Interim results from Stoke Therapeutics’ Phase 1/2a US study of STK-001 in children and adolescents (aged 2-18) with Dravet Syndrome.

We’re excited to share encouraging interim results from Stoke Therapeutics’ Phase 1/2a US study of STK-001 in children and adolescents (aged 2-18) with Dravet Syndrome.

STK-001 is first gene-specific (to SCN1A), disease-modifying approach to treating Dravet Syndrome, with the aim of improving both seizures and comorbidities.

Although these are very early findings with only a small number of patients (11 in total), they represent a promising step forward in our knowledge about the potential safety and effectiveness of this new genetic therapy. They indicate no safety concerns so far and a trend towards seizure reduction in 8 out of the 11 study participants analysed so far in the study. As this is early data, there are no updates as yet on the impact on comorbidities and quality of life.

The key findings show:

• Single doses of STK-001 up to 30mg and multiple doses of 20mg were well-tolerated with no safety concerns related to the study drug

• 8/11 patients experienced a reduction in convulsive seizure frequency

• Trend more evident in patients 2 to 12 years old

• Patients in the study were taking multiple anti-seizure medicines: 85% on 3 or more medications and 67% on 4 or more medications

• Analysis indicates that most (>95%) of patients are anticipated to achieve pharmacologically active levels of STK-001 with three monthly doses of 30mg

These findings are from the US-based MONARCH study. A similar study, called ADMIRAL, has recently started recruiting in the UK, with the first patient already receiving their first 30 mg dose (this study will evaluate multiple doses up to 70mg.

To find out more about the UK ADMIRAL study, including information on how to take part in this trial, please see our FAQs here . This study is open for children and young people with Dravet Syndrome, aged 2-18 years. Recruiters are particularly hoping to increase numbers of participants in the older (age 12-18) category.

You can read more information about the MONARCH results here. Further data was released by Stoke Therapeutics at the American Epilepsy Society annual meeting in early December 2021.