Stoke Therapeutics Appoints Ian F. Smith as Chief Executive Officer

Leader with Long Track Record in Building World-Class Biopharma Companies and Driving Late-Stage Development and Commercial Execution

Arthur Tzianabos Resumes Chairman of the Board Role

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 Board of Directors has appointed Ian F. Smith as Chief Executive Officer (CEO). Mr. Smith has served as Interim CEO since March 2025 and as a member of Stoke's Board of Directors and advisor to the Company since 2023. He will continue to serve as a Director on the Company's Board of Directors. Arthur Tzianabos, Ph.D., has been a member of the Board of Directors since 2018. He will resume his role as Chairman of the Board after serving as Executive Chairman while the CEO search was conducted.

A global Phase 3 study of the Company's lead investigational medicine, zorevunersen, is underway. Zorevunersen is a first-in-class potential disease-modifying medicine for Dravet syndrome. Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) characterized by severe, recurrent seizures as well as significant cognitive and behavioral impairments. In February 2025, Stoke entered a collaboration with Biogen Inc. to develop and commercialize zorevunersen for Dravet syndrome. Under the collaboration, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights.

Dr. Tzianabos said, "After conducting a comprehensive search, it became clear that Ian is uniquely qualified to lead Stoke and deeply committed to our science, our mission, and our people. Under his leadership over the last six months, the team and the business have thrived, achieving multiple milestones, including the start of our pivotal Phase 3 EMPEROR study in Dravet syndrome, generation of new data to drive understanding of zorevunersen, and the expansion of our pipeline with the start of a second clinical program in Autosomal Dominant Optic Atrophy. This strong execution has translated into meaningful value creation for our shareholders. Ian's deep experience in rare disease drug development and commercialization, regulatory know-how, exceptional track record of building and leading teams, and comprehensive, multi-faceted understanding of our business are unmatched and will be invaluable as we continue to build Stoke into a world-class biotechnology company."

Mr. Smith said, "As a Board member and advisor, I had established an appreciation for Stoke, and I have enjoyed my time working even more closely with the team as Interim CEO since March. I look forward to transitioning to the CEO and to continuing with the opportunity to build a company that has the potential to deliver first-in-class medicines to patients with severe genetic diseases – a mission both unique and deeply familiar to me. Over the last six months, we have strengthened our business and positioned Stoke for rapid advancement and growth. I look forward to continuing to work with this outstanding team to realize the full potential of our science and medicines."

Mr. Smith has spent most of his career delivering life-changing medicines for patients with rare diseases. He previously served as Executive Vice President, Chief Operating Officer, and Chief Financial Officer of Vertex Pharmaceuticals. Over the course of nearly two decades, he was instrumental in steering Vertex through key strategic pivots, transforming it from a research stage company into a global biopharmaceutical leader in rare disease innovation. Since 2019, Mr. Smith has held numerous board and advisory roles at several leading biotechnology companies.

Ian F. Smith Biography

Ian F. Smith is a biotechnology leader with more than 30 years of finance and operations experience and extensive relationships in the industry. Mr. Smith previously served as Executive Vice President, Chief Operating Officer, and Chief Financial Officer of Vertex Pharmaceuticals from 2001 to 2019. Prior to his tenure at Vertex, he was a partner in the Life Science and Technology Practice of the accounting firm Ernst & Young LLP. Mr. Smith currently serves as a senior advisor to Bain Capital Life Sciences, a position he has held since January 2021, and provides other advisory and consulting services to life science companies. He currently serves as Executive Chairman of the Board at Solid Biosciences and Chairman of the Board at Rivus Pharmaceuticals. He also sits on the Board of Directors for Foghorn Therapeutics, Alkeus Pharmaceuticals, Areteia Therapeutics, and Odyssey Therapeutics. Ian holds a B.A. with honors in accounting and finance from Manchester Metropolitan University (UK).

Arthur Tzianabos, Ph.D. Biography

Dr. Arthur Tzianabos was appointed to Stoke's Board of Directors in 2018 and was appointed Chair in 2024. He is currently CEO of Lifordi Immunotherapeutics and a venture partner at 5AM Ventures. He was previously CEO and later Chair of the Board of Directors at Homology Medicines, where he led the company from inception through a successful public offering. While at Homology, Dr. Tzianabos led efforts to develop genetic medicines by leveraging its in vivo gene therapy and nuclease-free gene editing platform for patients with rare genetic diseases. Dr. Tzianabos currently serves on the Board of Directors of Q32 Bio following its merger with Homology earlier this year. He was formerly Chair of the Board of Directors of Akouos, a publicly traded gene therapy company acquired by Lilly in 2022. Prior to this, he spent nine years at Shire, where he worked on the development and launches of multiple treatments for patients with rare genetic disorders and worked closely with the business development team to build Shire's product pipeline through investments and acquisitions.

Earlier in his career, Dr. Tzianabos was a principal investigator and faculty member at Harvard Medical School for 15 years, reaching the rank of associate professor of medicine and maintaining laboratories at the Channing Laboratory, Brigham and Women's Hospital and the Department of Microbiology and Molecular Genetics at Harvard Medical School. He has published more than 80 scientific papers, reviews, book chapters and patents. He holds a B.S. in biology from Boston College and a Ph.D. in microbiology from the University of New Hampshire.

About Stoke Therapeutics
Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine. Using Stoke's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke's first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study. Stoke's initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for Stoke's proprietary approach. Stoke is headquartered in Bedford, Massachusetts. For more information, visit https://www.stoketherapeutics.com/ .

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the design, timing and results of the Phase 3 study; the timing and expected progress of regulatory filings and regulatory decisions; the ability of zorevunersen to treat the underlying causes of Dravet syndrome and reduce seizures or show improvements in behavior and cognition at the indicated dosing levels or at all; and the expectations regarding the business operations. Statements including words such as "anticipate," "expect," "plan," "will," or "may" and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they prove incorrect or do not fully materialize, could cause the Company's results to differ materially from those expressed or implied by such forward-looking statements, including, but not limited to, risks and uncertainties related to: the Company's ability to advance, obtain regulatory approval and ultimately commercialize its product candidates; that if Biogen were to breach or terminate the collaboration, the Company would not obtain the anticipated financial or other benefits; the possibility that the Company and Biogen may not be successful in their development of zorevunersen and that, even if successful, they may be unable to successfully commercialize zorevunersen; positive results in a clinical trial may not be replicated in subsequent trials or successes in early stage clinical trials may not be predictive of results in later stage trials; the Company's ability to protect its intellectual property; the Company's ability to fund development activities and achieve development goals through mid-2028; and the other risks and uncertainties described under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the year ended December 31, 2024, its quarterly reports on Form 10-Q, and the other documents it files with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the Company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.

Stoke Media & Investor Contacts:
Dawn Kalmar
Chief Communications Officer
dkalmar@stoketherapeutics.com
781-303-8302

Doug Snow
Director, Communications & Investor Relations
IR@stoketherapeutics.com
508-642-6485

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