Stoke Therapeutics (STOK) on Friday released data from Phase 1/2a and open-label extension studies of zorevunersen for the treatment of Dravet syndrome.
The data analyzed nine patients who initially received two or three doses of 70 mg, followed by 45 mg maintenance dosing. Patients experienced substantial and sustained reductions in convulsive seizure frequency when zorevunersen was added to their existing anti-seizure medications.
Participants in the studies also showed continuous improvements in cognitive and behavioral measures over two years of treatment. Zorevunersen was generally well tolerated, reinforcing the company's proposed regimen for the upcoming Phase 3 registrational study, according to the company.
The latest data from nine patients treated with at least two doses of 45 mg in the study revealed a sustained median seizure reduction of at least 50% each month, culminating in an 87% median reduction by the eighth month of assessment.
The drug was generally well-tolerated in the phase 1/2a and OLE studies, with 81 patients treated with zorevunersen at the time of the analysis, and safety findings showed that 30% experienced treatment-related adverse events, mainly CSF protein elevations and procedural vomiting, the company said.
Shares of Stoke Therapeutics were up more than 6% in recent trading.
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