Alignment achieved with U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA) and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for EMPEROR One-year study of zorevunersen will evaluate reductions in major motor seizure frequency as well as improvements in behavior and cognition in children and adolescents ages 2 to <18 years old FDA Breakthrough Therapy designation positions zorevunersen on... Read More

BEDFORD, Mass.--( BUSINESS WIRE )-- Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine, today announced that its management team will host a webcast and conference call for investors and analysts to discuss successful alignment with global regulatory agencies related to a Phase 3 study of zorevunersen as potentially... Read More

Substantial and durable reductions in convulsive seizure frequency observed in patients treated with 2 or 3 doses of 70mg followed by 45mg maintenance dosing on top of the best available anti-seizure medicines Patients experienced continuous improvements in multiple measures of cognition and behavior with ongoing treatment... Read More

Supported by evidence from clinical studies that indicate that zorevunersen may demonstrate substantial improvement over available therapies Update on the company’s plans for a global, randomized, controlled Phase 3 registrational study anticipated by year-end BEDFORD, Mass. / Dec 04, 2024 / Business Wire / Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by... Read More

Company to host a virtual educational event for investors and research analysts on Monday, December 9 at 8:30 am Eastern (5:30 am Pacific) BEDFORD, Mass. / Dec 02, 2024 / Business Wire / Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine, today announced several presentations at the American Epilepsy Society (AES)... Read More

Company plans to provide seizure as well as cognition and behavior data from all patients treated with initial 70mg doses followed by 45mg maintenance dosing in studies of zorevunersen by year-end Discussions with FDA and global regulatory agencies related to a single global Phase 3 study of zorevunersen continue to progress; Company to provide an update by year-end As of September 30, 2024, Company had $269.2 million in... Read More

New data showed improvements in cognition and behavior during the first year of treatment with additional increases demonstrated as treatment continued Clinical effects observed across the Phase 1/2a and open-label extension studies (OLEs) of zorevunersen are a first in the treatment of Dravet syndrome and support plans for the Company’s Phase 3 registrational study Zorevunersen generally well-tolerated across the studies... Read More

Mr. Olson brings two decades of successful corporate business development experience with an emphasis on rare diseases and RNA therapeutics BEDFORD, Mass. / Sep 04, 2024 / Business Wire / Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine, today announced the appointment of Eric Olson as Chief Business Officer. Mr.... Read More

BEDFORD, Mass. / Sep 03, 2024 / Business Wire / Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine, today announced that management will present at the following upcoming investor conferences: H.C. Wainwright 26th Annual Global Investment Conference Date: Tuesday, September 10, 2024 Time: 12:00 p.m. ET 2024 Cantor... Read More

First presentation of landmark data from Phase 1/2a and OLE studies of zorevunersen that showed substantial reductions in seizures and continued improvements in multiple measures of cognition and behavior BEDFORD, Mass. / Sep 03, 2024 / Business Wire / Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine, today... Read More

BEDFORD, Mass. / Aug 16, 2024 / Business Wire / Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine, today announced that, effective on August 15, 2024, the company granted stock options to purchase an aggregate of 48,400 shares of common stock to three new employees, as a material inducement to their employment in... Read More

FDA removes Partial Clinical Hold as company advances toward a Phase 3 registrational study of zorevunersen (STK-001) in children and adolescents with Dravet syndrome Company to provide an update on Phase 3 registrational plans for zorevunersen in the second half of 2024 Company on track to initiate the Phase 1 study (OSPREY) of STK-002 for the treatment of Autosomal Dominant Optic Atrophy (ADOA) this year As of June 30,... Read More

BEDFORD, Mass. / Jul 16, 2024 / Business Wire / Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine, today announced that, effective on July 15, 2024, the company granted stock options to purchase an aggregate of 122,900 shares of common stock to three new employees, as a material inducement to their employment in... Read More

BEDFORD, Mass. / Jun 18, 2024 / Business Wire / 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 announced that, effective on June 17, 2024, the company granted stock options to purchase an aggregate of 10,400 shares of common stock to one new employee, as a material inducement to... Read More

BEDFORD, Mass. / Jun 03, 2024 / Business Wire / 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 announced that management will present at the following upcoming investor conferences: Goldman Sachs 45th Annual Global Healthcare Conference Date: Monday, June 10, 2024 Time: 4:00... Read More

BEDFORD, Mass. / May 17, 2024 / Business Wire / 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 announced that, effective on May 15, 2024, the company granted stock options of common stock to Thomas Leggett, the Company’s newly-appointed Chief Financial Officer, and four other... Read More

Recent data demonstrating reductions in seizure frequency and improvements in cognition and behavior support the potential for STK-001 as a disease-modifying potential medicine for the treatment of Dravet syndrome Company plans to meet with regulatory agencies to discuss registrational study design for STK-001; Update anticipated in the second half of 2024 As of March 31, 2024, Company had $178.6 million in cash and cash... Read More

Mr. Leggett brings more than two decades of experience as an investment banker and corporate executive leading financial strategy and operations for life science companies BEDFORD, Mass. / Apr 29, 2024 / Business Wire / 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 announced... Read More

Mr. Hoitt brings more than two decades of successful commercial experience with an emphasis on rare diseases BEDFORD, Mass. / Apr 17, 2024 / Business Wire / 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 announced the appointment of Jason Hoitt as Chief Commercial Officer. Mr.... Read More

BEDFORD, Mass. / Apr 04, 2024 / Business Wire / 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 announced that management will present at the 23rd Annual Needham Virtual Healthcare Conference on Wednesday, April 10, 2024, at 10:15 a.m. ET. A live webcast of the presentation,... Read More

BEDFORD, Mass. / Mar 27, 2024 / Business Wire / 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 announced the pricing of its upsized underwritten public offering of 5,555,557 shares of its common stock at a price to the public of $13.50 per share and, in lieu of common stock to... Read More

BEDFORD, Mass. / Mar 26, 2024 / Business Wire / 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 announced a proposed underwritten public offering in which it intends to offer and sell, subject to market and other conditions, up to $75 million of shares of its common stock. In... Read More

As of December 31, 2023, Company had $201.4 million in cash, cash equivalents, and marketable securities, anticipated to fund operations to the end of 2025 BEDFORD, Mass. / Mar 25, 2024 / Business Wire / 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... Read More

Phase 1/2a End of Study Data: 70mg doses demonstrated substantial and sustained reductions in convulsive seizure frequency on top of the best available anti-seizure medicines; Median reductions of 85% (n=10) at 3 months and 74% (n=9) at 6 months after last dose Open Label Extension Studies: Durable reductions in seizures and clinically meaningful improvements in multiple measures of cognition and behavior were maintained... Read More

BEDFORD, Mass. / Jan 17, 2024 / Business Wire / 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 announced that, effective on January 16, 2024, the company granted stock options to purchase an aggregate of 13,300 shares of common stock to 2 new employees, as a material inducement... Read More

Data anticipated in Q1 2024 from studies of STK-001 for the treatment of Dravet syndrome End of Phase 1/2a study results and OLE data from patients receiving ongoing treatment; Analyses to include effects of STK-001 on seizure frequency, cognition and behavior Pending Q1 data, Company to request Phase 3 planning meetings with regulators As of September 30, 2023, Company had $214.7 million in cash, cash equivalents and... Read More

BEDFORD, Mass. / Jan 04, 2024 / Business Wire / 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 announced that management will present at the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 10, 2024, at 6:45 p.m. ET (3:45 p.m. PT). A live audio webcast of the... Read More

Data from clinical studies of STK-001 demonstrated clinical benefit for patients ages 2 to 18 years old, including reductions in seizures and improvements in cognition and behavior that support the potential for disease modification Analysis of 72 patients treated in STK-001 clinical trials suggests that higher STK-001 drug exposure in brain leads to greater seizure reductions Two-year data from the longest prospective... Read More

New data include 24-month results from the largest and longest prospective natural history study of children and adolescents with Dravet syndrome, which showed a lack of improvement despite treatment with available medicines Additional presentations include data that support advancement of STK-001 as potentially the first disease-modifying new medicine for Dravet syndrome, including data showing substantial reductions in... Read More

STK-001: Q1 2024 readout expected to include end of Phase 1/2a study results and longer-term data on the effects of repeat dosing on seizure frequency, cognition, and behavior from the ongoing open-label extension (OLE) studies STK-001: Company then plans to meet with regulators to discuss a Phase 3 study design; Update expected in 1H 2024 As of September 30, 2023, Company had $214.7 million in cash, cash equivalents and... Read More

BEDFORD, Mass. / Oct 17, 2023 / Business Wire / 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 announced that, effective on October 16th, 2023, the company granted a stock option to purchase an aggregate of 32,800 shares of common stock to 1 new employee, as a material... Read More

BEDFORD, Mass. / Sep 21, 2023 / Business Wire / 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 announced that management will participate in a panel presentation at the 2023 Cantor Global Healthcare Conference on Tuesday, September 26, 2023, at 9:55 a.m. ET. A live webcast of... Read More

Mr. Smith brings significant experience in the successful development and commercialization of medicines for rare genetic diseases BEDFORD, Mass. / Sep 20, 2023 / Business Wire / 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 announced the appointment of Ian Smith to its Board... Read More

Data from multiple ongoing clinical studies of STK-001 show reductions in convulsive seizure frequency and improvements in cognition and behavior in children and adolescents with Dravet syndrome Data support the potential for STK-001 to be the first disease-modifying treatment for Dravet syndrome New pharmacokinetic (PK) modeling of clinical data from ongoing studies demonstrate that higher STK-001 drug exposures in brain... Read More

STK-001: Additional data from patients treated with single and multiple doses (70mg) along with data from open-label extension studies (30mg, 45mg) anticipated in Q1 2024 STK-001: Company plans to share an update on Phase 3 planning in 1H 2024 STK-002: Company received authorization to initiate a Phase 1 study in the UK for the treatment of Autosomal Dominant Optic Atrophy (ADOA) As of June 30, 2023, Company had $231.4... Read More

BEDFORD, Mass. / Aug 02, 2023 / Business Wire / 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 announced that management will present at the Canaccord Genuity 43rd Annual Growth Conference on Wednesday, August 9, 2023, at 4:30 p.m. ET. A live webcast of the presentation, which... Read More

Totality of data from these ongoing studies suggest clinical benefit for patients ages 2 to 18 years old, including reductions in seizures and improvements in cognition and behavior that support the potential for disease modification Phase 1/2a ADMIRAL Study Data STK-001 (70mg): Patients treated with 2 or 3 initial doses experienced substantial and sustained reductions in convulsive seizure frequency; Median reductions at 3... Read More

BEDFORD, Mass. / Jul 21, 2023 / Business Wire / 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 announced that management will hold a webinar and conference call for analysts and investors at 8:00 a.m. Eastern Time on Tuesday, July 25, 2023, to present new data from the two... Read More

BEDFORD, Mass. / Jun 16, 2023 / Business Wire / 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 announced that, effective on June 15, 2023, the company granted stock options to purchase an aggregate of 26,900 shares of common stock to 2 new employees, as a material inducement to... Read More

Company on-track to report new data from the ongoing Phase 1/2a clinical studies of STK-001 in children and adolescents with Dravet syndrome in mid-2023 Data readout will focus on safety and seizure frequency results for up to 16 patients who received three doses of 45mg of STK-001 Company plans to initiate a pivotal study in 2024, pending additional data from the Phase 1/2a studies Company received authorization to initiate... Read More

BEDFORD, Mass. / May 03, 2023 / Business Wire / 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 announced that management will present at the BofA Securities Health Care Conference on Thursday, May 11, 2023, at 11:00 a.m. ET. (8:00 a.m. PT) A live webcast of the presentation,... Read More

Company advancing second TANGO ASO into the clinic STK-002 has the potential to be the first disease-modifying therapy to address the root cause of ADOA, the most common inherited optic nerve disorder BEDFORD, Mass. / Apr 25, 2023 / Business Wire / Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based... Read More

BEDFORD, Mass. / Mar 15, 2023 / Business Wire / 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 announced that the FDA will allow the administration of a higher single dose of STK-001 (70mg) in its ongoing Phase 1/2a MONARCH study of children and adolescents with Dravet syndrome.... Read More

BEDFORD, Mass. / Mar 06, 2023 / Business Wire / 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 full year ended December 31, 2022 and provided business updates. “In 2022 we continued to advance the first potential new medicine to treat the... Read More

BEDFORD, Mass. / Feb 27, 2023 / Business Wire / 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 announced that management will be participating at the following upcoming investor conferences: Cowen 43rd Annual Health Care Conference Date: Monday, March 6, 2023 Time: 12:50 p.m. ET... Read More

BEDFORD, Mass. / Feb 07, 2023 / Business Wire / 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 announced that management will present at the virtual SVB Securities Global Biopharma Conference on Tuesday, February 14, 2023, at 4:20 p.m. ET. A live webcast of the presentation,... Read More

Additional safety and seizure frequency data from patients treated with multiple doses of STK-001 (45mg) anticipated in mid-2023 Data from the highest dose cohort (70mg) anticipated in the second half of 2023 Company expects to complete Phase 1/2a in 2023 in order to initiate a Phase 3 program in 2024 As of September 30, 2022, Company had $252.2 million in cash, cash equivalents, marketable securities, and restricted cash,... Read More

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 announced that management will present at the 41st Annual J.P. Morgan Healthcare Conference on Tuesday, January 10, 2023, at 2:15 p.m. ET (11:15 a.m. PT). A live audio webcast of the presentation will be available on the Investors &... Read More