Annexon (ANNX) Jefferies London Healthcare Conference 2024 summary

Company overview and differentiation

• Focuses on the classical complement system, specifically targeting C1q to provide comprehensive protection against neuroinflammatory and autoimmune diseases in the eye and brain.

• Most advanced program is for Guillain-Barré syndrome (GBS), with additional programs in geographic atrophy (GA) and a small molecule oral therapy for autoimmune conditions.

• Claims best-in-class potential by targeting complement upstream, with unique programs in GBS and GA, and the only small molecule in the classical complement space.

GBS Phase III program and regulatory strategy

• Completed the first placebo-controlled GBS Phase III study in nearly 40 years, showing rapid C1q blockade and significant clinical improvements from week 1 to week 26.

• Regulatory strategy includes matching Southeast Asian study patients to Western populations using the IGOS natural history dataset, with full regulatory sign-off.

• Comparison to IVIG is ongoing, focusing on key endpoints like muscle strength at week 1 and GBS disability score at week 4 and 8.

• Phase III data validated the optimal treatment window, with shorter complement inhibition proving more effective.

• Ongoing FDA discussions, with a pre-BLA meeting planned for early next year and BLA filing in the first half of next year.

Market opportunity and clinical advantages

• GBS market is sizable, with 7,000–22,000 patients annually in the U.S. and no approved drugs, presenting a significant commercial opportunity.

• IVIG, the current standard, lacks placebo-controlled studies, is dosed over five days, and has a less favorable safety profile compared to the new therapy.

• The new therapy is dosed over hours and has a safety profile similar to placebo, with fewer serious side effects.