Syfovre (pegcetacoplan intravitreal) / Biogen - LARVOL DELTA

Two-Year Experience with Intravitreal Pegcetacoplan for the Treatment of Geographic Atrophy in Clinical Practice. (PubMed, Am J Ophthalmol) - "Pegcetacoplan therapy reduced the growth rate of GA by about 41% over 2 years and continued to show accumulated efficacy during the second year."

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

Early Versus Delayed Pegcetacoplan Treatment for Geographic Atrophy Secondary to Age-Related Macular Degeneration in OAKS, DERBY, and GALE. (PubMed, Am J Ophthalmol) - "Pegcetacoplan demonstrated effectiveness in slowing GA progression and consistent safety over 48 months. Long-term anatomic and functional outcomes support the importance of early treatment to maximize the preservation of retinal tissue and function."

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

Predictive Value of Perilesional Fundus Autofluorescence Patterns and OCT-Based Ellipsoid Zone/Retinal Pigment Epithelium Loss Ratios in Geographic Atrophy Growth Rates. (PubMed, Ophthalmol Sci) - P3 | "OCT and FAF images from the OAKS (NCT03525613) and DERBY (NCT03525600) trials, 2 phase III, randomized controlled clinical trials of intravitreal pegcetacoplan for the treatment of GA secondary to age-related macular degeneration, were analyzed...There was a greater treatment effect in eyes with the diffuse-trickling pattern, presenting with higher EZ/RPE loss ratios on OCT in a small subgroup. Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article."

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

Intraocular inflammation and presumed retinal vasculitis following initial pegcetacoplan intravitreal injection. (PubMed, Digit J Ophthalmol) - "At day 83, visual acuity remained NLP. This patient developed presumed occlusive retinal vasculitis, progressing from visual acuity of 20/50 to NLP within 2 days of initial subjective vision loss and 12 days of a single intravitreal pegcetacoplan injection."

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

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=27 | Terminated | Sponsor: Hoffmann-La Roche | Completed ➔ Terminated; The study was terminated prematurely by the Sponsor due to observation of dose-limiting adverse events meeting the protocol-defined stopping criteria.

Trial termination • 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=27 | Completed | Sponsor: Hoffmann-La Roche | Active, not recruiting ➔ Completed | Trial completion date: Jan 2027 ➔ Mar 2026 | Trial primary completion date: Jan 2027 ➔ Feb 2026

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

Examining Real-World Attrition Among Patients Undergoing Complement Inhibitor Intravitreal Therapy for Geographic Atrophy. (PubMed, J Vitreoretin Dis) - "To identify characteristics of patients with geographic atrophy (GA) who received pegcetacoplan or avacincaptad pegol and discontinued treatment...nAMD treatment intervals of more than 6 weeks (OR, 0.59; 95% CI, 0.39-0.90; P = .014) and GA treatment intervals of 6 to 8 weeks (OR, 0.56; 95% CI, 0.43-0.74; P < .001) contributed to decreased odds of attrition. Poor baseline vision and development of new nAMD are associated with patient attrition, and the combination of nAMD and GA and treatment burden challenges patient retention."

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

Nonarteritic Anterior Ischemic Optic Neuropathy Associated With Retinal Nerve Fiber Layer Thickening Following Intravitreal Pegcetacoplan Therapy. (PubMed, J Vitreoretin Dis) - "Peripapillary RNFL thickness measured by OCT may be useful for monitoring patients receiving pegcetacoplan therapy and may allow earlier detection of nonarteritic anterior ischemic optic neuropathy. Changes in RNFL thickness may precede the development of nonarteritic anterior ischemic optic neuropathy."

Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain

Comparative Endophthalmitis Rates of Anti-VEGF, Corticosteroid Implants and Anti-Complement Injections: An IRIS® Registry Analysis. (PubMed, Am J Ophthalmol) - "The incidence of post-injection endophthalmitis is greater with corticosteroid implants and anticomplement therapies. Heightened aseptic vigilance may be warranted with injections that necessitate larger gauge needles, modulate the immune response and/or are prepared from a vial."

Journal • Ocular Infections • Ocular Inflammation • Ophthalmology • Pediatrics

One-Year Results of Geographic Atrophy Patients with Pre-Existing nAMD treated with both anti-VEGF therapy and pegcetacoplan. (PubMed, Retina) - "Concurrent anti-VEGF and pegcetacoplan therapy was associated with an overall reduction in GA lesion growth rate and stabilized nAMD."

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

Expert Opinion: Management of Intraocular Inflammation and Vasculitis After Intravitreal Pegcetacoplan for Geographic Atrophy. (PubMed, Ophthalmic Surg Lasers Imaging Retina) - "While IOI and vasculitis are rare, patients can be educated to call a retina specialist promptly if symptoms or any vision loss develop, especially within the 2 weeks following the first injection. In addition to excluding an infectious etiology, individualized management with multiroute corticosteroids can be considered."

Journal • Age-related Macular Degeneration • Cardiovascular • Dry Age-related Macular Degeneration • Endocrine Disorders • Hypertension • Infectious Disease • Inflammation • Novel Coronavirus Disease • Ocular Inflammation • Pain • Vasculitis

Virus-biomimetic melanin nanoparticles with spiky structures and neutral zwitterionic shell for non-invasive prevention of dry AMD. (PubMed, J Control Release) - "Notably, twice-daily topical administration of VMNPs markedly inhibited retinal degeneration in rabbits, achieving therapeutic efficacy comparable to a single intravitreal injection of pegcetacoplan. Collectively, these findings establish a promising paradigm for the non-invasive clinical treatment of dAMD."

Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Inflammation • Macular Degeneration • Ophthalmology • Retinal Disorders • HMOX1 • SLC7A11

Retinal dystrophies simulating geographic atrophy: A diagnostic challenge. (PubMed, Acta Ophthalmol) - "Misdiagnosis can lead to inappropriate treatment recommendations, impacting patient health and financial burden. This paper outlines the similarities and differences among prevalent diseases such as late-onset Stargardt disease, PRPH2-associated disease and maternally inherited diabetes and deafness (MIDD), resembling GA to aid in accurate diagnosis."

Journal • Review • Age-related Macular Degeneration • Diabetes • Dry Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Metabolic Disorders • Ophthalmology • Otorhinolaryngology • Retinal Disorders

Pharmacological Characterization of a Novel Complement C3 Inhibitor Antibody Fragment. (PubMed, Ophthalmol Ther) - "The antibody fragment is a potent and specific C3 inhibitor that displays dose-dependent inhibition of MAC formation across all three complement pathways and effectively crosses the BrM. In line with these results, the antibody fragment is being investigated in patients with GA. Graphical abstract available for this article."

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

Biogen ends Apellis research programs, cuts ‘small number’ of roles after $5.6B buyout (FierceBiotech) - "As part of the move, 'a small number of roles within the research organization have been eliminated,” the spokesperson added. Biogen continues to assess Apellis’ clinical and preclinical portfolio to “further evaluate strategic fit,' the company representative said...In cutting these research efforts, Biogen is making explicit its desire to focus on Apellis’ Empaveli and Syfovre, marketed drugs which share the active ingredient pegcetacoplan and which served as the centerpiece of the March deal....Besides the approved Apellis meds, the biotech had been conducting research on clinical-stage asset APL-3007 before the merger, according to its pipeline."

Licensing / partnership • Ophthalmology • Paroxysmal Nocturnal Hemoglobinuria

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. | Trial completion date: Mar 2028 ➔ Nov 2027 | Initiation date: Sep 2025 ➔ Jun 2025 | Trial primary completion date: Sep 2027 ➔ Jun 2027

Trial completion date • Trial initiation date • Trial primary 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. | Trial completion date: Nov 2027 ➔ Mar 2028 | Initiation date: Jun 2025 ➔ Sep 2025 | Trial primary completion date: Jun 2027 ➔ Sep 2027

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

Complement Inhibition in Geographic Atrophy: Clinical Evidence for Pegcetacoplan and Avacincaptad Pegol. (PubMed, Cureus) - "Despite favorable structural outcomes, functional endpoints like best-corrected visual acuity (BCVA) and low-luminance visual acuity (LLVA) have shown limited improvement, and concerns remain regarding their side effect profile. This narrative review provides an overview of GA, emphasizing its pathophysiology and the role of the complement cascade, and discusses the clinical relevance of complement inhibitors in the current management of the disease."

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

FCPGA-2025: Lesion-tailored, Fundus-controlled Perimetry in Geographic Atrophy (GA). (clinicaltrials.gov) - P=N/A | N=50 | Recruiting | Sponsor: University of Utah | Not yet recruiting ➔ Recruiting

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

Short-Term Intraocular Pressure Trends After Intravitreal Pegcetacoplan Injection. (PubMed, J Vitreoretin Dis) - " In this cohort of patients receiving pegcetacoplan IVI for GA, the initial IOP spike was high, but the majority of patients did not require AC paracentesis for acute IOP control. Eyes that had a PVD or severe IOP response in previous injections tended to have a higher IOP spike after subsequent injections."

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

Efficacy of Continuous Pegcetacoplan Treatment for Subfoveal Geographic Atrophy in Age-Related Macular Degeneration: 36-Month Results from OAKS, DERBY, and GALE Open-Label Extension. (PubMed, Clin Ophthalmol) - P3 | "To report efficacy of intravitreal pegcetacoplan treatment over 36 months in eyes with subfoveal geographic atrophy (GA)...Safety profile in GALE was consistent with OAKS and DERBY. Long-term efficacy of pegcetacoplan in slowing GA progression was demonstrated over 36 months in eyes with subfoveal GA."

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

FCPGA-2025: Lesion-tailored, Fundus-controlled Perimetry in Geographic Atrophy (GA). (clinicaltrials.gov) - P=N/A | N=50 | Not yet recruiting | Sponsor: University of Utah

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

Geographic atrophy: From slowing lesions to preserving vision. (PubMed, Curr Opin Ophthalmol) - "Structural slowing alone does not fully capture therapeutic value in geographic atrophy. Functional measures provide a more sensitive and patient-centered assessment of disease progression and treatment impact."

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

Biogen Completes Acquisition of Apellis Pharmaceuticals (GlobeNewswire) - "The acquisition adds two best-in-class commercialized products, EMPAVELI and SYFOVRE, significantly bolstering Biogen’s near-term growth outlook and accelerating the Company’s expansion into nephrology. Together, the products recorded $689 million in net product revenue in 2025. This transaction will strengthen Biogen’s revenue and EPS growth potential by being accretive to Biogen’s Non-GAAP diluted EPS in 2027 and is expected to materially increase Biogen’s non-GAAP EPS compound annual growth rate (CAGR) through the end of the decade. Updated financial guidance will be provided in conjunction with the Q2 earnings report in July."

M&A • Age-related Macular Degeneration • Complement-mediated Rare Disorders • Ophthalmology • Paroxysmal Nocturnal Hemoglobinuria

Real-world outcomes of patients with geographic atrophy developing neovascular age-related macular degeneration after receiving anti-complement therapy (ARVO 2026) - "Purpose Geographic atrophy (GA) treatments targeting complement inhibition, pegcetacoplan and avacincaptad pegol, have demonstrated efficacy in reducing GA lesion size growth but have also been associated with higher rates of conversion to neovascular age-related macular degeneration (nAMD) in clinical trials...We found that patients who received complement-inhibiting injections were more likely to develop nAMD, especially with increasing numbers of injections. These results highlight the importance of careful monitoring for patients receiving GA treatment."

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