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STOKE THERAPEUTICS Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates November 8 , 2021 - Company nominates STK - 002 as clinical candidate for the treatment of autosomal dominant optic atrophy ( ADOA ) , the most common inherited optic nerve disorder - - Additional data from an interim analysis of the Phase 1 / 2a MONARCH study of STK - 001 including 20mg multiple ascending dose ( MAD ) seizure data submitted for presentation at the American Epilepsy Society ( AES ) 2021 Annual Meeting - - Phase 1 / 2a studies MONARCH in the U.S. and ADMIRAL in the UK are ongoing ; enrollment and dosing continues in the MAD portion ( 30mg ) of both studies - - As of September 30 , 2021 , Company has $ 236.9 million in cash , cash equivalents , marketable securities , and restricted cash , anticipated to fund operations until the end of 2023- BEDFORD , Mass .-- ( BUSINESS WIRE ) -- Nov . 8 , 2021-- 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 reported financial results for the third quarter of 2021 and provided business updates . " The recent positive interim data from the Phase 1 / 2a MONARCH study in patients with Dravet syndrome continue to support our efforts to develop STK - 001 as the first potential new medicine to target the underlying cause of this severe and progressive genetic epilepsy , " said Edward M. Kaye , M.D. , Chief Executive Officer of Stoke Therapeutics . " Today , we are announcing continued pipeline progress with the nomination of STK - 002 as a clinical candidate for the treatment of autosomal dominant optic atrophy , the most common inherited optic nerve disorder . Like Dravet syndrome , ADOA is caused by insufficient protein production and there is a need for a treatment option that addresses the underlying cause of the disease . By leveraging RNA splicing with our TANGO approach , we believe we are uniquely positioned to treat this disease by restoring protein levels to near normal . We look forward to initiating preclinical studies in 2022 to support future clinical trial applications . " Third Quarter 2021 Business Updates • The Company announced positive interim data from the Phase 1 / 2a MONARCH study of STK - 001 in children and adolescents with Dravet syndrome . • The Phase 1 / 2a studies MONARCH and ADMIRAL continue to progress with enrollment and dosing of patients ongoing in the 30mg MAD cohorts . • The FDA will allow the evaluation of an additional higher dose level of STK - 001 ( 45mg ) in the MONARCH study . Recent Developments • The Company has nominated STK - 002 as the clinical candidate for the treatment of ADOA , a severe and progressive optic nerve disorder . There are currently no approved treatments for ADOA , which is primarily caused by loss - of - function mutations in the OPA1 gene , resulting in 50 % OPA1 protein expression and disease manifestation . STK - 002 , a proprietary antisense oligonucleotide ( ASO ) , is designed to upregulate OPA1 protein expression by leveraging the non - mutant ( wild - type ) copy of the OPA1 gene . Based on preclinical data generated to date , the Company believes that STK - 002 has the potential to be a disease - modifying therapy for people living with ADOA . Upcoming Anticipated Milestones • Additional data from an interim analysis of the Phase 1 / 2a MONARCH study of STK - 001 , including 20mg MAD seizure data , were submitted for presentation at the American Epilepsy Society ( AES ) 2021 Annual Meeting , December 3-7 , 2021 in Chicago . • The Company is on track to initiate the collection of natural history data of people living with ADOA by year - end . The data will be used to better understand the natural progression of this disease and to support future clinical development of STK - 002 for the treatment of ADOA . • The Company is on track to demonstrate in vivo proof of mechanism and safety for a third TANGO ASO program by the end of 2021 . • The Company plans to initiate preclinical studies in 2022 to support future clinical trial applications . • The Company expects to share preliminary clinical data on 30mg MAD doses of STK - 001 in the second half of 2022 . Third Quarter 2021 Financial Results • Net loss for the three months ended September 30 , 2021 was $ 22.6 million , or $ 0.61 per share , compared to $ 13.7 million ,