Annexon (ANNX) 2025 Conference Transcript

Summary of Inexon Conference Call Company Overview - Inexon is focused on complement, specifically classical complement C1Q, which is relevant in various neurodegenerative diseases and autoimmune conditions [5][6] Key Programs and Milestones - Guillain Barre Syndrome (GBS) - Late-stage program with a pivotal Phase III study [5] - No approved therapies in the U.S. for GBS; standard care is IVIG, which lacks substantial evidence [8][9] - Market opportunity: 150,000 patients worldwide, with 7,000 in the U.S. and 22,000 in Europe [8] - Phase III study showed a 2.5 times better likelihood of patients returning to normal health by week eight compared to placebo [12][14] - Expected BLA filing later this year [5][28] - Geographic Atrophy (GA) - Phase III program with 630 patients, aiming to treat 8 million patients worldwide [6] - Only program showing preservation of vision loss on multiple endpoints [6] - Small Molecule Program - First and only in the classical pathway, targeting various neuromuscular diseases [6] - Aiming to complete proof of concept study this summer [6] Regulatory and Market Considerations - FDA Engagement - Positive feedback from FDA regarding Phase III study and generalizability of data [30][31] - Upcoming pre-BLA meeting to discuss comparability of study patients to U.S. patients [31] - Potential for breakthrough designation to be discussed after the upcoming meeting [35] - Market Access and Pricing - Anticipated pricing for the therapy ranges from $75,000 to $150,000 per course [42] - Emphasis on health economics to demonstrate cost savings by reducing ICU and ventilator use [39][40] Safety and Efficacy - Safety profile of the drug is compelling, with treatment arms showing similar safety to placebo [52] - The Phase III study is the longest duration study ever conducted in GBS, with a primary endpoint at week eight [38] FORWARD Study - Open-label study enrolling Western patients to address physician concerns about U.S. experience with the drug [54][56] - Designed to provide real-time data on efficacy and safety [58] European Market - EMA discussions are advanced, with a target to file in Europe by the end of the year or early next year [62] Small Molecule Program Details - Enteric-coated tablet developed to improve tolerability [70] - Focus on treating patients with hemolysis in cold agglutinin disease [74] - Key biomarkers for success include safety, tolerability, complement reduction, and bilirubin levels [76][78] Conclusion - Inexon is positioned to make significant advancements in the treatment of GBS and other neuromuscular diseases, with promising data and regulatory engagement paving the way for potential market entry in the near future [5][6][28]