BEDFORD, Mass. - Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology firm focused on RNA-based medicines for severe diseases, today announced promising data from studies involving its investigational drug STK-001 for treating Dravet syndrome, a severe genetic epilepsy.
The Phase 1/2a studies and open-label extension (OLE) studies revealed significant seizure frequency reduction and cognitive and behavioral improvements in children and adolescents with Dravet syndrome already on existing anti-seizure medications.
The study data showed an 85% median reduction in convulsive seizure frequency at three months and a 74% reduction at six months among patients treated with 70mg doses of STK-001. Additionally, continued dosing at 30mg and 45mg in OLE studies maintained these reductions and improvements over 12 months.
STK-001 has been generally well-tolerated, with the most common treatment-emergent adverse events being CSF protein elevations and procedural vomiting. The U.S. Food and Drug Administration (FDA) has cleared patients to receive three doses of 70mg followed by continued dosing at 45mg. Stoke plans to discuss a registrational study design with regulatory agencies based on these findings.
Dravet syndrome, characterized by frequent, prolonged, and refractory seizures, often leads to intellectual disability, developmental delays, and a higher risk of sudden unexpected death in epilepsy (SUDEP). Current treatments focus on managing seizures but do not address the underlying genetic cause.
STK-001, a proprietary antisense oligonucleotide (ASO), aims to be the first disease-modifying therapy for Dravet syndrome by targeting the genetic root of the condition. The drug has received orphan drug designation by the FDA and the European Medicines Agency (EMA), and rare pediatric disease designation by the FDA.
Stoke Therapeutics will host a webcast and conference call for analysts and investors at 4:30 p.m. Eastern Time today to present these data. The information in this article is based on a press release statement from Stoke Therapeutics.
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