Stoke Therapeutics is set to commence a Phase I/IIa ADMIRAL study of its investigational new medicine, STK-001, for treating Dravet syndrome, a severe and progressive genetic epilepsy.
The latest development comes after the UK Medicines and Healthcare products Regulatory Agency (MHRA) authorised Stoke’s clinical trial application.
STK-001 is a proprietary antisense oligonucleotide (ASO) that could become the first disease-modifying therapy for the genetic cause of Dravet syndrome, characterised by recurrent, prolonged and refractory seizures.
The open-label, multi-centre study will evaluate the safety and tolerability of multiple doses of up to 70mg of STK-001 and characterise human pharmacokinetics.
Change in seizure frequency and quality of life measures will be included as secondary endpoints of the study.
It plans to enrol approximately 22 children and adolescents aged two to 18 years with Dravet syndrome across various clinical sites in the UK.
Stoke intends to initiate subject enrolment and dosing in the second half of this year.
Stoke Therapeutics CEO Edward Kaye said: “The ADMIRAL study complements our US-based MONARCH study, enabling the evaluation of higher dose levels of STK-001 and representing an initial step in the expansion of our global clinical development efforts.
“We look forward to working with the UK Dravet community, patients, families and healthcare providers, to add to our understanding of the potential for STK-001 to be the first disease-modifying therapy for Dravet syndrome.”
A similar MONARCH Phase I/IIa clinical trial is progressing in the US which has completed enrolment in the first two single ascending dose (SAD) cohorts (10mg and 20mg) and is currently enrolling and dosing in the third (30mg) SAD cohort.
The company anticipates preliminary safety and pharmacokinetic data from the SAD portion of the MONARCH study in the second half of this year. At present, the US Food and Drug Administration (FDA) has put a partial clinical hold on dosing above 30mg in the study.