BEDFORD - Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced that presentations related to the Company's work in Dravet syndrome will be presented at the American Epilepsy Society (AES) 2023 Annual Meeting, taking place December 1 - 5, in Orlando, Florida.

The company is advancing STK-001 as potentially the first medicine to address the genetic cause of Dravet syndrome.

'People living with Dravet syndrome typically experience high rates of seizures and debilitating effects on neurodevelopment, including behavioral and developmental delays, movement and balance issues, and delayed language and speech, among other life-altering challenges, which current treatments do not adequately address,' said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. 'We look forward to sharing the findings from more than 3 years of clinical studies, including important new data from the longest and largest prospective natural history study of patients with Dravet syndrome, which showed that despite treatment with multiple anti-seizure medicines, most patients continued to experience convulsive seizures over 24 months and that gaps in neurodevelopment persist between children with Dravet syndrome and their neurotypical peers. These data, together with the initial clinical findings from our Phase 1/2a and open label extension studies, as well as pharmacokinetic data to inform dosing, are giving us a very good understanding of how STK-001 works and highlight the critical need for a disease-modifying new medicine for Dravet syndrome.'

Details for the Company's poster presentations at AES are as follows

Title: 24-Month Analysis of BUTTERFLY: A Prospective, Observational Study to Investigate Cognition and Other Comorbidities in Children & Adolescents with Dravet Syndrome (DS)

Session Date & Time: Saturday, December 2 at 12:00 PM EST

Oral Presentation Date & Time: Monday, December 4 at 3:15 PM EST

Presenter: Joseph Sullivan, M.D., FAES, Professor of Neurology and Pediatrics and Director of the Pediatric Epilepsy Center of Excellence at the University of California San Francisco

Poster Number: 1.233

Title: MONARCH & ADMIRAL: Phase 1/2a Studies in US & UK Investigating Safety and Drug Exposure of STK-001, an Antisense Oligonucleotide (ASO), in Children & Adolescents with Dravet Syndrome (DS)

Session Date & Time: Saturday, December 2 at 12:00 PM EST

Presenter: Helen Cross, MB ChB, Ph.D., Professor, The Prince of Wales's Chair of Childhood Epilepsy and Head of the Developmental Neuroscience Programme at University College London Great Ormond Street Institute of Child Health, Honorary Consultant in Paediatric Neurology, President of the International League Against Epilepsy

Poster Number: 1.276

Title: SWALLOWTAIL & LONGWING: Open-Label Extension (OLE) Studies for Children and Adolescents with Dravet Syndrome (DS) who Previously Participated in a Study of Antisense Oligonucleotide (ASO) STK-001

Session Date & Time: Saturday, December 2 at 12:00 PM EST

Presenter: Archana Desurkar M.D., Consultant Paediatric Neurologist at Sheffield Children's Hospital National Health Service Foundation Trust

Poster Number: 1.279

Title: Utilization of a Pharmacokinetic (PK) Model for STK-001 in Patients with Dravet Syndrome (DS) To Support Selection of Dosing Regimens in Clinic

Session Date & Time: Monday, December 4 at 12:00 PM EST

Presenter: Meena, Ph.D., Senior Vice President of Translational DMPK and Clinical Pharmacology at Stoke Therapeutics

Poster Number: 3.110

About Dravet Syndrome

Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures, beginning within the first year of life. Dravet syndrome is difficult to treat and has a poor long-term prognosis. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. The effects of the disease go beyond seizures and often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system and mood disorders. The disease is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment associated with the disease. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. There are no approved disease-modifying therapies for people living with Dravet syndrome. One out of 16,000 babies are born with Dravet syndrome, which is not concentrated in a particular geographic area or ethnic group.

About STK-001

STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in ongoing clinical trials. 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. STK-001 has been granted orphan drug designation by the FDA and the EMA, and rare pediatric disease designation by the FDA as a potential new treatment for Dravet syndrome.

About the Phase 1/2a MONARCH Study (United States)

The MONARCH study is a Phase 1/2a open-label study of children and adolescents ages 2 to 18 who have an established diagnosis of Dravet syndrome and have evidence of a genetic mutation in the SCN1A gene. The primary objectives for the study are to assess the safety and tolerability of STK-001, as well as to determine the pharmacokinetics in plasma and exposure in cerebrospinal fluid. A secondary objective is to assess the efficacy as an adjunctive antiepileptic treatment with respect to the percentage change from baseline in convulsive seizure frequency. Stoke also intends to measure non-seizure aspects of the disease, such as quality of life, as secondary endpoints.

Patients who participated in the MONARCH study and meet study entry criteria are eligible to continue treatment in SWALLOWTAIL, an open-label extension (OLE) study designed to evaluate the long-term safety and tolerability of repeat doses of STK-001. We expect that SWALLOWTAIL will also provide valuable information on the preliminary effects of STK-001 on seizures along with non-seizure aspects of the disease, such as quality of life and cognition.

About the Phase 1/2a ADMIRAL Study (United Kingdom)

The ADMIRAL study is a Phase 1/2a open-label study of children and adolescents ages 2 to

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