There are currently no approved treatments to protect vision in people who have geographic atrophy (GA). But hope may be on the way for the 5 million people worldwide who have GA, the AAO said in a press release.
A new study presented at AAO 2024, shows that patients treated monthly with an investigational drug ANX007 (Annexon Biosciences) cut their risk of vision loss by 72%. Data also showed that significant areas of the retina critical for good vision were protected, the AAO said.
“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.”
Although the FDA 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, said the AAO.
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 GA.
In a Phase 2 clinical 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% in patients treated monthly (P = .006), and by 48% in patients treated every other month (P = .064).
Treatment also protected photoreceptors in the central fovea. After one year of treatment, ANX007 reduced ellipsoid zone area loss by approximately 30% in patients treated every month or every other month, compared with patients 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, the AAO said.
