Annexon Biosciences presented analyses of ANX007 from the completed Phase 2 ARCHER trial in geographic atrophy (GA) at the Floretina-ICOOR 2024 meeting in Florence, Italy. ANX007, a first-in-kind, non-pegylated antigen-binding fragment (Fab), is designed to block C1q locally in the eye via intravitreal formulation.
The Phase 2 ARCHER trial results indicate that ANX007 provides statistically significant, time and dose-dependent protection from vision loss, measured by a loss of ≥ 15 letters on reading an eye chart with best corrected visual acuity (BCVA). This protection was also observed in other pre-specified measures of BCVA and visual function, including low luminance visual acuity (LLVA) and low luminance visual deficit (LLVD).
Key Findings from ARCHER Trial
Dr. Jeffrey S. Heier from Ophthalmic Consultants of Boston presented on "Unlocking Structure/Function Relationships in GA: Central Subdomain Preservation and Visual Acuity Protection with C1q Inhibition." The data highlighted the relationship between structural preservation and functional vision protection with ANX007.
Dr. Paulo Eduardo Stanga from The Retina Clinic London and Institute of Ophthalmology, University College London, UK, presented on "Prevention of Visual Acuity Loss and Preservation of Photoreceptors by ANX007 in Dry Age-Related Macular Degeneration (AMD)/Geographic Atrophy (GA) in the Phase 2 ARCHER Trial, Including in Patients with Less Advanced Disease." The presentation emphasized ANX007's ability to protect key retinal structures, including photoreceptors, as measured by optical coherence tomography (OCT), and to slow the loss of retinal pigment epithelial cells (RPE) near the fovea, as measured by fundus autofluorescence (FAF).
Annexon Symposium Highlights
An Annexon symposium, "Protection of Vision and Structure in GA," featured presentations from leading experts:
- Dr. Peter Kaiser from the Cleveland Clinic of Ohio discussed "C1q Driven Neurodegeneration: Impacts on Structure and Function."
- Dr. Charles C. Wykoff from the Research Institute at Houston Methodist, Weill Cornell Medical College, Retina Consultants of Texas, presented "ANX007: Visual Acuity Protection and Safety in the Phase 2 ARCHER Trial."
- Dr. Anat Loewenstein from Tel Aviv Medical Center spoke on "Linking Structure to Function: Protection of Vision-Associated Structures with ANX007."
ANX007: Mechanism and Clinical Significance
ANX007 is designed to selectively inhibit C1q, the initiating molecule of the classical complement pathway, which is a key driver of neurodegeneration in dry AMD and GA. By blocking C1q, ANX007 aims to prevent the aberrant activation of the classical pathway, thereby reducing synapse loss, inflammation, and neuronal damage that leads to vision loss. The ARCHER trial demonstrated that ANX007 treatment effect increased over time, suggesting a growing and durable treatment effect.
Douglas Love, President and Chief Executive Officer of Annexon, presented on "C1q inhibition: Functional and Structural Protection in dry AMD / GA via a Novel Neuroprotective Mechanism," further emphasizing the potential of ANX007.
Ongoing Phase 3 ARCHER II Trial
The Phase 3 ARCHER II trial is actively enrolling approximately 630 patients with GA secondary to age-related macular degeneration. Patients are randomized 2:1 to receive monthly doses of ANX007 or a sham procedure. The primary endpoint is the prevention of ≥15-letter loss of best corrected visual acuity (BCVA). Topline data are expected in the second half of 2026.
