Retinal Disease Research Advances

Oct 6, 2021

Age-Related Macular Degeneration Research Advances

Recent developments in research on age-related macular degeneration.

Get updates on Retinal Disease Research Advances

Nutritional Supplement Reduces Risk of Advanced AMD

The Age-Related Eye Disease Study (AREDS) — a landmark investigation conducted by the National Eye Institute (NEI) — determined that antioxidant supplementation can slow the progression of AMD. The AREDS formulation is an over-the- counter antioxidant supplement recommended for people who are at risk of developing advanced forms of either dry or wet AMD. The formulation includes the antioxidants beta carotene, vitamin E, and vitamin C, as well as the nutrients zinc and copper.

The NEI recently completed a second AREDS study (AREDS2) to evaluate the potential benefits of the antioxidants lutein and zeaxanthin and the omega-3 fatty acids docosahexaenoic acid (DHA) and eicosapentaenoic acid (EPA).The results of AREDS2 showed that DHA and EPA did not confer additional benefit in reducing AMD risk. The researchers from AREDS2 did recommend that beta carotene in the original formula be replaced with lutein, because beta carotene can increase lung cancer risk in current and former smokers. For more information on the AREDS2 study, visit

Lucentis™ Preserves Vision in People with Wet AMD

Developed by Genentech, Lucentis is effective in reducing the risk of losing vision from the abnormal blood vessel growth under the retina associated with wet AMD. The drug was FDA approved in 2006. A two-year study showed that 95 percent of people with wet AMD who received monthly injections of Lucentis experienced no significant loss in visual acuity. Genentech also reported moderate visual improvement in 24.8 percent of participants treated with a 0.3 mg dose of Lucentis and 33.8 percent of participants treated with a 0.5 mg dose.

Genentech is reporting progress in the development of a device aimed at reducing the number of Lucentis injections required to inhibit wet AMD. The company is conducting a Phase II clinical trial for a sustained delivery device known as a port delivery system (PDS) is showing positive results for safety and the diffusion of Lucentis to affected areas of the retina. Investigators believe that the PDS can provide four months of therapy before needing to be refilled.

Avastin® Used Off-Label to Treat Wet AMD

A colorectal-cancer drug called Avastin® — a drug similar to Lucentis — has been used “off- label” by some ophthalmologists to treat wet AMD. The National Eye Institute conducted a clinical study of Avastin for the treatment of wet AMD to better determine the drug’s long-term safety and effectiveness. In the study, Avastin was compared to Lucentis. The two-year study showed that the drugs were similar in safety and efficacy.

EYLEA™ Preserves Vision in Wet AMD with Fewer Injections

Regeneron’s wet AMD treatment, Eylea, blocks the development of unhealthy blood vessels that lead to vision loss. Regeneron reports that in clinical trials, Eylea treated wet AMD as effectively as Lucentis, but with fewer eye injections. Genentech, maker of Lucentis, recommends monthly injections of their treatment. Regeneron, maker of Eylea, reports that their therapy can be injected every eight weeks after monthly dosing for the first 12 weeks of treatment. Eylea was approved by the FDA in November 2011.

Beovu Reduces Treatment Burden for Wet AMD Patients

Beovu®, a treatment for wet age-related macular degeneration (AMD) newly approved by the US Food and Drug Administration (FDA), often provides similar vision benefits to Eylea® when administered every three months. Eylea is frequently administered every two months. Novartis, developer of the treatment, reports that 30 percent of patients receiving Beovu every three months in Phase 3 clinical trials gained at least 15 letters (3 lines on an eye chart) at year one. Patients in the trials also showed greater reduction in central subfield thickness (CST) than patients in the trials receiving Eylea. CST is an indicator of harmful fluid in the retinas of wet AMD patients.

Vision Improvements Reported in Early Stem Cell Trial for Wet AMD

Two patients with advanced wet age-related macular degeneration (AMD) in a Phase I clinical trial demonstrated improved visual acuity sustained for one year after a sheet of retinal pigment epithelial (RPE) cells derived from embryonic stem cells was transplanted under their retinas. Each patient had one eye treated. Vision improvement for one patient was 29 letters or about 6 lines on an eye chart. The other had a gain of 21 letters or about 4 lines. Known as the London Project, the human study is taking place at Moorfields Eye Hospital in the UK.

Retinal Patch Performs Promisingly in Clinical Trial for Dry AMD

Regenerative Patch Technologies, a company developing stem-cell-derived treatments for people with retinal diseases, has reported encouraging results for the first five patients with advanced, dry age-related macular degeneration (AMD) participating in a Phase 1/2a clinical trial for its therapy – a patch comprised of a layer of retinal pigment epithelial (RPE) cells on a synthetic scaffold. One patient in the trial had visual acuity improvement of 17 letters (about 3 lines on an eye chart) in her treated eye. Three patients had vision maintained in their treated eyes. Two had improved fixation. No evidence of safety issues with the treatment was observed.

Apellis to Apply for FDA Approval for Dry AMD (GA) Treatment

The biopharmaceutical company Apellis announced that combined results from two Phase 3 clinical trials, DERBY and OAKS, showed that its drug APL-2 (pegcetacoplan) reduced the progression of geographic atrophy (GA), the advanced form of dry age-related macular degeneration (AMD). Monthly and every-other-month treatment with APL-2 reduced GA lesion growth by 17 percent and 14 percent, respectively, in the two trials. Treatments with the drug are made by injections into the vitreous, the soft gel in the middle of the eye. A total of 1,258 people were enrolled in the studies. The company plans to submit a New Drug Application (NDA) for APL-2 to the U.S. Food and Drug Administration (FDA) in the first half of 2022. If approved by the FDA, the drug would be the first treatment available for GA.

Genentech’s Port Delivery System for Wet AMD Meets Primary Endpoint in Phase 3 Clinical Trial

Genentech’s Port Delivery System (PDS), a permanent, refillable implant the size of a rice grain, is being developed to provide continual delivery of an anti-VEGF treatment to the retina. In a Phase 3 clinical trial, a single PDS implant performed as effectively as six monthly injections of Lucentis® (an FDA-approved anti-VEGF therapy). The PDS continually delivered a customized formulation of Lucentis for six months without being refilled. The primary outcome measure for the Phase 3 clinical trial was change in best-corrected visual acuity (measured by reading letters on an eye chart).

Zimura Slows Degeneration in Phase 3 Trial for Advanced Dry AMD

Iveric bio has reported that Zimura, its C5 complement inhibitor, slowed the progression of advanced dry AMD (also known as geographic atrophy) by 27% in a Phase 2/3 clinical trial. The treatment continues to be evaluated in a second Phase 3 trial. An overactive complement system, a part of the innate immune system, is implicated in the retinal degeneration associated with AMD. Zimura targets the C5 complement pathway.

Gyroscope Launches Phase 2 Clinical Trial for Dry AMD Gene Therapy

Gyroscope Therapeutics has dosed the first patient in its Phase 2 gene therapy clinical trial for people with advanced dry AMD (also known as geographic atrophy). The emerging one-time therapy is delivered through an injection underneath the retina. The therapy is designed to target the overactive complement system which is implicated in the retinal degeneration associated with AMD.

REGENXBIO's Wet AMD Gene Therapy Moves into Phase 3 Trial

REGENXBIO, a clinical-stage gene therapy biotechnology company, is planning two pivotal, Phase 3 clinical trials for RGX-314, its gene therapy designed to halt the growth of leaky blood vessels that cause retinal degeneration and central vision loss in people with the wet form of age-related macular degeneration (AMD). Dosing in the first trial, known as ATMOSPHERE™, is expected to begin in the first quarter of 2021. The second trial is expected to begin in the second half of 2021. Participants in the Phase 1/2 trial for RGX-314 had a significant reduction in their anti-VEGF treatment burden.