Annexon (ANNX) Goldman Sachs 47th Annual Global Healthcare Conference 2026 summary

Key clinical program updates

• Anticipates pivotal phase III data for vonaprument in geographic atrophy in Q4 2026, with a focus on vision preservation as the primary endpoint, differentiating from prior therapies targeting anatomical markers.

• Phase II data showed significant preservation of photoreceptor neurons and visual acuity, especially in the central macula, despite not meeting the primary anatomical endpoint.

• Patient selection in phase III targets those in the rapid vision loss phase, aiming to maximize the likelihood of demonstrating benefit on BCVA 15-letter loss.

• The phase III trial is powered above 90% for two independent sub-studies, aligned with FDA and EMA requirements.

• Safety profile of vonaprument is favorable, with minimal systemic exposure due to localized ocular delivery.

Scientific rationale and differentiation

• Vonaprument’s neuroprotective mechanism targets C1q to protect photoreceptors, addressing the root cause of vision loss in GA rather than just anatomical changes.

• Preclinical and clinical data support C1q inhibition as a viable neuroprotective strategy, with evidence of improved retinal function and anatomy.

• The drug’s small size and low viscosity allow for easier administration and reduced side effects compared to existing therapies.

• Vision preservation is emphasized as a more meaningful clinical outcome than lesion growth, aligning with regulatory and physician perspectives.

Commercial and market strategy

• Plans to commercialize vonaprument independently in the U.S., leveraging a focused sales force targeting retina specialists.

• Sees opportunity to expand market penetration beyond the current 20-25% of GA patients treated, driven by functional benefit and new imaging techniques.

• Physician activation and education are key to shifting the market narrative from anatomical to functional outcomes.

• Expects to displace current therapies due to higher efficacy and better patient experience.