Mycophenolate Mofetil

Systemic sclerosis and myositis interstitial lung disease subtypes flagged as most difficult to manage; specialists cite lack of effective treatments and diagnostic clarity EXTON, PA, June 17, 2025 (GLOBE NEWSWIRE) -- According to Spherix Global Insights’ newly published Special Topix™: Interstitial Lung Disease in Rheumatology (US) report, both rheumatologists and pulmonologists report significant challenges in diagnosing and managing connective tissue disease-associated interstitial lung disease (CTD-ILD), with systemic sclerosis (SSc) and myositis (IIM) cited as the most difficult subtypes to treat. The research, based on responses from 138 US specialists (n=68 rheumatologists and 70 pulmonologists), highlights persistent unmet needs, diagnostic ambiguity, and a call for more robust and collaborative management strategies. The findings reflect that undiagnosed and misdiagnosed patients make up a substantial portion of the CTD-ILD population, with nearly one-third of rheumatologists and over half of pulmonologists naming differential diagnosis as a key challenge. Specialists underscore a lack of specific biomarkers, logistical biopsy issues, and overlapping symptoms as primary obstacles. “The most definitive test (biopsy) is invasive. It is difficult to find the clinicians who are willing to do it in a timely manner, and to convince the patient it needs to be done.” – Rheumatologist “Ensuring the correct diagnosis to avoid misdiagnosing the type of ILD or source of ILD. I usually employ the assistance of a rheumatologist.” – Pulmonologist “Often it would be difficult to get patients biopsied for logistical reasons, and often the pathology report would not be precise enough.” – Pulmonologist Even once diagnosed, CTD-ILD patients face a treatment landscape with few options to slow or halt disease progression. When asked about the most difficult aspects of management, specialists consistently pointed to a lack of effective and targeted therapies, with SSc-ILD and myositis-ILD receiving the highest unmet need scores. For SSc-ILD specifically, not only is it associated with the most aggressive progression and worst long-term outcomes, but it also presents a uniquely complex therapeutic challenge. The clinical burden is especially pronounced in patients with diffuse cutaneous systemic sclerosis (dcSSc), where the use of corticosteroids is often limited due to the heightened risk of scleroderma renal crisis. This leaves physicians with a narrow band of anti-fibrotic and immunosuppressive options, many of which are perceived as only modestly effective in controlling ILD progression, let alone reversing it. Even therapies with regulatory approval for SSc-ILD, such as Boehringer Ingelheim’s OFEV (nintedanib), garner only measured enthusiasm from treating physicians. While rheumatologists acknowledge nintedanib’s potential to slow functional decline, they note it does little to address the underlying autoimmune pathology. Similarly, mycophenolate mofetil and rituximab remain staples of clinical practice, yet their real-world performance is viewed as inconsistent and difficult to monitor. Across the board, satisfaction ratings for currently utilized agents fall short of expectations, reinforcing a treatment landscape still dominated by trial-and-error approaches. This disconnect between disease complexity and treatment efficacy is fueling demand for dual-acting agents, therapies that can address both systemic autoimmunity and pulmonary fibrosis. Nowhere is this demand more visible than in systemic lupus erythematosus-associated ILD (SLE-ILD), where rheumatologists express growing interest in GSK’s Benlysta (belimumab). Though not currently approved for ILD, belimumab’s immunologic mechanism and expanded label for lupus nephritis have raised hopes that similar benefit may extend to pulmonary manifestations. Across interviews and survey feedback, physicians articulate a desire for treatments that reduce systemic activity while simultaneously preventing fibrotic progression—without the need for an antifibrotic add-on with limited synergy. Looking ahead, specialists are cautiously optimistic about the pipeline. While interest in agents with antifibrotic and immunomodulatory properties is rising, physicians emphasize that what’s most needed is not just another drug, but a paradigm shift—a treatment model that reflects the multifaceted nature of CTD-ILD. As one physician summarized, the goal is to "treat the patient, not just the lungs." In addition to tracking physician sentiment and practice patterns, the study includes target product profile evaluations for several pipeline therapies: Benlysta (GSK), BMS-986278 (Bristol Myers Squibb), Nerandomilast (Boehringer Ingelheim), and Treprostinil (United Therapeutics). These profiles offer manufacturers timely insights into clinical expectations, adoption potential, and differentiation opportunities in an increasingly complex ILD treatment landscape. Special Topix ™ is an independent service that includes access to a report or series of reports based on current events or topics of interest in specialty markets covered by Spherix. About Spherix Global Insights Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle. The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community. As a trusted advisor and industry thought leader, Spherix’s unparalleled market insights and advisory services empower clients to make better decisions and unlock opportunities for growth. To learn more about Spherix Global Insights, visit spherixglobalinsights.com or connect through LinkedIn . For more details on Spherix’s primary market research reports and interactive dashboard offerings, visit or register here: https://clientportal.spherixglobalinsights.com NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight’s analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release. Andy Stankus, Rheumatology Franchise Head Spherix Global Insights 4848794284 andy.stankus@spherixglobalinsights.com Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

News2025年6月12-15日，第30届欧洲血液学会（EHA）年会在意大利米兰隆重召开。EHA作为全球血液学工作者的盛会，汇集各个国家和地区知名血液病学专家，分享和探讨全球最前沿的研究进展和突破性临床研究及宝贵的临床实践经验。北京高博博仁医院李智慧教授主导的一项关于重组人源化抗CD25单克隆抗体替代MTX用于CAR-T桥接移植后GVHD预防研究成功入选口头报告。研究背景免疫治疗时代，嵌合抗原受体T细胞疗法（CAR-T）桥接移植已经成为一种新的治疗模式，为众多恶性血液病患者争取了长期生存的可能，但移植并发症移植物抗宿主病（GVHD）的防治仍是困扰临床的难题。甲氨蝶呤（MTX）是预防异基因造血干细胞移植（allo-HSCT）后aGVHD的基石药物，但MTX可引起多种不良反应，包括严重的黏膜炎、骨髓抑制、肝毒性等。接受CAR-T疗法后桥接HSCT的患者，由于细胞因子风暴、免疫缺陷和长期中性粒细胞减少，GVHD和口腔黏膜炎的发生率可能更高。本研究探索了重组人源化抗CD25单克隆抗体替代MTX在aGVHD预防中的可能性。研究方法研究回顾性分析了2020年8月至2024年7月在北京高博博仁医院接受CAR-T桥接造血干细胞移植治疗的217例患者，其中140例儿童、77例成人。诊断疾病包括急性B细胞或T细胞淋巴细胞白血病/淋巴瘤等。所有恶性血液肿瘤患者在CAR-T治疗后达到完全缓解。33.6%患者进行了第二次移植。抗胸腺细胞球蛋白（ATG）用于半相合和无关供者移植。对于GVHD预防方案，在（甲氨蝶呤）MTX组中，166例患者接受环孢素A（CsA）、霉酚酸酯（MMF）和MTX（第+1天15 mg/m²，第+3、+6、+11天，10 mg/m²），而重组人源化抗CD25单克隆抗体组中，51例患者接受CsA、MMF和人源化抗CD25单克隆抗体（第+4和+8天，1 mg/kg）。两组的基线特征相似。该研究中位随访时间为24.3个月。本研究主要终点是第100天aGVHD的累积发生率，次要终点包括总生存率（OS）、cGVHD发生率、EB病毒（EBV）、巨细胞病毒（CMV）、细菌及真菌感染发生率、口腔黏膜炎发生率等。研究结果重组抗CD25人源化单克隆抗体组100天时II-IV级aGVHD的累积发生率显著低于MTX组（21.2% vs. 41.0%，P=0.02），且慢性GVHD累积发生率有降低趋势（P=0.069）。重组抗CD25人源化单克隆抗体组的总口腔黏膜炎（OM；40.29% vs. 63.41%，P=0.003）和2-4级OM发生率显著降低（7.46% vs. 32.05%，P<0.001），OM中位持续时间更短（5天vs. 11天）。两组在巨细胞病毒血症、EB病毒血症、复发、移植相关死亡率（TRM）和总生存（OS）方面无显著差异（复发：P=0.41；TRM：P=0.85；OS：P=0.2）。结论该研究表明，对于CAR-T桥接移植后GVHD的预防，重组人源化抗CD25单克隆抗体替代MTX，显著降低了aGVHD和OM的发生率，同时在病毒感染、复发、移植相关死亡率（TRM）和生存率方面保持了相当的结果。该方案代表了一种有前景的GVHD预防策略，与传统的含MTX方案相比，有助于改善患者的生活质量，也为临床治疗带来了更大的可持续性，为CAR-T桥接移植后的并发症管理提供了个性化方案。参考文献：1. Zhihui Li, et al. Anti-CD25 antibody as an alternative to methotrexate for graft-versus-host disease prophylaxis resulted in better outcomes in patient of chimeric antigen receptor-T cell therapy bridging to transplant. 2025 EHA. abstract S262.关于三生国健三生国健是中国首批专注于抗体药物的创新型生物医药高新技术企业之一。公司拥有研、产、销一体化成熟平台，专注于单抗、双抗、多抗及多功能重组蛋白等新技术研究，拥有丰富的大规模抗体产业化和质量控制经验。公司是“免疫与炎症全国重点实验室”依托单位、拥有抗体药物国家工程研究中心、国家企业技术中心、上海市抗体技术创新中心、上海抗体工程技术研究中心等国家级和上海市级高新技术平台，承担过国家重大新药创制、“863”计划、国家发改委及上海市重大项目及各部委课题百余项，多次获得上海市级和浦东新区科学技术奖。目前，公司拥有22个处于不同开发阶段的在研创新药物项目，大部分为治疗用生物制品1类药品，部分在研药物为中美双报。About Sunshine Guojian警示说明及前瞻性陈述 本新闻稿包含前瞻性陈述，例如涉及业务和产品前景,或公司的意图、计划、认知、预期及策略。该等前瞻性陈述是根据本公司现有的资料，并按本新闻稿发布时的展望陈述。该等前瞻性陈述基于若干预测、假设及前提，其中一些是主观性的或不受我们控制。该等前瞻性陈述可能被证明是不正确的，或将来可能无法实现。就任何新产品或产品的新适应症, 我们无法确保其将能成功开发或最终上市销售。该等前瞻性陈述受各种风险及不明朗因素影响。我们的其他公开披露文件可能提供该等风险及不明朗因素的更多信息。所涉及之科学信息可能只是初步的和研究性的。本公司股东及潜在投资者在买卖本公司股份时，请务必谨慎行事。