Stoke Therapeutics (STOK) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Pipeline and platform overview

• Advancing a unique platform targeting haploinsufficiencies, with potential for 6,500 additional diseases.

• Zorevunersen is entering phase III for Dravet syndrome next year; ADOA and other programs are entering the clinic soon.

• Rett syndrome, SYNGAP1, and an undisclosed program with Acadia are progressing, with updates expected later this year or next.

Dravet syndrome program (zorevunersen)

• Dravet syndrome causes severe, frequent seizures and significant cognitive and behavioral deficits; current treatments leave 90% of patients inadequately controlled.

• Zorevunersen increases SCN1A gene expression, compensating for protein deficiency and showing disease-modifying potential.

• Phase I/IIa data showed 74–85% seizure reduction at higher doses, with durable effects seen in open-label extension studies up to three years.

• 80% of patients experienced over 50% seizure reduction; some achieved complete seizure freedom, with benefits seen across ages.

• Improvements in cognition and behavior were observed, as measured by Vineland-3, with meaningful impact for patients and caregivers.

Safety, regulatory, and commercial outlook

• Zorevunersen was generally well tolerated, with no clinically meaningful safety concerns at tested doses.

• Global phase III planning is underway, coordinating with EMA, FDA, and PMDA, with regulatory updates expected by year-end.

• Strong cash position of $282 million, providing runway through 2025 and supporting upcoming clinical and regulatory milestones.

• If approved, zorevunersen could address a significant unmet need and reduce healthcare system burden.