(MENAFN- GlobeNewsWire - Nasdaq) Dublin, Oct. 14, 2024 (GLOBE NEWSWIRE) -- The "Dravet Syndrome- Pipeline Insight, 2024" clinical trials has been added to ResearchAndMarkets.com's offering.
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.
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: 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.
Dravet Syndrome: Therapeutic Assessment
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.
Key Questions
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
Phases
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
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
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
For more information about this clinical trials report visit
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