Stoke Therapeutics, Inc. (STOK) Stock Analysis

Stoke Therapeutics dosed the first patient in the global Phase 3 EMPEROR study for zorevunersen (STK-001) in August 2025, targeting pivotal data in mid-2027 to support a rolling NDA submission. Dravet syndrome affects an estimated 38,000 patients across the US, UK, France, Germany, Italy, Spain, and Japan, and no approved disease-modifying therapy currently exists. A second program, STK-002 for autosomal dominant optic atrophy (ADOA), dosed its first patient in the Phase 1 OSPREY study in February 2026. Stoke currently generates no product revenue.

Stoke's revenue model depends entirely on eventual commercialization of zorevunersen or future product candidates. The company has invested substantially all its financial resources in its TANGO (Targeted Augmentation of Nuclear Gene Output) platform, which designs antisense oligonucleotides to selectively restore protein levels in haploinsufficiency diseases. Zorevunersen targets Dravet syndrome by upregulating Nav1.1 protein expression via the non-mutant copy of the SCN1A gene — an approach supported by Breakthrough Therapy Designation granted by the FDA in December 2024. The EMPEROR study enrolls approximately 150 patients aged 2 to under 18 at roughly 60 sites across the UK, US, EU, and Japan in a randomized, double-blind, sham-controlled design. Stoke retains North American commercialization rights while Biogen holds global rights outside the US, Canada, and Mexico. Manufacturing of drug substance relies on third-party contract manufacturing organizations, and the 10-K identifies the adequacy of CMO facilities as a condition of regulatory approval.