AAO 2024: New drug shows promise as first to protect vision in patients with dry AMD

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Key Takeaways

ANX007 significantly reduces vision loss risk in geographic atrophy patients by 72% with monthly treatment.
The drug targets the complement system, blocking C1q to prevent neurodegeneration and preserve retinal anatomy.
ANX007 has received Fast Track status from the FDA and PRIME designation from the EU.
A global Phase 3 trial is underway, with results expected in the second half of 2026.

According to a study presented at the American Academy of Ophthalmology’s annual meeting in Chicago, investigational treatment reduced vision loss and protected key structures in the eye essential for vision.

(Image credit: Adobe Stock/NickArt)

Currently, there are no approved treatments to preserve vision in people diagnosed with geographic atrophy, a severe and advanced form of dry age-related macular degeneration (AMD) that can result in permanent vision loss. However, there soon could be hope for the 5 million people worldwide affected by this condition.

A new study, being presented at the 128th annual meeting of the American Academy of Ophthalmology in Chicago, reveals that patients receiving monthly treatment with the investigational drug ANX007 reduced their risk of vision loss by 72 percent. The data also showed that significant areas of the retina essential for maintaining good vision were preserved.

“The data is impressive, showing that ANX007 significantly protects against vision loss from geographic atrophy and helps preserve important retinal anatomy to achieve this,” lead researcher Rahul N. Khurana, MD, of Northern California Retina-Vitreous Associates, said in an Academy news release. “This offers a lot of hope for our patients with geographic atrophy who are struggling for clinically meaningful treatments in practice.”

Geographic atrophy causes regions of cells in the retina to waste away and die resulting in a growing blind spot in the visual field. This makes it difficult to drive, read, and even recognize faces. Geographic atrophy causes about 20 percent of legal blindness in North America.

Although the FDA approved two new drugs last year that can slow the progression of geographic atrophy, there are still no treatments available that can protect vision from this debilitating condition, according to the release

ANX007, a novel neuroprotective agent, offers a potential breakthrough. Injected directly into the eye, it works by targeting the complement system, specifically blocking the immune molecule C1q, which is believed to drive neurodegeneration. By inhibiting C1q, researchers hope to prevent synapse loss, inflammation, and neuronal damage that lead to vision loss in geographic atrophy.

In a Phase 2 clinical trial, 161 patients were randomly assigned to receive either a 5 mg dose of ANX007 monthly, every other month, or a sham treatment. After one year, patients treated monthly had a 72 percent reduced risk of losing three or more lines of vision on an eye chart (P = .006), while those treated every other month saw a 48 percent reduction in risk (P = .064).

The treatment also preserved photoreceptors in the central fovea, the light-detecting neurons in the retina that are critical for vision. Protecting these photoreceptors is vital for maintaining eyesight. After one year of treatment, patients receiving ANX007 either monthly or every other month experienced approximately a 30 percent reduction in ellipsoid zone area loss compared to those who received sham injections.

Based on this and other data, the FDA has granted the treatment Fast Track status, and the European Union has given it Priority Medicine (PRIME) designation to speed up investigation of ANX007.

A global, randomized, sham-controlled Phase 3 trial of ANX007 is currently underway, with topline data expected in the second half of 2026.