STK-002 - (OSPREY)

October 1, 2026: Stoke Therapeutics (NASDAQ: STOK) said it will present preclinical and Phase 1 OSPREY data for STK-002, an antisense oligonucleotide for autosomal dominant optic atrophy (ADOA), at the AAO 2026 Annual Meeting (October 9-12, New Orleans). Two presentations are scheduled for October 11, 2026.

For STK-002/OSPREY in ADOA: the ongoing first-in-human Phase 1 open-label, dose-escalation study has completed dosing in the first two cohorts; the third cohort is expected to complete dosing by year-end 2026, with a fourth cohort to follow subject to safety. The company said cohorts 3 and 4 are expected to reach therapeutic dosing levels, and initial safety and efficacy data from these latter cohorts are anticipated in H1 2027. All eight planned trial sites are active across the UK, Germany, Denmark, Italy, and Austria.

The AAO presentations are described as providing translational evidence that increasing functional OPA1 protein may have disease-modifying potential in ADOA. STK-002 is designed to increase functional OPA1 mRNA/protein from the healthy copy of the OPA1 gene. The release also notes FDA orphan drug designation for STK-002 in ADOA.