AAO: New Drug May Be the First to Protect Vision in People With Dry AMD
Study shows investigational treatment reduced vision loss and protected key structures in the eye essential for vision. FDA has granted the treatment Fast Track status, and the European Union has given it Priority Medicine (PRIME) designation to speed up investigation.
There are currently no approved treatments to protect vision in people who have geographic atrophy, an advanced and severe form of dry age-related macular degeneration (AMD) that can lead to permanent vision loss. But hope may be on the way for the five million people worldwide who have geographic atrophy.
A new study that was presented at AAO 2024, the 128th annual meeting of the American Academy of Ophthalmology, shows that patients treated monthly with an investigational drug (ANX007 / Annexon Biosciences) cut their risk of vision loss by 72 percent. Data also showed that significant areas of the retina critical for good vision were protected.
Significant protection against vision loss
“The data is impressive, showing that ANX007 significantly protects against vision loss from geographic atrophy and helps preserve important retinal anatomy to achieve this,” said lead researcher Rahul N. Khurana, MD, of Northern California Retina-Vitreous Associates. “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 U.S. Food and Drug Administration last year approved two new drugs that can help slow worsening of the disease over time, there are currently no approved treatments that can protect vision from this devastating condition.
Novel neuroprotective agent
ANX007 is a novel neuroprotective agent that is injected into the eye. It works by blocking a part of the immune system known as the complement system, specifically the immune molecule C1q in the complement pathway. C1q is thought to be a key driver of neurodegeneration. The hope is that by blocking C1q it might be possible to prevent the synapse loss, inflammation and neuronal damage that results in vision loss from geographic atrophy.
In the Phase 2 ARCHER Trial, 161 patients were randomized to receive either a 5 mg dose of ANX007 every month, every other month, or sham treatment. After one year of treatment, the risk of losing three lines of vision or more as measured by an eye chart was reduced by 72 percent in patients treated monthly (P = .006), and by 48 percent in patients treated every other month (P = .064).
Protecting photoreceptors
Treatment also protected photoreceptors in the central fovea. Photoreceptors are neurons in the retina responsible for detecting light. Protecting photoreceptors is essential for protecting vision. After one year of treatment, ANX007 reduced ellipsoid zone area loss by approximately 30 percent in patients treated every month or every other month, compared with patients who received sham injections.
Fast Track status & Priority Medicine (PRIME) designation
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.