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Stoke Therapeutics Announces MHRA Authorization to Initiate Phase 1/2a Clinical Trial of STK-001 for Dravet Syndrome in the United Kingdom
– New ADMIRAL study complements ongoing U.S. studies and marks first step in global expansion of clinical development of STK-001 – – Study will evaluate
About this update from Stoke Therapeutics, Inc.
[{"type":"text","content":"\n– New ADMIRAL study complements ongoing U.S. studies and marks first step in global expansion of clinical development of STK-001 –\n\n– Study will evaluate safety and PK of multiple doses of STK-001 starting at 30mg; Enrollment and dosing anticipated to begin in 2H 2021 –\n\n– STK-001 has the potential to be the first disease-modifying therapy to address the root cause of Dravet syndrome, a severe and progressive genetic epilepsy –\n\n BEDFORD, Mass.--(BUSINESS WIRE)--\nStoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines, today announced the authorization of its Clinical Trial Application (CTA) by the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2a study (ADMIRAL) of STK-001 for the treatment of Dravet syndrome.\n\nSTK-001, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome, a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life. Dravet syndrome is classified as a developmental and epileptic encephalopathy resulting in developmental delay and cognitive impairment, in addition to seizure activity, that arise from the genetic mutation that causes the disease.\n\n“We are making excellent progress with our ongoing studies of STK-001 in the U.S. The high level of interest from the Dravet community underscores the urgent need for new treatment options that go beyond seizure control,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “The ADMIRAL study complements our U.S.-based MONARCH study, enabling the evaluation of higher dose levels of STK-001 and representing an initial step in the expansion of our global clinical development efforts. Together, we anticipate that these studies will generate a comprehensive set of data that will inform our future development plans. We look forward to working with the U.K. Dravet community – patients, families and healthcare providers – to add to our understanding of the potential for STK-001 to be the first disease-modifying therapy for Dravet syndrome.”\n\nADMIRAL is an open-label, multi...