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Stoke Therapeutics Presents Data from the Phase 1/2a MONARCH Study of STK-001 in Children and Adolescents with Dravet Syndrome at the American Epilepsy Society (AES) 2021 Annual Meeting
– Single doses of STK-001 up to 30mg and multiple doses of 20mg were well tolerated with no safety concerns related to study drug – – 70.6% (12/17) of
About this update from Stoke Therapeutics, Inc.
[{"type":"text","content":"\n– Single doses of STK-001 up to 30mg and multiple doses of 20mg were well tolerated with no safety concerns related to study drug –\n\n– 70.6% (12/17) of patients treated with STK-001 experienced a reduction from baseline in convulsive seizure frequency measured from Day 29 to Day 84 –\n\n– All patients ages 2-12 (n=7) experienced reductions in seizure frequency –\n\n– Additional data to be presented include pharmacokinetic (PK) modeling data of STK-001, initial safety data from the SWALLOWTAIL open-label extension study and three-month data from the BUTTERFLY observational study –\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 highlights from five presentations related to the ongoing clinical development of STK-001 being made at the American Epilepsy Society (AES) 2021 Annual Meeting, December 3 – 7. STK-001 has the potential to be the first disease-modifying therapy to target the underlying cause of Dravet syndrome, which is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures beginning within the first year of life. The disease is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment associated with the disease.\n\nInterim Safety, PK, and CSF Exposure Data from the Phase 1/2a MONARCH Study of STK-001, an Antisense Oligonucleotide (ASO), in Children and Adolescents with Dravet Syndrome (DS)\nSession Date & Time: Sunday, December 5 at 12:00 p.m. CT\nPresenter: Linda Laux, M.D., Associate Professor of Pediatrics (Neurology and Epilepsy) at Northwestern University Feinberg School of Medicine and Attending Physician at Ann & Robert H. Lurie Children's Hospital of Chicago\nPoster Number: 2.405\n\nHighlights include:\n\n\nSingle doses of STK-001 up to 30mg, and three 20mg doses of STK-001 given every four weeks, were found to be well tolerated with no safety concerns related to the study drug.\n\n\nThe most common treatment-emergent adverse events (TEAE) were headache, vomiting, seizure, irritability, and back pain.\n\n\n18.2% (4/22) of patients experienced a TEAE that was related to study drug, none of which were severe.\n...