Annexon (ANNX) investor relations material

Platform and pipeline overview

• Developing a next-generation complement platform targeting the neuroinflammatory cascade at the source, aiming for differentiated outcomes in vision, muscle strength, and cognition across multiple diseases.

• Leading programs include late-stage assets in Guillain-Barré syndrome (GBS) and geographic atrophy (GA), with a first-in-class oral small molecule (ANX1502) in early-stage development for autoimmune conditions.

• GA program is the only one to show significant vision preservation and photoreceptor protection on all endpoints, setting it apart from downstream approaches.

• Multiple regulatory and clinical milestones are expected in 2026, including filings and pivotal data readouts.

Guillain-Barré syndrome (GBS) program

• GBS affects 22,000–25,000 patients annually in the U.S. and Europe, with over 90% receiving treatment, representing a significant commercial opportunity.

• Pricing is estimated at $100,000–$150,000 per therapy course, with low cost of goods (<3%) and a concentrated sales model targeting fewer than 200 hospitals.

• Phase III data showed a safety profile similar to placebo and superior efficacy to standard care, with rapid functional recovery and reduced hospital stays.

• Regulatory filing in Europe is planned for January 2026, with U.S. filing contingent on additional FDA discussions and provision of U.S. patient data.

• An open-label study is proactively enrolling U.S. and European patients to address regulatory expectations, with initial data to be shared in cohorts.

Geographic atrophy (GA) program

• Phase III enrollment completed ahead of schedule with 650 patients across 30+ sites in the U.S. and Europe.

• The program targets the neurodegenerative root of GA, aiming to preserve photoreceptors and functional vision, unlike competitors focused on RPE cells.

• Regulatory strategy shifted from a head-to-head to a single protocol with two sub-analyses, increasing statistical power and efficiency.

• Approval in Europe requires one positive study, while the U.S. considers the totality of evidence from both sub-analyses.

• Primary endpoint is 15-letter best corrected visual acuity change, a clinically meaningful and regulatory-accepted measure.