Annexon (ANNX) Wells Fargo 20th Annual Healthcare Conference 2025 summary

Strategic program updates

• Advancing three first-in-kind programs targeting immune inflammation in body, brain, and eye, with a focus on Guillain-Barré syndrome (GBS), geographic atrophy (GA), and a novel oral small molecule for neuromuscular diseases.

• GBS program demonstrated landmark efficacy, with 90% of patients improving by week one in phase III trials.

• GA program is the only vision-sparing approach globally, recently completed a 650-patient phase III trial ahead of schedule.

• Oral C1s inhibitor program aims to disrupt established antibody markets with a more convenient pill form.

Regulatory and clinical progress

• GBS program has robust data from placebo-controlled studies, drug-drug interaction, and real-world evidence, supporting regulatory filings.

• EMA granted orphan drug designation for GBS based on proof of concept and real-world evidence; MAA filing anticipated by Q1 2026.

• Ongoing engagement with FDA, with a Type C meeting expected in the fall to clarify generalizability concerns before U.S. filing.

• FORWARD study in the U.S. is open-label, focusing on pediatric enrollment and understanding real-world patient management.

Commercialization and market strategy

• Intends to monetize ex-U.S. rights for GBS, seeking partners with established hospital-based neuro businesses in Europe.

• U.S. commercialization strategy targets a concentrated hospital market, emphasizing payer engagement and education to streamline patient flow.

• Health economics analysis for GBS to be released later this year, highlighting greater patient numbers and system costs than previously recognized.