Apellis Pharmaceuticals (APLS) FY 2025 Conference September 03, 2025 08:45 AM ET Speaker0All right, everyone, I think we'll get started with our next session. My name is Derek Archuleta. I'm one of the Senior Biotech Analysts here at Wells Fargo. Very excited to have Apellis Pharmaceuticals with us here for our next fireside discussion. We've got Tim Sullivan, the Chief Financial Officer, as well as David Acheson, the Chief Commercial Officer. Gentlemen, thank you so much for coming and looking forward to t ...

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]

Core Insights - InflaRx N.V. presented multiple data sets at the 2025 American Academy of Dermatology Annual Meeting, highlighting the potential of vilobelimab in treating inflammatory conditions such as pyoderma gangrenosum (PG) and hidradenitis suppurativa (HS) [1][2] Group 1: Vilobelimab in Pyoderma Gangrenosum (PG) - Safety data from a Phase 2a dose-finding study indicated that adverse events (AEs) were mostly mild to moderate, with vilobelimab being well tolerated across all doses [3] - Pharmacokinetic (PK) and pharmacodynamic (PD) analyses showed a significant reduction in C5a concentrations, with approximately 90% reduction observed by Day 15 across all dose groups [4] - The ongoing Phase 3 trial is utilizing vilobelimab dosed at 2400 mg bi-weekly to suppress C5a in ulcerative PG patients [4] Group 2: Vilobelimab in Hidradenitis Suppurativa (HS) - A post-hoc analysis from the Phase 2b SHINE trial demonstrated vilobelimab's effectiveness in reducing draining tunnels (dT) by 63.2% compared to 18.0% for placebo, with a 3.1x relative improvement in complete resolution of dT [5] - Safety analysis showed that vilobelimab was well tolerated, with similar rates and severity of AEs compared to placebo [6] - PK/PD analysis indicated that administration of 800 mg vilobelimab resulted in significant reductions in C5a concentrations, which remained lower than baseline during follow-up [7]