Stoke Therapeutics to Present New Preclinical Data on STK-001 at the American Epilepsy Society Annual Meeting
-- STK-001 is a potential new disease-modifying medicine for the treatment of Dravet syndrome, a severe and progressive form of genetic epilepsy --
Data will be presented from preclinical studies demonstrating the effects of STK-001, a proprietary antisense oligonucleotide (ASO), in the Scn1a-linked Dravet syndrome mouse model and in non-human primates. New results of EEG recordings used to measure the frequency of seizures in Dravet syndrome mice treated with STK-001 compared to placebo will be presented, as well as data on STK-001 biodistribution, target engagement, pharmacodynamics, safety and tolerability in non-human primates.
Dravet syndrome is a severe and progressive form of genetic epilepsy that affects approximately 35,000 people in
“These data support our belief that by restoring the Nav1.1 protein to physiological levels, STK-001 has the potential to provide a gene-specific, disease-modifying therapy for people living with Dravet syndrome,” said
Details on the presentations are as follows:
Presentation Title: Targeted Augmentation of Nuclear Gene Output (TANGO) of SCN1A Prevents SUDEP in a Mouse Model of Dravet Syndrome
Session Date & Time:
Session Title: Poster Session 1
Presenter:
Poster Number: 1.116
Location:
Presentation Title: TANGO Oligonucleotides for the Treatment of Dravet Syndrome: Safety, Biodistribution and Pharmacology in the Non-Human Primate
Session Date & Time:
Session Title: Poster Session 2
Presenter:
Poster Number: 2.195
Location:
The abstracts for these presentations are now available online on the Events and Presentations section of Stoke’s website at https://investor.stoketherapeutics.com/ or through the
About STK-001
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 by leveraging 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
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. The effects of the disease go beyond seizures and often include cognitive regression or developmental stagnation, ataxia, speech impairment and sleep disturbances. 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 in
About
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: 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 TANGO 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 set forth in our filings with the
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Source:
Stoke Media & Investor:
Dawn Kalmar
Vice President, Head of Corporate Affairs
dkalmar@stoketherapeutics.com
781-303-8302