Annexon to Present Promising Data on ANX007's Neuroprotective Effects in Geographic Atrophy at Major Ophthalmology Conferences
核心洞察
Phase 2 ARCHER trial data demonstrates ANX007's ability to preserve vision in patients with dry AMD (搜索) and geographic atrophy (搜索), showing significant protection across multiple visual function measures.
ANX007, a first-in-kind C1q (搜索) inhibitor, has received Fast Track designation from the FDA and is the first GA treatment to receive PRIME designation in the EU, highlighting its potential therapeutic advantage.
The global Phase 3 ARCHER II trial is currently enrolling approximately 630 patients, with topline data expected in the second half of 2026.
Annexon, Inc. (Nasdaq: ANNX) has announced upcoming presentations on the neuroprotective effects of its investigational therapy ANX007 at two major ophthalmology conferences in May 2025. The presentations will highlight data from the Phase 2 ARCHER trial supporting ANX007's potential to preserve vision in patients with dry age-related macular degeneration (搜索) (AMD (搜索)) with geographic atrophy (搜索) (GA).
The company will present at both the Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting in Salt Lake City, Utah (May 4-8) and the 2025 Retina World Congress in Fort Lauderdale, Florida (May 8-11).
ANX007 is described as a first-in-kind, non-pegylated antigen-binding fragment (Fab) designed to block C1q (搜索) locally in the eye with an intravitreal formulation. According to Annexon, it is the only investigational therapy in GA to demonstrate significant vision preservation on both best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA) endpoints.
Mechanism of Action and Clinical Significance
ANX007 selectively inhibits C1q (搜索), the initiating molecule of the classical complement pathway that plays a key role in neurodegeneration. In advanced dry AMD (搜索) or GA, C1q binds to photoreceptor synapses, triggering classical pathway activation that leads to synapse loss, inflammation, and neuronal damage resulting in vision loss.
Dr. Ajay E. Kuriyan, Retina Specialist at Wills Eye Hospital and Associate Professor of Ophthalmology at Sidney Kimmel Medical College of Thomas Jefferson University, will present a poster titled "Microglia-induced neuronal injury attenuation with C1q (搜索) Inhibition: Outcomes in Geographic Atrophy (搜索) (GA) and Huntington's Disease (搜索) (HD)" at the ARVO Annual Meeting on May 7.
At the Retina World Congress, Dr. Priya Vakharia, Retina Specialist at Retina Vitreous Associates of Florida, will deliver an oral presentation on "Visual Function Outcomes in the Phase 2 ARCHER Trial of ANX007, a C1q (搜索) Inhibitor, in Participants with dry AMD (搜索) with GA: Number Needed to Treat Analysis" on May 10.
Phase 2 ARCHER Trial Results
The Phase 2 ARCHER trial was a randomized, multi-center, double-masked, sham-controlled study that evaluated ANX007 in patients with dry AMD (搜索) and GA. Results demonstrated consistent protection against vision loss across multiple measures.
Key findings from the trial include:
- 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 in other prespecified measures of BCVA and visual function
- Treatment effect that increased over the course of the on-treatment portion of the study
- Maintained benefit against vision loss during the six-month off-treatment period
- Protection of key retinal structures important for vision, including photoreceptors and retinal pigment epithelial cells
- Generally well-tolerated safety profile through month 12, with no increase in choroidal neovascularization rates between treated and sham arms
Regulatory Status and Phase 3 Trial
ANX007 has received Fast Track designation from the U.S. Food and Drug Administration (FDA) and is the first therapeutic candidate for GA to receive Priority Medicine (PRIME) designation in the European Union. These designations acknowledge the potential therapeutic advantage ANX007 may offer over existing treatments or its benefit to patients without treatment options.
The company is currently enrolling patients in the Phase 3 ARCHER II trial, a global, randomized, double-masked, sham-controlled study expected to include approximately 630 patients with GA secondary to age-related macular degeneration. Participants will be randomized 2:1 to receive either a monthly dose of ANX007 or a sham procedure.
The primary endpoint of ARCHER II is the prevention of ≥15-letter loss of best corrected visual acuity (BCVA), representing three lines on the standard Early Treatment of Diabetic Retinopathy Study (ETDRS) eye chart. This is a well-established functional endpoint that has served as the basis for numerous ophthalmology drug approvals.
Secondary endpoints include safety, low-luminance visual acuity (LLVA), and photoreceptor integrity (EZ). Topline data from the Phase 3 trial are expected in the second half of 2026.
Unmet Medical Need in Geographic Atrophy
Dry AMD (搜索) is the most common form of AMD, and geographic atrophy (搜索) represents an advanced form of this condition. GA is a leading cause of blindness in the elderly, affecting an estimated one million people in the United States and eight million people globally.
The condition is characterized as a chronic progressive neurodegenerative disorder of the retina involving the loss of photoreceptor synapses and cells in the outer retina. It severely limits patients' independence and causes significant emotional hardship.
Despite the substantial impact of GA, effective treatments that preserve vision remain an unmet need. According to Annexon, no currently approved therapies have demonstrated significant prevention of vision loss in clinical trials.
About Annexon
Annexon Biosciences is developing therapeutics targeting classical complement-driven neurodegeneration as first-in-kind treatments for neuroinflammatory diseases affecting the body, brain, and eye. The company's scientific approach focuses on C1q (搜索), aiming to stop the neuroinflammatory cascade in disease before it starts.
The company's pipeline spans three therapeutic areas – autoimmune, neurodegenerative, and ophthalmic diseases – with investigational drug candidates designed to address the unmet needs of over 8 million people worldwide.