Stoke presents data for STK-001 development to treat Dravet Syndrome at IEC

Stoke Therapeutics announced highlights from presentations related to the ongoing clinical development of STK-001, the first potential new medicine to treat the underlying cause of Dravet syndrome. Four posters from the Company’s work in Dravet syndrome are being presented at the International Epilepsy Congress IEC 2023, September 2-6, in Dublin, Ireland. Data from the recently announced analysis of results from the ongoing Phase 1/2a studies MONARCH & ADMIRAL and the SWALLOWTAIL open-label extension study are being presented in a scientific forum for the first time. In addition, a new pharmacokinetic PK analysis of 61 patients treated in STK-001 clinical trials is being presented for the first time, and demonstrates a correlation between higher STK-001 drug exposure in brain and greater reductions in seizure frequency over time. “The data from ongoing studies of STK-001 provide the first evidence of a disease modifying medicine for Dravet syndrome,” said Helen Cross, MB ChB, Ph.D., Professor, The Prince of Wales’s 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. “The 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.” The 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.