Business
Stoke Therapeutics Reports Full Year 2020 Financial Results and Provides Business Updates
– First patient dosed in the multiple ascending dose portion of Phase 1/2a MONARCH study of STK-001 in Dravet syndrome – – Preliminary safety and PK data
About this update from Stoke Therapeutics, Inc.
[{"type":"text","content":"\n– First patient dosed in the multiple ascending dose portion of Phase 1/2a MONARCH study of STK-001 in Dravet syndrome –\n\n– Preliminary safety and PK data from the single ascending dose portion of MONARCH still expected in H2 2021 –\n\n– As of December 31, 2020, Company had $287.5 million in cash, cash equivalents and restricted cash, anticipated to fund operations into 2024 –\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 reported financial results for the full year ended December 31, 2020 and provided business updates.\n\n“During the last year, the power and potential of RNA medicines became clear to the world. Simultaneously, the Stoke team advanced our efforts to discover and develop RNA-based medicines that aim to address the underlying cause of severe genetic diseases by selectively boosting protein production,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “Our Phase 1/2a MONARCH study in children and adolescents with Dravet syndrome is well underway and patients are now being treated with STK-001 in both the single and multiple ascending dose portions of this study. We also recently initiated an open-label extension study called SWALLOWTAIL that will provide ongoing treatment with STK-001 for patients who complete MONARCH. We expect that SWALLOWTAIL and the MAD portion of MONARCH will provide important information on the potential effects of repeat doses of STK-001.”\n\nDr. Kaye continued, “Looking ahead to 2021, we are on track to report preliminary safety and PK data from the SAD portion of MONARCH in the second half of the year. Our pipeline continues to advance as well. Lead optimization efforts are underway to identify a clinical candidate for OPA1 protein deficiency, the primary cause of autosomal dominant optic atrophy, and our research team is interrogating several targets of interest with the goal of demonstrating in vivo proof of mechanism and safety for another program by year end.”\n\nFourth Quarter 2020 Business Highlights and Recent Developments\n\n\nThe Company announced today continued clinical progress with the Phase 1/2a MONARCH study of children and adolescents with Dravet syndrome. En...