Stoke Therapeutics announced positive data from Phase 1/2a and open-label extension (OLE) studies of zorevunersen, a drug candidate for Dravet syndrome. The studies showed substantial and durable reductions in convulsive seizure frequency in patients given 70mg followed by 45mg maintenance doses of zorevunersen, alongside existing anti-seizure medications. Furthermore, patients demonstrated continuous improvements in cognitive and behavioral measures over two years of treatment.
Currently, no disease-modifying therapies exist for this severe and progressive genetic epilepsy. Zorevunersen’s potential to address the underlying genetic cause of the disease, rather than just managing symptoms, represents a substantial advancement in the treatment landscape. Positive clinical data strengthens Stoke’s position in developing a first-in-class therapy, potentially offering a significantly improved quality of life for patients and their families.
Patients in the OLE study who received initial 70mg doses of zorevunersen sustained a median seizure reduction of at least 50% from baseline at every observed time point. At eight months, the latest assessed time point for nine patients, the median reduction reached 87%. In addition to seizure reduction, patients also experienced ongoing cognitive and behavioral improvements throughout the two-year treatment period, as measured by the Vineland-3 assessment. These findings support the dosing regimen proposed for the upcoming Phase 3 registrational study. Importantly, zorevunersen has been generally well-tolerated across the studies, with 82% of eligible Phase 1/2a participants continuing into the OLE studies as of June 2024.
The positive data from these studies sets a promising stage for zorevunersen’s development. The observed seizure reduction, combined with the cognitive and behavioral improvements, suggest the potential for a disease-modifying effect. This reinforces the rationale for the upcoming Phase 3 study and strengthens the possibility of zorevunersen becoming a valuable treatment option for Dravet syndrome. These results hold significant implications for the future management of this challenging condition, offering hope for a more effective and comprehensive approach to treatment.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.