Annexon (ANNX) investor relations material

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 primary neuroprotective marker.

• Vonaprument showed 50-60% protection in the central fovea, outperforming other therapies in this critical region.

Clinical trial design and execution

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

• Study design incorporates learnings from phase II, including exclusion of patients with very poor vision and matching key subgroups.

• High compliance (>90%) and low dropout (<10%) attributed to strong physician/patient belief and simple, tolerable dosing.

• Powering strategy leverages over-enrollment and extended follow-up to maximize statistical strength at both endpoints.

• Data integrity maintained by masking; DMC will report only win/go-forward/futility at month 15, with full unmasking after month 24 if all endpoints are met.