Stoke Therapeutics (STOK) Jefferies Global Healthcare Conference 2026 summary

Product and clinical development updates

• Lead medicine targets Dravet syndrome by boosting NaV1.1 expression, addressing the root cause and reducing seizures.

• Four years of data show durable 75% seizure reduction and continuous cognitive and behavioral improvements, with statistical significance (P<0.01).

• Phase III study nearing enrollment completion; 52-week data expected mid-2027, with rolling NDA submission starting Q1 2027.

• Additional pipeline includes treatments for ADOA and SYNGAP1, focusing on haploinsufficient diseases.

• Strong financial position with over $400 million in cash and runway into 2028.

Market opportunity and pricing strategy

• U.S. Dravet prevalence estimated at 16,000, with 6,000 patients under 25 identified via claims data; screening expected to expand this number.

• Market research with payers and physicians indicates high value placed on seizure suppression and cognitive benefits.

• Pricing analogs include Spinraza and exon skippers, suggesting potential for rare disease premium pricing.

• Global Dravet population in major geographies estimated at 40,000, with ex-U.S. commercialization partnered with Biogen.

Regulatory and clinical endpoints

• Ongoing, consistent engagement with FDA, focusing on education and submission of new data, including cognition and behavioral outcomes.

• Primary endpoint for approval is seizure reduction; secondary endpoints include expressive and receptive communication, measured by Vineland.

• Phase III is powered to detect a clinically meaningful two-point Vineland improvement with high statistical confidence.

• Natural history study (BUTTERFLY) shows untreated patients stagnate, highlighting the significance of observed gains.

• Label expected to include both phase III and long-term OLE data, supporting claims of durable efficacy and functional improvement.