Annexon (ANNX) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Company overview and late-stage programs

• Focuses on complement inhibition to address neuroinflammation in diseases of the brain, body, and eye, with two late-stage programs: one in Guillain-Barré syndrome (GBS) and one in geographic atrophy (GA).

• Achieved success in a large phase III GBS study, with filings in Europe and planned U.S. submission.

• GA program is the only one globally to show significant vision preservation and photoreceptor protection in late-stage trials.

• Mission is to impact millions with neuroinflammatory diseases lacking disease-modifying therapies.

Geographic atrophy (GA) program insights

• GA affects 8 million globally; current drugs offer only symptomatic benefit, not vision preservation.

• Vonaprument demonstrated statistically significant, dose-dependent vision preservation in phase II, with a 73% reduction in risk of major vision loss at 12 months.

• Functional benefit is linked to photoreceptor neuron protection, a key differentiator from other therapies.

• Structural endpoints have shifted from RPE to ellipsoid zone (EZ), now recognized by regulators as the key neuroprotective marker.

• Vonaprument showed 50–60% protection of central foveal photoreceptors at one year, outperforming competitors.

Clinical trial design and execution

• Phase III ARCHER II study: 659 patients, 2:1 randomization (monthly vonaprument vs. sham), dual primary endpoints at months 15 and 24.

• Patient cohorts and enrollment criteria refined based on phase II learnings, excluding those with very poor vision.

• Dropout rate is below 10%, compliance above 90%, attributed to strong physician and patient belief and simple, tolerable dosing.

• Study is powered above 90% for U.S. sub-analyses and even higher for the European filing.

• Month 24 endpoint adds commercial and regulatory value, creating a competitive moat and leveraging increased power from over-enrollment and retention.