Stoke Therapeutics announces 2-year data from the natural history study, FALCON

Stoke Therapeutics announced 2-year data from the FALCON study, a prospective natural history study in people with autosomal dominant optic atrophy (ADOA).

FALCON was a multicenter, 24-month, prospective natural history study of those aged 8 to 60 living with ADOA. According to the company, FALCON aimed to provide “a better understanding of how ADOA disease parameters change over time to inform potential future interventional clinical trials.”

The company describes ADOA as “a rare disease that causes progressive and irreversible vision loss in both eyes starting in the first decade of life,” and 46% of those with ADOA are registered as legally blind.

The study enrolled 47 patients across 10 sites in the US, UK, Italy, and Denmark and underwent assessments at baseline, 6 months, 12 months, 18 months, and 24 months.

Results from the study showed that OPA1-associated ADOA progresses slowly; however, 24% of patients experienced at least a 5-letter loss in low-contrast visual acuity (LCVA). Additionally, higher levels of mitochondrial dysfunction were shown in people with ADOA when compared with healthy individuals. The company also noted that no significant anatomic changes in the retina were observed, which the company says could suggest retinal dysfunction may be reversible with treatment intervention.

Patrick Yu-Wai-Man, MD, PhD, professor of ophthalmology and honorary consultant neuro-ophthalmologist at the University of Cambridge, Moorfields Eye Hospital, and the UCL Institute of Ophthalmology, United Kingdom, and the primary investigator on the Phase 1 OSPREY study, commented on the study in a press release from the company.

“These findings suggest that impaired function in the retina and the optic nerve occurs before permanent cell loss. By increasing the level of naturally occurring OPA1 protein to improve mitochondrial function, it may be possible to stabilize and even restore vision in people with ADOA.”

Additionally, Barry Ticho, MD, PhD, Chief Medical Officer of Stoke Therapeutics, added, “The FALCON study is the largest prospective natural history study to evaluate the effects of ADOA, a rare genetic disease that leads to progressive vision loss and, for many patients, results in blindness,” said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. “These data will provide important context as we initiate our Phase 1 study of STK-002 as the first potential disease-modifying medicine for ADOA.”

According to the company, the data from FALCON will support the clinical development of STK-002, the company’s proprietary antisense oligonucleotide (ASO) currently being evaluated in the phase 1 OSPREY study. STK-002 is designed to upregulate OPA1 protein expression by leveraging the non-mutant (wild-type) copy of the OPA1 gene to restore OPA1 protein expression with the aim of maintaining or improving vision in patients with ADOA, according to the company. STK-002 has been granted orphan drug designation by the FDA as a potential new treatment for ADOA

References:

1. Stoke Therapeutics Presents Two-Year Natural History Data from Patients with Autosomal Dominant Optic Atrophy (ADOA). Published October 20, 2025. Accessed October 20, 2025. https://investor.stoketherapeutics.com/news-releases/news-release-details/stoke-therapeutics-presents-two-year-natural-history-data