Stoke Therapeutics, Inc. announced the presentation of 2-year data from the FALCON study, a prospective natural history study in people with autosomal dominant optic atrophy (ADOA) (n=47). Results were presented at the annual meeting of the American Academy of Ophthalmology (AAO) and provide 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 (ASO) STK-002, currently being evaluated in the phase 1 OSPREY study, the company said in a press release.

While OPA1-associated ADOA progresses slowly, 24% of patients experienced at least a 5-letter loss in low-contrast visual acuity (LCVA). Higher levels of mitochondrial dysfunction were shown in people with ADOA compared with healthy individuals. No significant anatomic changes in the retina were observed, suggesting that retinal dysfunction may be reversible with treatment intervention, the company said.

FALCON was a multicenter, 24-month, prospective natural history study of people ages 8 to 60 who are living with ADOA. FALCON aimed to provide a better understanding of how ADOA disease parameters change over time to inform potential future interventional clinical trials and was designed to evaluate the rate of change in structural and functional ophthalmic assessments. No investigational medications or other treatments were provided.

The study enrolled 47 patients across 10 sites in the United States, United Kingdom, Italy and Denmark. All participants had a confirmed diagnosis of ADOA caused by an OPA1 variant. Patients underwent assessments at baseline, 6 months, 12 months, 18 months and 24 months. Data from the FALCON study support the clinical development of STK-002, Stoke’s proprietary antisense oligonucleotide (ASO) currently being evaluated in the phase 1 OSPREY study, the company said.