A Non-interventional, Observational Study to Evaluate Treatment Patterns and Safety of Avacincaptad Pegol (ACP/IZERVAY™) in Routine Clinical Practice in Patients With Geographic Atrophy Secondary to Age-related Macular Degeneration

This study is for people who have geographic atrophy due to age-related macular degeneration (AMD). AMD happens when the macula, the light-sensitive layer at the back of the eye called the retina, becomes damaged and causes a person's central vision to worsen. Geographic atrophy is an advanced form of AMD where cells in the retina waste away and die. Over time this can lead to permanent loss of vision.
Avacincaptad pegol can help slow down the worsening or progression of geographic atrophy. Avacincaptad pegol is a treatment approved in the US to treat geographic atrophy.
This study is about collecting information on how people with geographic atrophy are treated in routine clinical practice. This includes recording any medical problems from avacincaptad pegol. This is known as an observational study. Information will be collected from the peoples' medical records during and after treatment. The people in this study will have geographic atrophy in 1 or both eyes and they and their doctor has decided they will be treated with avacincaptad pegol. The individual's doctor decides on treatment, not the study sponsor (Astellas).
People that want to take part in the study will have eye examinations that they would usually have as part of their routine care. People will also be asked to complete surveys about their eye health. These surveys will occur when treatment starts and then every 6 months for the first 2 years. After 2 years the surveys will happen once a year. The people on the study can take part if their doctor provides treatment with avacincaptad pegol and they want to continue with the study. The people on the study can take part for up to 3 years or up to 5 years, depending on when they start the study.

Start Date22 Jan 2025

Sponsor / Collaborator

Astellas Pharma Global Development, Inc.

NCT05536297

/ Active, not recruitingPhase 3

An Open-label Extension (OLE) Phase 3 Trial to Assess the Safety of Intravitreal Administration of Avacincaptad Pegol (Complement C5 Inhibitor) in Patients With Geographic Atrophy Who Previously Completed Phase 3 Study ISEE2008 (GATHER2)

The purpose of this study is to assess long-term safety of avacincaptad pegol intravitreal administration for patients with geographic atrophy (GA) who completed Study ISEE2008 (GATHER2) through the Month 24 visit on study treatment (either avacincaptad pegol or Sham).

Start Date26 Sep 2022

Sponsor / Collaborator

Astellas Pharma Global Development, Inc.

NCT04435366

/ CompletedPhase 3

A Phase 3 Multicenter, Randomized, Double Masked, Sham- Controlled Clinical Trial to Assess the Safety and Efficacy of Intravitreal Administration of Zimura (Complement C5 Inhibitor) in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration

The objectives of this study was to evaluate the safety and efficacy of avacincaptad pegol intravitreal administration in participants with geographic atrophy secondary to age-related macular degeneration (AMD)

Start Date22 Jun 2020

Sponsor / Collaborator

IVERIC bio, Inc.

100 Clinical Results associated with Avacincaptad pegol

100 Translational Medicine associated with Avacincaptad pegol

100 Patents (Medical) associated with Avacincaptad pegol

189

Literatures (Medical) associated with Avacincaptad pegol

01 Feb 2025·EYE

Gene therapy for geographic atrophy in age-related macular degeneration: current insights

Review

Author: Waheed, Nadia K ; Waheed, Nadia K. ; Jamil, Muhammad Usman

Geographic atrophy (GA) is the advanced stage of non-neovascular (dry) age-related macular degeneration, defined by the presence of sharply demarcated atrophic lesions of the outer retina. The complement system is integral to the body's natural immune response, and hence its overactivation can lead to tissue damage and inflammation. It has been shown to play a significant role in GA lesion development and progression, and therefore, complement inhibition is emerging as a promising avenue for therapeutic intervention. With the recent approval by the Food and Drug Administration of drugs like SYFOVRE™ (pegcetacoplan injection) and IZERVAY™ (avacincaptad pegol intravitreal solution), there is hope for the development of interventions capable of slowing down or arresting the progression of GA. In particular, gene therapy intervention is gaining traction for halting GA atrophy at the source of our genes. The concept is to insert a gene into the eye that will act as an ocular "bio-factory," producing a desired protein. This can either lead to overproduction of an already available protein or produce a substance not typically generated in the eye. This review aims to provide an overview of the present understanding of GA, encompassing risk factors, prevalence, pathophysiology, and genetic associations. It will also highlight the current landscape of GA treatment, with particular emphasis on gene therapy intervention.

01 Feb 2025·Pharmacology Research & Perspectives

Intravitreal Administration of Avacincaptad Pegol in a Nonhuman Primate Model of Dry Age‐Related Macular Degeneration

Article

Author: Natsume, Takahiro ; Itani, Yoshitaka ; Fujii, Rintaro ; Takamatsu, Hiroyuki ; Matsushita, Mayumi ; Hama, Aldric

ABSTRACT:

The lack of effective treatments for dry age‐related macular degeneration (AMD) is in part due to a lack of a preclinical animal model that recapitulates features of the clinical state including macular retinal pigment epithelium (RPE) degeneration, also known as geographic atrophy (GA). A nonhuman primate model of GA was developed and its responsiveness to an approved treatment, avacincaptad pegol (ACP), a complement C5 inhibitor, was evaluated. Intravitreal (ivt) administration of sodium iodate (SI) into one eye of male Macaca fascicularis leads to retinal areas (mm2) of hyper‐ or hypo‐autofluorescence. Qualitative changes to the retinal structure over time were observed with spectral domain optical coherence tomography (OCT). Six days after SI administration, prior to treatment, mean (± SEM) GA of all eyes was 8.2 ± 1.8 mm2. Following randomization to treatment groups, either vehicle or ACP was ivt injected and treatment was continued every 4 weeks, for a total of four treatments. Sixteen weeks after SI administration, the GA area in vehicle‐treated eyes was 18.9 ± 6.6 mm2, whereas GA in ACP‐treated eyes was 11.4 ± 4.0 mm2, a reduction by about 36%. Increased, followed by decreased, overall macular thickness was observed with OCT over time following SI administration. Treatment with ACP did not change alter macular thickness thinning. Geographic atrophy‐like lesions that expand over time are observed following SI administration. The current macaque model could be utilized to further explore the mechanism of dry AMD and to develop more novel therapeutics.

01 Jan 2025·INDIAN JOURNAL OF OPHTHALMOLOGY

Dry and neovascular “wet” age-related macular degeneration: Upcoming therapies

Review

Author: Yan, Audrey ; Hasan, Nasiq ; Chhablani, Jay

The age-related macular degeneration (AMD) field is witnessing promising advancements in therapeutic options. Breakthrough drugs such as pegcetacoplan and avacincaptad have been FDA-approved for dry AMD, marking a significant development as there were no treatment options until August 2023. While several antivascular endothelial growth factor (VEGF) inhibitors have been approved for wet AMD, challenges persist with the need for frequent dosing. New treatments such as gene therapy, cell therapy, WNT pathway agonists, complement inhibitors, and anti-VEGF combination drugs are under development to address these issues. These developments are exciting and hold promise for transforming the field of medicine, offering hope for improved outcomes and enhanced patient care in managing AMD.

119

News (Medical) associated with Avacincaptad pegol

13 Feb 2025

·www.astellas.com

U.S. FDA Approves Expanded Label for Astellas’ IZERVAY™ (avacincaptad pegol intravitreal solution) for Geographic Atrophy

TOKYO, February 13, 2025 – Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, “Astellas”) today announced the U.S. Food and Drug Administration (FDA) approved expanded U.S. Prescribing information for IZERVAY™ (avacincaptad pegol intravitreal solution) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). As a result, IZERVAY is now approved without a limitation on duration of dosing—providing physicians and patients with greater flexibility when managing GA. The approval follows Astellas’ resubmission of the supplemental New Drug Application (sNDA) for IZERVAY on December 26, 2024, within days of meeting with the FDA to clarify the Agency’s feedback provided in the Complete Response Letter (CRL) issued in November 2024. Marci English, Senior Vice President, Biopharma and Ophthalmology Development, Astellas Pharma“We are pleased with the FDA’s decision to extend the use of IZERVAY for longer-term administration—further solidifying IZERVAY’s status as a trusted choice for thousands of GA patients since its launch in 2023. To date, IZERVAY remains the only FDA-approved treatment to demonstrate a statistically significant slowing of GA across two pivotal studies.” The approved label update was based on positive results from the GATHER2 Phase 3 clinical trial, which evaluated the efficacy and safety of IZERVAY through year 2. Since receiving a permanent J-code in April 2024, IZERVAY has had month-over-month growth in the U.S. with more than 143,000 vials distributed through the end of September 2024. Post-marketing safety reporting remains consistent with that observed in the clinical trial program, with no new or significant safety signals identified, providing confidence to prescribers in IZERVAY’s safety profile. Veeral S. Sheth, MD, MBA, FACS, FASRS, Partner and Director of Clinical Trials University Retina“This is a welcome update for retina specialists, providing continued management options for treatment of this chronic, progressive disease that can lead to irreversible vision loss.” The GATHER2 study demonstrated that IZERVAY continued to reduce the rate of GA lesion growth in patients with GA secondary to AMD through 2 years versus sham. The treatment benefit with IZERVAY versus sham was observed as early as 6 months, continued to increase over time through 2 years, and more than doubled over 2 years compared to year 1. IZERVAY was well tolerated over 2 years in GATHER2, with one case of non-serious intraocular inflammation and culture-positive endophthalmitis each, and zero cases of ischemic neuropathy or serious intraocular inflammation, including retinal vasculitis. Over 2 years, the incidence of choroidal neovascularization was slightly increased between IZERVAY (11.6%) versus sham (9%). IZERVAY was approved by the U.S. Food and Drug Administration on August 4, 2023, for the treatment of GA secondary to AMD. The impact of this matter on Astellas’ financial results for the fiscal year ending March 31, 2025, is expected to be minor. About IZERVAY™ (avacincaptad pegol intravitreal solution) U.S. 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 AtrophyAge-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 GATHER2 Clinical TrialGATHER2 (NCT04435366) was a randomized, double-masked, sham-controlled, multicenter Phase 3 clinical trial to evaluate the safety and efficacy of intravitreal administration of avacincaptad pegol (ACP) in 448 enrolled patients with GA secondary to AMD. ACP met its primary objective at 12 months, for which patients were randomized to receive either ACP or sham procedure monthly. In year 2 of the study, patients treated with ACP in year 1 were re-randomized to receive either ACP 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 AstellasAstellas 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 NotesIn 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. Click below for a copy of the full press release

Clinical ResultPhase 3Drug ApprovalFinancial Statement

12 Feb 2025

·www.prnewswire.com

U.S. FDA Approves Expanded Label for Astellas' IZERVAY™ (avacincaptad pegol intravitreal solution) for Geographic Atrophy

IZERVAY dosing approved beyond 12 months TOKYO, Feb. 12, 2025 /PRNewswire/ -- Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas") today announced the U.S. Food and Drug Administration (FDA) approved expanded U.S. Prescribing information for IZERVAY™ (avacincaptad pegol intravitreal solution) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). As a result, IZERVAY is now approved without a limitation on duration of dosing—providing physicians and patients with greater flexibility when managing GA. The approval follows Astellas' resubmission of the supplemental New Drug Application (sNDA) for IZERVAY on December 26, 2024, within days of meeting with the FDA to clarify the Agency's feedback provided in the Complete Response Letter (CRL) issued in November 2024. Marci English, Senior Vice President, Biopharma and Ophthalmology Development, Astellas Pharma "We are pleased with the FDA's decision to extend the use of IZERVAY for longer-term administration—further solidifying IZERVAY's status as a trusted choice for thousands of GA patients since its launch in 2023. To date, IZERVAY remains the only FDA-approved treatment to demonstrate a statistically significant slowing of GA across two pivotal studies." The approved label update was based on positive results from the GATHER2 Phase 3 clinical trial, which evaluated the efficacy and safety of IZERVAY through year 2. Since receiving a permanent J-code in April 2024, IZERVAY has had month-over-month growth in the U.S. with more than 210,000 vials distributed through the end of December 2024. Post-marketing safety reporting remains consistent with that observed in the clinical trial program, with no new or significant safety signals identified, providing confidence to prescribers in IZERVAY's safety profile. Veeral S. Sheth, MD, MBA, FACS, FASRS, Partner and Director of Clinical Trials University Retina "This is a welcome update for retina specialists, providing continued management options for treatment of this chronic, progressive disease that can lead to irreversible vision loss." The GATHER2 study demonstrated that IZERVAY continued to reduce the rate of GA lesion growth in patients with GA secondary to AMD through 2 years versus sham. The treatment benefit with IZERVAY versus sham was observed as early as 6 months, continued to increase over time through 2 years, and more than doubled over 2 years compared to year 1. IZERVAY was well tolerated over 2 years in GATHER2, with one case of non-serious intraocular inflammation and culture-positive endophthalmitis each, and zero cases of ischemic neuropathy or serious intraocular inflammation, including retinal vasculitis. Over 2 years, the incidence of choroidal neovascularization was slightly increased between IZERVAY (11.6%) versus sham (9%). IZERVAY was approved by the U.S. Food and Drug Administration on August 4, 2023, for the treatment of GA secondary to AMD. The impact of this matter on Astellas' financial results for the fiscal year ending March 31, 2025, is expected to be minor. About IZERVAY ™ (avacincaptad pegol intravitreal solution) U.S. 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 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 GATHER2 Clinical Trial GATHER2 (NCT04435366) was a randomized, double-masked, sham-controlled, multicenter Phase 3 clinical trial to evaluate the safety and efficacy of intravitreal administration of avacincaptad pegol (ACP) in 448 enrolled patients with GA secondary to AMD. ACP met its primary objective at 12 months, for which patients were randomized to receive either ACP or sham procedure monthly. In year 2 of the study, patients treated with ACP in year 1 were re-randomized to receive either ACP 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 . 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. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultPhase 3Drug Approval

05 Feb 2025

·www.prnewswire.com

Astellas Submits New Drug Application for Conditional Approval of Avacincaptad Pegol for Geographic Atrophy in Japan

- Ministry of Health, Labour and Welfare to evaluate ACP as potential first and only treatment for patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) in Japan - TOKYO, Feb. 5, 2025 /PRNewswire/ -- Astellas Pharma Inc. (TSE: 4503) (President and CEO: Naoki Okamura, "Astellas") today announced the submission of a New Drug Application (NDA) to Japan's Ministry of Health, Labour and Welfare (MHLW) for Conditional Approval of avacincaptad pegol intravitreal solution (ACP), a synthetic aptamer that inhibits the complement C5 protein, for the treatment of GA secondary to AMD. If approved, ACP has the potential to become the first and only GA treatment available in Japan. GA is a progressive form of AMD that can cause irreversible vision loss, with no treatments currently approved outside the US or Australia.1,2 Globally, over five million people are estimated to have GA and, without timely treatment, an estimated 66% of people living with GA may become legally blind or severely visually impaired.1,3 As a result, GA secondary to AMD has a substantial impact on patients' daily lives and psychological wellbeing.4,5 Marci English, Vice President, Head of BioPharma and Ophthalmology Development, Astellas "Today's submission comes as good news to people in Japan living with geographic atrophy who have no approved treatment options for this devastating disease. If approved, avacincaptad pegol has the potential to be the first and only treatment to slow disease progression for eligible patients in Japan. As such, we are committed to working with regulatory authorities in Japan to ensure that patients can benefit from this vital new treatment." The NDA submission is based on results of overseas clinical trials, including the GATHER1 and GATHER2 randomized, sham-controlled clinical trials, which evaluated the safety and efficacy of monthly 2mg intravitreal administration of ACP in patients with GA secondary to AMD.6 The data from both trials demonstrates that ACP slows GA lesion growth and has a favorable safety profile.6 Sustained efficacy of ACP, as shown in the restriction of lesion growth over time, was observed over a follow up of two years in GATHER1 and GATHER2 studies.7 This submission will have no impact on the financial forecasts of the current fiscal year ending March 31, 2025. About avacincaptad pegol Avacincaptad pegol (ACP) is an investigational drug for treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) that has been submitted for Conditional Approval in Japan. ACP is approved in the U.S. as IZERVAY for the treatment of GA secondary to AMD.6 ACP is a synthetic aptamer that inhibits the complement C5 protein.8 Overactivity of the complement system and the C5 protein play a critical role in the development and growth of scarring and vision loss associated with GA secondary to AMD.8 By targeting C5, ACP is considered to decrease activity of the complement system known to cause the degeneration of retinal cells and thus slow the progression of GA.8 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.1,8,9 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.10 Geographic atrophy (GA), associated with AMD, leads to further irreversible loss of vision in these patients.11 About the GATHER2 Clinical Trial GATHER2 (NCT04435366) was a randomized, double-masked, sham-controlled, multicenter Phase 3 clinical trial to evaluate the safety and efficacy of intravitreal administration of avacincaptad pegol (ACP) in 448 enrolled patients with GA secondary to AMD.6 ACP met its primary objective at 12 months, for which patients were randomized to receive either ACP or sham procedure monthly.6 In year 2 of the study, patients treated with ACP in year 1 were re-randomized to receive either ACP 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).7 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 . 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. References: 1 Keenan TDL, Cukras CA, Chew EY. Age-Related Macular Degeneration: Epidemiology and Clinical Aspects. Adv Exp Med Biol. 2021;1256:1-31. 2 Apellis. Apellis Receives Approval of SYFOVRE® (pegcetacoplan) in Australia for Geographic Atrophy (GA). Available at: . Last accessed: February 2025. 3 Colijn JM, Liefers B, Joachim N, et al. Enlargement of geographic atrophy from first diagnosis to end of life. JAMA Ophthalmol. 2021;139(7):743–750. 4 Lundeen EA, Saydah S, Ehrlich J, Saaddine J. Self-reported vision impairment and psychological distress in U.S. adults. Ophthalmic Epidemiol. 2022;29(2):171–181. 5 World Health Organization. Blindness and vision impairment. Available at: . Last accessed: February 2025. 6 IZERVAY™ (avancincaptad pegol intravitreal solution) Prescribing Information. February 2024. 7 Khanani AM, Patel SS, Staurenghi G, et al. GATHER2: Two-Year Data. Presented at AAO 2023 127th Annual Meeting. San Francisco, CA. 11-03-2023 to 11-06-2023. 8 Desai D and Dugel PU. Complement cascade inhibition in geographic atrophy: a review. Eye. 2022;36(2):294–302. 9 Ayoub T and Patel N. Age-related macular degeneration. J R Soc Med. 2009;102(2):56–61. 10 Jaffe GJ, Westby K, Csaky KG, et al. C5 Inhibitor avacincaptad pegol for geographic atrophy due to age-related macular degeneration: a randomized pivotal phase 2/3 trial. Ophthalmology. 2021;128(4):576–586. 11 Patel PJ, Ziemssen F, Ng E, et al. Burden of illness in geographic atrophy: a study of vision-related quality of life and health care resource use. Clin Ophthalmol. 2020;14:15–28. SOURCE Astellas Pharma Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Clinical ResultNDADrug Approval

100 Deals associated with Avacincaptad pegol

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KEGG	Wiki	ATC	Drug Bank
D11748	-	-	DB15165

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Geographic Atrophy	US	Astellas Pharma US, Inc.	04 Aug 2023

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Age Related Macular Degeneration	Phase 3	US	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	AR	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	AU	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	AT	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	BE	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	BR	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	CA	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	CO	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	HR	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	CZ	Astellas Pharma Global Development, Inc.	26 Sep 2022

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02686658 \| NCT04435366 (GATHER2, EURETINA2024) Manual	Phase 2/3	Geographic Atrophy	-	Avacincaptad Pegol 2 mg	zrmupxvcdz(nszomcljlz) = atyfewaoxb wklobhnikj (ehnuclmoqq )	Positive	19 Sep 2024
NCT04435366 (GATHER2, PRNewswire) Manual	Phase 3	Age Related Macular Degeneration	-	IZERVAY	thtsyxlwbh(spgqtiebzk) = IZERVAY was well tolerated over 2 years, with one case each of non-serious intraocular inflammation (IOI) and culture-positive endophthalmitis. There were no cases of ischemic neuropathy or retinal vasculitis. yzeppkqmhd (ycjtnuitjr ) View more	Positive	04 Nov 2023
				IZERVAY
EURETINA2023	Not Applicable	Geographic Atrophy	-	Avacincaptad pegol 2 mg	wkrgcbkxhb(lhthfzwnzx) = zixweddmxv tfrqowkexw (ciysucphtm, 0.56 - 0.15-2.06)	-	05 Oct 2023
EURETINA2023	Not Applicable	-	-	Avacincaptad pegol 2 mg	tyhhvyvlpf(qbgoeyleye) = vbqrqkalro ligvxyfzgn (npknyreteq ) View more	-	05 Oct 2023
NCT02686658 \| NCT04435366 (GATHER2, FDA) Manual	Phase 2/3	Geographic Atrophy	624	IZERVAY (GATHER1)	osfgiocgfn(qpyhfufbbi) = mblhgjmhkc ewohinusnh (frakvmhynj )	Positive	04 Aug 2023
				Sham (GATHER1)	osfgiocgfn(qpyhfufbbi) = nwvlgbzzjg ewohinusnh (frakvmhynj )
NCT05571267 (CTgov)	Phase 2	Age Related Macular Degeneration	1	Zimura+avastin (Zimura and Avastin)	lkyyrahltl(haczygltwc) = cjnbvvdwmr gtfxdmlvih (ykkmhndvsn, zmmhgzuayv - rwfjxnpvxf) View more	-	03 Apr 2023
				Zimura+Lucentis (Zimura and Lucentis)	tcbtxqfzii(zaxhqlpfex) = judrpcrjdp bkznmrpgmc (dqedcpcbtq, vvhgflzxof - dzbsbvjhhw) View more
NCT04435366 (GATHER2, Corporate Publications) Manual	Phase 3	Geographic Atrophy	447	Avacincaptad pegol	yxdupglnhg(zkdjcxlrku) = grsdjarxmn gqxudxssdi (qwismlplfx ) View more	Positive	06 Sep 2022
				Sham	yxdupglnhg(zkdjcxlrku) = aojbchgtfb gqxudxssdi (qwismlplfx ) View more
NCT02686658 (GATHER1, ARVO2022)	Phase 2/3	Geographic Atrophy	-	Avacincaptad pegol 2 mg	jwigwseplq(ukhlhsflnj) = hyaaddamid zzqqwajzzh (lgpxkdcyzt )	-	01 May 2022
				avacincaptad pegol (Sham)	jwigwseplq(ukhlhsflnj) = qqkqqxdgee zzqqwajzzh (lgpxkdcyzt )
NCT03362190 (CTgov)	Phase 2	Wet age-related macular degeneration	64	Zimura+Lucentis (Cohort 1)	pqugfnsxgj(ymdffyzfse) = xzillvamok siufcjyqcy (ebureibkwq, xnzrevvizi - hjnrigrynb) View more	-	02 Feb 2022
				Zimura+Lucentis (Cohort 2)	pqugfnsxgj(ymdffyzfse) = inajbwpatf siufcjyqcy (ebureibkwq, nqxtjtnwba - eiuugmnkry) View more
NCT02686658 (GATHER1, AAO2021)	Phase 2/3	dry age-related macular degeneration \| Geographic Atrophy	286	Avacincaptad Pegol 2mg	qeylkjujzc(pqrvrzbyqb) = dociewzggx ihrisevxih (qdgwybcdib )	-	13 Nov 2021
				Avacincaptad Pegol 4mg	qeylkjujzc(pqrvrzbyqb) = khwswwbnlr ihrisevxih (qdgwybcdib )

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