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Avacincaptad pegol reduces geographic atrophy growth compared to sham in Age-Related Macular Degeneration patientsThis Eye Injection Slows Blindness for Two Years Straight

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Key Takeaway
Note avacincaptad pegol reduces geographic atrophy growth, choroidal neovascularization was higher versus sham.

This phase 3, randomized, sham-controlled study enrolled patients with non-center point-involving geographic atrophy. The sample size included n=225 (ACP group) and n=222 (sham group) over 24.0 months. The intervention involved ACP 2 mg monthly (year 1); ACP 2 mg every month (EM) or every other month (EOM) (year 2).

Primary outcomes focused on reduction in GA growth (slope) and safety. Mean rate of GA area growth was 4.46 mm (ACP EM) vs 5.18 mm (sham), a 0.724 mm difference (95% CI 0.133-1.315 mm; P = 0.0165). For ACP EOM, mean rate of GA area growth was 4.20 mm (ACP EOM) vs 5.18 mm (sham), a 0.976 mm difference (95% CI 0.377-1.575 mm; nominal P = 0.0015).

Safety outcomes included incidence of choroidal neovascularization, which was 11.6% (ACP all treated) vs 9.0% (sham). There were no events of retinal vasculitis, ischemic optic neuropathy, or serious intraocular inflammation. Discontinuations showed 175 patients completed study in ACP group; 184 patients completed study in sham group. Tolerability showed no new safety signals compared with year 1.

Limitations were not explicitly reported in the provided data. Funding or conflicts note that proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article. Practice relevance was not reported. Clinicians should weigh the statistically significant reduction in growth against the safety profile in this specific population.

Imagine going to the eye doctor and hearing that your vision is slowly fading. There is no pain, but the details you need to read or drive are slipping away. This is the reality for millions of older adults facing a condition called geographic atrophy.

For years, patients have had very few options to stop this damage. Now, new research shows a treatment that works for two years in a row.

A Slow, Silent Loss

Geographic atrophy (GA) is often called “dry” macular degeneration. It is a leading cause of blindness in older adults. It affects the central part of the retina, called the macula. This area is responsible for sharp, straight-ahead vision.

Over time, GA causes blind spots to grow in the center of your sight. These spots can merge, making it hard to recognize faces or read. It is a progressive disease, meaning it usually keeps getting worse. Currently, there are very few approved treatments for GA.

What frustrates patients most is the lack of control. Lifestyle changes can help, but they cannot stop the damage. This new study offers a different path.

A New Approach to Protection

For a long time, doctors thought the immune system played a minor role in this type of vision loss. But recent science shows that the body’s immune system can actually attack the retina by mistake.

Think of the eye like a delicate camera. Inside, there is a constant cleanup crew. In GA, this crew gets overactive. It starts to damage healthy cells in the back of the eye.

The old way of thinking was to simply protect the eye with vitamins. The new way is to step in and turn down the overactive immune response.

This treatment uses a special molecule called an aptamer. You can think of an aptamer like a key. It is designed to fit perfectly into a specific lock.

In this case, the "lock" is a protein in the blood called complement C5. This protein is part of the immune system. When it is overactive, it causes inflammation and cell death in the retina.

The treatment, called avacincaptad pegol (ACP), acts like a key that blocks the lock. By stopping C5, it reduces the damage to the retina. It is given as an injection directly into the eye. This stops the "cleanup crew" from destroying healthy tissue.

The Study Snapshot

Researchers ran a Phase 3 trial called GATHER2. They enrolled patients with GA who did not have the center of their vision affected yet.

Patients were split into two groups. One group received a monthly injection of ACP. The other group received a "sham" injection, which is a fake procedure used to test if the real treatment works better.

After one year, the patients who got the real medicine were split again. Some kept getting it every month. Others switched to getting it every other month. The study followed everyone for a full two years.

The results showed a clear benefit that lasted. The goal was to slow down the growth of the blind spots.

At the two-year mark, the data was strong. Patients who received ACP every month saw their blind spots grow 14% slower than those who got the sham injection. Even more promising, patients who switched to getting the shot every other month saw a 19% reduction in growth compared to the sham group.

Here is what that means in real life. The treatment did not cure the blindness. But it significantly slowed the rate of damage. For someone losing their sight, buying time is a major victory.

The study also looked closely at safety. The injection is given into the eye, which always carries risks. However, the researchers found no new safety issues in the second year. The rates of side effects were similar to the first year.

But Here’s the Catch

While the results are encouraging, this treatment is not a cure. The blind spots did not disappear; they just grew more slowly. Also, the study focused on a specific group of patients—those whose central vision was still intact.

Experts in retinal disease view this as a significant step forward. For a condition with limited options, having a treatment that works for two years is important. The fact that it can be given every other month is also a plus. Fewer injections mean less burden for patients and doctors.

This doesn’t mean this treatment is available yet.

If you or a loved one has geographic atrophy, this research is hopeful. However, ACP is not yet approved for widespread use in all countries. It is still under review by regulatory agencies.

You should not ask for this specific drug by name at your next appointment. Instead, talk to your ophthalmologist about clinical trials or current options for GA. They can tell you what is available now and what is on the horizon.

This study has some limits. It was done over two years, which is good, but longer data is needed to see if the benefits last a lifetime. The study also focused on a specific type of GA (non-center involving). Results might differ for patients with more advanced disease.

What happens next? The data from this two-year study supports the case for approval. Regulatory agencies will review the safety and efficacy data. If approved, this could become a standard treatment for GA.

Research takes time, but this study brings us closer to a future where vision loss can be managed more effectively. For now, it offers a glimmer of hope for those facing a difficult diagnosis.

Study Details

Study typeRct
Sample sizen = 225
EvidenceLevel 2
Follow-up24.0 mo
PublishedApr 2026
View Original Abstract ↓
PURPOSE: Avacincaptad pegol (ACP) is a pegylated RNA aptamer that inhibits complement C5. The efficacy and safety of ACP 2 mg was investigated in GATHER2, with positive year 1 results published. Herein, 2-year results are reported. DESIGN: Phase 3, randomized, sham-controlled study (ClinicalTrials.gov identifier, NCT04435366). PARTICIPANTS: Patients with non-center point-involving geographic atrophy (GA). METHODS: Eligible patients were randomized 1:1 to receive monthly ACP 2 mg (n = 225) or sham (n = 222) for 1 year. At month 12, patients who received ACP 2 mg were randomized again 1:1 to dosing every month (EM; n = 96) or every other month (EOM; n = 93) with ACP 2 mg. Patients who had received monthly sham continued with sham (n = 203). MAIN OUTCOME MEASURES: The safety and efficacy of ACP versus sham administration over 2 years and the effect of ACP EM or EOM dosing in year 2. RESULTS: Overall, 175 and 184 patients in the ACP and sham group completed the study at year 2, respectively. At 2 years, treatment with ACP demonstrated a continued reduction in GA growth (slope) with both ACP EM and EOM versus sham. From baseline to year 2, the mean rate of GA area growth was 4.46 mm (standard error [SE], 0.25 mm) with ACP EM and 5.18 mm (SE, 0.17 mm) with sham, a difference in growth of 0.724 mm (95% confidence interval [CI], 0.133-1.315 mm; P = 0.0165), representing a 14% difference. From baseline to year 2, the mean rate of GA area growth was 4.20 mm (SE, 0.25 mm) with ACP EOM, a difference in growth of 0.976 mm (95% CI, 0.377-1.575 mm; nominal P = 0.0015) versus sham, representing a 19% difference. The incidence of choroidal neovascularization (study eye) was 11.6% with ACP (all treated) versus 9.0% with sham over 2 years. No events of retinal vasculitis, ischemic optic neuropathy, or serious intraocular inflammation occurred over 2 years. CONCLUSIONS: Dosing of ACP 2 mg, either EM or EOM, continued to reduce GA growth versus sham therapy over 2 years with no new safety signals compared with year 1. FINANCIAL DISCLOSURE(S): Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.
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