Stoke Therapeutics Announces Plans to Present New Data that Support Zorevunersen as Potentially the First Disease-Modifying Medicine for Dravet Syndrome at the American Epilepsy Society 2024 Annual Meeting
STOK 12.02.2024
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“We look forward to presenting positive new data from patients with Dravet syndrome who were already receiving the best available anti-seizure medicines and then were treated with initial doses of 70mg followed by 45mg maintenance dosing of zorevunersen,” said
New data will include the nine patients who received two or three initial doses of 70mg of zorevunersen in a Phase 1/2 study and then continued treatment in an open-label extension (OLE) study where they received at least two doses of 45mg. In addition, data from patients treated in the OLEs (n=73) were consistent with earlier findings and showed durable reductions in seizures and continuous improvements in multiple subdomains of the Vineland-3 through 24 months. Treatment was generally well tolerated.
Details of the Company’s presentations at AES are as follows:
- Title:Zorevunersen (STK-001) Demonstrates Potential for Disease Modification Including Reductions in Seizures and Improvements in Cognition and Behavior in Children and Adolescents with Dravet Syndrome (DS)Poster Session Date & Time:
Sunday, December 8 at12:00 PM PST Oral Presentation Date & Time:Monday, December 9 at3:15 PM PST Presenter:Linda Laux , M.D., Associate professor of Pediatrics (Neurology and Epilepsy) atNorthwestern University Feinberg School of Medicine and Attending Physician at Ann & Robert H. Lurie Children’s Hospital ofChicago Poster Number:2.379 - Title:Patients with Dravet Syndrome in Open-Label Extension Studies of Zorevunersen (STK-001) Have Durable Reductions in Seizure Frequency and Clinically Meaningful Improvements in Cognition and BehaviorPoster Session Date & Time:
Sunday, December 8 at12:00 PM PST Presenter:Joseph Sullivan , M.D., FAES, Professor of Neurology and Pediatrics and Director of thePediatric Epilepsy Center of Excellence at theUniversity of California San Francisco Poster Number:2.364 - Title:Small Changes on the Vineland-3 are Meaningful to Caregivers of Patients with Dravet SyndromePoster Session Date & Time:
Sunday, December 8 at12:00 PM PST Presenter:Carrie Condon , Director of Clinical Science atStoke Therapeutics Poster Number:3.383 - Title:Spectral EEG Analysis Demonstrates Decreased Slow-wave Activity in Patients with Dravet Syndrome after Treatment with Zorevunersen, an Antisense OligonucleotidePoster Session Date & Time:
Monday, December 9 at12:00 PM PST Presenter:Nigel Colenbier , Senior Data Scientist, Epilog, Clouds ofCare NV Poster Number:3.407
Stoke Therapeutics Corporate Symposium
Title:The Potential of Disease-Modifying Treatments for Dravet Syndrome: Entering a New EraDate and Time:
Stoke Therapeutics Investor and Analyst Virtual Event
Stoke will also host a virtual event led by leading clinicians and patient advocates to discuss the effects of Dravet syndrome, the current treatment landscape, including the latest data from studies of zorevunersen, and the potential real-world impacts of a disease-modifying medicine. The presentation will be conducted virtually and include an opportunity for research analysts to ask questions of the clinician presenters.
Title:Understanding Dravet Syndrome: The Unmet Need and Potential for Disease-ModificationDate and Time:
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 intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system and mood disorders. The disease is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment associated with the disease. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. There are no approved disease-modifying therapies for people living with Dravet syndrome. One out of 16,000 babies are born with Dravet syndrome, which is not concentrated in a particular geographic area or ethnic group.
About Zorevunersen
Zorevunersen is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in ongoing clinical trials. Stoke believes that zorevunersen, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. Zorevunersen is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant (wild-type) copy of theSCN1Agene to restore physiological NaV1.1 levels, thereby reducing both occurrence of seizures and significant non-seizure comorbidities. Zorevunersen has been granted orphan drug designation by the FDA and the EMA, and rare pediatric disease designation by the FDA as a potential new treatment for Dravet syndrome.
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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, the ability of zorevunersen to treat the underlying causes of Dravet syndrome and reduce seizures or show improvements in behavior and cognition at the indicated dosing levels or at all, the timing and expected progress of clinical trials, data readouts, regulatory meetings, regulatory decisions and other presentations, and the participation of scientists associated with Stoke making presentations at AES 2024 and the presentation of data at AES 2024. Statements including words such as “plan,” “potential,” “will,” “continue,” “expect,” or similar words 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 the Company’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 advance, obtain regulatory approval of, and ultimately commercialize its product candidates, including zorevunersen; the timing of data readouts and interim and final results of preclinical and clinical trials; the receipt and timing of potential regulatory decisions; positive results in a clinical trial may not be replicated in subsequent trials or successes in early stage clinical trials may not be predictive of results in later stage trials; the Company’s ability to fund development activities and achieve development goals, including expectations regarding its collaboration with Acadia Pharmaceuticals; the Company’s ability to protect its intellectual property; the direct or indirect impact of global business, political and macroeconomic conditions, including inflation, interest rate volatility, cybersecurity events, uncertainty with respect to the federal budget, instability in the global banking system and volatile market conditions, and global events, including public health crises, and ongoing geopolitical conflicts, such as the conflicts in
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