Stoke Therapeutics to Present Zorevunersen Data at 15th European Epilepsy Congress
Stoke Therapeutics, Inc., a biotechnology firm listed on Nasdaq (STOK), is committed to restoring protein expression through RNA medicine. The company recently announced it would present findings from its Dravet syndrome clinical program at the 15th European Epilepsy Congress (EEC) in Rome, Italy, from September 7 to 11.
Stoke Therapeutics is pioneering zorevunersen (STK-001), an antisense oligonucleotide (ASO) designed to target the genetic roots of Dravet syndrome. The upcoming session will feature a platform presentation and two poster presentations, displaying an extensive dataset from various Dravet syndrome studies conducted by the company.
Dr. Barry Ticho, Chief Medical Officer of Stoke Therapeutics, emphasized that their research strongly indicates current treatments fail to meet the needs of Dravet syndrome patients. He expressed optimism that zorevunersen could potentially revolutionize the treatment of this condition by addressing its root causes rather than just alleviating its symptoms. The company aims to collaborate with global Dravet syndrome experts to prepare for a Phase 3 registrational study of zorevunersen.
Key presentations at the EEC include:
1. MONARCH and ADMIRAL Studies:
- Title: "MONARCH and ADMIRAL: Open-label, Phase 1/2a studies in USA and UK investigating safety, drug exposure, and clinical effect of zorevunersen in children and adolescents with Dravet syndrome"
- Session: Platform Session – Paediatric Epileptology
- Date & Time: September 10, 12:22 – 12:30 PM CEST
- Presenter: Helen Cross from University College London
2. BUTTERFLY Study:
- Title: "24-month analysis of BUTTERFLY: A prospective, observational study to investigate seizures and comorbidities in children and adolescents with Dravet syndrome"
- Event Type: In-Person Posters
- Date & Time: September 8, 2:30 PM - 2:33 PM CEST
- Presenter: Joseph Sullivan from the University of California San Francisco
- Poster Number: P788
3. SWALLOWTAIL and LONGWING Studies:
- Title: "SWALLOWTAIL and LONGWING: Open-Label Extension (OLE) studies for children and adolescents with Dravet syndrome who previously participated in a study of Antisense Oligonucleotide zorevunersen"
- Event Type: In-Person Posters
- Date & Time: September 8 - 10; Q&A from 1:30 - 3:00 PM CEST each day
- Presenter: Andreas Brunklaus from the Royal Hospital for Children, Glasgow
- Poster Number: P875
Dravet syndrome is a severe, genetic epilepsy marked by frequent, prolonged seizures beginning in the first year of life. The condition is notoriously difficult to treat and is associated with various complications, including cognitive impairment, developmental delays, and an increased risk of sudden unexpected death in epilepsy (SUDEP). It affects about one in 16,000 infants globally.
Zorevunersen, currently under clinical evaluation, seeks to be the first disease-modifying treatment for Dravet syndrome. It operates by upregulating NaV1.1 protein expression, leveraging the non-mutant copy of the SCN1A gene to restore proper physiological levels, thereby potentially reducing seizures and other comorbidities. The drug has received orphan drug designation from both the FDA and EMA, along with rare pediatric disease designation from the FDA.
Stoke Therapeutics has conducted several studies to evaluate zorevunersen. In the U.S., the MONARCH Phase 1/2a study focused on children and adolescents with Dravet syndrome, aiming to assess the drug's safety, tolerability, and pharmacokinetics. Following MONARCH, eligible patients could continue in the SWALLOWTAIL open-label extension (OLE) study for long-term evaluation. Similarly, the UK-based ADMIRAL Phase 1/2a study, followed by the LONGWING OLE study, examined the drug's safety, tolerability, and long-term effects.
Additionally, the BUTTERFLY study was a two-year observational study that tracked neurodevelopmental status and seizure activity in children and adolescents with Dravet syndrome. Despite ongoing treatment with the best available anti-seizure medications, participants showed no meaningful improvement in seizure frequency and exhibited widening cognitive and behavioral gaps compared to their neurotypical peers.
Stoke Therapeutics aims to leverage its TANGO (Targeted Augmentation of Nuclear Gene Output) approach to develop ASOs that restore protein levels selectively. Their pipeline includes STK-002, targeting autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. The company’s research indicates broad potential applications for its proprietary approach across various organs, tissues, and systems. Stoke Therapeutics is headquartered in Bedford, Massachusetts, with additional offices in Cambridge, Massachusetts.
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