Jan 8, 2024

Age-Related Macular Degeneration Research Advances

Retinal Disease Research Advances

Recent developments in research on age-related macular degeneration.

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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 www.areds2.org.

 

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.

 

GENENTECH RECEIVES FDA APPROVAL FOR VABYSMO FOR TREATMENT OF WET AMD

Genentech, a global biotechnology company and member of the Roche Group, has received approval from the US Food & Drug Administration (FDA) for VabysmoTM (faricimab-svoa) for the treatment of wet age-related macular degeneration (AMD) and diabetic macular edema (DME). The treatment is injected into the patient’s vitreous, the soft gel in the middle of the eye, as infrequently as every 16 weeks at an eye doctor’s office.

 

GENENTECH’S SUSVIMO (PORT DELIVERY SYSTEM) FOR WET AMD RECEIVES FDA APPROVAL

Genentech’s Susvimo, a permanent, refillable implant the size of a rice grain, has received FDA

approval 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). Susvimo greatly reduces the burden of regular injections to treat wet 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.

 

APELLIS’ SYFOVRE APPROVED BY FDA FOR DRY AMD (GEOGRAPHIC ATROPHY)

The biopharmaceutical company Apellis received FDA approval to market SYFOVRE®, a treatment for geographic atrophy (GA), the advanced from of dry age-related macular degeneration. Combined results from two Phase 3 clinical trials, DERBY and OAKS, showed that SYFOVRE (pegcetacoplan) reduced the growth rate of atrophic lesions associated with GA geographic atrophy (GA). SYFOVRE was the first treatment available for GA. An overactive complement system, a part of the innate immune system, is implicated in the retinal degeneration associated with AMD. SYFOVRE targets the C3 complement pathway.

 

ASTELLAS’ IZERVAY APPROVED BY FDA FOR ADVANCED DRY AMD (GEOGRAPHIC ATROPHY)

Astellas has received FDA approval for IZERVAY™ (avacincaptad pegol) for the treatment of geographic atrophy (GA) secondary to advanced dry age-related macular degeneration. The drug was developed by Iveric Bio, which was acquired by Astellas. The drug, a C5 complement inhibitor, slowed the progression of lesion growth in two Phase 3 clinical trials. An overactive complement system, a part of the innate immune system, is implicated in the retinal degeneration associated with AMD. IZERVAY targets the C5 complement pathway.

 

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.

 

LUXA DOSES FIRST DRY AMD PARTICIPANT IN CLINICAL TRIAL OF RPE STEM CELLS

Luxa Biotechnology has announced transplantation of retinal pigment epithelial stem cell-derived RPE (RPESC-RPE-4W) in the first participant in its Phase 1/2 clinical trial for people with dry age-related macular degeneration (dry AMD). Dr. Rajesh Rao, the trial principal investigator at the Kellogg Eye Center, University of Michigan, transplanted 50,000 RPESC-RPE-4W cells under the macula of a legally blind participant with dry AMD. The 18-participant trial starting in Ann Arbor, Michigan, is the first in human study of RPESC-RPE-4W for the treatment of a retinal disease. The RPESC-RPE-4W cell product is derived from adult human RPE stem cells that haven’t quite become mature RPE cells but whose fate is determined to produce RPE. 

 

JANUARY 2024