Annexon (ANNX) 2024 Wells Fargo Healthcare Conference summary

Scientific and clinical highlights

• Focused on targeting C1q in the classical complement pathway, differentiating from C3/C5 approaches, with robust data across autoimmune and neurodegenerative diseases, including Guillain-Barré syndrome (GBS) and geographic atrophy (GA).

• Phase III GBS data showed highly significant improvement on the Guillain-Barré Disability Scale at multiple time points, with patients recovering faster and leaving the hospital and ventilator 30 days sooner than placebo.

• Lower dose of the drug outperformed the higher dose, attributed to optimal complement inhibition during the acute disease phase, aligning with prior proof-of-concept and IVIG studies.

• Phase III GBS program conducted in Southeast Asia due to U.S. regulatory requirements, with comparability to U.S. patients being established via the IGOS dataset.

• GA program (ANX007) received PRIME designation in the EU, demonstrating statistically significant preservation of vision and structural impact in the center fovea, unmatched by competitors.

Regulatory and development strategy

• For GBS, regulatory path includes demonstrating comparability of Southeast Asian and U.S. patients using the IGOS dataset, with data and regulatory feedback expected by year-end 2024.

• Not required to show outcomes versus IVIG for approval, but will provide such data for commercial positioning and potential European requirements.

• Engaged in Type C FDA meetings to clarify regulatory expectations, aiming for a smooth pre-BLA process and considering breakthrough designation if data supports superiority over standard of care.

• GA phase III study is global, with strong alignment from both EMA and FDA, and recruitment is progressing well.

Market opportunity and commercialization

• GBS market estimated at 7,000 U.S. patients, 15,000 in Europe, with most receiving IVIG; rapid treatment is critical due to the acute nature of the disease.

• GBS treatment costs are driven by hospitalization and ICU stays, not just drug costs; the new therapy aims to reduce these by accelerating recovery.

• Commercial strategy leverages concentration of GBS cases in major states and centers of excellence, with plans for J-code reimbursement and active payer engagement.

• GA market in Europe is substantial, with over three million patients, and the program’s functional vision preservation is expected to drive adoption.