Syfovre (pegcetacoplan intravitreal) / Apellis - LARVOL DELTA

VERDANT: A Study to Test Whether BI 771716 Helps People With an Advanced Form of Age-related Macular Degeneration (AMD) Called Geographic Atrophy (clinicaltrials.gov) - P2 | N=180 | Recruiting | Sponsor: Boehringer Ingelheim | Initiation date: Jan 2025 ➔ May 2024

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

Visual Acuity and Quality of Life Outcomes with Pegcetacoplan Treatment: A Post Hoc Analysis from the OAKS and DERBY Trials. (PubMed, Am J Ophthalmol) - "Pegcetacoplan treatment demonstrated a potentially meaningful slower decline in visual acuity and QoL for patients with GA lesions ≥250 μm from the foveal center. The change in visual acuity and QoL seen with pegcetacoplan treatment for patients with GA lesions <250 μm from the foveal center were within the limits of variability."

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

A Prospective, Multicenter, Open-Label, Observational Phase 4 Study to Evaluate Real-World Safety, Tolerability, and Treatment Patterns of Pegcetacoplan (Syfovre) in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration (clinicaltrials.gov) - P=N/A | N=300 | Recruiting | Sponsor: Apellis Pharmaceuticals, Inc. | Trial completion date: Oct 2027 ➔ Jun 2028 | Trial primary completion date: Oct 2027 ➔ Jun 2028

Real-world evidence • Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

A Study to Investigate Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD), and Immunogenicity of RO7669330 in Participants With Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD) (clinicaltrials.gov) - P1 | N=132 | Recruiting | Sponsor: Hoffmann-La Roche | Not yet recruiting ➔ Recruiting

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

Pegcetacoplan Reduces Rate of Geographic Atrophy Growth, 1-Year Trial Data Shows (HCPLive) - P3 | N=1,200 | GALE (NCT04770545) | Sponsor: Apellis Pharmaceuticals, Inc. | "12-month results from the ongoing GALE open-label extension study have indicated that pegcetacoplan successfully reduced the mean rate of geographic atrophy (GA) growth with increasing efficacy...Of the 792 patients who enrolled, 790 were included in the safety population and 780 in the modified full analysis set. During the first 12 months, 6305 pegcetacoplan injections were administered – 88.1% of patients (696/790) received ≥75% of planned injections, defined as ≥9 of 12 in PM-PM and SM-PM groups and 5 of 6 in the PEOM-PEOM and SEOM-PEOM groups...The most common treatment-emergent adverse events, which were defined as occurring in ≥5% of patients in ≥1 treatment group, included vitreous floaters, ocular discomfort, new-onset eAMD..."

Clinical data • Age-related Macular Degeneration

Pegcetacoplan for Geographic Atrophy Over 30 Months: Data From OAKS, DERBY, and the GALE Long-Term Extension Study. (PubMed, Ophthalmic Surg Lasers Imaging Retina) - "Pegcetacoplan reduced GA growth rate up to 45% with increasing efficacy over 30 months and demonstrated a favorable safety profile. [Ophthalmic Surg Lasers Imaging Retina 2025;56:XX-XX.]."

Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Inflammation • Macular Degeneration • Ocular Infections • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain • Retinal Disorders • Wet Age-related Macular Degeneration

A Study to Investigate Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD), and Immunogenicity of RO7669330 in Participants With Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD) (clinicaltrials.gov) - P1 | N=132 | Not yet recruiting | Sponsor: Hoffmann-La Roche

New P1 trial • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Innovative technologies for the treatment of dry age-related macular degeneration (AMD) - modern therapeutic perspectives and their future. (PubMed, Rom J Ophthalmol) - "Advances include molecularly targeted therapies (Pegcetacoplan, Avacincaptad Pegol) that reduce inflammation, gene therapy (HMR59) protecting RPE cells, and mitochondria-targeted drugs (SS-31) mitigating oxidative stress...However, further clinical trials are needed to optimize these strategies, assess long-term outcomes, and expand patient access to effective treatments. These advancements have the potential to significantly improve the quality of life for individuals affected by dry AMD."

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

Ocular Adverse Events Associated with Pegcetacoplan and Avacincaptad Pegol for Geographic Atrophy: A Population-Based Pharmacovigilance Study: Ocular Adverse Events of Pegcetacoplan & Avacincaptad Pegol. (PubMed, Am J Ophthalmol) - "Based on current prescribing patterns, a broader spectrum of ocular AEs were reported for pegcetacoplan than avacincaptad pegol. These findings aim to enhance clinicians' understanding of the safety profiles of these agents, enabling informed patient care and heightened vigilance of these novel GA treatments."

Adverse events • Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Hematological Disorders • Macular Degeneration • Macular Edema • Ocular Infections • Ocular Inflammation • Ophthalmology • Retinal Disorders • Vasculitis

Clinical Outcomes of Treatment of Geographic Atrophy: A Narrative Review. (PubMed, Ophthalmol Ther) - "The management of geographic atrophy has advanced with therapies like pegcetacoplan and avacincaptad pegol showing clear benefits in slowing lesion growth. However, safety concerns, such as neovascular complications, persist. Photobiomodulation and dietary supplementation provide alternative options with modest benefits, particularly in the early stages. Larger studies are needed to confirm long-term safety, efficacy, and optimal treatment strategies."

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

Pegcetacoplan Treatment for Geographic Atrophy in Age-Related Macular Degeneration Over 36 Months: Data From OAKS, DERBY, and GALE. (PubMed, Am J Ophthalmol) - P1, P3 | "Over 36 months, pegcetacoplan continued to reduce GA growth with increasing efficacy over time and reduced formation of new scotomatous points. The safety profile of pegcetacoplan in the first 12 months of GALE was consistent with the prior 24-month OAKS and DERBY studies."

Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Inflammation • Macular Degeneration • Ocular Infections • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain • Retinal Disorders • Vasculitis • Wet Age-related Macular Degeneration

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