Stoke Therapeutics Receives Authorization to Initiate a Phase 1/2 Study of STK-002 for ADOA in the UK

Stoke Therapeutics, Inc. received authorization to initiate a UK-based Phase 1/2 study of STK-002, a treatment of ADOA, for which there is currently no approved treatments. (Image Credit: Adobe Stock/adriano_cz)

Stoke Therapeutics, Inc. announced authorization of its Clinical Trial Application by the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 study (OSPREY) of STK-002, a treatment of autosomal dominant optic atrophy (ADOA). An estimated 65% to 90% of ADOA cases are caused by mutations in the OPA1 gene, most of which lead to a haploinsufficiency, resulting in 50% OPA1 protein expression and disease manifestation. There are currently no approved treatments for ADOA.

STK-002 is a proprietary antisense oligonucleotide (ASO) being developed as the first potential disease-modifying therapy to address the genetic cause of ADOA. It is designed to restore OPA1 protein expression by upregulating protein production from the non-mutant (wild-type) copy of the OPA1 gene. By doing this, the company hopes to slow or even stop vision loss in patients with ADOA.

“We are advancing a second TANGO ASO into the clinic which speaks to the potential of our unique approach to treat the underlying cause of a variety of genetic diseases, particularly of the central nervous system and the eye,” said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. “As we look to initiate the clinical studies of STK-002, our ongoing natural history study called FALCON is progressing well and will provide important information about the progression of this disease, which often leads to legal blindness. We look forward to working with the ADOA community as we prepare to start the first clinical study of STK-002 in early 2024.”

The OSPREY study is a Phase 1/2 open-label study of children and adults ages 6 to 55 who have an established diagnosis of ADOA and have evidence of a genetic mutation in the OPA1 gene. 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 serum. A secondary objective is to assess efficacy following intravitreal (IVT) administration of STK-002 in one eye of each patient as measured by changes in visual function and ocular structure as well as quality of life in patients with ADOA. Enrollment and dosing are anticipated to begin in early 2024.