Stoke Therapeutics, Inc. has recently been granted breakthrough therapy designation by the FDA for its investigational medicine, zorevunersen, aimed at treating Dravet syndrome. This designation was based on promising clinical data from the phase 1/2a and open-label extension (OLE) studies, indicating substantial and sustained reductions in seizure frequency and continuous improvements in multiple measures of cognition and behavior. It is noted that the effects were observed on top of the best available anti-seizure medicines, the current standard of care.
The company reported that zorevunersen was generally well tolerated across the studies, with more than 600 doses administered to patients, some of whom have been on treatment for more than three years. Stoke Therapeutics is currently in discussions with the FDA and other global regulatory agencies regarding a global, randomized, controlled phase 3 registrational study of zorevunersen. The company plans to provide an update on its phase 3 registrational plans by the end of the year.
Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged, and refractory seizures, with a poor long-term prognosis and a range of complications that contribute to a poor quality of life for patients and their caregivers. Currently, there are no approved disease-modifying therapies for people living with Dravet syndrome.
Zorevunersen, also known as STK-001, is an investigational new medicine being evaluated in ongoing clinical trials for the treatment of Dravet syndrome. It is a proprietary antisense oligonucleotide (ASO) designed to upregulate Nav1.1 protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological Nav1.1 levels, thereby reducing both the occurrence of seizures and significant non-seizure comorbidities. Zorevunersen has been granted orphan drug designation by the FDA and the EMA, as well as rare pediatric disease designation by the FDA as a potential new treatment for Dravet syndrome.
Stoke Therapeutics is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine. The company's first compound, zorevunersen, is in clinical testing for the treatment of Dravet syndrome, and they are also pursuing the development of STK-002 for the treatment of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder.
As of the press release, Stoke Therapeutics is headquartered in Bedford, Massachusetts, with offices in Cambridge, Massachusetts. As a result of these announcements, the company's shares have moved 5.6% on the market, and are now trading at a price of $13.27. For the full picture, make sure to review Stoke Therapeutics's 8-K report.
