\nDravet 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.<\/p>\n
\n\u201cThe data from ongoing studies of STK-001 provide the first evidence of a disease modifying medicine for Dravet syndrome,\u201d said Helen Cross, MB ChB, Ph.D., Professor, The Prince of Wales\u2019s Chair of Childhood Epilepsy and Director of University College London Great Ormond Street Institute of Child Health, Honorary Consultant in Paediatric Neurology, and the ADMIRAL study lead investigator. \u201cThe primary goal of these studies is to assess safety and tolerability as well as to inform dose selection for future studies. Importantly, within these data we can now see a differentiated pattern of response emerging among the 11 patients treated with an initial two or three doses of 70mg. Additionally, data from the open-label extension study that is evaluating ongoing dosing at lower levels showed, for the first time, improvements in multiple measures of cognition and behavior among patients who have been highly refractory to standard anti-seizure medicines.\u201d<\/p>\n
\nThe potential of STK-001, a proprietary antisense oligonucleotide (ASO), to be the first disease-modifying medicine for Dravet syndrome is supported by multiple presentations of clinical and other data at IEC.<\/p>\n
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- \nMONARCH and ADMIRAL Interim Analyses: Single and multiple doses of STK-001 up to 70mg were generally well tolerated. Patients treated with 2 or 3 initial doses of 70mg experienced substantial and sustained reductions in convulsive seizure frequency with median reductions of 80% (n=6) at 3 months and 89% (n=3) at 6 months after last dose, compared to baseline.<\/p>\n<\/li>\n<\/ul>\n
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- \nSWALLOWTAIL open label extension (OLE): Safety findings among patients, who are continuing to be dosed with STK-001, were consistent with the findings from MONARCH and ADMIRAL with the exception of a greater incidence of CSF protein elevation. Durable reductions in convulsive seizure frequency were observed throughout the course of treatment in SWALLOWTAIL. Additionally, data indicated substantial improvements from baseline through 12 months of continued STK-001 dosing in multiple assessments of cognition and behavior.<\/p>\n<\/li>\n<\/ul>\n
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- \nBUTTERFLY (natural history study): Small but significant improvements in receptive communication were observed at Month 12; however, little to no change was observed across other measures of cognition and behavior. Data from this study will inform future Dravet syndrome studies.<\/p>\n<\/li>\n<\/ul>\n
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- \nPK Model for STK-001: A relationship between STK-001 brain exposure and convulsive seizure frequency was evaluated based on patients treated in the two ongoing Phase 1\/2a studies (MONARCH and ADMIRAL) and the SWALLOWTAIL OLE study in children and adolescents with Dravet syndrome. The exposure-seizure relationship showed a significant negative trend based on simulated brain Cavg (R=-0.23, P<0.001) demonstrating that higher STK-001 brain exposure leads to greater reductions in convulsive seizure frequency. This PK model is anticipated to help identify an optimal dosing regimen for a Phase 3 study.<\/p>\n<\/li>\n<\/ul>\n
\n\u201cOur clinical findings to date show that patients treated with STK-001 experienced substantial and sustained reductions in seizures and importantly, improvements in cognition and behavior,\u201d said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. \u201cThe new data from our pharmacokinetic model provide additional confidence in the observed effects of STK-001 and provide helpful information as we design the pivotal program for STK-001. We look forward to sharing additional data in the first quarter of 2024 that are anticipated to provide greater clarity on dose and dose regimen, and to updating the Dravet community on our Phase 3 plans in the first half of 2024.\u201d<\/p>\n
\nDetails of the Company\u2019s four presentations can be found in the table below. All presentations are available for download on the Stoke Therapeutics website under the Investors & News tab.<\/p>\n
- \nPK Model for STK-001: A relationship between STK-001 brain exposure and convulsive seizure frequency was evaluated based on patients treated in the two ongoing Phase 1\/2a studies (MONARCH and ADMIRAL) and the SWALLOWTAIL OLE study in children and adolescents with Dravet syndrome. The exposure-seizure relationship showed a significant negative trend based on simulated brain Cavg (R=-0.23, P<0.001) demonstrating that higher STK-001 brain exposure leads to greater reductions in convulsive seizure frequency. This PK model is anticipated to help identify an optimal dosing regimen for a Phase 3 study.<\/p>\n<\/li>\n<\/ul>\n
- \nBUTTERFLY (natural history study): Small but significant improvements in receptive communication were observed at Month 12; however, little to no change was observed across other measures of cognition and behavior. Data from this study will inform future Dravet syndrome studies.<\/p>\n<\/li>\n<\/ul>\n
- \nSWALLOWTAIL open label extension (OLE): Safety findings among patients, who are continuing to be dosed with STK-001, were consistent with the findings from MONARCH and ADMIRAL with the exception of a greater incidence of CSF protein elevation. Durable reductions in convulsive seizure frequency were observed throughout the course of treatment in SWALLOWTAIL. Additionally, data indicated substantial improvements from baseline through 12 months of continued STK-001 dosing in multiple assessments of cognition and behavior.<\/p>\n<\/li>\n<\/ul>\n
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