Stoke Therapeutics(US:STOK)2026-02-11 20:32Summary of Stoke Therapeutics Conference Call Company Overview - Company: Stoke Therapeutics (NasdaqGS:STOK) - Focus: Development of treatments for genetic diseases, specifically Dravet syndrome and Autosomal Dominant Optic Atrophy (ADOA) [2][6] Key Points on Dravet Syndrome - Disease Understanding: Dravet syndrome is a severe form of epilepsy affecting young children, leading to significant developmental challenges [2][3] - Clinical Data: Initial phase 1/2 data showed an 80% reduction in seizures on top of standard care medications, with durable effects observed over nearly four years [4][9] - Cognitive and Behavioral Benefits: Improvements in cognition and behavior were noted, measured by the Vineland-3 assessment, indicating potential for neurodevelopmental progress in children who typically plateau after 18 months [5][10] - Phase 3 Program: Enrollment for the phase 3 study is expected to complete in Q2 2026, with data anticipated in mid-2027 [6][28] Market Opportunity - Patient Population: Approximately 36,000-40,000 patients globally, with 16,000 in the U.S. and 6,000 currently addressable [50][51] - Pricing Strategy: The company aims to position its treatment as a disease-modifying therapy, potentially aligning pricing with similar therapies like Spinraza [52][54] Regulatory Pathway - FDA Interaction: The company is pursuing accelerated approval but faced challenges due to the FDA's request for additional data. A follow-up discussion is planned to present data against patient baseline rather than cross-study comparisons [34][35][36] - Optimal Label: The goal is to achieve a label that reflects both seizure reduction and cognitive improvements, which is crucial for market positioning and pricing discussions [41][42] ADOA Development - New Indication: The first patient for ADOA was dosed recently, targeting a genetic loss of eyesight by upregulating the OPA1 gene to improve mitochondrial function and vision [56][57] - Market Size: The ADOA market is estimated at around 16,000 patients in the top seven geographies [58] Financial Position - Cash Reserves: The company reported approximately $400 million in cash at the end of 2025, with funding from Biogen covering 30% of Dravet R&D costs, providing a runway into 2028 [60]