Stoke Therapeutics (STOK) Canaccord Genuity's 46th Annual Growth Conference summary

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 is sham-controlled, with primary endpoint at week 28 (seizure reduction) and secondary at week 52 (cognition/behavior via Vineland-3 scale).

• Long-term open-label extension (OLE) data show sustained gains in cognition, behavior, and seizure reduction over up to five years.

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

• 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.

Product differentiation and value proposition

• Zorevunersen targets the underlying genetic cause of Dravet syndrome, aiming to treat the syndrome itself, not just symptoms.

• Breakthrough designation was granted based on evidence of both seizure reduction and cognitive/behavioral improvements.

• Longitudinal data indicate compounding functional gains over multiple years, a unique outcome in this disease area.

• Payers and physicians view long-term OLE data as the most compelling evidence for value and medical policy decisions.

• Pricing strategy will reflect the totality of clinical benefit, including quality of life and functional improvements beyond seizure control.

Pipeline and partnerships

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

• Collaboration with Biogen provides global reach, manufacturing, and commercial expertise, with a strong ongoing partnership structure.