Exhibit 99.1
Stoke Therapeutics Presents Data From Multiple Studies of Children and Adolescents With Dravet Syndrome
at the American Epilepsy Society (AES) 2023 Annual Meeting
Data from clinical studies of
STK-001 demonstrated clinical benefit for patients ages 2 to 18 years old, including reductions in seizures and improvements in cognition and behavior that support the potential for disease modification
Analysis of 72 patients treated in STK-001 clinical trials suggests that higher STK-001 drug exposure in brain leads to greater seizure reductions
Two-year data from the longest prospective natural history study of Dravet syndrome showed that, on average, patients experienced no meaningful improvement in convulsive seizure frequency and exhibited widening gaps
in cognition and behavior despite treatment with the best available anti-seizure medicines
BEDFORD,
Mass., December 1, 2023 Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced highlights from presentations of clinical data at the American Epilepsy Society (AES) 2023 Annual Meeting December 1 5, in Orlando, Florida. Together, these data
support the companys continued progress to develop STK-001 as the first disease-modifying medicine for the treatment of Dravet syndrome.
The comprehensive set of data being presented at AES are giving us a very good understanding of how STK-001
works and its potential to address not only seizures, but many of the non-seizure effects of Dravet syndrome, said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. The
substantial and sustained reductions in seizure frequency and improvements in cognition and behavior observed in our STK-001 clinical studies give us confidence that we are addressing the root cause of Dravet
syndrome. In addition, the correlation between higher STK-001 exposure levels in brain and reductions in seizure frequency shown in our modeling data provides additional confidence in the clinical benefits
observed among patients treated with STK-001 at higher doses and for longer periods of time. These findings are in stark contrast to the data from our two-year natural
history study that show a lack of improvement among patients who are taking the best available anti-seizure medicines.
Dravet syndrome goes far beyond seizures, and as children grow up, they experience a complex array of life-altering challenges, including developmental
delays, movement and balance issues and delayed language and speech, said Joseph Sullivan, M.D., FAES, Professor of Neurology and Pediatrics and Director of the Pediatric Epilepsy Center of Excellence at the University of