Emerging Treatments Offer New Hope for Dry and Wet Age-Related Macular Degeneration

An estimated 20 million Americans over age 40 are living with age-related macular degeneration (AMD), the leading cause of vision loss in people 60 and older. Over the past two decades, several treatments have been developed to slow vision loss in AMD.

There are now drugs on the market to treat advanced forms of dry AMD, in which waste deposits called drusen accumulate in the retina, damaging the macula. Lifestyle modification and supplement regimens, like AREDS2, are also recommended for many individuals in early to intermediate stages of dry AMD. Additionally, there are treatments for wet AMD, which is characterized by abnormal blood vessels that leak into the retina, causing a loss of central vision.

While these treatments have been proven to slow the progression of AMD, scientists working on the next generation of treatments are aiming higher, and in some cases, even hoping to restore vision.

Take a closer look at some of the pivotal Phase 3 trials happening in 2025 below. A comprehensive list of interventional clinical trials is available here, or you can search for trials in your area.

The Macular Degeneration Treatment Pipeline

New Targeted Drugs Show Promise in Wet AMD

The progression of wet AMD can be slowed by inhibiting (slowing down or blocking) the protein vascular endothelial growth factor (VEGF), and drugs that inhibit this protein have become the standard of care in treating the disease. However, receiving anti-VEGF treatments can be a hassle. Most people with wet AMD need to receive eye injections every four to 12 weeks.

To reduce the need for frequent injections, some drug developers are pursuing new targets that work upstream from VEGF. Two drugs in late-stage trials inhibit enzymes called tyrosine kinases, for example. Blocking these enzymes interferes with VEGF signaling, which in turn helps prevent the growth of abnormal blood vessels.

Axpaxli is an implant containing a tyrosine kinase inhibitor that’s designed to be injected into the eye once or twice a year. In an early trial, 60% of patients who received the implant did not need any additional treatment for a full year. Initial results from a Phase 3 trial are expected later this year. Another experimental implant containing a tyrosine kinase inhibitor, Duravyu, showed lasting results for 14 months in an early trial. The Phase 3 study of that treatment is expected to be completed in 2027.

Other promising late-stage treatments aim to combat wet AMD by combining VEGF targeting with other approaches to reducing retinal damage. An investigational eye injection called tabirafusp tedromer contains both a VEGF inhibitor and a second drug that inhibits an inflammatory protein called IL-6. In early trials, patients who received the drug showed sustained improvements in visual acuity. A Phase 3 trial will be completed next year.

A second two-pronged treatment, efdamrofusp alfa, blocks VEGF and the complement pathway, a network of proteins that activate each other in the blood and retina, causing inflammation and cell death. Participants in a Phase 2 trial who received the drug every 12 weeks showed improvements in visual acuity. A Phase 3 trial is now underway.

Gene Therapy Advances for Wet Macular Degeneration

What if a single injection in the eye could inhibit VEGF and slow the progression of wet AMD for a lifetime? That’s the goal of gene therapy. There are now three gene therapies for AMD in late-stage clinical trials. The most advanced candidate, ABBV-RGX-314, contains a gene that produces a protein that destroys VEGF. In early trials, the therapy was safe and produced stable or improved visual acuity over two years in people with wet AMD. Most did not require additional injections over that time period. A larger Phase 3 trial is ongoing, with results expected next year.

Two additional gene therapies for wet AMD recently started Phase 3 trials: ixo-vec and 4D-150. Among people participating in a Phase 2 trial testing two different doses of ixo-vec, less than half needed supplemental injections of anti-VEGF treatments in the year following the gene therapy treatment, and most who did need them received fewer than two injections. In a Phase 2 trial of 4D-150, 57% of people were injection-free one year after receiving the gene therapy.

Making Inroads in Dry Macular Degeneration

While there are still no treatments on the market to treat the earliest stages of dry AMD (other than the AREDS2 supplementation to assist in slowing down progression to later stages), there are innovative therapies coming for more advanced stages of the disease. Several treatments are in clinical trials to treat geographic atrophy (GA), an advanced form of dry age-related macular degeneration in which retinal cells die, causing a blind spot in the visual field. There are two drugs on the market to slow the progression of GA, Syfovre and Izervay, eye injections that work by inhibiting complement proteins (which are important for immunity but can cause inflammation damage if levels get imbalanced).

Three drugs in Phase 3 clinical trials take novel approaches to slowing the progression of GA and are showing promise in improving upon the first generation of treatments. AVD-104 targets not only the complement system, but also additional inflammation caused by immune cells called macrophages/microglia. It is anticipated that this will be even more effective than the current drugs that only target the complement. In an early trial, a single injection in the eye prompted a 48% reduction in GA lesion growth after three months, as well as an improvement in vision.

ANX007 is an eye injection that works by inhibiting C1q, a protein complex that activates the complement system. In a Phase 2 trial, the drug significantly protected against vision loss, and the level of protection grew over time. A Phase 3 trial is ongoing, with initial data expected in the second half of 2026.

Elamipretide, which is in late-stage clinical trials for geographic atrophy, is distinctive in that it’s a once-daily drug that people can inject themselves subcutaneously, or just under the skin. It works by stabilizing mitochondria—the power centers inside of cells—through interaction with cardiolipin proteins coating the inner wall, with the goal of relieving stress that can lead to cell death. Nearly 15% of people who received the drug in a Phase 2 trial showed improved vision after 48 weeks, versus just 2% of people who received a placebo.

Another drug designed for people with dry AMD to take at home is tinlarebant, which is in Phase 3 trials to treat both geographic atrophy and Stargardt disease, an inherited form of macular degeneration. The drug, which is taken orally, works by reducing levels of RBP4, which is a vitamin A transport protein, which in turn reduces levels of vitamin A-based toxins (known as bisretinoids) that can build up in the eye, to ultimately reduce the cell death and vision loss caused by these disease byproducts. The FDA granted breakthrough therapy status to tinlarebant based on early data from a Phase 3 trial in a particular type of Stargardt disease, called STGT1, showing that the drug was well tolerated and stabilized visual acuity in most people participating in the trial. Clinical trials are currently ongoing for both GA and STGT1.

Summary

New wet AMD therapies in development—including long-lasting implants, gene therapies, and combination drugs—aim to reduce treatment burden and, in some cases, even restore vision. For the dry form of the disease, promising new treatments are being tested that may better protect vision and slow the progression to wet AMD. With several of these treatments now in Phase 3 clinical trials, people with AMD can look forward to a host of new treatment options in the near future.

Scientists funded by BrightFocus’ Macular Degeneration Research program are investigating several novel approaches to treating the disease. Find out more about the exciting work we’re funding below and learn how to support our work.

• Identifying FDA-Approved Drugs to Reverse Dry AMD

• Dark Matter: Developing a Nanoantioxidant-Based Therapeutic System for AMD

• Stem Cell Therapy for Early AMD

• The Development of a Transplant-Independent Therapy for RPE Dysfunction

• A Novel Method for Treating Wet AMD Reversibly with a Single Intraocular Injection

• Regeneration of Cone Photoreceptors in the Human Retina

• New Drug Delivery Approach to Transform Macular Degeneration Treatment

• A Novel Implantable Device to Treat Wet Macular Degeneration

• A Blood Test to Measure Genes Associated with Macular Degeneration

• Storing Fat in the Eye: A Pathway for Tackling AMD

• Tracking Biological Responses to Lifestyle Changes in AMD Patients

Disclaimer: This article focuses on Phase 3 trials for macular degeneration, primarily taking place in the U.S.