Stoke announces new safety, efficacy data from patients treated with STK-001

Stoke Therapeutics announced new safety and efficacy data from patients treated with STK-001 in the two ongoing Phase 1/2a studies — MONARCH and ADMIRAL — and the SWALLOWTAIL open-label extension, or OLE, study in children and adolescents with Dravet syndrome. These new data suggest clinical benefit for patients 2 to 18 years of age treated with multiple doses of STK-001. The observed reductions in convulsive seizure frequency as well as substantial improvements in cognition and behavior support the potential for disease modification in a highly refractory patient population. The combined efficacy analysis reported was based on clinically evaluable data from 45 patients who were treated with multiple doses in either of these two ongoing studies. The greatest reduction in convulsive seizure frequency has been observed among the small number of patients treated with two or three doses of 70mg in the ADMIRAL study. The analysis of the 70mg multiple dose cohort from ADMIRAL study consists primarily of patients treated with three doses of STK-001. The company anticipates that the remaining ADMIRAL study data will consist primarily of patients treated with two doses of 70mg. The safety analysis for the Phase 1/2a studies reported was based on data from 74 patients who were treated with single or multiple doses of STK-001 and followed for up to six months after their last dose. STK-001 was generally well-tolerated among 74 patients treated with single and multiple doses of 10mg to 70mg in the Phase 1/2a studies and there were no discontinuations related to study drug. One patient who received multiple doses of 70mg STK-001 in the ADMIRAL study experienced Suspected Unexpected Serious Adverse Reactions that were attributed by the investigator to STK-001. The patient went on to complete the study. Subsequently, the study protocol for ADMIRAL was amended to allow investigators to decide whether to administer two or three doses of STK-001 in the ADMIRAL study before patients would be eligible to enroll in the LONGWING OLE. Safety findings from patients who continued treatment in SWALLOWTAIL OLE were consistent with the findings from MONARCH and ADMIRAL with the exception of a greater incidence of CSF protein elevation.