Stoke Therapeutics Highlights Strategic Priorities and Anticipated Milestones for 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 outlined its expected milestones and the following strategic priorities for 2023:

• Complete Phase 1/2a studies of STK-001 for Dravet syndrome in 2023 in order to initiate a Phase 3 program in 2024;
• Continue to advance STK-002 for autosomal dominant optic atrophy (ADOA) toward the clinic; and
• Develop and expand the pipeline with a focus on the central nervous system and the eye.

“Our recent data showed the first evidence that upregulating protein expression with STK-001 to target the underlying cause of Dravet syndrome can reduce seizure frequency in children and adolescents who are already taking several anti-seizure medicines, including fenfluramine,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “These data support our belief that we are entering a new era in the treatment of this disease – a shift from seizure management to syndrome management – one that could have a profound impact for patients and caregivers. With additional data anticipated in 2023, we expect to be in a position to select a dose level and dose frequency in order to initiate a Phase 3 program in 2024.”

“In recent years our understanding of Dravet syndrome has improved dramatically. We now know what causes it and the effects it has on patients as they age. Even though three new medications have been approved by the FDA in the last five years for the treatment of seizures associated with Dravet syndrome, none of them address the full scope of the syndrome," said Joseph Sullivan, M.D., Professor of Neurology and Pediatrics and Director of the Pediatric Epilepsy Center of Excellence at the University of California San Francisco, and a prominent researcher into Dravet Syndrome. "That leaves a significant gap in our ability to effectively care for our patients. By restoring native gene expression, potential disease-modifying approaches are positioned to improve seizure control and address the non-seizure aspects of the disease that negatively impact health and quality of life for people living with this disease.”