DelveInsight’s, “Encephalopathy Pipeline Insight, 2025” report provides comprehensive insights about 50+ companies and 55+ pipeline drugs in Encephalopathy pipeline landscape. It covers the Encephalopathy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Encephalopathy pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
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Key Takeaways from the Encephalopathy Pipeline Report
- In July 2025, Longboard Pharmaceuticals conducted a (DEEp OCEAN Study) is a double-blind, randomized, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of LP352 in the treatment of seizures in children and adults with DEE. The study consists of 3 main phases: Screening, Titration period, Maintenance period, followed by a Taper period and Follow-Up. The total duration of the study will be approximately 24 months.
- In July 2025, Capsida Biotherapeutics Inc. conducted a clinical trial is to learn about the safety of CAP-002 gene therapy in children with Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy. It will also provide information about whether CAP-002 demonstrates efficacy in treating children with STXBP1 with and without seizures.
- In July 2025, CereMark Pharma LLC conducted a clinical trial to evaluate the safety and efficacy of [F-18]Flornaptitril-PET (F-18 FNT-PET) for the prediction of clinical progression of Mild Cognitive Impairment (MCI) with either Suspected Chronic Traumatic Encephalopathy (CTE) or Alzheimer’s Disease (AD).
- In July 2025, Neurocrine Biosciences organized a study to evaluate how safe and tolerable the drug NBI-921352 is when used as adjunctive therapy in participants with SCN8A developmental and epileptic encephalopathy syndrome (SCN8A-DEE).
- In July 2025, Stoke Therapeutics Inc. conducted a study is to evaluate the efficacy, safety, and tolerability of zorevunersen in Patients with Dravet syndrome.Zorevunersen is an investigational new medicine for the treatment of Dravet syndrome. It is an antisense oligonucleotide (ASO) that is intended to increase the level of productive SCN1A messenger RNA (mRNA) and consequently increase the expression of the sodium channel Nav1.1 protein. This RNA-based approach is not gene therapy, but rather RNA modulation, as it does not manipulate nor insert genetic deoxyribonucleic acid (DNA).
- In July 2025, Zogenix International Limited Inc. announced a study of ZX008 in pediatric and young adult subjects with Dravet syndrome who participated in one of the core studies (ZX008-1501 and ZX008-1502) and are candidates for continuous treatment for an extended period of time. This trial will consist of a 36-month Open-Label Extension (OLE) Treatment Period and a 2-week Post-Dosing Period.
- DelveInsight’s Encephalopathy pipeline report depicts a robust space with 50+ active players working to develop 50+ pipeline therapies for Encephalopathy treatment.
- The leading Encephalopathy Companies such as Takeda, SK Life Science, Inc, Epygenix, Stoke Therapeutics, Inc, Encoded Therapeutics, Genfit, TenNor Therapeutics and others.
- Promising Encephalopathy Pipeline Therapies such as TAK-935, PRAX-562, Rifaximin SSD, VSL#3, AST-120, VE303, HPN-100 and others.
Discover how the Encephalopathy treatment paradigm is evolving. Access DelveInsight’s in-depth Encephalopathy Pipeline Analysis for a closer look at promising breakthroughs @ Encephalopathy Clinical Trials and Studies
Encephalopathy Emerging Drugs Profile
- STK-001: Stoke Therapeutics, Inc
STK-001, also known as zorevunersen, is Stoke Therapeutics’ lead investigational therapy for Dravet syndrome, a severe genetic epilepsy. It’s an antisense oligonucleotide designed to upregulate NaV1.1 protein expression by targeting the non-mutant SCN1A gene, aiming to restore physiological NaV1.1 levels and reduce both seizures and associated comorbidities. Clinical trials have shown substantial and sustained reductions in seizure frequency and improvements in cognition and behavior among patients already on standard anti-seizure medications. Recognized for its potential, zorevunersen has received FDA Breakthrough Therapy Designation, as well as orphan drug and rare pediatric disease designations. A global Phase III registrational study is planned to commence in Q2 2025, following a collaboration agreement with Biogen for international development and commercialization.
- EPX-100: Epygenix
EPX-100, clemizole hydrochloride, is under development for the treatment of Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS). EPX-100 acts by targeting central 5-hydroxytryptamine receptors to modulate serotonin signaling. The drug candidate is administered orally twice a day in a liquid formulation and has been developed based on a proprietary phenotype-based zebrafish drug screening platform. DS is caused by a loss of function mutation in the SCN1A gene, and scn1 mutant zebrafish replicate the genetic etiology and phenotype observed in the majority of DS patients. The scn1Lab mutant zebrafish model that expresses voltage gated sodium channels has been used for high-throughput screening of compounds that modulate Nav1.1 in the central nervous system. EPX-100, clemizole hydrochloride, is under development for the treatment of Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS). EPX-100 acts by targeting central 5-hydroxytryptamine receptors to modulate serotonin signaling. The drug candidate is administered orally twice a day in a liquid formulation and has been developed based on a proprietary phenotype-based zebrafish drug screening platform. DS is caused by a loss of function mutation in the SCN1A gene, and SCN1 mutant zebrafish replicate the genetic etiology and phenotype observed in the majority of DS patients. The scn1Lab mutant zebrafish model that expresses voltage gated sodium channels has been used for high-throughput screening of compounds that modulate Nav1.1 in the central nervous system. Currently, the drug is in Phase II trial for the treatment of Dravet syndrome.
- ETX101: Encoded Therapeutics
ETX101 as a potential one-time, disease-modifying gene regulation therapy targeting the underlying cause of SCN1A+ Dravet syndrome. In ETX101, a transgene encoding an engineered transcription factor under the control of a cell-selective regulatory element is delivered within a clinically-validated capsid (AAV9) to upregulate, or increase, the expression of the endogenous SCN1A gene. This approach is expected to increase production of NaV1.1 protein sodium channels in target neurons in the brain, leading to restored function. By targeting the underlying mechanism, ETX101 has the potential to address the full range of symptoms associated with Dravet syndrome. ETX101 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation by the US Food and Drug Administration and Orphan Designation by the European Medicines Agency. Currently, the drug is in Phase I/II trial for the treatment of Dravet syndrome.
The Encephalopathy pipeline report provides insights into
- The report provides detailed insights about companies that are developing therapies for the treatment of Encephalopathy with aggregate therapies developed by each company for the same.
- It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Encephalopathy Treatment.
- Encephalopathy Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
- Encephalopathy Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
- Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Encephalopathy market.
Get a detailed analysis of the latest innovations in the Encephalopathy pipeline. Explore DelveInsight’s expert-driven report today! @ Encephalopathy Unmet Needs
Encephalopathy Companies
Takeda, SK Life Science Inc, Epygenix, Stoke Therapeutics, Inc, Encoded Therapeutics, Genfit, TenNor Therapeutics and others.
Encephalopathy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
- Intra-articular
- Intraocular
- Intrathecal
- Intravenous
- Ophthalmic
- Oral
- Parenteral
- Subcutaneous
- Topical
- Transdermal
Encephalopathy Products have been categorized under various Molecule types such as
- Oligonucleotide
- Peptide
- Small molecule
Download DelveInsight’s latest report to gain strategic insights into upcoming Encephalopathy Therapies and key Developments @ Encephalopathy Market Drivers and Barriers, and Future Perspectives
Scope of the Encephalopathy Pipeline Report
- Coverage- Global
- Encephalopathy Companies- Takeda, SK Life Science, Inc, Epygenix, Stoke Therapeutics, Inc, Encoded Therapeutics, Genfit, TenNor Therapeutics and others.
- Encephalopathy Pipeline Therapies- TAK-935, PRAX-562, Rifaximin SSD, VSL#3, AST-120, VE303, HPN-100 and others.
- Encephalopathy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
- Encephalopathy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
Which companies are leading the race in Encephalopathy drug development? Find out in DelveInsight’s exclusive Encephalopathy Pipeline Report—access it now! @ Encephalopathy Emerging Drugs and Major Companies
Table of Contents
- Introduction
- Executive Summary
- Encephalopathy: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Encephalopathy– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- XEN496: Xenon Pharmaceuticals
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- RBX 7455: Rebiotix
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- Drug Name: Company Name
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Drug Name; Company Name
- Drug profiles in the detailed report…..
- Inactive Products
- Encephalopathy Key Companies
- Encephalopathy Key Products
- Encephalopathy – Unmet Needs
- Encephalopathy – Market Drivers and Barriers
- Encephalopathy – Future Perspectives and Conclusion
- Encephalopathy Analyst Views
- Encephalopathy Key Companies
- Appendix
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