Stoke Therapeutics Inc

Stoke Therapeutics, Inc. develops treatments for severe genetic diseases by upregulating protein expression. The company uses its proprietary Targeted Augmentation of Nuclear Gene Output (TANGO) approach to develop antisense oligonucleotides (ASOs) that selectively restore protein levels. Its lead candidates include STK-002, in Phase 1 trials for autosomal dominant optic atrophy, and Zorevunersen (STK-001), an investigational medicine for Dravet syndrome being evaluated in Phase 3 trials. The company also develops programs targeting haploinsufficiency diseases of the central nervous system and eye, and has collaborations with Biogen Inc. and Acadia Pharmaceuticals Inc. It was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Incorporated in 2014, it is headquartered in Bedford, Massachusetts.

Biotech & Genomic Medicine▲2

Stoke Therapeutics completes enrollment in Phase III EMPEROR study for zorevunersen

Stoke Therapeutics has completed enrollment of 162 patients in its Phase III EMPEROR study of zorevunersen for Dravet syndrome, positioning the company for a data readout in the third quarter of 2027. The study has seen zero patient discontinuations to date, compared to the 15% rate assumed in the original statistical powering, and 60 patients have already reached the week 28 primary endpoint milestone. Management plans to initiate a rolling New Drug Application submission in the first quarter of 2027, supported by a pro forma cash position of approximately $420 million, which is expected to fund operations through a potential U.S. launch in early 2028. The company also reported progress in its pipeline, including dose escalation in the Phase I study of STK-002 for autosomal dominant optic atrophy and lead optimization for SYNGAP1-related disorders.

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