Annexon Shares ANX007 Neuroprotection Data for Vision in Geographic Atrophy at ARVO 2024

Annexon, Inc., a biopharmaceutical company listed on Nasdaq under the ticker ANNX, has recently shared promising findings regarding their therapeutic candidate ANX007 in the treatment of geographic atrophy (GA). GA is an advanced form of dry age-related macular degeneration (AMD) that severely affects the vision of millions of people worldwide, leading to significant loss of independence and emotional distress for those afflicted.

ANX007, a first-in-class, non-pegylated antigen-binding fragment (Fab), is designed to inhibit C1q, a key component in the classical complement cascade associated with neurodegenerative diseases. The drug has been specifically formulated for intravitreal administration, targeting the eye to halt the activation of this pathway. This therapeutic candidate has the distinction of being the first GA treatment to receive Priority Medicine (PRIME) designation from the European Medicines Agency, highlighting its potential as a significant advancement in combating this debilitating condition.

The latest data presented at the Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting, including new analyses from the Phase 2 ARCHER trial, reveal significant benefits of ANX007 in preserving vision and retinal structures in patients with GA. The data showed that ANX007 treatment resulted in broad-based protection against vision loss, particularly in challenging conditions such as low light settings and in both foveal and non-foveal patients. Specifically, the treatment demonstrated a 73% relative risk reduction in losing 15 letters or more in the best-corrected visual acuity (BCVA) tests, signifying a time-dependent protective effect.

One of the standout findings of the study was the significant reduction in photoreceptor loss over a 12-month period as measured by OCT Ellipsoid Zone assessment, a direct measure of photoreceptor structure. The treatment also slowed the progression of retinal pigment epithelium (RPE) loss, particularly in areas near the fovea, which are closely linked to visual acuity. More than 50% of patients treated with ANX007 experienced reduced significant RPE loss in the fovea compared to the overall population.

ANX007's efficacy extends beyond clinical measures of visual acuity. Preclinical studies reinforce these findings by demonstrating that C1q blockade via ANX007 protects photoreceptor synapses and preserves retinal function. In animal models simulating GA, inhibiting C1q prevented photoreceptor damage, further supporting the drug's neuroprotective mechanism.

The safety profile of ANX007 has also been encouraging, with no reported increase in choroidal neovascularization (CNV) or instances of retinal vasculitis in treated patients compared to the sham group.

Douglas Love, President and CEO of Annexon, expressed optimism about the potential of ANX007, emphasizing that the combined clinical and preclinical data support the drug’s unique ability to preserve visual function by safeguarding photoreceptor synapses. Dr. David Boyer, from the Retina-Vitreous Associates Medical Group in California, highlighted the statistically significant preservation of both vision and underlying anatomical structures associated with ANX007 treatment.

Looking forward, Annexon is planning two major Phase 3 trials to further validate the efficacy of ANX007 in GA. The global pivotal Phase 3 ARCHER II trial, expected to start mid-2024, will compare ANX007 to a sham control. Additionally, the pivotal Phase 3 ARROW trial, slated to begin in the second half of 2024, will evaluate ANX007 against SYFOVRE®, a pegcetacoplan injection.

These forthcoming trials aim to confirm the findings from the Phase 2 ARCHER trial, which showed ANX007 as a promising treatment for preserving vision in GA patients through its novel neuroprotective mechanism.

Overall, the advancements with ANX007 reflect Annexon’s commitment to addressing unmet medical needs in neuroinflammatory diseases, offering hope to millions of patients affected by GA and potentially transforming the therapeutic landscape for other related conditions.