Stoke Therapeutics, Inc.

Good day and thank you for standing by. Welcome to the Stoke Therapeutics third quarter 2025 business and financial update. At this time, all participants are in listen only mode. After the speaker's presentation, there will be a question and answer session to ask a question. During this session, you need to press star one one on your telephone. You would then hear an automated message advising your hand is raised to withdraw your question. Please press star one one again. Please be advised that today's conference is being recorded. I would now like to hand the conference over to your first speaker today, Thomas Leggett, Chief Financial Officer. Please go ahead.

Good afternoon and welcome to Stoke Therapeutics' third quarter 2025 conference call. I'm Thomas Leggett, Chief Financial Officer at Stoke Therapeutics. Joining me are Ian Smith, our Chief Executive Officer, and Dr. Barry Tico, Chief Medical Officer. Jason Hoyt, Chief Patient Officer, will join us for the Q&A portion. Today's webcast presentation is available in the Investor News section of our website. It is being recorded and will be available for replay later today. Before we begin, please note that today's discussion includes forward-looking statements. These are subject to risks and uncertainties, and actual results may differ materially. Please refer to our filings of the SEC for additional information. With that, I will turn the call over to Ian.

Welcome, everyone, and thank you for joining us today. I'm honored to have been asked to serve as the CEO of Stoke Therapeutics. This is an incredible company with significant opportunity ahead of it. I could not be more excited to be part of the team as we advance Zoriva Nursing through phase three and build the company to deliver long-term value to patients, our employees, and shareholders. On today's call, we will review our third quarter progress. I'll start by summarizing where the company has come from and how that has brought us to today. I will talk about what priorities are next for us. Those progress in 2025 has put us in an important growth trajectory. There are several drivers of our momentum. With four years of Xeribinersen clinical experience and two years of natural history data, we have compiled a wealth of data to support our understanding of Dravet syndrome and the potential for Xeribinersen to treat this devastating disease. In addition, to support the Phase III study, our long-term longitudinal data give us a clearer picture of the disease-modifying potential of cerebronursome and the benefits it could provide for people living with Dravet syndrome and the families and caregivers who support these people. One of our priorities for 2025 has been to bring broader and more consistent understanding of cerebronursome to clinicians, the patient community, and the investment community. We have done so primarily through scientific and medical communications and our interactions at medical congresses. These activities, along with increasing awareness of the severity of Dravet syndrome, are contributing to significant interest in our phase three study. Back in August, we told you 150 patients had been identified through the investigator pre-screening process. That number has now grown to more than 200 patients. In a few minutes, Barry will share an update on progression of those patients through the formal screening process and into enrollment and study randomization. As we continue to move forward with EMPEROR, we are preparing to meet with the FDA to discuss potential expedited regulatory pathways to bring Zoriva Nursing to patients as soon as possible. This multidisciplinary meeting is part of the standard process for investigational medicines granted breakthrough therapy designation. Although Zariva-Nersen was granted breakthrough designation last December, we did not request the meeting until we had phase three OLE longitudinal data. This now allows us to bring a comprehensive and current safety and efficacy data set to the discussion. The breakthrough process was created to expedite the development and review of drugs that are intended to treat serious conditions. As part of that, the FDA commits to the involvement of senior FDA managers. The meeting will be guided by a briefing book that is submitted in advance by the company. We have submitted our briefing book, which includes data and a series of questions, one of which relates to the potential for an expedited regulatory pathway for zarivinersen. With the growing awareness of Dravet syndrome and the data to support the disease-modifying potential for zarivinersen, the market opportunity is taking shape. As we move through our phase three and prepare to potentially launch Cerebral Nursing in the U.S., we are investing in foundational efforts that will enable us to rapidly scale our business to reach as many patients as quickly as possible. Through 2025, we have prioritized leadership in our core areas of our business, namely medical affairs and commercial, as well as the executive team. The medical affairs field has increased the level and quality of our interactions with the clinical community. Our commercial leadership team is now in place with experienced leaders in market access, pricing, marketing, commercial operations, and new product planning. TMC and supply activities remain on schedule to support the phase three and potential launch. We're in the initial stage of market shaping activities, starting with Unseen, a disease awareness campaign to increase awareness of diagnosis of Dravet syndrome through genetic testing. You'll be hearing more about this effort, which is designed to confront the gap in disease diagnosis. Derevynosin demonstrates the potential of our platform. We see a broad opportunity to develop additional disease-modifying medicines, particularly in haploid-sufficient disease areas of CNS, eye, and heart. In terms of the eye, we recently presented data from our two-year ADOA natural history study that offer insights into underlying biology of disease progression and clinical assessments that support clinical development of STK002. Recruitment has begun for our phase one dose escalating study of STK002 as a potential treatment for autosomal dominant optic atrophy. Sites are active in the UK, and European sites are expected to initiate in early 2026. While moving through this dose escalating study, the earliest we would anticipate potential efficacy data from Osprey would be toward the end of 2026 or early 2027. Next in the pipeline is our Syngap program. Similar to Dravet, Syngap is a neurodevelopment disorder that is primarily caused by a haploid insufficiency. Preclinical work continues as we work towards the potential nomination of a clinical development candidate in 2026. Our experience with Zoriva Nursing and relationships with treating physicians and patient advocates have been helpful in our early planning for potential Fingap clinical development program. In addition to these programs, we continue to invest in our early stage pipeline across multiple therapeutic areas. We look forward to providing updates as our pipeline programs advance. I will now turn the discussion over to Barry for a brief review of key data and an update on progress of EMPEROR.