Stoke Therapeutics Receives FDA Breakthrough Therapy Designation for Zorevunersen for the Treatment of Dravet Syndrome
Stoke Therapeutics has received FDA Breakthrough Therapy Designation for zorevunersen in treating Dravet syndrome with confirmed SCN1A gene mutations. Clinical data from Phase 1/2a and open-label extension studies showed substantial and sustained reductions in seizure frequency, along with improvements in cognition and behavior, while being generally well-tolerated. Over 600 doses have been administered, with some patients on treatment for more than three years.
The company plans to provide updates on its global Phase 3 registrational study by year-end. The Breakthrough Therapy designation grants zorevunersen access to Fast Track features, intensive FDA guidance, and organizational commitment from senior FDA managers.
Stoke Therapeutics ha ricevuto la Designazione di Terapia Innovativa dalla FDA per zorevunersen nel trattamento della sindrome di Dravet con mutate confermate del gene SCN1A. I dati clinici derivanti dagli studi di Fase 1/2a e dalle estensioni in aperto hanno mostrato riduzioni sostanziali e sostenute nella frequenza delle crisi, insieme a miglioramenti nella cognizione e nel comportamento, risultando generalmente ben tollerati. Sono state somministrate oltre 600 dosi, con alcuni pazienti in trattamento da più di tre anni.
L'azienda prevede di fornire aggiornamenti sul suo studio registrativo globale di Fase 3 entro la fine dell'anno. La designazione come Terapia Innovativa consente a zorevunersen di accedere a caratteristiche di Fast Track, a una guida intensiva da parte della FDA e all'impegno organizzativo dei dirigenti senior della FDA.
Stoke Therapeutics ha recibido la Designación de Terapia Innovadora de la FDA para zorevunersen en el tratamiento del síndrome de Dravet con mutaciones confirmadas en el gen SCN1A. Los datos clínicos de los estudios de Fase 1/2a y la extensión a etiqueta abierta mostraron reducciones sustanciales y sostenidas en la frecuencia de las convulsiones, junto con mejoras en la cognición y el comportamiento, siendo generalmente bien tolerados. Se han administrado más de 600 dosis, con algunos pacientes en tratamiento durante más de tres años.
La compañía planea proporcionar actualizaciones sobre su estudio registrativo global de Fase 3 para fin de año. La designación de Terapia Innovadora permite a zorevunersen acceder a características de Fast Track, orientación intensiva de la FDA y compromiso organizativo por parte de gerentes senior de la FDA.
스톡 제약 회사는 드라벳 증후군의 SCN1A 유전자 돌연변이가 확인된 환자를 위한 혁신 치료제 지정을 FDA로부터 받았습니다. 임상 데이터는 1/2a 단계와 오픈 레이블 확장 연구에서 발작 빈도의 상당하고 지속적인 감소와 인지 및 행동 개선을 보여주었으며, 전반적으로 잘 견디는 것으로 나타났습니다. 600회 이상의 투여가 이루어졌으며, 일부 환자는 3년 이상 치료를 받고 있습니다.
회사는 연말까지 글로벌 3상 등록 연구에 대한 업데이트를 제공할 계획입니다. 혁신 치료제 지정을 통해 zorevunersen은 패스트트랙 기능, 집중적인 FDA 지침 및 고위 FDA 관리자의 조직적 지원에 접근할 수 있습니다.
Stoke Therapeutics a reçu la Désignation de Thérapie Innovante de la FDA pour zorevunersen dans le traitement du syndrome de Dravet avec des mutations du gène SCN1A confirmées. Les données cliniques des études de Phase 1/2a et d’extension à étiquetage ouvert ont montré des réductions substantielles et durables de la fréquence des crises, ainsi que des améliorations de la cognition et du comportement, tout en étant généralement bien tolérées. Plus de 600 doses ont été administrées, certains patients recevant un traitement depuis plus de trois ans.
L’entreprise prévoit de fournir des mises à jour sur son étude d'enregistrement mondiale de Phase 3 d'ici la fin de l'année. La désignation de Thérapie Innovante permet à zorevunersen d'accéder à des caractéristiques de voie rapide, à des directives intensives de la FDA et à un engagement organisationnel de la part de responsables seniors de la FDA.
Stoke Therapeutics hat von der FDA die Breakthrough-Therapie-Bezeichnung für zorevunersen zur Behandlung des Dravet-Syndroms mit bestätigten SCN1A-Genenmutationen erhalten. Klinische Daten aus Phase 1/2a und offenen Verlängerungsstudien zeigten erhebliche und nachhaltige Reduzierungen der Anfallsfrequenz, sowie Verbesserungen in Kognition und Verhalten, wobei die Behandlung allgemein gut vertragen wurde. Über 600 Dosen wurden verabreicht, wobei einige Patienten seit mehr als drei Jahren in Behandlung sind.
Das Unternehmen plant, bis zum Jahresende Updates zu seiner globalen Phase-3-Registrierungsstudie bereitzustellen. Die Breakthrough-Therapie-Bezeichnung ermöglicht zorevunersen den Zugang zu Fast-Track-Funktionen, intensiven FDA-Richtlinien und organisatorischem Engagement von leitenden FDA-Managern.
- Received FDA Breakthrough Therapy Designation, potentially expediting development and review
- Clinical data showed substantial reduction in seizure frequency
- Demonstrated improvements in cognition and behavior
- Strong safety profile with over 600 doses administered
- Potential to be first disease-modifying treatment for Dravet syndrome
- Phase 3 study still pending, with details yet to be announced
- Regulatory approval not guaranteed despite Breakthrough designation
Insights
The FDA's Breakthrough Therapy Designation for zorevunersen represents a significant milestone for Stoke Therapeutics. The designation is particularly noteworthy because it's supported by compelling clinical data showing substantial and sustained reductions in seizure frequency and improvements in cognition and behavior in Dravet syndrome patients.
Several key factors make this development impactful:
- Zorevunersen could become the first disease-modifying treatment for Dravet syndrome, addressing the underlying cause rather than just symptoms
- The drug has demonstrated efficacy on top of current standard-of-care treatments
- Safety profile appears favorable with over 600 doses administered, some patients receiving treatment for 3+ years
- The Breakthrough designation will accelerate development and review processes, potentially bringing this treatment to market faster
The upcoming Phase 3 trial announcement by year-end will be important for determining the timeline to potential commercialization. This news significantly strengthens Stoke's position in the rare disease treatment space.
This regulatory milestone substantially derisks Stoke's lead program and could accelerate the path to market. For a company with a market cap of
- Reduces development costs through expedited review processes
- Increases probability of eventual FDA approval
- Strengthens market position in the Dravet syndrome space
- Could attract potential strategic partnerships or investment interest
The upcoming Phase 3 trial announcement will be a key catalyst for the stock. The addressable market for Dravet syndrome treatments is significant, with current treatments generating substantial revenues. As a potential first-in-class disease-modifying therapy, zorevunersen could command premium pricing if approved.
– Supported by evidence from clinical studies that indicate that zorevunersen may demonstrate substantial improvement over available therapies –
– Update on the company’s plans for a global, randomized, controlled Phase 3 registrational study anticipated by year-end –
Clinical data from the Phase 1/2a and open-label extension (OLE) studies of zorevunersen demonstrated substantial and sustained reductions in seizure frequency and continuous improvements in multiple measures of cognition and behavior. These effects were observed on top of the best available anti-seizure medicines, the current standard of care. Zorevunersen was generally well tolerated across the studies. To date, more than 600 doses of zorevunersen have been administered to patients, some of whom have been on treatment for more than three years.
Discussions with the FDA and other global regulatory agencies regarding a global, randomized, controlled Phase 3 registrational study of zorevunersen continue to progress. The Company plans to provide an update on its Phase 3 registrational plans by the end of the year.
“The FDA’s Breakthrough Therapy designation for zorevunersen is supported by promising clinical data that suggest that zorevunersen has the potential to demonstrate substantial improvement over current treatments for Dravet syndrome,” said Shamim Ruff, Chief Regulatory Affairs Officer, Stoke Therapeutics. “By helping the body restore naturally occurring NaV1.1 protein levels, zorevunersen is designed to treat the underlying cause of the disease. We thank the FDA for their support and look forward to continuing to work together closely to efficiently advance zorevunersen into a registrational Phase 3 study.”
“This designation brings new hope to the many patients with Dravet syndrome who continue to experience treatment-resistant seizures and a myriad of health and quality of life complications despite the availability of symptomatic treatments,” said Mary Anne Meskis, Executive Director, Dravet Syndrome Foundation. “Our organization has been engaging with the FDA to ensure greater awareness and understanding of Dravet syndrome. We are encouraged by the Agency’s shared sense of urgency for the development of innovative new medicines that could help address the gaps left by current treatments by treating the underlying cause of the disease.”
Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically-significant endpoint(s). This designation grants zorevunersen access to all Fast Track designation features, intensive guidance on an efficient drug development program and an organizational commitment involving senior FDA managers.
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 (STK-001)
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 the SCN1A gene 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.
About Stoke Therapeutics
Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine. Using Stoke’s proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore protein levels. Stoke’s first compound, zorevunersen, is in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. Dravet syndrome is one of many diseases caused by a haploinsufficiency, in which a loss of ~
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; and the timing and expected progress of clinical trials, data readouts, regulatory meetings, regulatory decisions and other presentations. Statements including words such as “expect,” “plan,” “will,” “continue,” 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 prove incorrect or do not fully materialize, could cause our results to differ materially from those expressed or implied by such forward-looking statements, including, but not limited to, risks and uncertainties related to: the Company’s ability to advance, obtain regulatory approval of, and ultimately commercialize its product candidates, including zorevunersen; fast track or breakthrough designations by the FDA may not lead to faster development or regulatory review or approval process and do not increase the likelihood that zorevunersen will receive marketing approval; 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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Stoke Media & Investor Contacts:
Dawn Kalmar
Chief Communications Officer
dkalmar@stoketherapeutics.com
781-303-8302
Doug Snow
Director, Communications & Investor Relations
IR@stoketherapeutics.com
508-642-6485
Source: Stoke Therapeutics, Inc.
FAQ
What is the significance of FDA Breakthrough Therapy Designation for STOK's zorevunersen?
What clinical results has STOK's zorevunersen shown in Dravet syndrome treatment?
When will STOK announce its Phase 3 trial plans for zorevunersen?
