DelveInsight’s, “Dravet Syndrome – Pipeline Insight, 2020,” report provides comprehensive insights about 20+ companies and 20+ 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 (DS) is a severe form of epilepsy characterized by frequent, prolonged seizures often triggered by high body temperature (hyperthermia), developmental delay, speech impairment, ataxia, hypotonia, sleep disturbances, and other health problems. DS is thought to be at the severe end of a spectrum of disorders associated with changes (mutations) in genes for the sodium ion channel. The sodium ion channel is a gated pore-like structure in the cell membrane that regulates the movement of sodium ions into and out of the cell, helping to propagate electrical signals along neurons. Sodium ion channels are critical components of any tissue requiring electrical signals including the brain and heart. More than 80% of patients with Dravet syndrome have a mutation in the SCN1A gene, but not all SCN1A mutations lead to Dravet syndrome.
Symptoms
Signs and symptoms include:
• Many different types of seizures
• Sudden muscle jerking (myoclonus)
• Loss of developmental skills
• Intellectual disability
• Problems with walking
• Speech impairment
• Autistic-like behavior
Diagnosis
Dravet syndrome is diagnosed based on the results of a clinical exam looking for specific symptoms that have been previously seen in this condition. Genetic testing can also be helpful. Diagnosis is important because certain anti-seizure medications (sodium-channel agents) can make the seizures worse in Dravet syndrome.
Treatment
Although there is no cure for Dravet syndrome, most treatments aim to reduce seizures. First line anti-seizure medications include clobazam (Onfi, Frisium) and valproic acid (Depakote, Depakene). Second line treatments include stiripentol (Diacomit), topiramate (Topamax), and the ketogenic diet. Variations of the ketogenic diet including the Modified Atkins Diet may also be beneficial in DS. Third line treatments include clonazepam (Klonopin), levetiracetam (Keppra), zonisamide (Zonegran), ethosuximide (Zarontin), and vagal nerve stimulator (VNS).
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: Ovid Therapeutics
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. 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 excitatory/inhibitory balance of NMDA channel activity.
• STK-001: Stoke Therapeutics
STK-001 is an investigational new medicine for the treatment of Dravet syndrome. Stoke believes that STK-001, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. STK-001 is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing both occurrence of seizures and significant non-seizure comorbidities. Stoke has generated preclinical data demonstrating proof-of-mechanism and proof-of-concept for STK-001. STK-001 has been granted orphan drug designation by the FDA as a potential new treatment for Dravet syndrome.
Further product details are provided in the report……..
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. 20+ 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, Celltrion.
• Phases
DelveInsight’s 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
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
• Parenteral
• intravitreal
• Subretinal
• Topical.
• Molecule Type
Products have been categorized under various Molecule types such as
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• 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.

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.
• In August 2019, the U.S. Food and Drug Administration (FDA) has granted orphan drug designation — allowing various development incentives — to Stoke Therapeutics’ STK-001 for the treatment of Dravet syndrome.
• In August 2020, Stoke Therapeutics announced the publication of preclinical data from studies of STK-001 that demonstrated significant improvements in survival and reductions in seizure frequency in a mouse model of Dravet syndrome.
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
• Stoke Therapeutics
• Zogenix International
• Takeda
• Ovid Therapeutics Inc.
• NYU Langone Health
• PTC Therapeutics
• Marinus Pharmaceuticals
• INSYS Therapeutics Inc
• Epygenix
• GreenLight Clinical PTY LTD
• XERIS Pharmaceuticals
• Encoded Therapeutics

Key Products
• Diazepam
• STK-001
• ZX008
• TAK-935
• ataluren
• ganaxolone
• Cannabidiol Oral Solution
• EPX-100 (Clemizole HCl)
• ETX 101