Stoke Therapeutics Enrolls First Patient in an Observational Study of Children and Adolescents Living with Dravet Syndrome
Dravet syndrome is a severe and progressive genetic epilepsy generally caused by mutations in the SCN1A gene
The BUTTERFLY study is designed to evaluate seizure frequency and non-seizure comorbidities; data will support clinical development plans for the company’s lead therapeutic candidate, STK-001
BUTTERFLY is a two-year observational study that is designed to evaluate seizure frequency and non-seizure comorbidities associated with the disease, including motor and speech impairment, intellectual and developmental disabilities, behavioral deficits and abnormal sleep patterns. Data from the study will support clinical development plans for the company’s lead therapeutic candidate, STK-001, an investigational new treatment for Dravet syndrome.
“Our goal is to develop the first medicine to treat the underlying cause of Dravet syndrome,” said
Approximately 85% of Dravet syndrome cases are caused by spontaneous, heterozygous loss of function mutations in the SCN1A gene. This gene encodes the voltage-gated sodium channel type 1 alpha subunit (NaV1.1). Currently available treatments do not address the underlying cause of Dravet syndrome. Even when taking the current standard-of-care regimen, which includes multiple antiepileptic medicines, more than 90% of patients still report suffering from incomplete seizure control.
“Increased awareness along with more widespread availability of genetic testing have allowed us to diagnose genetic epilepsies like Dravet syndrome earlier, allowing physicians to help patients and their families better manage the disease,” said
About the BUTTERFLY Study
The BUTTERFLY study is an observational study of children and adolescents ages 2 to 18 who have been diagnosed with Dravet syndrome as a result of an SCN1A gene mutation. The study is designed to evaluate seizure frequency and non-seizure comorbidities associated with the disease, including motor and communication impairment, intellectual and developmental disabilities, behavioral deficits and abnormal sleep patterns. No investigational medications or other treatments will be provided. Participants will continue to receive their usual care, and they will be observed by a team of doctors and nurses over time for up to two years. The study is expected to enroll at approximately 20 sites in
Additional Information About Dravet Syndrome
Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures, beginning within the first year of life. Dravet syndrome is difficult to treat and has a poor long-term prognosis. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. Dravet syndrome affects approximately 35,000 people across
STK-001 is an investigational new medicine for the treatment of Dravet syndrome. Stoke believes that STK-001, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. STK-001 is designed to upregulate NaV1.1 protein expression from the non-mutant (wild-type) copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing both occurrence of seizures and significant non-seizure comorbidities. Stoke has generated preclinical data demonstrating proof-of-mechanism for STK-001. STK-001 has been granted orphan drug designation by the
Cautionary Note Regarding Forward-Looking Statements
This press release contains “forward-looking” statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: enrollment in the BUTTERFLY study and the study’s ability to support Stoke’s clinical development plans; Stoke’s expectation about timing and execution of anticipated milestones; Stoke’s ability to use study data to advance the development of STK-001; the ability of STK-001 to treat the underlying causes of Dravet syndrome; and the ability of Stoke to design medicines to increase protein production. Statements including words such as “plan,” “continue,” “expect,” or “ongoing” and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they do not fully materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. Forward-looking statements are subject to risks and uncertainties that may cause Stoke’s actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the company’s ability to develop, obtain regulatory approval for and commercialize STK-001 and its future product candidates, the timing and results of preclinical studies and clinical trials, the company’s ability to protect intellectual property; and other risks and uncertainties described under the heading “Risk Factors” in documents the company files from time to time with the
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