Annexon, Inc. announced that the European Medicines Agency (EMA) has selected vonaprument (formerly ANX007) to participate in the Product Development Coordinator (PDC) pilot program, marking a significant regulatory milestone for the potential first-in-class treatment for dry age-related macular degeneration with geographic atrophy.
The PDC pilot, launched by the EMA in July 2025, strengthens stewardship through development support activities by appointing a designated coordinator with therapeutic area expertise. The program assists Priority Medicine (PRIME) designation holders in navigating regulatory interactions, including expedited scientific advice, marketing authorization application submission readiness activities, and ad-hoc queries throughout development.
Unique Mechanism and Clinical Promise
Vonaprument represents a first-in-kind, non-pegylated antigen-binding fragment (Fab) designed to block C1q locally in the eye through intravitreal administration. The drug selectively inhibits C1q, the initiating molecule of the classical complement pathway and a key driver of neurodegeneration in advanced dry AMD.
"We are honored that vonaprument has been selected by the EMA to participate in the PDC pilot," said AJ Acker, senior vice president of regulatory, quality and clinical safety at Annexon. "This selection reflects the EMA's commitment to fostering development support of vonaprument through faster, more flexible and expert-driven mechanisms."
The therapy has achieved notable regulatory recognition, receiving both Priority Medicine (PRIME) designation in Europe and Fast Track Designation from the U.S. Food and Drug Administration. Vonaprument is the first therapeutic candidate for geographic atrophy treatment to receive PRIME designation in the EU and stands as the only investigational therapy in geographic atrophy to show significant vision preservation on the endpoints of best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA).
Phase 3 ARCHER II Trial Design
The pivotal Phase 3 ARCHER II trial represents a global, randomized, double-masked, sham-controlled study that has enrolled more than 630 patients with advanced dry AMD/geographic atrophy. Patients are randomized 2:1 to receive monthly vonaprument doses or sham procedures.
The primary endpoint focuses on preventing ≥15-letter loss of best corrected visual acuity, representing three lines on the standard Early Treatment of Diabetic Retinopathy Study (ETDRS) eye chart. This proportion of patients experiencing BCVA ≥15-letter loss serves as a well-established functional endpoint that has formed the basis for numerous ophthalmology drug approvals by both the FDA and EMA.
Secondary endpoints include safety assessments, low-luminance visual acuity (LLVA), and photoreceptor integrity (EZ). The primary analysis will occur at least 12 months from dosing, with topline data expected in the second half of 2026.
Phase 2 Clinical Success
In the randomized, multi-center, double-masked, sham-controlled Phase 2 ARCHER clinical trial, vonaprument demonstrated consistent protection against vision loss across multiple measures in a broad population of patients with dry AMD and geographic atrophy. The therapy provided statistically significant, time and dose-dependent protection from vision loss as measured by ≥15 letter loss on best corrected visual acuity (BCVA≥15).
Significant protection from vision loss was also demonstrated in other prespecified measures of BCVA and visual function, including low luminance visual acuity (LLVA) and low luminance visual deficit (LLVD). The treatment effect increased over the course of the on-treatment portion of the study, suggesting vonaprument may provide a growing and durable treatment effect over time.
The therapy also protected key retinal structures important for vision, including significant protection of photoreceptors as measured by optical coherence tomography (OCT) and supported by slowing of loss of retinal pigment epithelial cells (RPE) near the fovea, as measured by fundus autofluorescence (FAF). Vonaprument was generally well-tolerated through month 12, with no increase in choroidal neovascularization (CNV) rates between treated and sham arms and no events of retinal vasculitis reported.
Addressing Significant Unmet Medical Need
Dry age-related macular degeneration represents the most common form of AMD, with geographic atrophy as its advanced form and the leading cause of blindness in the elderly. These chronic progressive neurodegenerative disorders of the retina involve the loss of photoreceptor synapses and cells in the outer retina.
Geographic atrophy affects an estimated one million people in the United States and eight million people globally, severely limiting their independence and causing frustration, anxiety and emotional hardship. Effective treatments that preserve vision remain critically needed, as no currently approved therapies have been shown in clinical trials to significantly prevent vision loss.
The potential approval of vonaprument could represent the first treatment approved in both Europe and the United States for dry AMD with geographic atrophy based on protection of visual acuity and visual structures, addressing a substantial unmet medical need in ophthalmology.
