Annexon to Present Phase 2 ANX007 Data on Vision and Photoreceptor Protection in Geographic Atrophy at Floretina-ICOOR 2024

Annexon, Inc., a biopharmaceutical company, is presenting analyses of its drug ANX007 from the completed Phase 2 ARCHER trial for geographic atrophy (GA) at the Floretina-ICOOR 2024 meeting in Florence, Italy. ANX007 is designed to inhibit C1q locally in the eye using an intravitreal formulation. This inhibition aims to prevent vision loss and preserve photoreceptors in patients with GA.

Key presentations at the event include:
- "Unlocking Structure/Function Relationships in GA: Central Subdomain Preservation and Visual Acuity Protection with C1q Inhibition" by Dr. Jeffrey S. Heier.
- "Prevention of Visual Acuity Loss and Preservation of Photoreceptors by ANX007 in Dry Age-Related Macular Degeneration (AMD)/GA in the Phase 2 ARCHER Trial, Including in Patients with Less Advanced Disease" by Dr. Paulo Eduardo Stanga.
- An Annexon Symposium on "Protection of Vision and Structure in GA," which involves discussions on neurodegeneration, visual acuity protection, and the relationship between structure and function related to vision.
- "C1q inhibition: Functional and Structural Protection in dry AMD / GA via a Novel Neuroprotective Mechanism" by Douglas Love, President and CEO of Annexon.

ANX007 has shown promising results in the Phase 2 ARCHER trial. It's designed as a first-in-kind therapeutic antibody to selectively block C1q, the molecule that initiates the classical complement pathway and is a key driver of neurodegeneration. This pathway's activation can lead to synapse loss, inflammation, and neuronal damage, causing vision loss in dry AMD/GA. Animal models have demonstrated that ANX007 can protect against the loss of photoreceptor synapses and cells, preserving retinal function.

The randomized, multi-center, double-masked, sham-controlled Phase 2 ARCHER trial showed that ANX007 provided significant protection against vision loss in a broad patient population with GA. It demonstrated a time and dose-dependent protection from vision loss, as measured by the loss of 15 letters on an eye chart with best-corrected visual acuity (BCVA). This protection was also observed in other measures of visual function, including low luminance visual acuity (LLVA) and low luminance visual deficit (LLVD). The treatment effect of ANX007 increased over the course of the treatment period, with benefits maintained during a six-month off-treatment period, although the rate of vision decline began to parallel that of the sham group after treatment stopped.

ANX007 also showed protection of retinal structures crucial for vision, including photoreceptors and retinal pigment epithelial cells, as observed through optical coherence tomography (OCT) and fundus autofluorescence (FAF). The drug was generally well-tolerated, with no increase in choroidal neovascularization (CNV) rates and no retinal vasculitis events.

Dry AMD is the most common form of AMD, with GA being an advanced stage causing severe vision loss. It affects millions globally, with significant impacts on daily life and emotional well-being. Effective treatments that can preserve vision are still needed, as no currently approved therapies have shown significant prevention of vision loss in clinical trials.

The Phase 3 ARCHER II trial is a global, randomized, double-masked, sham-controlled study aiming to enroll approximately 630 patients with GA secondary to age-related macular degeneration. The primary endpoint is the prevention of a 15-letter loss in BCVA. Secondary endpoints include safety and measures of visual function. Topline data are expected in the second half of 2026.

Annexon Biosciences is focused on developing treatments for neuroinflammatory diseases by targeting C1q, aiming to prevent tissue damage and loss caused by misdirected inflammation. The company's pipeline includes therapies for autoimmune, neurodegenerative, and ophthalmic diseases, addressing the needs of millions of people worldwide.