Stoke Therapeutics (NasdaqGS:STOK) FY Conference Transcript

Summary of Stoke Therapeutics FY Conference Call Company Overview - Company: Stoke Therapeutics (NasdaqGS:STOK) - Focus: Development of treatments for Dravet syndrome and Autosomal Dominant Optic Atrophy (ADOA) Key Points on Dravet Syndrome - Market Opportunity: Approximately 35,000-40,000 patients in the core geographies (U.S., Japan, U.K., EU 4) with about 16,000 in the U.S. and a similar number in the EU 4 [85][86] - Addressable Patients: 6,000 patients are immediately addressable upon launch, with 4,000 being pediatric patients aged 18 and younger [85][86] - FDA Interactions: Ongoing discussions with the FDA regarding the potential success of the phase 3 study and the importance of the label for pricing and promotion [15][71] - Clinical Data: Phase 1, 2 studies show robust seizure reduction and improvements in cognition and behavior, with a focus on longitudinal data over a 5-year period [17][19][20] - Primary Endpoint: Seizure reduction at 26 weeks, with secondary endpoints including cognition and behavior assessed at 52 weeks [48][55] - Vineland Assessment: Statistical improvements observed in Vineland scores, indicating cognitive and behavioral benefits [23][25] Phase 3 Study (EMPEROR) - Enrollment Status: Approximately 160 patients enrolled in the U.S. and Europe, with completion expected in mid-2027 [26][27] - Study Design: Includes lumbar puncture sham control patients and additional cohorts for regulatory compliance [27][35] - Powering of Study: Designed for a 90% confidence level with a p-value of 0.01 for secondary endpoints, ensuring robust statistical significance [48][52] Pricing and Market Dynamics - Pricing Strategy: Aiming for a price reflective of the value provided by the treatment, potentially aligning with other genetically targeted disease-modifying treatments rather than traditional anti-seizure medications [103][105] - Comparison with SPINRAZA: Pricing expected to be in line with other ASOs like SPINRAZA, which is priced around $600 per dose [118][105] Autosomal Dominant Optic Atrophy (ADOA) - Patient Population: Approximately 13,000 patients in the U.S. and Europe, with a significant risk of legal blindness [127] - Phase 1 Study: Ongoing dose escalation study with initial dosing completed, aiming to demonstrate efficacy in improving eyesight [128][129] Additional Insights - Long-term Development: The treatment aims to provide more years of neurotypical development for children with Dravet syndrome, addressing both seizure control and cognitive development [20][71] - Genetic Testing: Increased access and utilization of genetic testing are expected to enhance patient identification, particularly in adults who may have been misdiagnosed [88][90] Conclusion - Stoke Therapeutics is positioned to address significant unmet needs in the treatment of Dravet syndrome and ADOA, with ongoing clinical studies and strategic planning for market entry and pricing. The company emphasizes the importance of robust clinical data to support its treatment's efficacy and market potential.