Stoke Therapeutics, Inc.

Good morning. My name is Audra and I will be your conference operator today. At this time, I would like to welcome everyone to the Stoke Therapeutics interim analysis of STK001 for the treatment of Dravet syndrome. All lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question and answer session. If you would like to ask a question during this time, simply press the star key followed by the number one on your telephone keypad. If you would like to withdraw your question, press star one again. At this time, I'd like to turn the conference over to Eric Rojas, Head of Investor Relations. Please go ahead.

Thanks, Audra, and good morning. This is Eric Rojas, Head of Investor Relations at Stokes Therapeutics. On today's call, we will discuss results from the interim analysis of the Phase 1-2A clinical studies of our investigational medicine STK001 for the treatment of Gervais syndrome. Our speakers today are Dr. Edward Kay, Stokes CEO, and Dr. Barry Tico, Stokes Chief Medical Officer. As you listen to this call, we recommend that you access the webcast slides in the Investors section on our website. This call is being recorded, and a replay will also be available on the Stoke website later today. We will make forward-looking statements on this call that are subject to the risks and uncertainties discussed in detail in today's press release and in our filings with the SEC. These statements, including without limitation those regarding Stoke's Dervet Syndrome Program, are based on management's current intentions beliefs, and assumptions. Actual outcomes and events can differ materially. Except as required by law, we assume no obligation to update any such forward-looking statements after the date of this presentation or to conform these forward-looking statements to actual results. I will now turn the call over to Dr. Kaye. Thank you, Eric, and good morning, everyone.

We're pleased to share with you today the positive interim results from our ongoing phase 1 to 8 clinical studies of STK001. And to frequent and prolonged seizures, patients experience significant non-seizure comorbidities. What you will see today is new evidence that STK001 continues to be well-tolerated and is reducing seizures in children and adolescents with Dravet syndrome. Importantly, these improvements are on top of the best available anti-seizure medicines with more than half of the patients taking fenfluramines. Today, we are also sharing very preliminary evidence of the drug's effect on non-seizure comorbidities. Together, these results give us confidence that we are honing in on an effective dose level and dose frequency. We look forward to sharing additional data in 2023 that will inform our plans for pivotal studies. Before Barry walks you through the specifics of the data, I'd like to share a bit of background on the disease and patient populations. Privé syndrome is a severe and progressive genetic epilepsy. It's caused by a haploid insufficiency, which results in half of the normal NAV1.1 protein expression. One out of 16,000 babies around the world are born with this devastating disease, and it is not concentrated on any particular geographic area. There is a real and urgent need for better medicines that treat seizures and the myriad of non-seizure comorbidities. The non-seizure aspects of this disease, some of which are listed on slide six, significantly impact the quality of life for patients and their caregivers. The combination of anti-seizure medicines that make up the current standard of care are burdensome, do not control seizures for the vast majority, and come with a host of side effects. With our butterfly observational study, we are learning more about the natural progression of Dravet syndrome and the clinical assessments that we can use to measure changes in neurodevelopment. Born without symptoms, these children develop fairly normally, but begin to regress with age as the disease advances. The goal of treatment today is seizure management, but what these patients need is syndrome management. Our number one priority at Stoke is to advance STK001 as potentially the first disease-modifying approach for the treatment of Dravet syndrome. one that will control seizures and address the non-seizure comorbidities. From the start, we have set a high bar for success. The majority of patients enrolled in our studies are taking at least three and often four anti-seizure medicines. Despite this, they continue to experience seizures and many of the debilitating effects that characterize this disease. Based on what we are seeing in the clinic, we believe that producing a consistent and durable clinical effect for patients will require reaching a steady state of drug levels in the brain to boost and sustain normal protein levels. Three factors play a role in this process, dose level, dose frequency, and time on drug. We expect that additional data in 2023 will answer our remaining questions and unlock the path to pivotal studies. I'll now hand over the call to Barry.