Stoke Therapeutics (STOK) investor relations material

Clinical development updates

• EMPEROR phase III trial for zorevunersen in Dravet syndrome enrolled 162 patients, surpassing the target, with zero dropouts and strong patient retention through key endpoints.

• The trial measures both seizure reduction (primary endpoint at week 28) and cognitive/behavioral gains (secondary endpoint at week 52) using Vineland-3, reflecting a holistic approach to treating the syndrome.

• Long-term open-label extension (OLE) data show sustained and compounding gains in function and behavior over four to five years, supporting the drug's disease-modifying potential.

• Rolling NDA submission is planned to start in Q1 2027 and complete in Q3 2027, with the aim of leveraging breakthrough designation for expedited review.

• The study is powered to detect a 40–45% reduction in seizures and a clinically meaningful 2–3 point gain in receptive communication on Vineland-3.

Value proposition and market access

• The value proposition centers on both seizure reduction and meaningful improvements in cognition and behavior, which are highly valued by caregivers, physicians, and payers.

• Payers and prescribers view long-term OLE data as the most compelling evidence for both medical policy and pricing decisions.

• Pricing strategy will be informed by analogs such as SPINRAZA and DAYBUE, with emphasis on the totality of evidence and disease-modifying impact.

• Education efforts with payers highlight the unique, syndrome-modifying profile of zorevunersen compared to symptomatic treatments.

Pipeline and partnerships

• The OSPREY program for Autosomal Dominant Optic Atrophy (ADOA) is in phase I/II, targeting the OPA1 gene to improve vision, with efficacy data expected in H1 2027.

• Collaboration with Biogen provides global reach, manufacturing, and regulatory expertise, with a strong governance structure and positive ongoing relationship.