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STOKE THERAPEUTICS Stoke Therapeutics Reports Second Quarter Financial Results and Provides Business Updates August 10 , 2021 - Dosing up to 30mg complete in the single ascending dose ( SAD ) portion in the Phase 1 / 2a MONARCH study of STK - 001 in children and adolescents with Dravet syndrome ; Enrollment and dosing in the multiple ascending dose ( MAD ) portion ongoing - - Third quarter data readout will include 3 - month safety , pharmacokinetic ( PK ) , and cerebrospinal fluid ( CSF ) drug exposure data from patients in the SAD portion of MONARCH , along with modeling of predicted brain exposure - - Company on - track to initiate an additional Phase 1 / 2a study ( ADMIRAL ) of STK - 001 up to 70mg in patients with Dravet syndrome across multiple sites in the United Kingdom - - As of June 30 , 2021 , Company has $ 251.4 million in cash , cash equivalents , marketable securities , and restricted cash , anticipated to fund operations into 2024- BEDFORD , Mass .-- ( BUSINESS WIRE ) -- Aug . 10 , 2021-- Stoke 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 reported financial results for the second quarter of 2021 and provided business updates . " During the first half of 2021 , the Stoke team laid the groundwork for important clinical milestones in the second half of this year , " said Edward M. Kaye , M.D. , Chief Executive Officer of Stoke Therapeutics . " With dosing now complete up to 30mg in the single ascending dose portion of MONARCH , we are on track to report preliminary safety , PK and CSF data from a planned interim analysis of this portion of the study in the third quarter . By comparing these first in - human results with our modeling data , we expect to get insight into the predicted brain exposure and projected therapeutic dose range of STK - 001 in patients with Dravet syndrome . In the coming months , we also expect to treat the first patient with STK - 001 in our ADMIRAL study in the United Kingdom , which together with MONARCH , is anticipated to provide a robust early understanding of STK - 001 to inform future development plans . " Dr. Kaye continued , " We continue to advance our pipeline of TANGO antisense oligonucleotides ( ASO ) and remain on - track to identify a clinical candidate to address the underlying cause of autosomal dominant optic atrophy ( ADOA ) , a severe and progressive genetic disease that causes irreversible vision loss and for which there are no treatment options , by year end . " Second Quarter 2021 Business Highlights and Recent Developments • Dosing is complete in the 10mg , 20mg , and 30mg SAD portion of the Phase 1 / 2a MONARCH study of STK - 001 in children and adolescents with Dravet syndrome . • Enrollment and dosing in the MAD portion of MONARCH is ongoing at the 20mg dose level . Dosing of patients is ongoing in SWALLOWTAIL , an open label extension ( OLE ) study designed to evaluate the long - term safety and tolerability of repeat doses of STK - 001 in patients who participated in the Phase 1 / 2a MONARCH study . • In May 2021 , the Company presented new preclinical efficacy data for TANGO ASO at The Association for Research in Vision and Ophthalmology ( ARVO ) Annual Meeting and The American Society of Gene and Cell Therapy ( ASGCT ) Annual Meeting . The new data , which demonstrated TANGO ASO - mediated OPA1 protein upregulation and improved mitochondrial function in human cells derived from ADOA patients with different OPA1 mutations , supports the Company's work to develop the first potential disease modifying approach for the treatment of ADOA , the most common inherited optic nerve disorder . Upcoming Anticipated Milestones • Enrollment and dosing in the Phase 1 / 2a ADMIRAL study of STK - 001 for the treatment of children and adolescents ages 2 to up to 18 with Dravet syndrome in the United Kingdom is on - track to begin in the second half of 2021 . • As part of a planned interim analysis , the Company expects to report modeling data and 3 - month safety , PK , and CSF drug exposure data from the SAD portion of the Phase 1 / 2a MONARCH study of STK - 001 in the third quarter of 2021 . • In the second half of 2021 , the Company plans to initiate natural history data collection of people living with ADOA to better understand the natural progression of this disease and to support future clinical development of a TANGO ASO for the treatment of ADOA . • Lead optimization on track to identify a clinical candidate for the treatment of ADOA by the end of 2021 . • The Company remains on track to demonstrate in vivo proof of mechanism and safety for a third TANGO ASO program by the end of 2021 . Second Quarter 2021 and Year - to - Date Financial Results