– Data from the positive interim analysis of the Phase 1/2a MONARCH study of STK-001 in children and adolescents with Dravet syndrome will be presented –
BEDFORD, Mass.--(BUSINESS WIRE)-- Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced that five abstracts related to the Company’s work in Dravet syndrome will be presented at the American Epilepsy Society (AES) 2021 Annual Meeting, taking place December 3 – 7, 2021 in Chicago.
“At Stoke, our goal is to develop the first medicine to target the underlying cause of Dravet syndrome, a severe and progressive genetic epilepsy,” said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. “In addition to our progress in the clinic with STK-001, we continue to build a strong foundational understanding of the non-seizure aspects of the disease with data from our BUTTERFLY Observational study to help evaluate the potential of disease-modifying medicines. We look forward to sharing updates on our progress at this year’s AES annual meeting.”
Details for the Company’s presentations at AES are as follows:
- Title: Interim Safety, PK, and CSF Exposure Data from the Phase 1/2a MONARCH Study of STK-001, an Antisense Oligonucleotide (ASO), in Children and Adolescents with Dravet Syndrome (DS) Session Date & Time: Sunday, December 5 at 12:00 p.m. CT Presenter: Linda Laux, M.D., Associate Professor of Pediatrics (Neurology and Epilepsy) at Northwestern University Feinberg School of Medicine and Attending Physician at Ann & Robert H. Lurie Children's Hospital of Chicago Poster Number: 2.405
- Title: SWALLOWTAIL: An Open-Label Extension (OLE) Study for Patients with Dravet Syndrome (DS) who Previously Participated in Studies of STK-001 Session Date & Time: Sunday, December 5 at 12:00 p.m. CT Presenter: Colin Roberts, M.D., Director of the Doernbecher Childhood Epilepsy Program at Oregon Health & Science University Poster Number: 2.220
- Title: ADMIRAL: A UK Open-Label Study to Investigate the Safety and Pharmacokinetics (PK) of Multiple Ascending Doses of Antisense Oligonucleotide (ASO) STK-001 in Children and Adolescents with Dravet Syndrome Session Date & Time: Sunday, December 5 at 12:00 p.m. CT Presenter: Helen Cross, MB ChB, Ph.D., Professor, The Prince of Wales’s Chair of Childhood Epilepsy and Head of the Developmental Neuroscience Programme at University College London Great Ormond Street Institute of Child Health, Honorary Consultant in Paediatric Neurology, President of the International League Against Epilepsy Poster Number: 2.219
- Title: BUTTERFLY, An Observational Study to Investigate Cognition and Other Non-seizure Comorbidities in Children and Adolescents with Dravet Syndrome Session Date & Time: Monday, December 6 at 12:00 p.m. CT Presenter: Elaine Wirrell, M.D., Director of Pediatric Epilepsy at Mayo Clinic, Director of the Child and Adolescent Neurology Residency Training Program at Mayo Clinic Poster Number: 3.278
- Title: A Pharmacokinetic (PK) Model for STK-001, an Antisense Oligonucleotide (ASO), Based on Data from Non-human Primates (NHP) Enables Dose Selection in Patients with Dravet Syndrome (DS) Session Date & Time: Monday, December 6 at 12:00 p.m. CT Presenter: Meena, Ph.D., Vice President of Bioanalytical, DMPK and Biomarker Development at Stoke Therapeutics Poster Number: 3.264
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 STK-001
STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in ongoing clinical trials. 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 and proof-of-concept for STK-001. STK-001 has been granted orphan drug designation by the FDA as a potential new treatment for Dravet syndrome.
About Phase 1/2a MONARCH Study (United States)
The MONARCH study is a Phase 1/2a open-label study of children and adolescents ages 2 to 18 who have an established diagnosis of Dravet syndrome and have evidence of a pathogenic genetic mutation in the SCN1A gene. The primary objectives for the study are to assess the safety and tolerability of STK-001, as well as to determine the pharmacokinetics in plasma and exposure in cerebrospinal fluid. A secondary objective is to assess the efficacy as an adjunctive antiepileptic treatment with respect to the percentage change from baseline in convulsive seizure frequency over a 12-week treatment period. Stoke also intends to measure non-seizure aspects of the disease, such as quality of life, as secondary endpoints. Stoke plans to enroll approximately 90 patients in the study across 20 sites in the United States. Additional information about the MONARCH study can be found at https://www.monarchstudy.com/.
Patients who participated in the MONARCH study are eligible to continue treatment in SWALLOWTAIL, an open label extension (OLE) study designed to evaluate the long-term safety and tolerability of repeat doses of STK-001. Enrollment and dosing in SWALLOWTAIL are underway.
About Phase 1/2a ADMIRAL Study (United Kingdom)
The ADMIRAL study is a Phase 1/2a open-label study of children and adolescents ages 2 to