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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.

回复@今夕复河西:$键凯科技(SH688356)$键凯科技：AI制药赛道蓄势待发，技术壁垒与创新基因驱动未来增长一、AI+PEG：技术协同打开研发新范式键凯科技深耕PEG材料研发近20年，其技术壁垒覆盖从基础合成到临床应用的全链条。目前，公司已支持国内80%以上的PEG修饰药物上市，并为全球30余个临床项目提供核心原料。面对药物研发周期长、成本高的痛点，公司自2025年起加速布局AI技术，重点突破药物结构设计与分子筛选两大环节。通过构建计算生物学团队，键凯尝试将AI算法融入PEG修饰位点预测、分子稳定性模拟等场景，以提升研发效率并降低试错成本。例如，AI可快速筛选出与靶点高亲和力的PEG衍生物结构，缩短传统实验验证周期，助力创新药管线快速迭代。二、小核酸递送系统：AI赋能下一代技术制高点小核酸药物（如siRNA、mRNA）被视为未来十年最具颠覆性的治疗领域，而递送系统是其研发的核心壁垒。键凯科技凭借国内唯一的递送系统全产业链能力，已参与国内超2/3的mRNA疫苗项目，提供关键PEG脂质成分。AI技术的引入将进一步提升其竞争力：1.递送系统优化：通过机器学习预测核酸-脂质复合物的稳定性与靶向性，设计更高效的纳米载体；2.工艺降本增效：AI模拟生产流程中的关键参数，优化合成路径，降低规模化生产成本；3.适应症拓展：结合疾病靶点数据库，AI可加速开发针对肿瘤、罕见病等领域的定制化递送方案。这一布局使键凯不仅限于原料供应商，更有望向“递送系统技术授权+联合开发”模式升级，抢占千亿级市场先机。三、商业化潜力：从技术储备到业绩爆发尽管AI技术尚处投入期，键凯科技的短期业绩已呈现复苏态势。2025年上半年海外订单激增36.96%，支撑营收同比增长0.89%，而下半年1.49亿元待确认订单将推动全年收入突破3亿元。长期来看，AI驱动的研发效率提升有望释放管线价值：•PEG-伊立替康：全球首个BIC（同类最佳）潜力的小细胞肺癌药物，III期临床数据优异，预计商业化后生命周期收入超50亿元；•小核酸管线：覆盖肿瘤、免疫等领域的10余个专利药物，AI加速临床前研究进程；•医美与器械：PEG交联玻尿酸、长效水光针等产品进入临床，AI辅助配方优化或缩短上市周期。四、估值逻辑重构：低估值与高成长的错配机遇当前键凯科技市值约57亿元，显著低于其技术平台价值。对比同类AI制药企业，其核心优势在于：•稀缺性：国内唯一同时具备PEG材料量产能力与AI研发布局的企业；•国际化：海外收入占比超60%，受益于全球创新药产业链转移；•轻资产模式：以技术服务与材料销售为主，现金流稳定且风险可控。若AI技术在未来3-5年实现商业化落地，叠加小核酸药物等管线兑现，估值修复空间可达5—10倍。查看图片//@今夕复河西:回复@今夕复河西:$键凯科技(SH688356)$Q3业绩数据还不错，基本按之前的预期走，由于半年报已经披露了在手订单，有了明确的托底，因此也就不算有多超预期了。拆解下数据：最大的亮点是国内市场，Q3同比增长21%，环比增长95%，彻底摆脱高新自产的阴霾，最大的功臣无疑是器械销量的崛起，今年国内一口气新上市3个新产品，让人咋舌（要知道键凯自上市直到今年4月才支持1个产品），而且从项目储备看，器械的业绩比重预计还会继续提升，其中还有瑞瞳这样的彩蛋可以期待。Q3技术服务费532万，同比增长423.5%，环比增长141%，特宝佩金和益佩生终于开始有点像样的输出了，按这个趋势明年技术服务费上2000万难度不大，这都是纯利润。国外市场作为当前业绩大腿，也没掉链子，UCB的Zilbrysq还在快速放量期，波科稳中有升。今年Instylla公司的重磅产品EmbraceHES上市，其市场潜力不输SpaceOAR，业绩预计在明年开始释放，还有即将上市的SOBI的NASP、北极星的ADZODI，明后年的业绩都不用愁。再说说PEG伊立替康，从去年10月开始将临床三期费用资本化，去年花了2081万，今年三个季度分别花了845万、949万、1389万，今年Q3自从苏亚辉主持临床工作以来，临床进度明显加速，盲猜小肺三期入组人数120+，虽然老赵总被诟病慢，但只要拿出手的，都还是靠谱的。