IZERVAY™ (avacincaptad pegol intravitreal solution) Showed Increased Benefit in Reducing Geographic Atrophy Progression Over Time and Consistent Long-Term Safety

In GATHER2 open-label extension study, IZERVAY reduced GA growth 37-40.5% vs. projected sham, with earlier intervention resulting in greater protection of retinal tissue area  

Study results found no new safety signals and no cases of retinal vasculitis or occlusive vasculitis after IZERVAY treatment of up to 3.5 years

New U.S. real-world data from more than 10,000 GA patients support safety and tolerability of IZERVAY with  low rates of discontinuation and adverse events

TOKYO, Oct. 19, 2025 /PRNewswire/ -- Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas") today announced the first results from the open-label extension trial of the Phase 3 GATHER2 study, which demonstrated monthly treatment with IZERVAY™ (avacincaptad pegol intravitreal solution) in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) continued to reduce GA lesion growth for up to 3.5 years, with earlier intervention resulting in greater protection of retinal tissue area. IZERVAY was well tolerated, with no cases of retinal vasculitis or occlusive vasculitis. These findings, which indicate a cumulative treatment benefit with IZERVAY, were presented at the American Academy of Ophthalmology 2025 Annual Meeting (AAO 2025) during the Retina Subspeciality Day in Orlando, Fla.

During the 18-month open-label extension (OLE) trial, GA patients who previously completed the GATHER2 study switched from IZERVAY monthly (EM) or every-other-month (EOM) treatment to IZERVAY EM, or from sham to IZERVAY EM.

In the OLE study, disease progression, as measured by mean change in GA lesion growth area was reduced by 40.5% from month 24 (mm²/year) vs. projected sham in participants who switched from IZERVAY EM or EOM to IZERVAY EM at 3.5 years, and by 37% vs. projected sham in the IZERVAY EM group who previously received sham (p<0.001 for each). Earlier intervention with IZERVAY consistently resulted in greater protection of retinal tissue area: 2.92 mm² in the group that switched from IZERVAY EM or EOM to IZERVAY EM at 3.5 years and 1.83 mm² in participants treated with IZERVAY EM following sham. IZERVAY was well-tolerated with no new safety signals, no cases of retinal vasculitis or occlusive vasculitis, and no increased risk of intraocular inflammation.

Arshad M. Khanani, MD, MA, FASRS, Director of Clinical Research at Sierra Eye Associates, Clinical Professor at University of Nevada, Reno, Nevada
"The first reported findings from this open-label extension study corroborate the favorable efficacy and safety outcomes previously demonstrated in the 2-year GATHER2 trial. IZERVAY maintains a consistent safety profile and is associated with meaningful reduction in disease progression over time, with the most pronounced benefits observed in patients who initiated treatment at an earlier stage."

A separate real-world study highlighted treatment patterns and safety of IZERVAY in patients with GA (n=10,181 patients, 13,391 eyes) treated in routine clinical practice. Based on data from the American Academy of Ophthalmology IRIS^® Registry (Intelligent Research In Sight), this observational analysis included data from U.S. patients who received IZERVAY during its first year of approval by the U.S. Food and Drug Administration (FDA). This retrospective study found no new safety signals and low rates of discontinuation, supporting the safety and tolerability of IZERVAY.

Key real-world study results include:

• In patients who received [IZERVAY/ACP] at least twice, the mean interval between injections per patient eyes (n=11,799) was 7.0 weeks
• While nearly 9 in 10 patients (87.1%) had GA in both eyes, 68.5% received IZERVAY in one eye
• 22.7% of patient eyes received anti-VEGF therapy in addition to IZERVAY, of which nearly 90% had previously been on anti-VEGF treatment
• Overall, 3.6% of patients (2.9% of eyes) experienced any adverse event (AE) during the 25-week study follow-up period, for an event rate of 103 per 10,000 IZERVAY injections

Marci English, Senior Vice President, Biopharma and Ophthalmology Development, Astellas Pharma
"IZERVAY continues to be a trusted treatment option for countless patients living with GA and the fear of vision loss. At Astellas, we are committed to deepening our understanding of this condition, and our latest findings shed light on both disease progression and treatment strategies. The ongoing research and data continue to suggest the crucial need for earlier detection and intervention to support patients in their journey to preserve their vision for as long as possible."

Another Astellas study at AAO 2025 examined the relationship between structure and function in GA by analyzing ellipsoid zone (EZ) integrity and low luminance deficit (LLD) in eyes with GA treated with IZERVAY. This post hoc analysis of pooled 18-month data from the GATHER1 and GATHER2 studies demonstrated a structure-function link in GA between photoreceptor health, measured by EZ integrity, and visual function, captured by LLD. Treatment with IZERVAY slowed progressive loss of photoreceptor signal intensity in the EZ compared to sham over 18 months, particularly in eyes with greater baseline LLD, i.e., more severe disease. The findings support the structure-function link between EZ integrity and LLD as biomarkers to help measure GA progression and the impact of treatment. The results also suggest LLD may be a better indicator of visual function in GA than best-corrected visual acuity (BVCA).

IZERVAY is approved for the treatment of GA in the United States, Australia and conditionally in Japan. Astellas continues to engage with regulatory authorities around the world to bring ACP to GA patients globally.

About IZERVAY ™  (avacincaptad pegol intravitreal solution)

INDICATION AND IMPORTANT SAFETY INFORMATION

What is IZERVAY™?
IZERVAY (avacincaptad pegol intravitreal solution) is a prescription eye injection, used to treat geographic atrophy (GA), the advanced form of dry age-related macular degeneration (AMD). 

What is the most important information I should know about IZERVAY?

Do NOT receive IZERVAY if you: 

• Have an infection in or around your eye 
• Have active swelling in or around your eye that may include pain and redness 

IZERVAY can cause serious side effects: 

• Eye injections like the one for IZERVAY can cause an eye infection (endophthalmitis) or separation of layers of the retina (retinal detachment). 
• Call your healthcare provider right away if you have redness of the eye, eye pain, increased discomfort, worsening eye redness, blurred or decreased vision, an increased number of small specks floating in your vision, flashes of light, or increased sensitivity to light. 
• There is a risk of developing wet AMD with IZERVAY. You should report any symptoms (visual distortions such as straight lines seeming bent, deterioration in vision, dark spots, loss of central vision) to your healthcare provider to monitor. 
• IZERVAY may cause a temporary increase in eye pressure after the injection. Your healthcare provider will monitor this after each injection. 

Before receiving IZERVAY tell your healthcare provider about all of your medical conditions including if you: 

• Have a history of seeing flashes of light or small specks floating in your vision and if you have a sudden increase of size and number of these specks. 
• Have high pressure in the eye or if you have glaucoma. 
• Are pregnant or breastfeeding, think you may be pregnant, or are planning to have a baby, ask your doctor for advice before taking this medicine. 
• Are taking any medications, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Tell your healthcare provider about any medicine you take. 

What should I avoid while receiving IZERVAY? 

• Your vision may be impaired after receiving an eye injection or after an eye exam. Do not drive or use machinery until your vision has recovered sufficiently. 

What are the most common side effects of IZERVAY? 

• Blood in the white of the eye 
• Increase in eye pressure 
• Blurred vision 
• Wet age-related macular degeneration 

These are not all the possible side effects of IZERVAY. Tell your healthcare provider about any side effect that bothers you or that does not go away.

Call your healthcare provider for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please see full Prescribing Information for more information.

About Geographic Atrophy 
Age-related macular degeneration (AMD) is the major cause of moderate and severe loss of central vision in aging adults, affecting both eyes in the majority of patients. The macula is a small area in the central portion of the retina responsible for central vision. As AMD progresses, the loss of retinal cells and the underlying blood vessels in the macula results in marked thinning and/or atrophy of retinal tissue. Geographic atrophy, associated with AMD, leads to further irreversible loss of vision in these patients.  

About the GATHER Clinical Trials
IZERVAY met its primary endpoint in the GATHER1 (NCT02686658) clinical trial and the GATHER2 (NCT04435366) clinical trial, both of which were randomized, double-masked, sham-controlled, multicenter Phase 3 clinical trials. These trials evaluated the safety and efficacy of monthly 2 mg intravitreal administration of IZERVAY in patients with GA secondary to AMD. For the first 12 months in both trials, patients were randomized to receive either IZERVAY 2 mg or sham monthly. There were 286 participants enrolled in GATHER1 and 448 participants enrolled in GATHER2. The primary efficacy endpoints in both pivotal studies were based on GA area measured by fundus autofluorescence at three time points: baseline, month 6, and month 12. Safety was evaluated in over 700 patients with GA across the two trials.

In year 2 of the GATHER2 study, patients treated with IZERVAY in year 1 were re-randomized to receive either IZERVAY dosed monthly (EM, n=96) or every other month (EOM, n=93); patients who received sham in year 1 continued to receive sham in year 2 (n=203). IZERVAY is continuing to be evaluated in an open-label extension study.  

About Astellas
Astellas is a global life sciences company committed to turning innovative science into VALUE for patients. We provide transformative therapies in disease areas that include oncology, ophthalmology, urology, immunology and women's health. Through our research and development programs, we are pioneering new healthcare solutions for diseases with high unmet medical need. Learn more at www.astellas.com.

Cautionary Notes
In this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management's current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas' intellectual property rights by third parties. Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice.

SOURCE Astellas Pharma Inc.