Stoke Therapeutics Appoints Ian Smith as CEO as Phase 3 Trial for Dravet Syndrome Treatment Advances
Key Insights
Stoke Therapeutics has appointed Ian Smith as Chief Executive Officer after he served as Interim CEO since March 2025, bringing over 30 years of rare disease drug development experience.
The company's lead candidate zorevunersen, a first-in-class potential disease-modifying treatment for Dravet syndrome (search), is currently being evaluated in a global Phase 3 EMPEROR study.
Stoke has entered a collaboration with Biogen for zorevunersen development and commercialization, retaining exclusive rights in the US, Canada, and Mexico while Biogen receives rest-of-world rights.
Stoke Therapeutics has officially appointed Ian Smith as Chief Executive Officer, formalizing his leadership role after serving as Interim CEO since March 2025. The appointment comes as the biotechnology company advances its lead investigational medicine zorevunersen through a pivotal Phase 3 trial for Dravet syndrome (search).
Leadership Transition and Strategic Direction
Smith, who has served on Stoke's Board of Directors and as an advisor since 2023, will continue his board role while taking the helm as CEO. Arthur Tzianabos, Ph.D., will resume his position as Chairman of the Board after serving as Executive Chairman during the CEO search process.
"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," said Dr. Tzianabos. He highlighted Smith's leadership over the past six months, during which the company achieved multiple milestones including the start of the pivotal Phase 3 EMPEROR study and expansion of the pipeline.
Zorevunersen Phase 3 Development
The company's lead candidate, zorevunersen, represents a first-in-class potential disease-modifying medicine for Dravet syndrome (search), a severe developmental and epileptic encephalopathy characterized by severe, recurrent seizures as well as significant cognitive and behavioral impairments. The global Phase 3 study is currently underway, marking a critical milestone in the drug's development.
Zorevunersen utilizes Stoke's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, employing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. The therapy has demonstrated potential for disease modification in patients with Dravet syndrome (search) in earlier studies.
Strategic Partnership with Biogen
In February 2025, Stoke entered into a collaboration agreement with Biogen Inc. for the development and commercialization of zorevunersen. Under this partnership structure, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico, while Biogen receives exclusive commercialization rights for the rest of the world.
Executive Leadership Experience
Smith brings extensive rare disease drug development expertise to his new role, having spent nearly two decades at Vertex Pharmaceuticals where he served as Executive Vice President, Chief Operating Officer, and Chief Financial Officer from 2001 to 2019. During his tenure at Vertex, he was instrumental in transforming the company from a research-stage entity into a global biopharmaceutical leader in rare disease innovation.
"I look forward 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," Smith stated regarding his appointment.
Pipeline Expansion
Under Smith's interim leadership, Stoke has expanded its pipeline beyond Dravet syndrome (search), initiating a second clinical program targeting Autosomal Dominant Optic Atrophy (search). This expansion demonstrates the company's commitment to leveraging its TANGO platform across multiple rare genetic diseases caused by haploinsufficiency, where patients have approximately 50% of normal protein levels.
The company's approach focuses primarily on diseases of the central nervous system and the eye, though proof of concept has been demonstrated in other organs, tissues, and systems, suggesting broader potential for the proprietary platform.