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New one-year data reinforces the robust and sustained efficacy of Aspaveli/Empaveli (pegcetacoplan) in patients with C3G and primary IC-MPGN. “Patients on Aspaveli/Empaveli maintained proteinuria reduction, irrespective of taking immunosuppressants and independent of their baseline proteinuria levels. One third of patients achieved complete proteinuria remission compared to 3% for placebo at week 26. These benefits were maintained through one year, and patients who switched from placebo experienced similar improvements. This demonstrates the potential of Aspaveli/Empaveli to transform care for people living with these rare diseases, who have a high risk of kidney failure,” said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. Sobi will also present data showing NASP maintained stable renal function throughout the 6-month study period, with a higher proportion of patients experiencing improvement in CKD stage compared to placebo among those with uncontrolled gout and stage 3 chronic kidney disease. Additionally, NASP treatment led to a reduction in gout-related clinical manifestations and was generally well tolerated. Key data to be presented at ASN Kidney Week 2025  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  Presenting author:Carla M. Nester, MD, MSA, FASN Pegcetacoplan for adolescents and adults with C3 glomerulopathy or primary immune-complex membranoproliferative glomerulonephritis: enrolment status of the VALE extension Presentation Author: Eliyah Khankin, MD Poster Presentation Session title: Informational Posters- 1Date: 6 November 2025 Time: 10:00 AM to 12:00 PM (CT)Location: Exhibit Hall, Convention Center  Pegcetacoplan for 52 weeks maintained proteinuria reduction regardless of immunosuppressant use or nephrotic range proteinuria at baseline: VALIANT subgroup analysis  Presenting Author: Carla M. Nester MD; MSA, FASN  Poster Presentation Session title:Glomerular Clinical Trials: From Data to ImpactDate: 7 November 2025Time: 10:00 AM to 12:00 PM (CT)Location: Exhibit Hall, Convention Center  Pegcetacoplan for 52 weeks maintains proteinuria reduction and eGFR stabilisation for pediatric patients: Phase 3 VALIANT subgroup analysisPresenting author: Larry A. Greenbaum, MD Poster Presentation Session title: Pediatric Nephrology: CKD, ESKD and Glomerular DiseasesDate: 7 November 2025Time: 10:00 AM to 12:00 PM (CT)Location: Exhibit Hall, Convention Center  Human factors validation of an on-body autoinjector for pegcetacoplan demonstrates ease of use by adolescents with C3 glomerulopathy/primary immune-complex membranoproliferative glomerulonephritis  Presenting author: Eliya Khankin, MD Poster Presentation Session title: Glomerular Outcomes: From Proteinuria to PrognosisDate: 7 November 2025Time: 10:00 AM to 12:00 PM (CT)Location: Exhibit Hall, Convention Center  Clinical efficacy of Pegcetacoplan versus Iptacopan in patients with C3 Glomerulopathy: Indirect Treatment Comparisons   Presenting author: Bradley P Dixon, MD Poster Presentation Session title: Glomerular Management: Real-World Lessons and Emerging TherapiesDate: 7 November 2025Time: 10:00 AM to 12:00 PM (CT)Location: Exhibit Hall, Convention Center  A thematic analysis of patient perspectives on care and unmet needs in C3G and primary IC-MPGN  Presenting author: Sayna Nourouzi, MD Poster Presentation Session title: Glomerular Research: Design, Registries, Surveys and EpidemiologyDate: 8 November 2025 Time: 10:00 AM to 12:00 PM (CT)Location: Exhibit Hall, Convention Center Management of Uncontrolled Gout Among Nephrology Professionals: Findings from a Medical Chart AuditPresenting author: Bhavisha Desai, PharmD, Bhavisha Desai, PharmD, Sobi Poster Presentation  Session title: CKD: Epidemiology, Risk Factors, and Other Conditions [PO2301-1] Date: 6 November 2025Time: 10:00 AM to 12:00 PM (CT)Location: Exhibit Hall, Convention Center Effect of Nanoencapsulated Sirolimus plus Pegadricase (NASP) on Kidney Outcomes: Results from the Phase 3 DISSOLVE Studies   Presenting author: Minesh Rajpal, MD Southwest Kidney Institute, Phoenix, AZ, USA Poster Presentation  Session title: CKD: Therapies, Innovations, and Insights [PO2302-1] Date: 6 November 2025 Time: 10:00 AM to 12:00 PM (CT)Location: Exhibit Hall, Convention Center Nanoencapsulated Sirolimus Plus Pegadricase (NASP) Reduces Gout Clinical Manifestations in Patients with Uncontrolled Gout (UG) and Stage 3 CKD Presenting authors: Nissreen Elfadawy, MD, University Hospitals Geauga Medical Center, Chardon, OH, and Case Western Reserve University, Cleveland, OH, USA  Poster Presentation Session title: CKD: Therapies, Innovations, and Insights [PO2302-1]Date: 6 November 2025 Time: 10:00 AM to 12:00 PM (CT)Location: Exhibit Hall, Convention Center About Aspaveli/Empaveli (pegcetacoplan) Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 and C3b therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. It is the first treatment approved in the United States for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older, to reduce proteinuria. Aspaveli/Empaveli is also approved for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) in the United States, European Union, and other countries globally, and is under investigation for other rare diseases.  About the VALIANT Study The VALIANT Phase 3 study (NCT05067127) was a randomised, placebo-controlled, double-blinded, multi-center study that evaluated efficacy and safety of Aspaveli/Empaveli (pegcetacoplan) in 124 patients who were 12 years of age and older with C3G or primary IC-MPGN. It is the largest single trial conducted in these populations and the only study to include paediatric and adult patients, with native or post-transplant kidneys. Study participants were randomised to receive Aspaveli/Empaveli or placebo twice weekly for 26 weeks. Following this 26-week randomised controlled period, patients were able to proceed to a 26-week open-label phase in which all patients received Aspaveli/Empaveli. The primary endpoint of the study was the log transformed ratio of urine protein-to-creatinine ratio (UPCR) at Week 26 compared to baseline.  About C3 Glomerulopathy (C3G) and Primary Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN) C3G and primary IC-MPGN are rare and debilitating kidney diseases that can lead to kidney failure. Excessive C3 deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. Approximately 50% of people living with C3G and primary IC-MPGN suffer from kidney failure within five to 10 years of diagnosis, requiring a burdensome kidney transplant or lifelong dialysis therapy.1-3 Additionally, approximately 90% of patients who previously received a kidney transplant will experience disease recurrence.4 The diseases are estimated to affect 5,000 people in the United States and up to 8,000 in Europe.5   About the Sobi and Apellis Collaboration Apellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan, and Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy.  About NASP NASP is a novel investigational medicine designed to reduce serum uric acid (sUA) levels in people living with uncontrolled gout, potentially reducing harmful tissue urate deposits which when left untreated can lead to debilitating gout flares and joint deformity. NASP is administered every 4-weeks as a sequential, two-component, infusion therapy consisting of tolerogenic nanoencapsulated sirolimus (NAS) which mitigates the formation of anti-drug antibodies (ADAs) and a uricase, pegadricase (P), which reduces serum uric acid. ADAs develop due to unwanted immune responses to biologic medicines, reducing their efficacy and tolerability, which remains an issue across multiple therapeutic modalities and disease states including uncontrolled gout. About Uncontrolled Gout Gout is the most common form of inflammatory arthritis with more than 8.3 million people in the United States having been diagnosed. Gout is caused by high levels of uric acid in the body that accumulate around the joints and other tissues and can result in flares that cause intense pain. Approximately 200,000 people in the United States suffer from uncontrolled gout, with serum uric acid (sUA) levels above 6 mg/dL despite treatment with oral urate lowering therapies. This leads to debilitating flares andtophi. Elevated sUA levels have also been associated with diseases of the heart, vascular system, metabolism, kidney and joints. Sobi®Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. ContactsFor details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. References 1. Smith RJH, et al. Nat Rev Nephrol. 2019;15(3):129-143. 2. Servais A, et al. Kidney Int. 2012;82(4):454-464. 3. Zand L, et al. J Am Soc Nephrol. 2014;25(5):1110-1117. 4. Tarragón, B, et al. C3 Glomerulopathy Recurs Early after Kidney Transplantation in Serial Biopsies Performed within the First 2 Years after Transplantation. Clinical Journal of the American Society of Nephrology. August 2024; 19(8)1005-1015. doi: 10.2215/CJN.0000000000000474. 5. Data on file using literature consensus. We are a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Our therapies are concentrated within the areas of Haematology, Immunology and Specialty Care. We contribute to societies by improving access to treatment of rare diseases. Find out more about our business and financial performance. Here we present our most recent press releases, news articles, and images. Every day, we work actively to find better ways to understand and meet patient needs.

STOCKHOLM, Oct. 24, 2025 /PRNewswire/ -- Sobi® (STO: SOBI), a global biopharmaceutical company dedicated to delivering innovative treatments for patients with rare disease, will be presenting new data across its immunology portfolio at the annual American College of Rheumatology (ACR) Convergence 2025 meeting in Chicago, October 24-29. Sobi is presenting a total of 15 scientific abstracts, with six oral presentations featuring new clinical data from completed and ongoing studies of NASP, pacritinib, and emapalumab-lzsg. "The data Sobi is presenting highlight the distinct clinical benefit of emapalumab-lzsg in MAS in Still's disease, the ongoing study of pacritinib in VEXAS Syndrome, and the potential for NASP to redefine the treatment of uncontrolled gout. The results underscore the strength of our portfolio in advancing care for complex and rare inflammatory diseases where progress is urgently needed," said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. Summary of full Sobi data to be presented at ACR 2025: About NASP NASP is a novel investigational medicine designed to reduce serum uric acid (sUA) levels in people living with uncontrolled gout, potentially reducing harmful tissue urate deposits which when left untreated can lead to debilitating gout flares and joint deformity. NASP is administered every 4-weeks as a sequential, two-component, infusion therapy consisting of tolerogenic nanoencapsulated sirolimus (NAS) which mitigates the formation of anti-drug antibodies (ADAs) and a uricase, pegadricase (P), which reduces serum uric acid. ADAs develop due to unwanted immune responses to biologic medicines, reducing their efficacy and tolerability, which remains an issue across multiple therapeutic modalities and disease states including uncontrolled gout. About Uncontrolled Gout Gout is the most common form of inflammatory arthritis with more than 8.3 million people in the United States having been diagnosed. Gout is caused by high levels of uric acid in the body that accumulate around the joints and other tissues and can result in flares that cause intense pain. Approximately 200,000 people in the United States suffer from uncontrolled gout, with serum uric acid (sUA) levels above 6 mg/dL despite treatment with oral urate lowering therapies. This leads to debilitating flares and tophi. Elevated sUA levels have also been associated with diseases of the heart, vascular system, metabolism, kidney and joints. About Gamifant (emapalumab-lzsg) Gamifant is the only approved anti-interferon gamma (IFNy) monoclonal antibody. Gamifant works by binding to and neutralizing interferon gamma (IFNy). When interferon gamma (IFNy) is secreted in an uncontrolled manner, hyperinflammation occurs within the body. Gamifant is indicated for administration through intravenous infusion over one hour. Gamifant is approved in the US for the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Gamifant is also approved in the US for the treatment of adult and pediatric (newborn and older) patients with hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in known or suspected Still's disease with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS. About macrophage activation syndrome (MAS) Macrophage activation syndrome (MAS) is a severe complication of rheumatic diseases, most frequently in Still's disease including systemic juvenile idiopathic arthritis (sJIA) – a rare systemic disorder of auto-inflammatory nature with common clinical manifestations such as daily spiking fever, typical transient cutaneous rash, arthritis, lymphadenopathy, hepatosplenomegaly and serositis. MAS is characterized by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities and hyperferritinaemia, possibly progressing to multiple organ failure and death. MAS is classified as a secondary form of haemophagocytic lymphohistiocytosis (HLH). About Vonjo (pacritinib) Vonjo is a kinase inhibitor that is indicated in the US for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). About VEXAS VEXAS syndrome is a disease that causes inflammatory and hematologic (blood) manifestations. The syndrome is caused by mutations in the UBA1 gene of blood cells and acquired later in life. The condition is not genetically inherited. About Sobi Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi's share (STO: SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED