ANX007: Neuroprotective Efficacy in Geographic Atrophy Presented at ARVO 2024

ANX007, a novel treatment developed by Annexon, Inc., has shown promising results in protecting against vision loss in patients with geographic atrophy (GA), an advanced form of age-related macular degeneration. The drug, a non-pegylated antigen-binding fragment (Fab), is designed to block C1q, a key initiator of the classical complement pathway, which is implicated in neurodegeneration. In the Phase 2 ARCHER trial, ANX007 demonstrated a significant reduction in photoreceptor loss and a meaningful slowing of lesion growth, particularly in the fovea, which is crucial for visual acuity.

The treatment has been well-tolerated, with no increase in choroidal neovascularization or reported cases of vasculitis. The Phase 2 data also revealed that ANX007 provided broad-based protection against vision loss, with statistically significant and dose-dependent protection observed in best corrected visual acuity (BCVA) measurements. Furthermore, ANX007 showed protection for both foveal and non-foveal patients and in low light settings, indicating its potential to be a valuable addition to the current treatment landscape for GA.

Preclinical studies have further supported the neuroprotective mechanism of ANX007, showing that C1q blockade protected photoreceptor synapses and preserved retinal function. This aligns with the findings from the ARCHER trial and enhances the understanding of how ANX007 could protect these critical structures and maintain visual function in GA patients.

Annexon is planning to initiate two pivotal Phase 3 trials for ANX007 in mid-2024. The ARCHER II trial will be a global pivotal Phase 3 study against a sham control, while the ARROW trial will be a head-to-head comparison with SYFOVRE®. These trials aim to confirm the Phase 2 findings and highlight the unique neuroprotective action of ANX007.

GA is a significant cause of blindness in the elderly, affecting millions worldwide. It is characterized by the loss of photoreceptor synapses and cells in the outer retina, leading to severe vision impairment. With no approved therapies shown to significantly prevent vision loss in clinical trials, the development of effective treatments like ANX007 is of utmost importance.

Annexon Biosciences is a biopharmaceutical company focused on developing novel therapies for various diseases driven by classical complement-mediated inflammation. The company's approach targets C1q to halt the inflammatory cascade, offering potential benefits across a range of conditions. With positive data from the ARCHER trial and a clear path forward with upcoming Phase 3 trials, Annexon is poised to make significant contributions to the treatment of GA and other neuroinflammatory diseases.