Financial Data and Key Metrics Changes - The company ended the second quarter with $355 million in cash, cash equivalents, and marketable securities, expected to fund operations through mid-2028 [42] - Total revenue for the quarter was $13.8 million, driven by collaborations with ACADIA and Biogen, with expectations for revenue from Biogen to increase [43] - The net loss for the quarter was $23.5 million, or $0.40 per share, slightly improved from the prior year despite a $6.9 million year-over-year increase in operating expenses [43] Business Line Data and Key Metrics Changes - The Phase III EMPORER study for Dravet syndrome is underway, with the first patient dosed and strong enrollment anticipated due to high awareness and urgent patient need [5][21] - The company has advanced STK002 into Phase I clinical development for autosomal dominant optic atrophy (ADOA), indicating a growing pipeline [7][35] Market Data and Key Metrics Changes - The collaboration with Biogen enhances the company's ability to deliver zurivanersen globally and strengthens its balance sheet [8] - The company estimates approximately 13,000 patients currently living with ADOA across key geographies, indicating a significant market opportunity [38] Company Strategy and Development Direction - The key priority remains the development of zurivanersen for Dravet syndrome, aiming to deliver a disease-modifying medicine [4] - The company is focused on establishing internal capabilities and enhancing leadership to support growth and value creation [9] - The strategic collaboration with Biogen is expected to provide global expertise in commercializing high-value disease-modifying medicines [8] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term potential of zurivanersen to modify the course of Dravet syndrome, supported by positive data from ongoing studies [7][31] - The company is committed to advancing zurivanersen to patients as quickly as possible, leveraging breakthrough therapy designation from the FDA [50][52] Other Important Information - The company has seen a favorable safety profile for zurivanersen, with no clinical manifestations associated with elevated CSF protein levels observed in patients [32][94] - The Vineland-three assessment is being used to measure changes in cognition and behavior in patients with Dravet syndrome, with significant improvements noted over time [28][30] Q&A Session Summary Question: Can you help us understand the potential for accelerated approval for zurivanersen? - Management confirmed that zurivanersen has breakthrough designation and they are collecting further data to discuss with the FDA in the second half of the year [50][52] Question: What is the magnitude of cognition and behavior improvements in the Vineland-three data? - Management noted that changes in cognition and behavior are clinically meaningful, with caregivers identifying even small improvements as significant [62][63] Question: Can you explain the data used to inform the powering assumptions for the Phase III EMPOR study? - Management indicated that data from previous studies and natural history studies were used to inform the powering assumptions, ensuring robust statistical significance [70][72] Question: Are there trends in seizure reduction and neurodevelopmental benefits among different age groups? - Management acknowledged variability in responses but emphasized the importance of treating younger patients to potentially change the course of their development [86][87] Question: Can you discuss the higher incidence of CSF protein elevations in the OLE study? - Management clarified that elevated CSF protein levels are a laboratory finding and have not been associated with clinical effects, supporting the safety profile for moving into Phase III [92][94] Question: What prompted the decision to explore STK-two in ADOA? - Management explained that a thorough evaluation of the opportunity in ADOA, including promising nonhuman primate data, led to the decision to pursue clinical studies [98][100]