Stoke Therapeutics has recently announced that the global Phase III EMPEROR study for its potential disease-modifying therapy, zorevunersen, targeting Dravet syndrome, has completed enrollment of all 162 patients. This achievement of a key milestone represents a significant step toward submitting a New Drug Application and potentially benefiting patients as soon as possible.

Previously, the company had anticipated completing enrollment of approximately 150 patients in the second quarter of 2026. The announced enrollment of 162 patients has surpassed this expectation. The enrolled patients, from multiple countries and regions including the United States, United Kingdom, and Japan, will be randomly assigned to receive either zorevunersen or a placebo.

Zorevunersen is a first-in-class antisense oligonucleotide drug designed to treat Dravet syndrome at its root by increasing the expression of functional NaV1.1 protein from the non-mutant SCN1A gene copies in brain cells. The drug has received Breakthrough Therapy designation and Orphan Drug status from the U.S. Food and Drug Administration.

The company aims to report topline data from the EMPEROR study in mid-2027 and plans to initiate a rolling New Drug Application submission in the first half of 2027, with the goal of potential market approval by the end of 2027 or early 2028. The company currently holds approximately $411 million in cash, which is sufficient to support the drug's development through to its potential commercialization.