Annexon (ANNX) 25th Annual Needham Virtual Healthcare Conference summary

Strategic priorities and milestones

• 2026 is positioned as a pivotal year, with multiple late-stage registrational programs advancing after a 12-year development journey.

• Key 2026 milestones include anticipated European approval for GBS in early 2027, FDA BLA filing for GBS in 2024, and phase III readouts for both ANX1502 and vonaprument.

• ANX1502, a first-in-class oral small molecule, is in proof-of-concept studies with data expected in 2024.

• Vonaprument’s phase III readout in geographic atrophy (GA) is expected in Q4 2024.

Pipeline and clinical program insights

• Vonaprument targets C1q in the classical complement pathway, aiming to halt inflammation at the disease site, differentiating it from C3 and C5 approaches.

• Phase II ARCHER trial in GA showed significant preservation of photoreceptors and vision, despite missing the primary endpoint on lesion growth at 12 months.

• Phase III ARCHER II trial is enriched for patients likely to lose vision, with a primary endpoint of 15-letter BCVA loss at 15 months and robust powering assumptions.

• Secondary endpoints include structural (EZ) and functional (low-light visual acuity) measures, with regulatory alignment on primary endpoints.

Regulatory and commercial strategy

• For GA, FDA and EMA require statistical significance on BCVA 15-letter loss; the phase III trial is designed to meet these standards.

• FDA recommended a single protocol with two sub-study analyses, each individually powered, simplifying execution and reducing risk.

• In GBS, European approval is pending, while FDA requires additional PK/PD and efficacy data from Western patients, being collected in the FORWARD study.

• ANX1502’s target product profile includes BID dosing and normalization of complement markers and bilirubin, with proof-of-concept data expected in 2024.