Stoke Therapeutics (STOK)
Pitch Summary: Stoke’s zorevunersen (ASO) aims to treat Dravet syndrome by increasing SCN1A/NaV1.1 expression via its TANGO splicing-modulation platform. Open-label Ph1/2 (MONARCH/ADMIRAL) showed compelling ≥50% median seizure reduction at the 70mg dose, with durability and further improvement in OLE (often 70–80%+ reductions). Non-seizure benefits (Vineland-3 behavior/cognition) trended positive, which—if replicated vs placebo—could justify premium pricing […]
