Stoke Therapeutics Commences Initial-Stage Optic Nerve Disorder Study

Stoke Therapeutics STOK has enrolled first patient in a prospective natural history study of people ages 8 to 60 who are living with autosomal dominant optic atrophy (ADOA).

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 and expected to enroll approximately 45 patients across 10 sites in the U.S and globally.

The data readout from the FALCON study will support the clinical development of STK-002, proprietary antisense oligonucleotide (ASO) in preclinical development for the treatment of ADOA.

Barry Ticho, Chief Medical Officer, stated, "ADOA is a severe and progressive disease that, for many patients, leads to legal blindness. 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."

Autosomal dominant optic atrophy (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.

Price Action : Stoke shares closed Wednesday’s trading higher 2 percent at $17.19.