trials expected to report results, the earliest in Q4 2026.
Upcoming Milestones
- Q4 2026A Phase 3, Randomized, Multi-Center, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Relutrigine in Participants With DEE Followed by an Open-Label Extension
- Q1 2027EMPEROR: 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
- Q1 2027Exploring the Impact of Genetic Variations on The Clinical Efficacy of Nalbuphine in Postoperative Pain Management
- Q2 2027A 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)
- Q2 2028A Prospective, Case-control Evaluation of Ketogenic Dietary Therapy for New-onset Childhood Absence Epilepsy
Major developments
Upcoming trial readoutHigh impact
Results expected Q2 20322026-07-10
Upcoming trial readoutHigh impact
Results expected Q1 20272026-07-01
Clinical MilestonesView all 9Hide
Results posted
Results expected
- 2026-07-10Phase III Trial Of Neoadjuvant Durvalumab (NSC 778709) Plus Chemotherapy Versus Chemotherapy Alone For Adults With MammaPrint High 2 Risk (MP2) Hormone Receptor (HR) Positive / Human Epidermal Growth Factor Receptor (HER2) Negative Stage II-III Breast CancerResults expected Q2 2032
- 2026-07-01EMPEROR: 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 SyndromeResults expected Q1 2027
- 2026-06-30A 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)Results expected Q2 2027
- 2026-05-11A Phase 3, Randomized, Multi-Center, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Relutrigine in Participants With DEE Followed by an Open-Label ExtensionResults expected Q4 2026
- 2026-05-07A Prospective, Case-control Evaluation of Ketogenic Dietary Therapy for New-onset Childhood Absence EpilepsyResults expected Q2 2028
Other updates
- 2025-11-12A Prospective, Long-Term, Interventional, Active Extension Study to Evaluate the Safety and Tolerability of NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)Terminated
- 2025-09-24A Phase 3, Prospective, Open-Label, Multisite, Extension of Phase 3 Studies To Assess the Long-Term Safety and Tolerability of Soticlestat as Adjunctive Therapy in Subjects With Dravet Syndrome or Lennox-Gastaut Syndrome (ENDYMION 2)Terminated
+1 more in the activity timeline below
Activity timeline9
- 2026-07-10ClinicalPhase III Trial Of Neoadjuvant Durvalumab (NSC 778709) Plus Chemotherapy Versus Chemotherapy Alone For Adults With MammaPrint High 2 Risk (MP2) Hormone Receptor (HR) Positive / Human Epidermal Growth Factor Receptor (HER2) Negative Stage II-III Breast CancerResults expected Q2 2032
- 2026-07-01ClinicalEMPEROR: 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 SyndromeResults expected Q1 2027
- 2026-06-30ClinicalA 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)Results expected Q2 2027
- 2026-05-11ClinicalA Phase 3, Randomized, Multi-Center, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Relutrigine in Participants With DEE Followed by an Open-Label ExtensionResults expected Q4 2026
- 2026-05-07ClinicalA Prospective, Case-control Evaluation of Ketogenic Dietary Therapy for New-onset Childhood Absence EpilepsyResults expected Q2 2028
- 2026-04-15ClinicalExploring the Impact of Genetic Variations on The Clinical Efficacy of Nalbuphine in Postoperative Pain ManagementResults expected Q1 2027
- 2025-11-17ClinicalAn Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy for Seizures in Patients With Rare Seizure Disorders Such as Epileptic Encephalopathies Including Dravet Syndrome and Lennox-Gastaut SyndromeResults posted
- 2025-11-12ClinicalA Prospective, Long-Term, Interventional, Active Extension Study to Evaluate the Safety and Tolerability of NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)Terminated
- 2025-09-24ClinicalA Phase 3, Prospective, Open-Label, Multisite, Extension of Phase 3 Studies To Assess the Long-Term Safety and Tolerability of Soticlestat as Adjunctive Therapy in Subjects With Dravet Syndrome or Lennox-Gastaut Syndrome (ENDYMION 2)Terminated
Clinical trials
The current development programme across all trial phases.
Clinical programme
20
6
15
6
Late-stage studies
Recruiting
Recently completed
Associated genes
Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.
Disease biology
Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.
Gamma-aminobutyric acid receptor subunit beta-2via GABRB20.78Sodium channel protein type 1 subunit alphavia SCN1A0.77Potassium voltage-gated channel subfamily KQT member 2via KCNQ20.73Voltage-dependent R-type calcium channel subunit alpha-1Evia CACNA1E0.71Gamma-aminobutyric acid receptor subunit alpha-2via GABRA20.66Potassium voltage-gated channel subfamily A member 2via KCNA20.64Gamma-aminobutyric acid receptor subunit alpha-5via GABRA50.63BDNF/NT-3 growth factors receptorvia NTRK20.61Voltage-dependent calcium channel subunit alpha-2/delta-2via CACNA2D20.58Potassium voltage-gated channel subfamily B member 1via KCNB10.52Voltage-gated delayed rectifier potassium channel KCNH5via KCNH50.51Synaptosomal-associated protein 25via SNAP250.50
Reference
Authoritative identity, definition & identifiers.
Synonyms
hereditary developmental and epileptic encephalopathy, developmental and epileptic encephalopathy
References & data sources
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Related entities are derived from literature co-mention (studied together) — associative, not causal.