Annexon (ANNX) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Therapeutic strategy and pipeline overview

• Focus on targeting the classical complement pathway, specifically C1q, to address autoimmune and neurodegenerative diseases in the body, brain, and eye.

• Lead programs include ANX005 for Guillain-Barré syndrome (GBS), ANX007 for geographic atrophy (GA), and ANX1502, a first-in-class oral small molecule for autoimmune conditions.

GBS phase III data and regulatory pathway

• Phase III GBS study showed statistically significant improvement on the primary endpoint at week 8, with benefits seen as early as week 1 and sustained through week 26.

• Patients treated with ANX005 came off ventilators and regained ability to walk about a month earlier than placebo.

• Lower dose (30 mg/kg) was more effective than higher dose, aligning with the need for complement activity to return for recovery.

• Real-world evidence study matches Southeast Asian trial patients with Western patients using the IGOS dataset to demonstrate generalizability for regulatory approval.

• Outcomes data versus IVIG and matching analysis expected by end of year, supporting BLA submission in 1H 2025 and potential approval by year-end 2025.

Commercial opportunity and differentiation in GBS

• U.S. sees about 7,000 new GBS cases annually, with 15,000 in the EU; 90% receive IVIG, often in multiple courses.

• ANX005 offers single-infusion treatment, rapid effect, and a safety profile comparable to placebo, unlike IVIG's black box warning and lengthy infusion.

• Targeted commercial approach focuses on 400 hospitals treating 80% of U.S. GBS patients, especially in large states.