Business
Stoke Therapeutics Reports Full Year 2019 Financial Results and Provides Business Updates
– Company receives FDA clearance to begin dosing STK-001 in Part A of Phase 1/2a “Monarch” clinical trial in Dravet syndrome; enrollment and dosing expected
About this update from Stoke Therapeutics, Inc.
[{"type":"text","content":"\n– Company receives FDA clearance to begin dosing STK-001 in Part A of Phase 1/2a “Monarch” clinical trial in Dravet syndrome; enrollment and dosing expected to start in 2H 2020 –\n\n\n– Part B of study, designed to evaluate higher doses of STK-001, on partial clinical hold pending preclinical data to more fully characterize the safety profile of STK-001 at doses higher than the current NOAEL; partial clinical hold not due to any identified manufacturing or safety issue – \n\n\n– Additional toxicology studies now underway; preliminary data from Monarch study still anticipated in 2021 –\n\n\n– As of December 31, 2019, company has approximately $222.7 million in cash, cash equivalents and restricted cash, which is anticipated to fund operations into 2023 –\n\n BEDFORD, Mass.--(BUSINESS WIRE)--\nStoke Therapeutics, Inc., (Nasdaq: STOK), a biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today reported financial results for the full year ended December 31, 2019, and provided business updates.\n\n\n“In 2019 we advanced our understanding of the potential applications of our TANGO technology to a variety of genetic targets, and generated preclinical data that underscore our confidence in STK-001 as a promising potential disease-modifying treatment for Dravet syndrome,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “As we look ahead to 2020, we are poised to become a clinical-stage company with the anticipated start of our first in-human study of STK-001, and a growing portfolio as we work toward identifying our next preclinical product candidate later this year.”\n\n\nToday, the company also provided an update on its lead product candidate, STK-001, for Dravet syndrome. In the first quarter of 2020, Stoke received communication from the U.S. Food and Drug Administration (FDA) confirming that it may proceed with clinical dosing in Part A of its planned Phase 1/2a “Monarch” study of STK-001 in children and adolescents ages 2 to 18 years old with Dravet syndrome. Part A of the study is designed to evaluate two dose cohorts of STK-001.\n\n\nAs part of that communication, the FDA also informed the company that it has placed a partial clinical hold on Part B of the study, which is designed to evaluate higher doses of STK-001...