Stoke Therapeutics, Inc. Common Stock Canaccord Genuity's 46th Annual Growth Conference
Review the key takeaways and the transcript of this earnings call.
- Stoke Therapeutics Inc is conducting a phase three, sham-controlled study called Emperor for Dravet syndrome with 162 patients enrolled, exceeding the target of 150.
- The primary endpoint is at week 28 and the secondary endpoint at week 52, with 60 patients already through the primary endpoint and zero dropouts reported so far, indicating good drug tolerability.
- The study is powered to detect a 40-45% treatment benefit in seizure reduction on top of standard of care, with phase 1/2 data showing 70-80% seizure reduction in the higher dose group.
- Secondary endpoints include cognition and behavioral gains measured by Vineland-3 scales, with a clinically meaningful improvement defined as a 2-3 point increase in receptive communication.
- Longitudinal data from the phase 1/2 open-label extension shows sustained and compounding cognitive and behavioral improvements over four to five years of treatment.
- The company plans to initiate a rolling NDA submission in Q1 2027 and complete it by Q3 2027, with CMC data submitted first, followed by preclinical and clinical data.
- Stoke is also conducting a phase 1/2 dose-escalation study for autosomal dominant optic atrophy (ADOA), targeting the OPA1 gene to improve mitochondrial function and vision, with potential efficacy data expected in the first half of 2027.
- Stoke has a partnership with Biogen for commercialization outside North America, leveraging Biogen's antisense oligonucleotide expertise, manufacturing capabilities, and global footprint.
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Transcript
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Good morning, everyone. I'm Sumant Kulkarni, a senior biotechnology analyst at Canaccord Genuity, and I'm really pleased to have Stoke Therapeutics with us here today. Thankfully, you didn't have to travel too far, but I'm still thankful that you made it to the event. We're really happy to have you, and thanks to everyone for attending and for tuning into the webcast. Stoke is, again, at a very interesting time in its evolution. They have a phase III study for a product called zorevunersen for Dravet syndrome. This is going to be one of the few products that potentially treats the entire syndrome and not just symptoms. With that, I'll turn it over to Ian Smith, the CEO. We also have Chief Patient Officer Jason Hoitt. That's a really important role because this is a different kind of paradigm for the treatment of Dravet.
In the audience, we have Dawn Kalmar, who is the Chief of Corporate Affairs as well. With that, Ian, please. We can actually launch into a direct Q&A if that's the way to do it.
Thank you. We also have a mic going around, so please feel free to raise your hands.
Don't be shy. We can get your questions if any in the audience have them. I'll start first. Zorevunersen, as I mentioned, very different. It's probably going to change the treatment paradigm if and when it's approved, hopefully when. You have an EMPEROR phase III trial now. What can you say about how the trial is going that gives you confidence in achieving what you would need to achieve to get the product approved?
Thanks for the question, and good morning, everybody. I do have to provide my safe harbor statement, so if you'd see our SEC filings for all the forward-looking statements are covered in our SEC filings. Just last week, we had the opportunity to have our second quarter call, and we did provide an update at that point on the phase III study. Just to kind of broadly, it's a phase III study, sham-controlled, 162 patients study with a primary endpoint at week 28 and a secondary endpoint at week 52. As far as the progress of the study, we couldn't be happier. First of all, 162 patients enrolled. We did target 150. We ended at 162. Of those 162, 145 are already through week 8. Why is that an important time point?
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