Syfovre (pegcetacoplan intravitreal) / Apellis - LARVOL DELTA

Novel drug development for geographic atrophy. (PubMed, Asia Pac J Ophthalmol (Phila)) - "A marked important therapeutic milestone with the first FDA approvals of complement inhibitors, Pegcetacoplan and Avacincaptad pegol, demonstrated a modest but statistically significant slowing of lesion growth...Key challenges include treatment burden associated with frequent intravitreal injections, safety considerations, and the persistent disconnect between anatomic endpoints and functional outcomes. These underscore the necessity for future treatments that not only alter disease anatomy but also effectively preserve patients' vision and improve their quality of life."

Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Gene Therapies • Inflammation • Macular Degeneration • Metabolic Disorders • Ophthalmology • Retinal Disorders

Deep learning in geographic atrophy: rethinking age-related macular degeneration progression and treatment. (PubMed, Ann Transl Med) - No abstract available

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

Guiding syringe selection for intravitreal injections: injectability and stability analysis of compounded pegcetacoplan (SYFOVRE) and the broader implications for high-viscosity ophthalmic therapies. (PubMed, Int J Retina Vitreous) - No abstract available

Journal • 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 | Active, not recruiting | Sponsor: Apellis Pharmaceuticals, Inc. | Trial completion date: Jun 2028 ➔ Jun 2027 | Trial primary completion date: Jun 2028 ➔ Jun 2027

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

Transient vitreous opacity following combined intravitreal injection of pegcetacoplan and faricimab-svoa in patients with neovascular age-related macular degeneration and geographic atrophy. (PubMed, Am J Ophthalmol Case Rep) - No abstract available

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

Ophthalmology : what's new in 2025 (PubMed, Rev Med Suisse) - "Presbyopia, atrophic age-related macular degeneration, and thyroid eye disease are ophthalmic conditions that can significantly affect patients' vision and quality of life. In this article, we present the most recent therapeutic innovations for their management, including the myotic eye drop aceclidine for presbyopia, complement inhibitors (pegcetacoplan, avacincaptad pegol) to slow the progression of atrophic AMD, and teprotumumab, an anti-IGF1-R antibody, to reduce proptosis and diplopia in thyroid eye disease."

Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Thyroid Eye Disease • IGF1

Integrative Landscape of Dry AMD Pathogenesis, Models, and Emerging Therapeutic Strategies. (PubMed, Int J Mol Sci) - "Although the recent U.S. Food and Drug Administration (FDA) approvals of pegcetacoplan and avacincaptad pegol represent major milestones, their therapeutic effects remain modest...Finally, we review current and emerging therapeutic strategies-including complement inhibitors, visual cycle modulators, and mitochondrial-protective approaches-and outline future directions for translational research. Collectively, this review synthesizes mechanistic insights, disease models, and therapeutic innovation to support the development of targeted and stage-specific interventions for dry AMD."

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

Study to Evaluate a Pegcetacoplan (Syfovre®) Prefilled Syringe (clinicaltrials.gov) - P3 | N=44 | Completed | Sponsor: Apellis Pharmaceuticals, Inc. | Not yet recruiting ➔ Completed

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

Central retinal vein occlusion following intravitreal injections: a case series highlighting multifactorial risk. (PubMed, Int J Retina Vitreous) - No abstract available

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

Strategic Response to Market Disruption: The Syfovre Case and Post-Launch Volatility in Biopharmaceutical Commercialization (ASHP 2025) - No abstract available

Clinical

Complement inhibitor use is not a significant predictor of cancer incidence: A real-world study of patients requiring chronic complement blockade. (ASH 2025) - "Sixwere treated with eculizumab and ravulizumab, 4 with eculizumab alone, 1 with ravulizumab alone, and 1with eculizumab, ravulizumab, pegcetacoplan, and iptacopan at various points...Factors such asfamily history, smoking/alcohol use, and past medical history of HPV, hepatitis B, hepatitis C, and HIVremain established predictors for cancer incidence. Further analysis in a larger cohort is needed tovalidate these initial results."

Clinical • Real-world • Real-world evidence • Age-related Macular Degeneration • Atypical Hemolytic Uremic Syndrome • Bladder Cancer • Breast Cancer • CNS Disorders • Complement-mediated Rare Disorders • Endocrine Cancer • Hepatitis B • Hepatitis C • Hepatology • Human Immunodeficiency Virus • Immunology • Infectious Disease • Kaposi Sarcoma • Macular Degeneration • Myasthenia Gravis • Nephrology • Neuromyelitis Optica Spectrum Disorder • Oncology • Ophthalmology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Renal Disease • Retinal Disorders • Sarcoma • Solid Tumor

Effect of Pegcetacoplan on Aqueous Humor Proteome in Geographic Atrophy: A Prospective Exploration. (PubMed, Invest Ophthalmol Vis Sci) - "Although no changes in the target protein C3 were detected following treatment, significant changes in proteins tightly connected to complement, inflammation, and coagulation were observed. These findings underscore the need for further investigation into the biological and clinical relevance of the observed molecular shifts."

Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Inflammation • Macular Degeneration • Ophthalmology • Retinal Disorders • C1QB

Geographic Atrophy Progression in Clinical Practice Before and After Pegcetacoplan Treatment. (PubMed, Vision (Basel)) - "This retrospective study evaluated changes in ocular characteristics and retinal pigment epithelium (RPE) and photoreceptor ellipsoid zone (EZ) depletion rates before and after intravitreal pegcetacoplan initiation in clinical practice...These real-world findings align with data from the pegcetacoplan phase 3 trials, showing reduced RPE and EZ depletion rates without changes in rates of BRVA loss. Additional studies are warranted."

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

Real-World Clinical Usage and Safety Profile of Intravitreal Pegcetacoplan in Age-Related Macular Degeneration-Associated Geographic Atrophy. (PubMed, J Vitreoretin Dis) - "Preserved or improved visual acuity while undergoing anti-VEGF therapy was noted in 384 of 396 patients (97%). Real-world data on intravitreal pegcetacoplan treatment identifies clinician practice patterns and demonstrates an acceptable safety profile, with complications leading to long-term vision loss following pegcetacoplan administration being rare in this cohort."

Journal • Real-world evidence • Age-related Macular Degeneration • Cardiovascular • Dry Age-related Macular Degeneration • Glaucoma • Hematological Disorders • Inflammation • Macular Degeneration • Ocular Inflammation • Ophthalmology • Retinal Disorders • Uveitis • Vasculitis

Short-Term Changes in Intraocular Pressure Following Intravitreal Injection of Pegcetacoplan. (PubMed, J Vitreoretin Dis) - "To determine short-term changes in intraocular pressure (IOP) after intravitreal injection of 0.1 mL pegcetacoplan (Syfovre; Apellis Pharmaceuticals) for the treatment of geographic atrophy (GA)...Following pegcetacoplan injections, immediate IOP elevation was observed, which gradually declined within the first 30 minutes. Eyes with higher baseline IOP or a history of glaucoma exhibited higher postinjection IOP."

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

Apellis Announces 5-Year GALE Data Showing SYFOVRE (pegcetacoplan injection) Delayed Progression of Geographic Atrophy by ~1.5 Years (GlobeNewswire) - "The results demonstrate that both every-other-month and monthly SYFOVRE delayed GA lesion growth by approximately 1.5 years in patients with nonsubfoveal GA when compared to sham/projected sham....The safety profile of SYFOVRE through five years remained consistent with previously reported data."

P3 data • Age-related Macular Degeneration

Comparative Efficacy and Safety of Avacincaptad Pegol vs. Pegcetacoplan in Treatment of Geographic Atrophy: Insights From a Multilevel Network Meta-Regression (ISPOR-EU 2025) - "ACP was associated with a significantly lower incidence of new onset CNV compared to PEG while maintaining similar efficacy in slowing GA lesion growth and visual acuity loss. The key strength of this analysis is the use of ML-NMR, which leverages all available data to achieve robust population adjustment across all EMs identified through published evidence and/or clinical opinion. These insights help address a critical evidence gap in the evolving treatment paradigm for GA."

Clinical • Age-related Macular Degeneration • Dry Age-related Macular Degeneration

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 | Active, not recruiting | Sponsor: Apellis Pharmaceuticals, Inc. | Recruiting ➔ Active, not recruiting

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

Plain language summary of disease activity and therapeutic response to pegcetacoplan for geographic atrophy identified by deep learning-based analysis of OCT. (PubMed, Immunotherapy) - No abstract available

Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration

Efficacy of Intravitreal Pegcetacoplan vs Avacincaptad Pegol in Patients With Geographic Atrophy. (PubMed, J Vitreoretin Dis) - P3 | "A numerically greater reduction in GA lesion growth was observed with pegcetacoplan every other month vs avacincaptad pegol monthly (95% CI, -1.130 to -0.300; P = .25). Matching-adjusted indirect comparisons support a greater reduction in GA growth with pegcetacoplan monthly vs avacincaptad pegol monthly and no significant difference between pegcetacoplan every other month and avacincaptad pegol monthly."

Journal • Age-related Macular Degeneration • Dry 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=792 | Completed | Sponsor: Apellis Pharmaceuticals, Inc. | Active, not recruiting ➔ Completed | N=1200 ➔ 792 | Trial completion date: Sep 2026 ➔ Sep 2025

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

A Phase 2, Randomized, Placebo Controlled, Multicenter, Masked Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Multidose APL 3007 in Combination With Syfovre/Pegcetacoplan (APL-2) in Patients Diagnosed With Geographic Atrophy Secondary to Age Related Macular Degeneratio (clinicaltrials.gov) - P2 | N=240 | Recruiting | Sponsor: Apellis Pharmaceuticals, Inc.

New P2 trial • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Study to Evaluate a Pegcetacoplan (Syfovre®) Prefilled Syringe (clinicaltrials.gov) - P3 | N=36 | Not yet recruiting | Sponsor: Apellis Pharmaceuticals, Inc.

New P3 trial • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

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=188 | Active, not recruiting | Sponsor: Boehringer Ingelheim | Recruiting ➔ Active, not recruiting

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

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 | Trial completion date: Feb 2027 ➔ Oct 2026

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