Stoke Therapeutics, Inc announced in a press release that the first patient has been dosed in the phase 1 OSPREY study of STK-002, an investigational medicine for the treatment of autosomal dominant optic atrophy (ADOA). ADOA is a rare genetic disease that causes progressive and irreversible vision loss in both eyes starting in the first decade of life.

The OSPREY study is a phase 1, dose-escalating open-label study of children and adults ages 6 to 55 who have an established diagnosis of ADOA and have a confirmed disease-causing variant in the OPA1 gene. According to the company, the primary objectives for the study are to assess the safety and tolerability of single ascending doses of STK-002, as well as to determine the exposure in blood. Secondary objectives are to assess changes in visual function, ocular structure and quality of life after single doses of STK-002.

The OSPREY study follows a standard dose-escalation design with participants enrolled into sequential cohorts receiving increasing dose levels of STK-002. Dose escalation of the first 4 cohorts will continue through 2026 and early 2027, pending safety and tolerability assessments. Data from the OSPREY study will help to inform potential future development of STK-002, the company said.

“Data from our natural history study suggest that for some people affected by ADOA, the disease progresses more rapidly than previously thought," said the lead principal investigator of the study Patrick Yu-Wai-Man, MD, PhD, professor of ophthalmology at the University of Cambridge and honorary consultant neuro-ophthalmologist at Addenbrooke’s Hospital, Moorfields Eye Hospital, and the UCL Institute of Ophthalmology, United Kingdom. "Based on a growing understanding of the disease biology, we believe that increasing naturally occurring OPA1 protein may help restore vision in people with ADOA. There are currently no medicines available for people living with ADOA, and there is a lot of interest in this study from the ADOA community given the potential for STK-002 to restore vision by addressing the root cause of the disease.”