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Stoke Therapeutics Announces That FDA Will Allow the Administration of a Higher Single Dose of STK-001 (70mg) in the Ongoing Phase 1/2a MONARCH Study of Children and Adolescents with Dravet Syndrome
– Dosing anticipated to start in the coming weeks – – Company remains on track for its planned data readouts from the Phase 1/2a MONARCH and ADMIRAL studies
About this update from Stoke Therapeutics, Inc.
[{"type":"text","content":"\n– Dosing anticipated to start in the coming weeks –\n\n– Company remains on track for its planned data readouts from the Phase 1/2a MONARCH and ADMIRAL studies in 2023 –\n\n BEDFORD, Mass.--(BUSINESS WIRE)--\nStoke 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 that the FDA will allow the administration of a higher single dose of STK-001 (70mg) in its ongoing Phase 1/2a MONARCH study of children and adolescents with Dravet syndrome. This new 70mg single dose cohort in MONARCH will complement the ongoing evaluation of multiple doses of 70mg in the ADMIRAL study.\n\nDravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures beginning within the first year of life. Complications of the disease often contribute to poor quality of life for patients and their caregivers. Despite available anti-seizure medicines, seizures are not adequately controlled for more than 90% of patients. STK-001 is a proprietary antisense oligonucleotide (ASO) being developed by Stoke as the first potential new medicine to address the genetic cause of Dravet syndrome.\n\n“There is an urgent need for a disease modifying approach to treat Dravet syndrome, a devastating disease with effects that go beyond seizures and impact quality of life for patients and their families,” said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. “We thank the FDA for its timely review of the clinical data from our ongoing studies. We expect data from this new dose cohort to provide information about the potential effects and durability of a single dose of 70mg. We look forward to dosing the first patient in the coming weeks and to be able to include preliminary findings in our anticipated data readout in the second half of 2023.”\n\nUpcoming Anticipated Milestones\n\n\nData from patients treated with multiple 45mg doses of STK-001 are anticipated in mid-2023.\n\n\nData from patients treated with 70mg of STK-001 in MONARCH (single dose) and ADMIRAL (multiple doses) are anticipated in the second half of 2023.\n\n\nThe Company expects to complete the Phase 1/2a studies of STK-001 in 2023 in order to initiate a Phase 3 program in 2024.\n\n\nThe MON...