Syfovre (pegcetacoplan intravitreal) / Apellis - LARVOL DELTA

Real-World Outcomes with Complement Inhibitors for Geographic Atrophy: A Comparative Study of Pegacetacoplan versus Avacincaptad Pegol. (PubMed, Clin Ophthalmol) - "This real-world study reports that treatment with IVA and IVP have similar visual and anatomic outcomes at 12-months, although subjects undergoing treatment with IVP may receive fewer injections to obtain these outcomes. Further studies comparing the safety and efficacy of IVA and IVP are warranted."

Journal • Real-world evidence • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Syfovre and Izervay Clinical Outcomes (ARVO 2025) - "One eye developed RV confirmed by angiography and was treated with prednisone, difluprednate, and pressure-lowering drops; at month 2, logMAR VA plateaued at 1.3 from 0.47. One eye underwent RD repair following IVS and IV faricimab...Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Clinical • Clinical data • Ocular Infections • Ocular Inflammation • Ophthalmology • Retinal Disorders

Discovering A Novel AAV Variant for Efficient RPE Transfection via Intravitreal Injection for Retinal Degenerative Disease (ARVO 2025) - "Recently the FDA approved the first drug for patients with dry age-related macular degeneration (AMD) and geographic atrophy (GA), SYFOVRETM, which must be injected intravitreally [IVT]) every 25-60 days...Currently, there is no published evidence of high RPE transfection via IVT injection, the preferred delivery method to ensure the least amount of surgical damage to the retina. In this study, I have identified a promising candidate for RPE transfection via IVT injection and quantitatively measured its effectiveness over an established AAV capsid, 7m8, capable of outer retinal transfection."

Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders

Complement Inhibition Treatment for Geographic Atrophy in Eyes with Exudative AMD (ARVO 2025) - "Methods A retrospective chart review was performed of eyes treated for GA with either intravitreal pegcetacoplan (Syfovre; Apellis; Waltham, MA) or avacincaptad pegol (Izervay; Astellas; Northbrook, IL) injections. Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Ophthalmology • Wet Age-related Macular Degeneration

Safety of Avacincaptad Pegol for Geographic Atrophy: Real-world experience. (ARVO 2025) - "These findings suggest that avacincaptad pegol is generally safe for managing GA, providing hope for patients with this challenging condition. However, we recommend careful monitoring during treatment to manage potential side effects effectively."

Clinical • Real-world • Real-world evidence • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ocular Infections • Ocular Inflammation • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Optimizing functional endpoints in geographic atrophy: Microperimetry insights from OAKS and GALE (ARVO 2025) - "The findings from the OAKS and GALE studies showed that pegcetacoplan helped preserve retinal sensitivity, meaning it could slow down the vision loss associated with geographic atrophy. This research suggests that microperimetry might be a better way to assess the effect of eye treatments, potentially leading to improved care and outcomes for patients with age-related vision problems."

Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

US retina specialists' preferences for treating geographic atrophy with gene therapy and complement inhibitors (ARVO 2025) - "Results The final dataset included 166 RS, 25% (n=42) had experience as investigators in GA clinical trials, the majority had utilised complement inhibitors (pegcetacoplan (77%; n=127) and/or avacincaptad pegol (58%; n=97)) and 48% (n=80) owned a private practice...They also preferred a one-time injection over monthly injections and would tolerate some increase in the risk of side effects for this. These insights into provider preferences may inform the development of new treatments that align with what retina specialists believe will best help their GA patients."

Gene therapy • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Ophthalmology • Wet Age-related Macular Degeneration

Automatic OCT segmentation biomarkers for disease staging in Geographic Atrophy (ARVO 2025) - "Patients had to be treated with pegcetacoplan (Syfovre, Apellis) to be included...Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Biomarker • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Protective Efficacy of ABI-201 in a Sodium Iodate-Induced Retinal Damage Model of Dry AMD in Non-Human Primates (ARVO 2025) - "Compared to an existing treatment, Syfovre®, ABI-201 performed better in maintaining vision-related functions.In lab tests, the protective component of ABI-201 also shielded human eye cells from damage caused by oxidative stress, a major factor in retinal diseases. Importantly, the treatment was safe, with no signs of inflammation or other side effects.These findings suggest ABI-201 has the potential to offer a new, highly effective way to protect vision in people at risk of losing sight due to conditions like dry AMD, providing hope for better outcomes and improved quality of life."

Clinical • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ocular Inflammation • Ophthalmology • Retinal Disorders • CD59

Growth Rates of Large Choroidal Hypertransmission Defects over a Wide Range of Lesion Sizes in AMD (ARVO 2025) - "Follow-up visits were censored once exudation developed or pegcetacoplan intravitreal injections were initiated...When hyperTDs first appear, their sqrt growth rates increase with lesion size until they reach a total size of about 2.5 mm2 (1 disc area (DA)), which is followed by a plateau in average sqrt growth rates, supporting the current inclusion criteria of 1-7 DAs for most clinical trials. However, if lesions smaller than 1 DA are included, they should be analyzed separately."

Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Utilization Patterns and Adverse Outcomes of Anti-Complement Therapy in Geographic Atrophy in the Clinical Setting (ARVO 2025) - "Methods A retrospective cohort study using Vestrum Health Retina Database including 13,988 eyes treated with pegcetacoplan and 5,720 treated with avacincaptad pegol between February 2023 to September 2024...However, they require regular eye injections and may carry risks like inflammation or infection, which the study highlights. This research provides insights into these treatments' real-world benefits and risks, helping doctors make informed decisions for this condition."

Adverse events • Clinical • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ocular Infections • Ocular Inflammation • Ophthalmology • Retinal Disorders

A Review of Early-Stage Clinical Trials for the Treatment of Geographic Atrophy (ARVO 2025) - "In 2023, Syfovre (Apellis) received the first FDA approval for the treatment of GA, followed by Izervay (Iveric). Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Clinical • Review • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Real-World Efficacy of Intravitreal Pegcetacoplan for the Treatment of Geographic Atrophy in AMD: 1-Year Report (ARVO 2025) - "Our real-world experience with pegcetacoplan showed a slowing of the annualized growth rate of GA by about 39% at one year, and this treatment benefit was the same for both foveal and non-foveal GA. Thus, our results suggest that pegcetacoplan is an effective therapy for slowing the growth of GA and is even better than the clinical trials because we enrolled patients with subjective, ongoing vision loss."

Clinical • Real-world • Real-world effectiveness • Real-world evidence • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Characteristics of Age-Related Macular Degeneration Patients with Inactive Choroidal Neovascularization Treated with Pegcetacoplan for Geographic Atrophy (ARVO 2025) - "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Clinical • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Real-World Safety of Intravitreal Pegcetacoplan for the Treatment of Geographic Atrophy in AMD: 1-Year Report (ARVO 2025) - "It appeared as though this macular fluid was the direct result of the pegcetacoplan. We found that when eyes developed macular fluid 4 weeks after an injection, we were able to reduce this fluid by extending the treatment interval to every 8 weeks."

Clinical • Real-world • Real-world evidence • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ocular Infections • Ocular Inflammation • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

GALE: An Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan (APL-2) in Subjects With Geographic Atrophy Secondary to AMD (clinicaltrials.gov) - P3 | N=1200 | Active, not recruiting | Sponsor: Apellis Pharmaceuticals, Inc. | Trial completion date: Sep 2025 ➔ Sep 2026

Trial completion date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Apellis Pharmaceuticals Reports Fourth Quarter and Full Year 2024 Financial Results (GlobeNewswire) - "On track to initiate two Phase 3 trials of EMPAVELI in focal segmental glomerulosclerosis (FSGS) and in delayed graft function (DGF) in 2H 2025...Total revenue was $212.5 million for the fourth quarter of 2024, which consisted of $167.8 million in U.S. net product revenue of SYFOVRE, $23.4 million in U.S. net product revenue of EMPAVELI and $21.4 million in licensing and other revenue associated with the Sobi collaboration...For the full year 2024, total revenue was $781.4 million, which consisted of $611.8 million in U.S. net product revenue of SYFOVRE, $98.1 million in U.S. net product revenue of EMPAVELI and $71.4 million in licensing and other revenue associated with the Sobi collaboration."

Commercial • New P3 trial • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Focal Segmental Glomerulosclerosis • Macular Degeneration • Paroxysmal Nocturnal Hemoglobinuria

Visual Function Benefit After Treatment With Pegcetacoplan: Microperimetry Analysis From The Phase 3 Oaks Trial: Microperimetry: Visual Function Benefit With Pegcetacoplan. (PubMed, Am J Ophthalmol) - "Microperimetry demonstrates a reduced rate of visual function loss in the central macula and junctional zone with pegcetacoplan treatment in GA due to AMD."

Journal • P3 data • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Modeling pegcetacoplan treatment effect for atrophic age-related macular degeneration with AI-based progression prediction. (PubMed, Int J Retina Vitreous) - "This model based on an 80% growth of RORA after two years illustrates the variable effect of treatment with Pegcetacoplan according to the individual situation, supporting personalized medical care."

Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Favorable Outcome After Pegcetacoplan (Syfovre)-Associated Occlusive Retinal Vasculitis. (PubMed, Ophthalmic Surg Lasers Imaging Retina) - "After intravitreal steroids and ceftazidime, the vision ultimately improved to 20/20. Periocular and systemic steroids and antibiotics were associated with a favorable visual outcome in this patient. [Ophthalmic Surg Lasers Imaging Retina 2025;56:XX-XX.]."

Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ocular Inflammation • Ophthalmology • Retinal Disorders • Vasculitis

Apellis Receives Approval of SYFOVRE (pegcetacoplan) in Australia for Geographic Atrophy (GA) (GlobeNewswire) - "Apellis Pharmaceuticals...announced today that the Therapeutic Goods Administration (TGA) has approved SYFOVRE (pegcetacoplan) for the every-other-month treatment of adult patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) with an intact fovea and when central vision is threatened by GA lesion growth. SYFOVRE is the first and only approved treatment for GA in Australia....The approval is based on results from the Phase 3 OAKS and DERBY studies at 24 months. In the studies, treatment with both every-other-month and monthly SYFOVRE slowed GA progression and showed a generally well-tolerated safety profile."

Approval • Age-related Macular Degeneration

Population Pharmacokinetics of Pegcetacoplan in Patients with Geographic Atrophy or Neovascular Age-related Macular Degeneration. (PubMed, Ophthalmol Sci) - "This population PK model adequately described the serum concentration-time profile of pegcetacoplan after intravitreal administration in adults with GA or nAMD. Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article."

Journal • PK/PD data • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Geographic Atrophy in Age-Related Macular Degeneration. (PubMed, Dtsch Arztebl Int) - "In Europe at present, there is no approved treatment for GA due to AMD. There is thus a continuing need for preventive and rehabilitative measures such as smoking cessation, a balanced diet, and magnifying visual aids for patients in the advanced stages of the disease."

Journal • Review • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Gene Therapies • Infectious Disease • Macular Degeneration • Ophthalmology • Retinal Disorders • Tobacco Cessation

Valeda Light Delivery System for age-related macular degeneration. (PubMed, Med Lett Drugs Ther) - No abstract available

Journal • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Pegcetacoplan for the treatment of geographic atrophy due to age-related macular degeneration: a plain language summary of OAKS and DERBY clinical studies. (PubMed, Immunotherapy) - No abstract available

Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders