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Stoke Therapeutics announces highlights from STK-001 presentation
The Fly

Stoke Therapeutics announces highlights from STK-001 presentation

Stoke Therapeutics announced highlights from presentations related to the ongoing clinical development of STK-001, the first potential new medicine to treat the underlying cause of Dravet syndrome. A total of seven posters will be presented at the American Epilepsy Society, or AES, 2022 Annual Meeting, December 2-6. Dravet syndrome 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. Topline data from a combined interim analysis of the Phase 1/2a MONARCH and ADMIRAL studies showed single and multiple doses of STK-001 up to 45mg were well-tolerated. Reductions in seizure frequency were observed among patients who received multiple doses of STK-001. The effects were more evident at the highest dose and in younger patients and were observed on top of standard treatments. Half of the patients in these studies were taking four or more anti-seizure medicines, including fenfluramine. An interim analysis of six patients treated with three doses of 45mg was presented and showed: A 55% median reduction from baseline in convulsive seizure frequency from Day 29 after the first dose to three months after receiving the last dose. Reductions from baseline in convulsive seizure frequency in 5/6 patients. A greater than 50% reduction in convulsive seizure frequency in 4/6 patients. Reductions in seizure frequency began after the first dose and continued with additional treatment, consistent with the anticipated mechanism of action of STK-001. The interim safety analysis of patients treated in MONARCH and ADMIRAL showed that 27% of patients experienced a treatment-emergent adverse event, or TEAE, that was related to study drug. All adverse events related to study drug were mild to moderate in severity. No TEAEs led to study drug withdrawal. Preliminary data from a small cohort of patients in the open-label SWALLOWTAIL study showed the effects of ongoing treatment with STK-001 (30mg), including: Reductions in convulsive seizure frequency that were observed in MONARCH were maintained with ongoing treatment in the SWALLOWTAIL open-label extension study. A trend toward improvement in non-seizure comorbidities as measured by the BRIEF-P, an assessment of executive function, was observed among patients in the SWALLOWTAIL open-label extension study. One-year data from the BUTTERFLY observational study, which did not include treatment with STK-001, showed little change from baseline to 12 months in the mean BRIEF-P scores and other commonly used cognition assessments. Safety profile consistent with MONARCH and ADMIRAL.

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