Stoke: FDA allows higher single dose of STK-001 in 1/2a MONARCH study

Stoke Therapeutics announced that the FDA will allow the administration of a higher single dose of STK-001 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. Dravet 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 being developed by Stoke as the first potential new medicine to address the genetic cause of Dravet syndrome. "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."