Stoke Therapeutics, Inc. announced highlights from presentations related to the ongoing clinical development of STK-001, the first potential new medicine to treat the underlying cause of Dravet syndrome. Four posters from the Company's work in Dravet syndrome are being presented at the International Epilepsy Congress (IEC) 2023, September 2-6, in Dublin, Ireland. Data from the recently announced analysis of results from the ongoing Phase 1/2a studies (MONARCH & ADMIRAL) and the SWALLOWTAIL open-label extension study are being presented in a scientific forum for the first time.

In addition, a new pharmacokinetic (PK) analysis of 61 patients treated in STK-001 clinical trials is being presented for the first time, and demonstrates a correlation between higher STK-001 drug exposure in brain and greater reductions in seizure frequency over time. Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures beginning within the first year of life. The disease is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment associated with the disease.

The effects of the disease go beyond seizures and often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, and sleep abnormalities. The disease is classified as an developmental and epileptic encephalopath 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. STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in ongoing clinical trials.

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 EEMA, and rare pediatric disease designation by the FDA as a potential new treatment for Dravet syndrome. 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 pharmacokinetics in plasma, and exposure in cerebrosp spinal fluid. A secondary objective is To assess the pharmacokinetics in blood and exposure in cerebrospal fluid.

A secondary objective is the potential for STK-001 is to assess the pharmacokinetic in plasma and exposure in Cerebrospinal fluid. A primary objective is to assess the pharmacetics in plasma and exposure in Cere Brospinal fluid. A secondary objectives is to assess the pharmacokin genetics in plasma and exposure in cerebspinal fluid.

A secondary goal is to assess the pharmacokin characteristics in plasma and exposure in brain and exposure in cerebrospina fluid. A secondary objective isto assess the pharmacokinetics in the plasma and exposure in cerebro Spinal fluid. A secondary objective was to assess the pharmacokinetics of STK-001 and exposure in the SWALLOWTAIL Open-label extension study.