The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted its approval to Stoke Therapeutics’ clinical trial application for commencing the Phase l/ll OSPREY trial of STK-002 to treat autosomal dominant optic atrophy (ADOA).

ADOA is the most common inherited optic nerve disorder and leads to progressive and irreversible vision loss in both eyes beginning in the first decade of life.

Currently, there are no approved treatments available for the disease.

An investigational proprietary antisense oligonucleotide (ASO) developed by Stoke, STK-002 is capable of restoring OPA1 protein expression by upregulating protein production from the non-mutant (wild-type) OPA1 gene copy.

This process is expected to slow or even stop vision loss in ADOA patients.

The OSPREY trial is an open-label study that will include children and adults aged six to 55 years with an established diagnosis of ADOA and have evidence of a genetic mutation in the OPA1 gene.

Its primary objectives are the safety and tolerability of single ascending doses of STK-002, in addition to determining the exposure in serum.

The secondary objective of the trials is to examine the efficacy after intravitreal (IVT) administration of STK-002 in one eye of each patient as calculated by changes in visual function and ocular structure, as well as the quality of life in ADOA patients.

The trial expects to start patient enrolment and dosing by early next year.

Stoke Therapeutics chief medical officer Barry Ticho said: “We are advancing a second TANGO ASO into the clinic, which speaks to the potential of our unique approach to treating the underlying cause of a variety of genetic diseases, particularly of the central nervous system and the eye.

“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.”