Stoke Therapeutics Enrolls First Patient in a Natural History Study of People Living with Autosomal Dominant Optic Atrophy (ADOA)

Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced enrollment of the first patient in a prospective natural history study of people ages 8 to 60 who are living with autosomal dominant optic atrophy (ADOA). ADOA is the most common inherited optic nerve disorder. It is a rare disease that causes progressive and irreversible vision loss in both eyes starting in the first decade of life. There are currently no approved treatments for ADOA.

Most cases of ADOA are caused by mutations in one allele of the OPA1 gene, which result in half of the normal OPA1 protein production. FALCON is a two-year prospective natural history study in patients who have a confirmed diagnosis of ADOA that is caused by an OPA1 mutation. The study is designed to evaluate the rate of change in structural and functional ophthalmic assessments. Data from the FALCON study will support the clinical development of STK-002, Stoke’s proprietary antisense oligonucleotide (ASO) in preclinical development for the treatment of ADOA.

“ADOA is a severe and progressive disease that, for many patients, leads to legal blindness,” said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. “There are currently no available treatments for ADOA. We look forward to partnering with the ADOA community and clinicians to learn more about this disease as we work to develop the first potential medicine to treat the underlying cause of ADOA.”

“Understanding what causes ADOA is helping diagnose the disease earlier and is, for the first time, giving us the opportunity to develop medicines that may be able to slow or even stop vision loss in these patients,” said Julie Falardeau, M.D., Professor of Ophthalmology and Neurology at Oregon Health & Science University School of Medicine. “We look forward to participating in the FALCON study and to generating data that will provide new insights into how the disease affects patients, which will be important as we study potential new treatments.”

About the FALCON Study
FALCON is a multicenter, prospective natural history study of people ages 8 to 60 who have an established clinical diagnosis of ADOA that is caused by a heterozygous OPA1 gene variant. No investigational medications or other treatments will be provided. The study is expected to enroll approximately 45 patients across 10 sites in the U.S., U.K., Italy and Denmark. Patients will undergo assessments at baseline, 6 months, 12 months, 18 months, and 24 months. There will be no additional follow-up period. For more information about enrolling in the study, please email [e-mail].