Annexon (ANNX) 44th Annual J.P. Morgan Healthcare Conference summary

Strategic Vision and Pipeline Overview

• Focused on immunotherapy for neuroinflammatory diseases using a proprietary C1q inhibition platform, aiming to halt neuroinflammation at its source and address significant unmet needs in large patient populations.

• Lead programs include ANX007/vonaprument for geographic atrophy in dry AMD and ANX005/tanruprubart for Guillain-Barré syndrome, both in late-stage development and targeting markets exceeding $10 billion annually.

• ANX1502, the first oral C1 inhibitor for autoimmune conditions, is advancing with proof-of-concept data expected in 2026.

• Multiple next-wave programs are in clinical development, with updates anticipated later in the year.

• Company is well-capitalized to fund key milestones into late 2027, supporting commercialization, pre-launch, and market education efforts.

Clinical and Pipeline Developments

• ANX007/vonaprument demonstrated 50–60% protection of photoreceptors and a 73% reduction in risk of vision loss at 12 months in phase II for GA, with robust protection of central retinal structure.

• Phase III ARCHER II trial for vonaprument is underway, powered >90%, with primary endpoint of best-corrected visual acuity at 15 months and global regulatory alignment.

• ANX005/tanruprubart is the first therapy in 40 years to show robust efficacy in GBS, with 90% of patients improving by week one and significant reductions in ICU and ventilator time.

• Early experience with tanruprubart in the US and EU suggests rapid, consistent effects; BLA filing planned for 2026.

• ANX1502 is advancing as a next-wave program for autoimmune indications, with proof-of-concept data in Cold Agglutinin Disease expected in 2026.

Market and Competitive Positioning

• C1q inhibition platform offers a differentiated approach, aiming to reset the standard of care in neuroinflammatory diseases with limited or no approved therapies.

• Market opportunity for GA is estimated at $7–10 billion annually, with over 8 million patients worldwide and no approved treatments for vision preservation.

• Tanruprubart addresses a significant burden in GBS, with over 150,000 cases annually and no FDA-approved therapies; current treatments are suboptimal.

• Vision-preservation medicines are projected to exceed $7 billion in global peak sales, with vonaprument offering a differentiated benefit-risk profile.

• Company is positioned to capture asymmetric value, drawing comparisons to major industry acquisitions in the complement inhibition space.