Stoke Therapeutics presents two-year data from FALCON study

Stoke Therapeutics (STOK) announced the presentation of two-year data from the FALCON study, a prospective natural history study in people with Autosomal Dominant Optic Atrophy. Results were presented at the 2025 American Academy of Ophthalmology annual meeting and provide important insights into ADOA, a rare, progressive disease for which there are no approved treatments. The data have informed the company’s clinical development program for the proprietary antisense oligonucleotide STK-002, currently being evaluated in the Phase 1 OSPREY study. A summary of findings from the FALCON study includes: while OPA1-associated ADOA progresses slowly, 24% of patients experienced at least a five-letter loss in low-contrast visual acuity. LCVA detects more subtle changes in optic nerve function, often before standard vision tests show a difference, making it a sensitive measure of disease progression. Higher levels of mitochondrial dysfunction were shown in people with ADOA compared with healthy individuals. Mitochondrial function is crucial for vision because mitochondria produce most of the energy required by the cells that make up the optic nerve. No significant anatomic changes in the retina were observed, suggesting that retinal dysfunction may be reversible with treatment intervention.