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New data from electroencephalography (EEG) recordings showed 76% of Dravet syndrome (DS) mice treated with STK-001 were seizure free compared to 48% of placebo-treated mice
An 80% reduction in the average number of spontaneous seizures was also observed among treated DS mice
Data support the clinical development of STK-001 as a potential new medicine for Dravet syndrome, a severe and progressive genetic epilepsy
BEDFORD, Mass.--(BUSINESS WIRE)-- Stoke Therapeutics, Inc. (Nasdaq:STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases by precisely upregulating protein expression, today announced preclinical data from studies of STK-001 that showed significant improvements in survival and reductions in seizure frequency in a mouse model of Dravet syndrome (DS). New data from electroencephalography (EEG) recordings showed 76% (16/21) of DS mice treated with STK-001 were seizure free compared to 48% (10/21) that were treated with a placebo. An 80% reduction in the average number of spontaneous seizures (3 seizures vs 16 seizures) was also observed among treated DS mice compared to placebo. EEG is a highly sensitive measure of seizure activity, which enables the detection of seizures that may not be otherwise visible. These data were presented in a poster session at the American Epilepsy Society (AES) Annual Meeting in Baltimore.
“The data on STK-001 from this mouse model give us confidence in our approach to treating the underlying cause of Dravet syndrome by restoring Nav1.1 protein expression to near normal levels,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “The reductions in mortality previously observed with STK-001 were compelling and the new EEG data provide further evidence of the potential for STK-001 to impact Dravet syndrome by reducing seizure frequency and possibly preventing seizures entirely. What is particularly remarkable is that these data were generated from a spontaneous seizure model, which we believe accurately reflects the clinical situation in people with Dravet syndrome.”
Dravet syndrome is a severe and progressive genetic epilepsy that begins within the first year of life. The effects of the disease go beyond seizures and often include cognitive regression or developmental stagnation, ataxia, speech impairment and sleep disturbances. Approximately 85% of Dravet syndrome cases are caused by spontaneous, heterozygous variants in the SCN1A gene. This gene encodes the voltage-gated sodium channel α subunit, NaV1.1. STK-001 is a proprietary antisense oligonucleotide (ASO) designed to increase – or upregulate – protein production by manipulating the cell’s RNA splicing process.
In the studies presented in today’s poster, the efficacy of STK-001 was evaluated in a Scn1a-linked mouse model of Dravet syndrome that results in Nav1.1 haploinsufficiency. Seizures and Sudden Unexpected Death in Epilepsy (SUDEP) in these mice occurred spontaneously and were not provoked by temperature changes or other manipulation. Mice were administered a single intracerebroventricular (ICV) injection of STK-001 or placebo at postnatal day 2 or postnatal day 14 and monitored to postnatal day 90. The data showed improvements among mice treated with STK-001 compared to placebo, including:
