Stoke Therapeutics (STOK) investor relations material

Company focus and clinical approach

• Focuses on haploinsufficient diseases, primarily Dravet syndrome, a severe genetic epilepsy caused by a lack of NAV 1.1 protein expression.

• Uses an antisense oligonucleotide (ASO) to upregulate NAV 1.1, addressing the root cause and reducing seizures and improving neurodevelopmental outcomes.

• Clinical data show significant seizure reduction and improvements in cognition and behavior, even on top of standard care.

Clinical trial progress and results

• Currently in phase 3, with the study initiated recently and patient recruitment underway; over 150 patients in prescreening for a 170-patient trial.

• Long-term data from phase 1/2 and open-label extension (OLE) studies show durable seizure reduction (~85%) and consistent cognitive and behavioral improvements over four years.

• Vineland-3 scores, measuring cognition and behavior, improved dramatically, with scores of 8–11 (clinically meaningful is 2), and benefits observed across a broad age range.

• Dosing in phase 3 involves two loading doses of 45mg four months apart, mirroring successful regimens from earlier studies.

Regulatory and market outlook

• Received FDA breakthrough designation for Dravet syndrome, with plans to discuss expedited approval pathways using mature four-year safety and efficacy data.

• Phase 3 is a 52-week, sham-controlled study with primary endpoint at week 28 (seizure reduction) and key secondary endpoints including Vineland-3 scores.

• Enrollment expected to complete in 2026, with readout in 2027; study powered for strong statistical significance (p=0.01) on both seizure and cognitive endpoints.

• High demand for trial enrollment driven by advocacy groups and physician interest.