Developmental And Epileptic Encephalopathies (DEE) - Pipeline Insight, 2025

This “Developmental and Epileptic Encephalopathies (DEE) - Pipeline Insight, 2025” report provides comprehensive insights about 15+ companies and 20+ pipeline drugs in Developmental and Epileptic Encephalopathies (DEE) 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.

Developmental and Epileptic Encephalopathies (DEE): Understanding

Developmental and Epileptic Encephalopathies (DEE): Overview

Developmental and Epileptic Encephalopathy (DEE) refers to a group of severe epilepsies that are characterized both by seizures, which are often drug-resistant, as well as encephalopathy, which is a term used to describe significant developmental delay or even loss of developmental skills. In the DEEs, there are two factors that contribute to the developmental delay. Developmental delay or even loss of developmental skills. In the DEEs, there are two factors that contribute to the developmental delay.

Developmental and Epileptic Encephalopathies represent a group of severe epilepsy syndromes characterized by early-onset seizures and significant cognitive impairment. Symptoms typically include frequent and severe seizures (such as myoclonic, tonic, or atonic seizures), developmental delays, behavioral issues (like hyperactivity and aggression), cognitive deficits, and motor impairments. These conditions often lead to a regression of previously acquired developmental skills, with many affected individuals experiencing lifelong challenges in cognitive and motor functions.

Developmental and epileptic encephalopathies represent a complex group of severe neurological disorders characterized by early-onset seizures and significant cognitive impairment. The pathophysiology of DEE is multifaceted, involving both genetic mutations and structural brain abnormalities that disrupt normal brain development and function. Key factors include the impact of frequent seizures on cognitive development, as well as the underlying genetic defects that can lead to varying degrees of neurodevelopmental delay. For instance, mutations in genes such as SCN1A and KCNQ2 are commonly associated with specific DEEs, influencing the severity of seizures and developmental outcomes.

Developmental and Epileptic Encephalopathies are severe epilepsy syndromes with frequent seizures and developmental impairments, often starting in infancy or early childhood. Diagnosis involves clinical evaluation, EEG, genetic testing, and neuroimaging like MRI. Symptoms can evolve, complicating diagnosis, and initial tests may not always reveal abnormalities. Management focuses on seizure control, which may improve cognitive outcomes but rarely resolves underlying developmental challenges.

Treatment for Developmental and Epileptic Encephalopathies focuses on managing seizures and improving quality of life. Anti-seizure medications like clobazam and vigabatrin are tailored to the specific syndrome. Refractory cases may require interventions like vagus nerve stimulation, ketogenic diets, or surgery. Emerging therapies, including gene therapies, aim to address genetic causes while balancing seizure control with minimizing medication side effects.

'Developmental and Epileptic Encephalopathies (DEE)- Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Developmental and Epileptic Encephalopathies (DEE) pipeline landscape is provided which includes the disease overview and Developmental and Epileptic Encephalopathies (DEE) treatment guidelines. The assessment part of the report embraces, in depth Developmental and Epileptic Encephalopathies (DEE) 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, Developmental and Epileptic Encephalopathies (DEE) 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 Developmental and Epileptic Encephalopathies (DEE) R&D. The therapies under development are focused on novel approaches to treat/improve Developmental and Epileptic Encephalopathies (DEE).

Developmental and Epileptic Encephalopathies (DEE) Emerging Drugs Chapters

This segment of the Developmental and Epileptic Encephalopathies (DEE) report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, 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.

Developmental and Epileptic Encephalopathies (DEE) Emerging Drugs

LP352: Lundbeck

LP352 is an oral, centrally acting, 5-HT2c superagonist in development for the potential treatment of seizures associated with DEEs such as Dravet syndrome, Lennox-Gastaut syndrome (LGS), tuberous sclerosis complex (TSC), CDKL5 deficiency disorder (CDD), and other epileptic disorders. LP352 is designed to modulate GABA and, as a result, suppress the central hyperexcitability that is characteristic of seizures. LP352 has demonstrated negligible observed impact on 5-HT2b and 5-HT2a receptor subtypes in preclinical studies to date. 5-HT2b and 5-HT2a receptor agonism have been associated with significant adverse side effects. LP352 has novel chemistry and attributes. It was designed to be more specific and selective for the 5-HT2c receptor subtype, giving it the potential to reduce seizures in patients with DEEs while overcoming the known or perceived safety limitations of available drugs in the 5-HT2 class. Currently, the drug is in Phase III stage of its clinical trial for the treatment of Developmental and Epileptic Encephalopathies.

NBI-921352: Neurocrine Biosciences

NBI-921352 is an investigational selective Nav1.6 sodium channel inhibitor for the potential treatment of SCN8A-DEE. Neurocrine acquired exclusive rights to NBI-921352 from Xenon Pharmaceuticals, Inc. and received orphan drug and rare pediatric disease designations from the U.S. Food and Drug Administration (FDA) for NBI-921352 in SCN8A-DEE. Currently, the drug is in Phase II stage of its clinical trial for the treatment of Developmental and Epileptic Encephalopathies.

CAP-002: Capsida Biotherapeutics

CAP-002 is a wholly owned first-in-class next-generation IV-administered gene therapy that is designed to stably supplement STXBP1 protein throughout the brain after a single intravenous infusion while providing a superior safety profile compared to traditional gene therapy approaches by lowering dose and de-targeting non-therapeutic organs. This potential disease modifying therapy aims to correct the underlying disease pathology to alleviate disease manifestations for patients with STXBP1-DEE. The STXBP1 program is in IND-enabling studies to support initiation of clinical trials in the first half of 2025.

Developmental and Epileptic Encephalopathies (DEE): Therapeutic Assessment

This segment of the report provides insights about the different Developmental and Epileptic Encephalopathies (DEE) drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Developmental and Epileptic Encephalopathies (DEE)

• There are approx. 15+ key companies which are developing the therapies for Developmental and Epileptic Encephalopathies (DEE). The companies which have their Developmental and Epileptic Encephalopathies (DEE) drug candidates in the most advanced stage, i.e. Phase III include, Lundbeck.

Phases

The report covers around 20+ 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

Developmental and Epileptic Encephalopathies (DEE) pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

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

Developmental and Epileptic Encephalopathies (DEE): Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Developmental and Epileptic Encephalopathies (DEE) 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 Developmental and Epileptic Encephalopathies (DEE) drugs.

Developmental and Epileptic Encephalopathies (DEE) Report Insights

• Developmental and Epileptic Encephalopathies (DEE) Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Developmental and Epileptic Encephalopathies (DEE) 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 Developmental and Epileptic Encephalopathies (DEE) drugs?
• How many Developmental and Epileptic Encephalopathies (DEE) drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Developmental and Epileptic Encephalopathies (DEE)?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Developmental and Epileptic Encephalopathies (DEE) 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 Developmental and Epileptic Encephalopathies (DEE) and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Lundbeck
• Neurocrine Biosciences
• Bright Minds Biosciences
• Praxis Precision Medicines
• Capsida Biotherapeutics
• Harmony Biosciences
• Q-State Biosciences, Inc

Key Products

• LP352
• NBI-921352
• BMB-101
• PRAX-222
• CAP-002
• EPX-200
• Research programme: DEE4 - STXBP1 (ASO)

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