Stoke Therapeutics Posts Encouraging Interim Safety Data From Early-Stage Dravet Syndrome Trial

Stoke Therapeutics Inc STOK announced interim analysis data from Phase 1/2a MONARCH study of STK-001 in children and adolescents with Dravet syndrome.
The interim analysis is based on data from 21 patients who were treated in the single 10mg (n=5), 20mg (n=4), or 30mg (n=6) dose cohorts.
A dose-proportional increase in study drug exposure was observed in plasma pharmacokinetics.
Cerebrospinal fluid (CSF) exposure was measurable up to six months following a single intrathecal dose.
A dose-proportional increase in CSF concentration was observed from 20mg to 30mg.
Based on data available from 11 patients in the single ascending doses cohorts, 8 out of 11 patients demonstrated a reduction in convulsive seizure frequency.
The most common treatment-emergent adverse events were headache, irritability, vomiting, seizure, and back pain.
Following recent interactions with the FDA related to the partial clinical hold on higher dose levels in the MONARCH study, the FDA will allow Stoke to add a higher dose level (45mg) to the SAD and MAD portions of the MONARCH study.
The Company expects to share clinical data from multiple doses of 30mg in 2H of 2022.
The Company will provide greater detail on data from the MONARCH study at the American Epilepsy Society annual meeting in December.
Related content: Benzinga's Full FDA Calendar.
Price Action: STOK stock is up 6.60% at $25.21 during the market session on the last check Tuesday.