Dravet Syndrome - Pipeline Insight, 2024

This “Dravet syndrome - Pipeline Insight, 2024” report provides comprehensive insights about 10+ companies and 12+ pipeline drugs in Dravet syndrome 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

Dravet syndrome: Understanding

Dravet syndrome: Overview

Dravet syndrome is a rare, severe form of epilepsy that begins in infancy, characterized by prolonged seizures that are often triggered by fever. It is associated with developmental delays, motor dysfunction, and a higher risk of sudden unexpected death in epilepsy (SUDEP). The condition is typically caused by mutations in the SCN1A gene. Children with Dravet syndrome initially show focal (confined to one area) or generalized (throughout the brain) convulsive seizures that start before 15 months of age (often before age one).

Dravet Syndrome presents with a variety of signs and symptoms, including Prolonged Seizures: Often triggered by fever, Developmental Delays: Delayed development of language, motor skills, and cognitive abilities, Motor Dysfunction: Issues such as poor coordination, balance problems, and a tendency to walk on the toes, Behavioral Issues: Hyperactivity, impulsiveness, and autistic-like behaviors, Sleep Disturbances: Difficulties with falling or staying asleep, Frequent Infections: Respiratory and ear infections are common, Sensitivity to Temperature Changes: Seizures can be triggered by hot baths or warm weather.

Dravet Syndrome's pathophysiology primarily involves mutations in the SCN1A gene, which encodes the Nav1.1 subunit of the sodium channel in the brain. These mutations lead to dysfunctional sodium channels, impairing neuronal excitability and communication, resulting in the hyperexcitability of neuronal networks and an increased propensity for seizures.

Dravet Syndrome is diagnosed through a combination of clinical evaluation, including detailed seizure history and developmental assessment, and genetic testing to identify mutations in the SCN1A gene. EEGs may show characteristic patterns, and MRI scans can help rule out other conditions. Early diagnosis is crucial for managing the disorder and implementing appropriate treatments.

Treatment for Dravet Syndrome focuses on managing seizures and includes a combination of antiepileptic drugs such as valproate, clobazam, and stiripentol. Cannabidiol (CBD) has also shown efficacy in reducing seizure frequency. A ketogenic diet may be beneficial for some patients. Additionally, supportive therapies, including physical, occupational, and speech therapy, are essential to address developmental delays and improve quality of life. Seizures in Dravet syndrome are difficult to control but can be reduced by anticonvulsant drugs. Some anticonvulsant medications (such as oxcarbazepine, carbamazepine, phenytoin, and lamotrigine) should not be used on a daily basis as they may make seizures worse. A ketogenic diet, high in fats and low in carbohydrates, also may be helpful.

“Dravet syndrome - Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Dravet syndrome pipeline landscape is provided which includes the disease overview and Dravet syndrome treatment guidelines. The assessment part of the report embraces, in depth Dravet syndrome 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, Dravet syndrome 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 Dravet syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Dravet syndrome.

Dravet syndrome Emerging Drugs Chapters

This segment of the Dravet syndrome 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.

Dravet syndrome Emerging Drugs

Soticlestat : Takeda

Soticlestat is a potent, highly selective, first-in-class inhibitor of the enzyme cholesterol 24-hydroxylase (CH24H), with the potential to reduce seizure susceptibility and improve seizure control. CH24H is predominantly expressed in the brain, where it converts cholesterol into 24S-hydroxycholesterol (24HC) to adjust the homeostatic balance of brain cholesterol. 24HC is a positive allosteric modulator of the NMDA receptor and modulates glutamatergic signaling associated with epilepsy. Currently, the drug is in the Phase III stage of its development for the treatment of Dravet syndrome and Lennox-Gastaut syndrome.

EPX-100: Harmony Biosciences

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.1 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 the Phase II stage of its development for the treatment of Dravet syndrome and Lennox-Gastaut syndrome.

Dravet syndrome: Therapeutic Assessment

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

Major Players in Dravet syndrome

There are approx. 10+ key companies which are developing the therapies for Dravet syndrome. The companies which have their Dravet syndrome drug candidates in the most advanced stage, i.e. Phase III include, Takeda.

Phases

The report covers around 12+ 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

Dravet syndrome 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.

Dravet syndrome: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Dravet syndrome 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 Dravet syndrome drugs.

Dravet syndrome Report Insights

• Dravet syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Takeda
• Harmony Biosciences
• Jazz Pharmaceuticals
• Stoke Therapeutics
• Encoded Therapeutics
• Xenon Pharmaceuticals
• reMYND

Key Products

• Soticlestat
• EPX-100
• Cannabidiol
• STK-001
• ETX101

Research programme: Nav1.1 sodium channel openers

• ReS3 T

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