Empaveli (pegcetacoplan SC) / Apellis, SOBI - LARVOL DELTA

Pegcetacoplan-induced remission in pediatric immune-complex membranoproliferative glomerulonephritis with comorbid autosomal recessive polycystic kidney disease: a case report. (PubMed, Front Med (Lausanne)) - "Initial treatment with prednisolone and mycophenolate mofetil failed to halt disease progression, and the patient became dialysis dependent. To our knowledge, this is the first pediatric case of IC-MPGN with genetically confirmed ARPKD successfully treated with pegcetacoplan. The case illustrates that renal recovery occurred following initiation of proximal complement inhibition with pegcetacoplan."

Journal • Antibody-mediated Rejection • Cardiovascular • Genetic Disorders • Glomerulonephritis • Hypertension • Lupus Nephritis • Nephrology • Pediatrics • Polycystic Kidney Disease • Renal Disease • PKHD1

SUCCESSFUL TREATMENT OF REFRACTORY IMMUNE COMPLEX-MEDIATED MEMBRANOPROLIFERATIVE GLOMERULONEPHRITIS (IC- MPGN) WITH PEGCETACOPLAN: A CASE REPORT (ISN-WCN 2026) - "These findings support mechanistic targeting of the alternative pathway in complement-dysregulated disease and justify prospective evaluation to define durability, optimal dosing and biomarkers of response.I have potential conflict of interest to disclose.Sobi provided funding for the medical writing activities of this abstract through MedDots FZC. The sponsor did not influence the content or direction of the manuscript but was allowed to review it as a courtesy.I did not use generative AI and AI-assisted technologies in the writing process."

Case report • Clinical • Glomerulonephritis • Lupus Nephritis • Nephrology

PEGCETACOPLAN FOR 52 WEEKS MAINTAINS PROTEINURIA REDUCTION AND EGFR STABILIZATION FOR PEDIATRIC PATIENTS: PHASE 3 VALIANT SUBGROUP ANALYSIS (ISN-WCN 2026) - P3 | "EK: is an employee of Apellis and may hold stocks or stock options AM: is an employee of Apellis and may hold stocks or stock options ES: is an employee of Sobi and may hold stocks or stock options JS: is an employee of Sobi and may hold stocks or stock options. MW: received consultancy fees from Novartis, Sobi, Travere, Roche, Apellis, Alexion, BioCryst, Purespring, Bayer, and WebMD; participates in clinical trials sponsored by Alexion, Bayer, Novartis, Roche, Chinook, Apellis, and Travere; and serves on speaker bureaus for Novartis, Roche, Vifor, Travere, Sobi, and GlaxoSmithKline.I did not use generative AI and AI-assisted technologies in the writing process."

Clinical • P3 data • Complement-mediated Rare Disorders • Glomerulonephritis • Lupus Nephritis • Nephrology • Otorhinolaryngology • Pediatrics • Rare Diseases • Renal Disease • EGFR

PEGCETACOPLAN FOR 52-WEEKS MAINTAINED PROTEINURIA REDUCTION REGARDLESS OF IMMUNOSUPPRESSANT USE OR NEPHROTIC RANGE PROTEINURIA AT BASELINE: VALIANT SUBGROUP ANALYSIS (ISN-WCN 2026) - P3 | "The safety profile of pegcetacoplan was consistent with previous studies.Previously presented at ASN 2025 (Journal of the American Society of Nephrology)I have potential conflict of interest to disclose.CMN: is the Associate Director for Molecular Otolaryngology and Renal Research Laboratory; receives NIH grant support (2R01DK110023-07); serves on advisory boards for Novartis, Apellis, BioCryst, and Alexion; participates as a site investigator for Novartis, Apellis, BioCryst, and Retrophin; is a member of the data safety monitoring board for Kira; serves as Chair of a data safety monitoring board for FIT4KID; and receives author royalties for UpToDate. FF: has received consulting fees from Apellis, Sobi, Novartis, Roche, Alexion, and AstraZeneca on behalf of the VALIANT investigatorsI did not use generative AI and AI-assisted technologies in the writing process."

Complement-mediated Rare Disorders • Glomerulonephritis • Inflammation • Lupus Nephritis • Nephrology • Otorhinolaryngology • Renal Disease

EFFICACY OF PEGCETACOPLAN IN PATIENTS WITH LOWER BASELINE PROTEINURIA: A SUBGROUP ANALYSIS FROM THE VALIANT TRIAL (ISN-WCN 2026) - "EK: is an employee of Apellis and may hold stocks or stock options. CMN: is the Associate Director for Molecular Otolaryngology and Renal Research Laboratory; receives NIH grant support (2R01DK110023-07); serves on advisory boards for Novartis, Apellis, BioCryst, and Alexion; participates as a site investigator for Novartis, Apellis, BioCryst, and Retrophin; is a member of the data safety monitoring board for Kira; serves as Chair of a data safety monitoring board for FIT4KID; and receives author royalties for UpToDate.I did not use generative AI and AI-assisted technologies in the writing process."

Clinical • Complement-mediated Rare Disorders • Glomerulonephritis • Infectious Disease • Lupus Nephritis • Meningococcal Infections • Nephrology • Otorhinolaryngology • Renal Disease

CLINICAL EFFICACY OF PEGCETACOPLAN VERSUS IPTACOPAN IN PATIENTS WITH C3 GLOMERULOPATHY: INDIRECT TREATMENT COMPARISONS (ISN-WCN 2026) - P3 | "The findings from this analysis may help guide clinicians managing patients with this rare condition. This abstract was also submitted for the ASN Kidney Week 2025 congress.I have potential conflict of interest to disclose.Bradley P Dixon reports consultancy from Apellis, Novartis, Alexion, AstraZeneca, Arrowhead, Calliditas; Andrew S Bomback reports consultancy and/or honoraria from Achillion, Alexion, Amgen, Apellis, Aurinia, Calliditas, Catalyst, Genentech, GSK, Kezar, Novartis, Otsuka, Q32, Silence Therapeutics, UpToDate; Carly Rich is an employee of Sobi; Mingyi Huang is an employee of Apellis; Piotr Wojciechowski is an employee of Clever Access; Rose Chang is an employee of the Analysis Group, Inc.; Fernando Caravaca-Fontán reports consultancy and/or honoraria from Alexion, Apellis, AstraZeneca, Novartis, Otsuka, Roche, Sobi, Vifor; Fadi Fakhouri reports consultancy and/or honoraria from Alexion, Apellis, AstraZeneca, Novartis, Roche, Sanofi, Sobi.I did..."

Clinical • Complement-mediated Rare Disorders • Glomerulonephritis

A pediatric case of C3 glomerulonephritis initially misclassified as IgA nephropathy with a favorable response to C3-targeted therapy. (PubMed, Pediatr Nephrol) - "Despite standard immunosuppressive treatment with prednisone and mycophenolic acid plus angiotensin-converting enzyme inhibitors, she achieved only partial remission and subsequently relapsed, exhibiting severe, persistent C3 consumption (6-8 mg/dl) and sustained C5b9 activation (1059 ng/ml)...After 1 year and 9 months without response to immunosuppressive treatment, the C3 inhibitor pegcetacoplan was initiated...This case highlights the inherent diagnostic complexity of C3GN, suggesting the critical role of repeat biopsies in treatment-refractory, complement-dysregulated glomerulonephritis. It strongly supports the potential efficacy of C3 inhibition as a targeted therapeutic approach for patients with C3GN."

Journal • Complement-mediated Rare Disorders • Glomerulonephritis • IgA Nephropathy • Lupus Nephritis • Nephrology • Pediatrics • Renal Disease

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

APL-2 and Pembrolizumab Versus APL-2, Pembrolizumab and Bevacizumab Versus Bevacizumab Alone for the Treatment of Recurrent Ovarian, Fallopian Tube, or Primary Peritoneal Cancer and Malignant Effusion (clinicaltrials.gov) - P2 | N=60 | Recruiting | Sponsor: Roswell Park Cancer Institute | Trial completion date: Apr 2027 ➔ Apr 2028 | Trial primary completion date: Apr 2026 ➔ Apr 2028

Trial completion date • Trial primary completion date • Carcinosarcoma • Fallopian Tube Cancer • Oncology • Ovarian Cancer • Ovarian Serous Adenocarcinoma • Peritoneal Cancer • Sarcoma • Solid Tumor

Indirect treatment comparison of iptacopan versus pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and residual anemia despite C5 inhibitor treatment. (PubMed, J Comp Eff Res) - "No significant difference was noted in CfB in LDH and FACIT-Fatigue score. These findings suggest that iptacopan may improve Hb levels and reduce transfusion dependency compared with pegcetacoplan in PNH patients with residual anemia despite C5i and must be interpreted in the context of ITCs."

Journal • Anemia • Complement-mediated Rare Disorders • Fatigue • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Pegcetacoplan use in Pediatric Immune Complex-Membranoproliferative Glomerulonephritis (IC-MPGN), Dense Deposit Disease (DDD), Complement 3 Glomerulopathy (C3G): a Single Center Case Series (PAS 2026) - No abstract available

Clinical • Complement-mediated Rare Disorders • Glomerulonephritis • Lupus Nephritis • Nephrology • Pediatrics

Low Risk for Meningococcal Infections with Systemically Administered Pegcetacoplan, a Complement C3 and C3b Inhibitor (THSNA 2026) - P3 | "Clinical benefits of the initially available C5 inhibitors that block terminal complement activation (eculizumab, ravulizumab, crovalimab) paved the way for the development of proximal inhibitors, including the C3/C3b inhibitor pegcetacoplan, the factor B inhibitor iptacopan, and the add-on (to C5 inhibitors) factor D inhibitor danicopan. Understanding the safety profile of pegcetacoplan and other complement-targeted therapies will help physicians and patients make informed treatment decisions for individuals with complement-mediated conditions. For over 7 years of total systemic pegcetacoplan exposure, no cases of encapsulated N. meningitidis and consistently low rates of infections by other encapsulated bacteria were observed in patients with PNH, C3G, primary IC-MPGN, or other conditions. These findings may reflect effective risk mitigation strategies."

Complement-mediated Rare Disorders • Glomerulonephritis • Hematological Disorders • Immunology • Infectious Disease • Influenza • Lupus Nephritis • Meningococcal Infections • Nephrology • Paroxysmal Nocturnal Hemoglobinuria • Pneumococcal Infections • Pneumonia • Primary Immunodeficiency • Rare Diseases • Respiratory Diseases • Septic Shock

Severe systemic lupus erythematosus with thrombotic microangiopathy: rescue therapy with pegcetacoplan. (PubMed, Pediatr Nephrol) - "This report describes the case of a 12-year-old female patient with severe lupus nephritis and refractory thrombotic microangiopathy, who required dialysis for several months despite receiving intensive immunosuppressive treatment, including terminal complement blockade with eculizumab. After more than 8 months of haemodialysis, the patient could be weaned off dialysis. This case study highlights the therapeutic potential of pegcetacoplan in the treatment of thrombotic microangiopathy in systemic lupus erythematosus."

Journal • Chronic Kidney Disease • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Renal Disease • Systemic Lupus Erythematosus

Pegcetacoplan for 52 weeks results in sustained proteinuria reduction to remission (≤0.5 g/g) and normalisation (≤0.2 g/g): Phase 3 VALIANT trial (UKKW 2026) - P3 | "At week 26, 20 (31.8%) pegcetacoplan-to-pegcetacoplan patients achieved proteinuria remission (≤0.5 g/g); 11 (17.5%) achieved proteinuria normalisation (≤0.2 g/g) (Figure). Results were maintained at week 52, with 23 (36.5%) and 11 (17.5%) achieving proteinuria remission and proteinuria normalisation, respectively. Similar results were seen at week 52 in placebo-to-pegcetacoplan patients with 20 (32.8%) achieving remission and 7 (11.5%) achieving normalisation (following switch to pegcetacoplan from placebo at week 26)."

Clinical • P3 data • Complement-mediated Rare Disorders • Glomerulonephritis • Inflammation • Lupus Nephritis • Nephrology • Renal Disease

CASE REPORT: THE USE OF PEGCETACOPLAN PERI-HAEMATOPOIETIC STEM CELL TRANSPLANT FOR PAROXYSMAL NOCTURNAL HAEMOGLOBINURIA / APLASTIC ANAEMIA (EBMT 2026) - "She did not have an adult fully matched sibling donor so underwent immunosuppression with horse anti-thymocyte globulin (hATG), cyclosporin and eltrombopag...The conditioning regimen consisted of rATG, fludarabine, cyclophosphamide and one fraction of 2.00 Gy total body irradiation as per the protocol by DeZern et al (Blood 2023)... Use of eculizumab during the peri-transplant period has been described. We are not aware of any previously described cases of pegcetacoplan as a bridging therapy to allograft for patients with PNH and AA. The case demonstrates that this novel complement inhibitor can be used during the transplant conditioning and continued after stem cell infusion with successful engraftment and clearance of the PNH clone."

Case report • Clinical • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Cardiovascular • Chronic Graft versus Host Disease • Complement-mediated Rare Disorders • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Infectious Disease • Paroxysmal Nocturnal Hemoglobinuria • Pneumonia • Septic Shock • Thrombosis • Transplantation

Pegcetacoplan in C3 Glomerulopathy and Immune-Complex MPGN. (PubMed, N Engl J Med) - No abstract available

Journal • Complement-mediated Rare Disorders • Glomerulonephritis

Pegcetacoplan in C3 Glomerulopathy and Immune-Complex MPGN. Reply. (PubMed, N Engl J Med) - No abstract available

Journal • Complement-mediated Rare Disorders • Glomerulonephritis

Cold Agglutinin Disease (ICKSH 2026) - "The diagnosis of CAD is established by evidence of hemolysis (anemia, elevated LDH and bilirubin, decreased haptoglobin) together with a direct antiglobulin test (DAT) showing C3d positivity (±IgG) , supportive cold agglutinin testing (commonly a titer ≥1:64), and assessment of thermal amplitude...Second, complement -directed therapy has emerged as an important option; the C1s inhib itor sutimlimab demonstrated improvements in hemoglobin and hemolysis markers and meaningful reduction in fatigue in clinical trials. More recently, complement C3 inhibition (e.g., pegcetacoplan) has shown potential activity in both CAD and warm AIHA, expa nding the therapeutic landscape...Berentsen S, Randen U, Oksman M, Birgens H, Tvedt THA, Dalgaard J, Galteland E, Haukas E, Brudevold R, Sorbo JH, Naess IA, Malecka A, Tjonnfjord GE (2017) Bendamustine plus rituximab for chronic cold agglutinin disease: results of a Nordic prospective multicenter trial...Blood 145:397 -408...."

Autoimmune Hemolytic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Immunology • Infectious Disease • HP

Pediatric Complement 3 Glomerulonephritis (C3GN) Responsive to Mycophenolate mofetil (NKF-SCM 2026) - "Prednisone (2 mg/Kg/day) and Lisinopril were started at 1.5 months...As UPCR remains elevated (~4.09 mg/mg), empagliflozin was added...RESULTS/CASE DISCUSSION Despite the availability of Pegcetacoplan or Iptacopan, these are not currently approved for children under 12 years of age. Complement inhibitors including Eculizumab/ Ravuluzimab will carry potential life threatening risk of infections. CONCLUSION This case highlights MMF as a viable long-term management strategy in refractory C3GN in young patients intolerant or resistant to corticosteroids."

Clinical • Glomerulonephritis • Infectious Disease • Lupus Nephritis • Nephrology • Pediatrics • Renal Disease

Comparative Efficacy of Complement Inhibitors in Complement Inhibitor-Naïve PNH: A Systematic Review With Supportive Exploratory Network Meta-Analysis of Randomized Trials. (PubMed, EJHaem) - "A total of four randomized controlled trials evaluating four complement inhibitor agents (ravulizumab, crovalimab, eculizumab and pegcetacoplan) were included in this systematic review and network meta-analysis, involving 589 complement inhibitor-naïve adults with PNH. However, the evidence network is sparse (four trials) and the cross-trial differences limit reliable inference regarding relative efficacy between active agents. Comparative findings should be interpreted as hypothesis-generating."

Journal • Retrospective data • Review • Complement-mediated Rare Disorders • Fatigue • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria

ECONOMIC EVALUATION OF PEGCETACOPLAN FOR PNH FROM THE BRAZILIAN PUBLIC HEALTHCARE SYSTEM PERSPECTIVE (ISPOR 2026) - "Comparators included eculizumab, ravulizumab, and iptacopan. From the Brazilian Public Healthcare System perspective, pegcetacoplan represents a cost-saving therapeutic strategy for PNH, yielding lower long-term treatment costs than available C5 inhibitors and offering meaningful reduction in projected national expenditures. These findings support pegcetacoplan as an economically advantageous option for managing previously treated PNH patients."

HEOR • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Primary membranoproliferative glomerulonephritis: natural history, pathogenesis, and treatment. (PubMed, Front Nephrol) - "Two agents are now FDA approved for C3G, the oral Factor B inhibitor iptacopan and the subcutaneous C3-inhibitor pegcetacoplan, the latter also approved for IC-MPGN. If complement inhibition is unavailable, MMF/steroids may be considered. Following transplantation, protocol biopsies are needed to detect early recurrence with the intent of complement inhibition."

Journal • Review • Complement-mediated Rare Disorders • Glomerulonephritis • Immunology • Infectious Disease • Nephrology • Oncology • Transplantation

Pegcetacoplan for 52 weeks maintained proteinuria reduction in key patient populations: VALIANT subgroup analyses (NKF-SCM 2026) - P3 | "Safety was consistent with previous reports. *on behalf of the VALIANT investigators"

Clinical • Complement-mediated Rare Disorders • Glomerulonephritis • Lupus Nephritis • Nephrology • Renal Disease

Low risk for meningococcal infections with systemically administered pegcetacoplan, a complement C3 and C3b inhibitor (NKF-SCM 2026) - "These findings suggest effective risk mitigation strategies. Continued follow-up and surveillance for serious infections with encapsulated bacteria remain warranted."

Complement-mediated Rare Disorders • Glomerulonephritis • Hematological Disorders • Immunology • Infectious Disease • Influenza • Meningococcal Infections • Paroxysmal Nocturnal Hemoglobinuria • Pneumococcal Infections • Pneumonia • Primary Immunodeficiency • Rare Diseases • Respiratory Diseases

Complement 3 Glomerulopathy Revisited: Bridging Pathogenesis and Therapy in the New Era. (PubMed, Adv Kidney Dis Health) - "Encouraging results from recent clinical trials provide hope for improving the overall prognosis of this entity, yet critical knowledge gaps persist. This review explores the heterogeneity of complement 3 glomerulopathy and examines the emerging therapeutic landscape, highlighting advancements in the management of this complex condition."

Journal • Review • Complement-mediated Rare Disorders • Glomerulonephritis • Inflammation • Nephrology • Renal Disease • Transplantation