Published on 27 Mar 2024 on Zacks via Yahoo Finance
Stoke Therapeutics, Inc. STOK reported encouraging new data from two phase I/IIa studies and two open-label extension (OLE) studies evaluating investigational candidate,STK-001, in children and adolescents aged two to 18 years with Dravet syndrome.
STK-001 is Stoke Therapeutics’ novel proprietary antisense oligonucleotide with the potential to become the first disease-modifying therapy to address the genetic cause of Dravet syndrome, rather than just treating the symptoms like currently available treatments.
Per the data readout from the studies, treatment with STK-001 demonstrated clinically meaningful effects, including substantial and durable reductions in convulsive seizure frequency and improvements in multiple measures of cognition and behavior that support the potential for disease modification.