Annexon (ANNX) investor relations material

Key program updates and clinical milestones

• Two major late-stage programs advancing: phase III in Guillain-Barré syndrome (GBS) and phase III in geographic atrophy (GA) with vonaprument, both showing strong clinical promise.

• GBS program: European approval filing underway, U.S. BLA filing expected soon, with FORWARD study in the West showing 100% response rate and strong safety profile.

• GA program: Phase III study (ARCHER II) added a 24-month dual endpoint, leveraging increased statistical power from over-enrollment and high patient retention.

• Data readouts for GA expected in Q4, with potential for European approval and U.S. discussions based on overall study success.

• Small molecule oral C1s program progressing, with phase II proof-of-concept data and next steps to be announced after ongoing analysis.

Clinical strategy and differentiation

• GA study uniquely focuses on vision preservation (BCVA 15 letter loss), differentiating from prior RPE structural studies and aiming for functional benefit.

• Mechanistic differentiation: C1q inhibition targets neurodegeneration earlier than C3/C5, potentially offering superior outcomes and earlier intervention.

• Vonaprument offers easier administration, lower viscosity, and potential for less frequent dosing, supporting lifecycle management and new IP.

• GBS program demonstrates rapid, meaningful clinical improvement, with a targeted commercial approach and strong health economic value.

• Oral C1s program leverages extensive assay development and aims to address large antibody-mediated autoimmune disorders.

Commercial outlook and financial position

• GA market opportunity seen as significant, with potential to replace existing therapies and expand into earlier-stage disease.

• GBS viewed as a blockbuster rare disease, with concentrated hospital-based commercial strategy and efficient footprint (~50 FTEs).

• Partnership strategy for ex-U.S. commercialization in GBS, with term sheets in place and announcement expected before year-end.

• Company funded into 2028, covering all late-stage programs, initial commercialization, and open-label extension studies.

• Ongoing education and advocacy efforts in GBS to support market uptake and regulatory engagement.