Annexon (ANNX) 43rd Annual J.P. Morgan Healthcare Conference 2025 summary

Strategic vision and scientific platform

• Focused on transforming the complement landscape with therapies for complement-mediated diseases, leveraging a clinically validated platform with broad applicability across neuroinflammatory, neurodegenerative, autoimmune, and ophthalmic conditions.

• Targeting C1q to halt neuroinflammation, with over 20 years of research and more than 30 novel assays developed to understand complement's role in disease.

• Strategic drug development approach starts with signal-finding studies, leading to game-changing data and a robust, diversified pipeline.

• Flagship programs have de-risked the next wave of candidates, supporting long-term value creation.

• Cash runway extends into the second half of 2026, supporting achievement of key milestones including BLA submission for ANX005 and completion of ARCHER II enrollment for ANX007.

Flagship programs and clinical progress

• ANX005 for Guillain-Barré Syndrome (GBS) showed rapid and durable improvement in muscle strength and functional recovery, outperforming standard of care (IVIg) in both phase III and real-world studies, with reduced ICU admissions, ventilation needs, and time to recovery.

• BLA submission for ANX005 planned for the first half of 2025, with ongoing regulatory discussions in the US and EU.

• ANX007 for geographic atrophy demonstrated significant vision preservation and robust photoreceptor protection in phase II, with PRIME designation in Europe and ongoing global phase III enrollment.

• ANX1502, a first-in-class oral C1s inhibitor, achieved target drug levels and tolerability in Phase 1, with proof-of-concept data in cold agglutinin disease expected in Q1 2025.

• The pipeline includes additional programs in Huntington's, ALS, and lupus nephritis, supporting a diversified late-stage portfolio.

Commercial and operational strategy

• GBS market is highly concentrated, with 440 hospitals and 3,000 physicians accounting for most US cases, enabling a focused commercial approach.

• Pre-commercial activities include intensive physician education, managed care engagement, and distribution planning to ensure rapid access upon approval.

• Drug distribution strategies include buy-and-bill, consignment, and rapid drop shipment, supported by a stable product shelf life.

• Ongoing publication and conference presentations to build awareness and support adoption among clinicians.

• Team culture emphasizes execution, adaptability, and a patient-centric mission, with a track record of on-time, on-budget delivery.