Encephalopathy- Pipeline Insight, 2025

DelveInsight's, "Encephalopathy- Pipeline Insight, 2025" report provides comprehensive insights about 50+ companies and 55+ pipeline drugs in Encephalopathy pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered:

• Global coverage

Encephalopathy: Understanding

Encephalopathy: Overview

Encephalopathy, a broad term encompassing various brain dysfunctions, presents a multifaceted challenge in clinical practice. Characterized by alterations in cognition, behavior, and consciousness, encephalopathy can stem from diverse etiologies, including metabolic imbalances, infectious agents, toxins, and vascular insults. Its manifestations range from subtle cognitive deficits to profound impairment, posing diagnostic dilemmas and necessitating tailored management strategies. Understanding the underlying mechanisms driving encephalopathy and its clinical manifestations is pivotal for effective intervention and improved patient outcomes.

The signs and symptoms of encephalopathy are diverse and can range from mild to severe, reflecting the broad spectrum of underlying causes. Common manifestations include confusion, memory loss, personality changes, and difficulty concentrating. Patients may also experience tremors, muscle weakness, and seizures. In more advanced cases, symptoms can progress to stupor or coma. Additional signs such as altered speech, uncoordinated movements, and visual disturbances may be observed.

Encephalopathy encompasses various types including Hepatic, Metabolic, Infectious, Hypoxic-Ischemic, Toxic, Uremic, Wernicke's, Traumatic, and Hypertensive, each linked to specific underlying causes.

The pathology of encephalopathy encompasses a wide array of structural and functional brain abnormalities resulting from various etiological factors. Metabolic encephalopathies, for example, arise from imbalances such as hypoglycemia or hepatic dysfunction, leading to neuronal injury due to inadequate energy supply or toxin accumulation. Infectious encephalopathies, caused by pathogens like viruses or bacteria, induce inflammation and direct neuronal damage. Toxin-induced encephalopathies involve exposure to harmful substances that disrupt normal brain function. Vascular causes, including strokes or chronic hypoperfusion, result in ischemic damage and neuronal death.

The diagnosis of encephalopathy involves a comprehensive evaluation of the patient's clinical history, symptoms, and physical examination findings. Diagnostic tests such as blood work, neuroimaging (CT or MRI scans), and electroencephalography (EEG) are essential to identify underlying causes and assess brain function. Additional tests may include lumbar puncture and metabolic panels to detect infections, toxins, or metabolic abnormalities contributing to the condition

The treatment of encephalopathy focuses on addressing the underlying cause, such as correcting metabolic imbalances, treating infections, or removing toxins. Supportive care, including hydration, nutrition, and managing symptoms like seizures or agitation, is crucial for patient stabilization. Early intervention and tailored therapies are essential to prevent further brain damage and improve recovery outcomes.

"Encephalopathy- Pipeline Insight, 2025" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Encephalopathy pipeline landscape is provided which includes the disease overview and Encephalopathy treatment guidelines. The assessment part of the report embraces, in depth Encephalopathy commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Encephalopathy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights:

• The companies and academics are working to assess challenges and seek opportunities that could influence Encephalopathy R&D. The therapies under development are focused on novel approaches to treat/improve Encephalopathy.

Encephalopathy Emerging Drugs Chapters

This segment of the Encephalopathy report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, II/III I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Encephalopathy Emerging Drugs

• 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.

Encephalopathy: Therapeutic Assessment

This segment of the report provides insights about the different Encephalopathy drugs segregated based on following parameters that define the scope of the report, such as:

• Major Players in Encephalopathy

There are approx. 50+ key companies which are developing the therapies for Encephalopathy. The companies which have their Encephalopathy drug candidates in the most advanced stage, i.e. Phase III include, Stoke Therapeutics, Inc.

• Phases

DelveInsight's report covers around 55+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• Route of Administration

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

• Intravenous
• Subcutaneous
• Oral
• Intramuscular
• Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal antibody
• Small molecule
• Peptide
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Encephalopathy: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Encephalopathy therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Encephalopathy drugs.

Encephalopathy Report Insights

• Encephalopathy Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Encephalopathy Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions:

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Encephalopathy drugs?
• How many Encephalopathy drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Encephalopathy?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Encephalopathy therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Encephalopathy and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Takeda
• SK Life Science, Inc
• Epygenix
• Stoke Therapeutics, Inc
• Encoded Therapeutics
• Genfit
• TenNor Therapeutics

Key Products

• Soticlestat
• Carisbamate
• EPX-100
• STK-001
• ETX101
• VS 02
• Rifaquizinone