Dravet Syndrome- Pipeline Insight, 2024

This “Dravet Syndrome- Pipeline Insight, 2024” report provides comprehensive insights about 12+ companies and 16+ 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 severe and progressive genetic epilepsy characterized by frequent, prolonged, and refractory seizures that usually begin within the first year of life. Dravet Syndrome is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment, in addition to seizure activity, that stem from the genetic mutation that causes the disease. The voltage-gated sodium channel is responsible for the initiation of action potentials and, therefore, is involved in neuronal excitability. The alpha subunit has four homologous domains with six transmembrane segments each that form the voltage sensor and ion-conducting pore. Mutations cause either a gain or a loss of function. Initially, researchers could not explain how loss-of-function mutations could lead to seizures. A mouse model of DS showed selective loss of sodium current in the hippocampal γ-aminobutyric acid-mediated inhibitory interneurons. Failure of inhibition leading to excitation is a proposed pathogenesis of this mutation in DS.

Approximately 85% of those diagnosed with Dravet Syndrome have a mutation of the SCN1A gene. Dravet Syndrome is not usually caused by an inherited mutation. In 90% of these patients, the mutation is not found in the patient’s parents Dravet Syndrome can be diagnosed, if a patient exhibits specific symptoms, genetic testing may be done via an epilepsy panel, to look for SCN1A and other genes commonly associated with epilepsy. However, the effects of Dravet syndrome are not limited to seizures, developmental delays, movement and balance issues and also language and speech disturbances. Dravet syndrome has a high rate of premature death due to the severity of this type of epilepsy. Up to 20% of children and adolescents living with Dravet die before adulthood.

The correct diagnosis of DS and appropriate follow-up are typically delayed. The EEG is normal at onset, and neuroimaging reveals no structural lesion. Early development is normal, but signs of regression appear in the second year of life and are often accompanied by convulsive status epilepticus, alternating hemiconvulsions, and myoclonic seizures. The diagnosis can be confirmed by genetic testing that is now available and shows mutations within the SCN1A gene. Early recognition and diagnosis of DS and management with appropriate anticonvulsants and a treatment plan may reduce the seizure burden and improve the long-term developmental outcome.

"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 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.

Dravet Syndrome Emerging Drugs

Soticlestat : Takeda

Soticlestat is a potent, highly selective, oral, 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. Glutamate is one of the main neurotransmitters in the brain and has been shown to play a role in the initiation and spread of seizure activity. Recent literature indicates that CH24H is involved in over-activation of the glutamatergic pathway through modulation of the NMDA channel and that increased expression of CH24H can disrupt the reuptake of glutamate by astrocytes, resulting in epileptogenesis and neurotoxicity. Inhibition of CH24H by soticlestat reduces the neuronal levels of 24HC and may improve the excitatory/inhibitory balance of NMDA channel activity. It is being investigated by Ovid and Takeda for the treatment of rare developmental and epileptic encephalopathies (DEEs), a group of highly refractory epilepsy syndromes including Dravet Syndrome and LGS. Soticlestat is in the Phase III stage of development for the treatment of Dravet Syndrome.

EPX-100: Harmony Biosciences

EPX-100 is a repurposed antihistamine that was used in the past to treat itchiness. The medication can suppress seizures via modulation of serotonin (5-HT) signaling pathways, which is different from its anti-histaminic properties. EPX-100 was a powerful suppressor of spontaneous convulsive behavior and electrographic seizures in zebrafish disease models for Dravet syndrome. EPX-100’s antiepileptic action is not through a histaminergic mechanism of action but acts via modulation of serotonin (5-HT) signaling pathways. Currently, the drug is in Phase II trial as a single pivotal study to compare the efficacy of EPX-100 against placebo as adjunctive therapy in patients with Dravet Syndrome. In April 2024, Harmony Biosciences announced the acquisition of Epygenix Therapeutics, Inc., accelerating its growth strategy by adding a rare epilepsy franchise to its expanding late-stage pipeline of innovative CNS assets.

STK-001: Stoke Therapeutics

STK-001 is an investigational new medicine for the treatment of Dravet syndrome. It is a proprietary antisense oligonucleotide (ASO) and has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. The drug is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing both the occurrences of seizures and significant non-seizure comorbidities. This RNA-based approach is not gene therapy but rather RNA modulation, as it does not manipulate nor insert genetic deoxyribonucleic acid (DNA). The drug is currently in Phase II clinical trial for the treatment of patients with Dravet 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. 12+ 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

DelveInsight’s report covers around 16+ 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

• 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.

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
• Stoke Therapeutics
• Longboard Pharmaceuticals
• Encoded Therapeutics
• Eisai
• Virpax Pharmaceuticals

Key Products

• Soticlestat
• EPX-100
• STK-001
• LP352
• ETX101
• Lorcaserin
• VRP324

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