DelveInsight’s, “Dravet syndrome Pipeline Insight 2025” report provides comprehensive insights about 10+ companies and 12+ pipeline drugs in Dravet syndrome pipeline landscape. It covers the Dravet Syndrome pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Dravet Syndrome pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
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Key Takeaways from the Dravet Syndrome Pipeline Report
- In July 2025, Encoded Therapeutics conducted a Phase 1/2 study in the UK to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet Syndrome aged 6 to < 48 months. The study follows and open-label, dose-escalation design.
- In July 2025, Longboard Pharmaceuticals announced a study to investigate the efficacy, safety, and tolerability of LP352 in the treatment of seizures in children and adults with DS. The study consists of 3 main phases: Screening, Titration period, and Maintenance period, followed by a Taper period and Follow-Up. Participants will be randomized to LP352 or placebo. The total duration of the study will be approximately 24 months.
- In July 2025, UCB BIOSCIENCES Inc. announced a study is evaluate the safety and tolerability of fenfluramine hydrochloride (HCl) 0.2 to 0.8 mg/kg/day in infants 1 year to less than 2 years of age with Dravet syndrome.
- DelveInsight’s Dravet Syndrome pipeline report depicts a robust space with 10+ active players working to develop 12+ pipeline therapies for Dravet Syndrome treatment.
- The leading Dravet Syndrome Companies such as Hinova Pharmaceuticals, Lantheus, Janux Therapeutics, Tagworks Pharmaceuticals, AstraZeneca, Xencor, Cardiff Oncology, Madison Vaccines, Bayer, Olema Oncology, Eli Lilly and Company, Curium, Pfizer, Regeneron Pharmaceuticals, PendreaBio, Momotaro-Gene, Norroy Bioscience, Orion Corporation, Epizyme, Barinthus Biotherapeutics, Twinpig Biolab, Oxford Vacmedix, Roche, ImaginAb, Halda THERAPEUTICS, Novartis, Kyinno Biotechnology and others.
- Promising Dravet Syndrome Pipeline Therapies such as Ganaxolone, Fenfluramine, Zorevunersen, ETX101, STK-001, Clemizole HCl, LP352, Verapamil, ZX008 and others.
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Dravet Syndrome Emerging Drugs Profile
- 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.
The Dravet Syndrome Pipeline Report Provides Insights into
- The report provides detailed insights about companies that are developing therapies for the treatment of Dravet Syndrome with aggregate therapies developed by each company for the same.
- It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Dravet Syndrome Treatment.
- Dravet Syndrome Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
- Dravet Syndrome Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
- Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Dravet Syndrome market
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Dravet Syndrome Companies
Hinova Pharmaceuticals, Lantheus, Janux Therapeutics, Tagworks Pharmaceuticals, AstraZeneca, Xencor, Cardiff Oncology, Madison Vaccines, Bayer, Olema Oncology, Eli Lilly and Company, Curium, Pfizer, Regeneron Pharmaceuticals, PendreaBio, Momotaro-Gene, Norroy Bioscience, Orion Corporation, Epizyme, Barinthus Biotherapeutics, Twinpig Biolab, Oxford Vacmedix, Roche, ImaginAb, Halda THERAPEUTICS, Novartis, Kyinno Biotechnology and others.
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
Dravet Syndrome Products have been categorized under various Molecule types such as
· Recombinant fusion proteins
· Small molecule
· Monoclonal antibody
· Peptide
· Polymer
· Gene therapy
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Scope of the Dravet Syndrome Pipeline Report
- Coverage- Global
- Dravet Syndrome Companies- Hinova Pharmaceuticals, Lantheus, Janux Therapeutics, Tagworks Pharmaceuticals, AstraZeneca, Xencor, Cardiff Oncology, Madison Vaccines, Bayer, Olema Oncology, Eli Lilly and Company, Curium, Pfizer, Regeneron Pharmaceuticals, PendreaBio, Momotaro-Gene, Norroy Bioscience, Orion Corporation, Epizyme, Barinthus Biotherapeutics, Twinpig Biolab, Oxford Vacmedix, Roche, ImaginAb, Halda THERAPEUTICS, Novartis, Kyinno Biotechnology and others.
- Dravet Syndrome Pipeline Therapies- Ganaxolone, Fenfluramine, Zorevunersen, ETX101, STK-001, Clemizole HCl, LP352, Verapamil, ZX008 and others.
- Dravet Syndrome Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
- Dravet Syndrome Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
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Table of Contents
- Introduction
- Executive Summary
- Dravet Syndrome: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Dravet Syndrome– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Soticlestat : Takeda
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- EPX-100: Harmony Biosciences
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I/II)
- Drug Name: Company Name
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Drug Name: Company Name.
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Dravet Syndrome Key Companies
- Dravet Syndrome Key Products
- Dravet Syndrome- Unmet Needs
- Dravet Syndrome- Market Drivers and Barriers
- Dravet Syndrome- Future Perspectives and Conclusion
- Dravet Syndrome Analyst Views
- Dravet Syndrome Key Companies
- Appendix
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