Phase 2 Study of 9-ING-41, a Glycogen Synthase Kinase 3 Beta (GSK 3β) Inhibitor, Plus Carboplatin in Patients With Advanced, Metastatic Salivary Gland Carcinoma

9-ING-41 is a small molecule potent selective GSK-3β inhibitor with antitumor activity. This study investigates 9-ING-41 in combination with carboplatin chemotherapy in patients with incurable, recurrent or metastatic salivary gland carcinomas (SGC). Patients with advanced SGC (including all histologic subtypes and adenoid cystic carcinoma [ACC]) will receive 9-ING-41 intravenously (IV) along with carboplatin IV at standard dosing together on Day 1, and 9-ING-41 alone on Day 4 of a 21-day cycle. Participants will be enrolled to two histologic cohorts: Cohort 1 will be comprised of those with ACC, and Cohort 2 will include patients with non-ACC SGC (or all other salivary gland cancer histologies). Treatment will continue until progression of disease, death, or discontinuation of therapy for any reason.

开始日期2030-12-18

申办/合作机构

Actuate Therapeutics, Inc.

NCT05056012

/ Not yet recruitingN/AIIT

Penn Sjögren's International Clinical Collaborative Alliance (SICCA) Follow-up Study

This study will involve the collection of follow-up data for patients who previously participated in the Sjogren's International Clinical Collaborative Alliance (SICCA) study at the University of Pennsylvania. Clinical data and specimens will be collected from subjects with objective evidence of dry eye who were or were not diagnosed with Sjogren's syndrome at the time of their initial participation in the SICCA study. Specimens will be collected from participants which will include tears, saliva, whole blood, serum, DNA and possible labial minor salivary gland biopsies when indicated. All individuals will participate in a standard evaluation protocol including an oral, ocular and physical examination, objective tests for dry eyes and dry mouth and, whenever necessary, a labial minor salivary gland biopsy. The biopsy requirement is waived for those who have already had positive lip biopsies in the past.

开始日期2026-01-01

申办/合作机构

University of Pennsylvania

NCT06297213

/ Not yet recruiting临床1期

A Single and Repeat Dosing Study of the Safety, Drug Exposure and Clinical Activity of R-2487 in Patients With Sjogren's Syndrome (SS)

The goal of this study is to determine the safety and tolerability of orally taken probiotic (R-2487) in patients with Sjogren's Syndrome.
Patients will take an oral dosage of probiotic (R-2487) and physicians will assess and measure their Sjogren's Syndrome. Blood and fecal evaluations of inflammation and assessment of probiotic (R-2487) on fecal level will also be measured.

开始日期2025-06-01

申办/合作机构

Rise Therapeutics LLC

100 项与涎液疾病相关的临床结果

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100 项与涎液疾病相关的转化医学

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0 项与涎液疾病相关的专利（医药）

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64,778

项与涎液疾病相关的文献（医药）

2025-12-31·Pulmonology

Early detection of interstitial lung disease in rheumatic diseases: A joint statement from the Portuguese Pulmonology Society, the Portuguese Rheumatology Society, and the Portuguese Radiology and Nuclear Medicine Society

Review

作者: Pereira, J.M. ; Freitas, S. ; Fernandes, M.O. ; Branco, J.C. ; Duarte, A.C. ; Marques, I.D. ; Cordeiro, A. ; Morais, A. ; Khmelinskii, N. ; Bernardes, M. ; Pinto, P. ; Borba, A. ; Pinto-Basto, R. ; Alfaro, T. ; Ruano, C. ; Calha, J. ; Campainha, S. ; Salvador, M.J.

INTRODUCTIONInterstitial lung disease (ILD) contributes significantly to morbidity and mortality in connective tissue disease (CTD). Early detection and accurate diagnosis are essential for informing treatment decisions and prognosis in this setting. Clear guidance on CTD-ILD screening, however, is lacking.OBJECTIVETo establish recommendations for CTD-ILD screening based on the current evidence.METHODFollowing an extensive literature research and evaluation of articles selected for their recency and relevance to the characterization, screening, and management of CTD-ILD, an expert panel formed by six pulmonologists from the Portuguese Society of Pulmonology, six rheumatologists from the Portuguese Society of Rheumatology, and six radiologists from the Portuguese Society of Radiology and Nuclear Medicine participated in a multidisciplinary discussion to produce a joint statement on screening recommendations for ILD in CTD.RESULTSThe expert panel achieved consensus on when and how to screen for ILD in patients with systemic sclerosis, rheumatoid arthritis, mixed connective tissue disease, Sjögren syndrome, idiopathic inflammatory myopathies and systemic lupus erythematous.CONCLUSIONSDespite the lack of data on screening for CTD-ILD, an expert panel of pulmonologists, rheumatologists and radiologists agreed on a series of screening recommendations to support decision-making and enable early diagnosis of ILD to ultimately improve outcomes and prognosis in patients with CTD.

2025-12-31·Autoimmunity

Anti-perilipin-1 autoantibodies in autoimmune Addison’s disease and related endocrine disorders

Article

作者: Anderson, Mark S. ; Oftedal, Bergithe E. ; Sulen, Andre ; Appel, Silke ; Wolff, Anette S. B. ; Rahman, Muhammad O. ; Breivik, Lars ; Husebye, Eystein S.

Immune-mediated lipodystrophy syndromes are rare autoimmune disorders characterized by complete or partial destruction of adipocytes in the body. Recently, autoantibodies against perilipin-1 (PLIN1-autoAbs) have been linked to lipodystrophy. Since various perilipins are expressed in the adrenal cortex and ovaries, we asked whether PLIN1-autoAbs were present in patients with adrenal dysfunction and other autoimmune endocrinopathies. Using a sensitive radiobinding immune assay we analyzed anti-PLIN1-autoAbs in 521 patients with endocrinopathies including Sjögren's syndrome. We identified 22 (4.2%) PLIN1-autoAbs positive patients, of whom 15% had autoimmune polyendocrine syndrome type 1 (4/27), 4% autoimmune Addison's disease and/or autoimmune polyendocrine syndrome type 2 (11/274), 8% type 1 diabetes patients (4/53), and 2% Sjögren's syndrome patients (1/50). However, none of them had known lipodystrophy. In conclusion, PLIN1-autoAbs are found in subgroups of autoimmune endocrinopathies and indicate autoimmunity against adipose tissue, but their pathogenic role if any, remains to be defined. Investigating their role in disease progression and their potential as therapeutic targets could pave the way for novel interventions in autoimmune endocrine diseases.

2025-12-31·Stress

Chronic stress resulting from stressful life events and its role in the onset of primary Sjögren’s syndrome: a comparative analysis using the modified Holmes-Rahe stress scale

Article

作者: Kościńska-Shukla, Iga ; Viti, Alessandro ; Jaskólska, Marta ; Jaskólski, Dawid ; Ziętkiewicz, Marcin ; Chmielewski, Michał

Recent years brought considerable attention to the connection between chronic stress and the development of autoimmune diseases. However, little is still known about the impact of prolonged stress reactions on the onset and course of primary Sjögren Syndrome (pSS). This study aimed to seek for associations between chronic stress, resulting from stressful life events, and pSS. In the study, 50 patients with diagnosed pSS, as well as 50 control patients with osteoarthritis underwent an assessment. Modified Holmes-Rahe (H-R) stress scale was used in order to evaluate the impact of stressful events within 12 months prior to the diagnosis. Patients with pSS had a significantly higher total score on H-R stress scale within one-year preceding the disease diagnosis (152 ± 66.3 vs 50 ± 54.6; p __-mml:math xmlns:mml="http://www.w3.org/1998/Math/MathML"-__< 0.001). Additionally, the pSS patients more commonly than the controls reported a subjectively perceived correlation between stressful events and the occurrence of disease symptoms (50% vs 12%; p __-mml:math xmlns:mml="http://www.w3.org/1998/Math/MathML"-__< 0.001). Moreover, the H-R score at the time of the assessment correlated with the disease activity. The results support the view that pSS belongs to the group of diseases which pathogenesis is closely related to stressful life events. The novelty of this work lies in focus on both the correlation of stress on the onset of autoimmune disease as well as the activity of previously diagnosed disorder. Our data contributes to finding evidence-based medicine (EBM) arguments to what has until recently been merely a thematic observation-the harmfulness of negative stress on individual's health status.

1,815

项与涎液疾病相关的新闻（医药）

2025-05-04

·药明康德

疾病控制率超90%的抗癌多肽偶联药物；癌症复发率降低4倍的放射性疗法…… | 一周盘点

本期看点1. 多肽偶联药物AVA6000的1期临床试验结果积极，在唾液腺癌患者中，≥250 mg/m²剂量水平的AVA6000治疗的疾病控制率达到91%。2. 放射性疗法Radspherin的1/2期临床试验18个月随访结果公布，在铂敏感复发性卵巢癌合并腹膜癌转移患者中，手术后接受Radspherin治疗的患者18个月后复发率为10%。而在类似人群中，接受最佳标准治疗的患者在同一随访时点预计复发率约为40%。AVA6000：公布1期临床试验结果Avacta Therapeutics公司日前公布了其主打在研多肽偶联药物AVA6000的1期临床试验积极数据。AVA6000是一种在肿瘤微环境中特异性激活的多柔比星（doxorubicin）前药，旨在减少传统化疗的全身性副作用。在1a期剂量递增研究中，无论是每三周一次（Q3W）还是每两周一次（Q2W）的给药方案，AVA6000均显示出良好的耐受性。即使剂量升高至每三周385 mg/m²，也未达到最大耐受剂量（MTD）。在唾液腺癌患者（n=11）中，接受≥250 mg/m²剂量水平的AVA6000治疗后，多位患者获得确认缓解，疾病控制率达到91%。中位无进展生存期（PFS）尚未达到，当前中位随访时间已超过25周。AVA6000是Avacta研发管线中的首个多肽偶联药物，由多柔比星与Avacta专有的pre|CISION多肽偶联而成，该多肽可在肿瘤微环境中由成纤维细胞激活蛋白-α（FAP）特异性切割。FAP在大多数实体瘤中，与健康组织相比表达水平显著升高。Avacta的多肽偶联药物利用这一特征在肿瘤微环境中特异性释放活性药物，从而降低系统暴露和毒性，为患者提供更好的治疗效果。CHS-114：公布1期临床试验结果Coherus BioSciences公布了其正在进行的1期临床试验的更新数据，该试验旨在评估选择性抗CCR8抗体CHS-114作为单药，以及与toripalimab联用，治疗复发/转移性头颈部鳞状细胞癌（HNSCC）患者的活性，以优化给药方案。数据显示，一例经过多线治疗且对PD-1抑制剂耐药的患者获得确认部分缓解，同时CCR8阳性调节性T细胞（Treg）减少超过50%、CD8阳性效应T细胞数量增加，与抗肿瘤活性一致，证明了药物的作用机制。这些数据支持继续评估CHS-114与包括toripalimab在内的其他疗法联用，并支持推进该研究第3部分的患者招募与开展，以进一步评估CHS-114与toripalimab在HNSCC中的疗效。CHS-114是一种去岩藻糖化的CCR8单克隆抗体，能优先杀伤肿瘤微环境中的CCR8阳性Treg，同时保留正常组织中的CD8阳性效应T细胞和Treg。Radspherin：公布1期临床试验18个月随访结果Oncoinvent日前公布了其1期临床试验RAD-18-001的18个月随访结果。该试验在铂敏感复发性卵巢癌合并腹膜癌转移患者中评估放射性疗法Radspherin的效果，该试验于2023年底完成患者招募，目前患者正接受长期随访。在本试验中，10例患者接受了单次腹腔内Radspherin给药。Oncoinvent在2024年11月公布的12个月数据中，10例患者中仅1例出现腹膜复发。18个月随访时，未见新增复发病例，即迄今为止仅10%的患者出现复发。而在类似人群中，接受最佳标准治疗的患者在同一随访时点预计约有40%的复发率。Radspherin是一种在研放射性药物，旨在局部治疗已扩散至体腔的癌症。它由数十亿个含放射性同位素镭-224的碳酸钙微粒组成。其作用机制是镭-224衰变发射的α粒子，这是一种高效能的电离辐射形式。Radspherin用于治疗卵巢癌和结直肠癌引起的腹膜癌转移，在外科切除所有可见肿瘤后，通过腹腔内给药。DT-168：公布1期临床试验积极数据Design Therapeutics公司日前公布了在研药物DT-168在健康志愿者中开展的1期单次及多次递增剂量（SAD/MAD）试验的积极数据。DT-168是一种新型GeneTAC小分子药物，制剂为滴眼液，旨在选择性靶向并降低导致角膜内皮细胞功能障碍的突变TCF4基因的表达，从而治疗Fuchs角膜内皮营养不良（FECD）。这项试验旨在评估DT-168滴眼液的安全性、耐受性和系统药代动力学（PK）。试验结果显示，DT-168滴眼液在所有受试者中均耐受良好。未观察到严重不良事件，也无眼部相关不良事件（AEs），亦无因不良事件导致的停药。所有观察到的不良事件均被研究者判定为与DT-168无关。PK分析结果表明，在所有时间点和所有剂量组中，受试者的系统性暴露均低于定量检测的下限。基于上述发现，Design公司计划开展DT-168的2期生物标志物临床试验，评估其在FECD患者中的安全性、耐受性及角膜内皮生物标志物表现。参考资料：[1] Bayer Initiates Phase I Study Targeting GPC3 With Actinium-225 Radiopharmaceutical in Patients With Advanced Hepatocellular Carcinoma. Retrieved April 29, 2025, from https://www.businesswire.com/news/home/20250429877323/en/Bayer-Initiates-Phase-I-Study-Targeting-GPC3-With-Actinium-225-Radiopharmaceutical-in-Patients-With-Advanced-Hepatocellular-Carcinoma[2] Coherus Presents Promising Early Clinical Data from Phase 1 Dose Expansion Study of CHS-114 in Patients with Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma at AACR 2025. Retrieved April 29, 2025, from https://www.globenewswire.com/news-release/2025/04/28/3069362/33333/en/Coherus-Presents-Promising-Early-Clinical-Data-from-Phase-1-Dose-Expansion-Study-of-CHS-114-in-Patients-with-Recurrent-Metastatic-Head-and-Neck-Squamous-Cell-Carcinoma-at-AACR-2025.html[3] BlackfinBio Announces FDA Clearance to Initiate Phase 1/2 Clinical Trial for Novel AAV Gene Therapy BFB-101 to Treat the Rare Neurological Disease Hereditary Spastic Paraplegia, Type 47 (SPG47). Retrieved April 29, 2025, from https://www.globenewswire.com/news-release/2025/04/28/3068935/0/en/BlackfinBio-Announces-FDA-Clearance-to-Initiate-Phase-1-2-Clinical-Trial-for-Novel-AAV-Gene-Therapy-BFB-101-to-Treat-the-Rare-Neurological-Disease-Hereditary-Spastic-Paraplegia-Typ.html[4] Oncoinvent Reports updated 18-Month Data from Phase 1 Ovarian Cancer Trial with Radspherin®: No new recurrences Demonstrate Continued Promise of Preventing Disease Progression. Retrieved April 29, 2025, from https://www.prnewswire.com/news-releases/oncoinvent-reports-updated-18-month-data-from-phase-1-ovarian-cancer-trial-with-radspherin-no-new-recurrences-demonstrate-continued-promise-of-preventing-disease-progression-302439415.html[5] Therini Bio Announces Positive Phase 1a Trial Results Evaluating THN391 for Neurodegenerative Diseases. Retrieved April 30, 2025, from https://www.globenewswire.com/news-release/2025/04/29/3070399/0/en/Therini-Bio-Announces-Positive-Phase-1a-Trial-Results-Evaluating-THN391-for-Neurodegenerative-Diseases.html[6] Design Therapeutics Announces Favorable Phase 1 Data for DT-168 Supporting Advancement into Phase 2 Biomarker Trial for Patients with Fuchs Endothelial Corneal Dystrophy. Retrieved May 1, 2025, from https://www.globenewswire.com/news-release/2025/05/01/3072316/0/en/Design-Therapeutics-Announces-Favorable-Phase-1-Data-for-DT-168-Supporting-Advancement-into-Phase-2-Biomarker-Trial-for-Patients-with-Fuchs-Endothelial-Corneal-Dystrophy.html[7] Nouscom Presents Positive Final Results from Completed Phase Ib/II Study of Neoantigen Immunotherapy NOUS-209 at AACR 2025, Demonstrating a Highly Potent and Durable Immune Response in Lynch Syndrome Carriers. Retrieved May 1, 2025, from https://www.globenewswire.com/news-release/2025/04/29/3070727/0/en/Nouscom-Presents-Positive-Final-Results-from-Completed-Phase-Ib-II-Study-of-Neoantigen-Immunotherapy-NOUS-209-at-AACR-2025-Demonstrating-a-Highly-Potent-and-Durable-Immune-Response.html[8] Avacta Therapeutics Presents Data from Lead pre|CISION® Candidate FAP-Dox (AVA6000) at the 2025 AACR Annual Meeting. Retrieved May 2, 2025, from https://avacta.com/avacta-therapeutics-presents-data-from-lead-precision-candidate-fap-dox-ava6000-at-the-2025-aacr-annual-meeting/免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新2

临床结果临床1期放射疗法临床2期

2025-05-04

·医脉通

女子无故腹痛、胃肠道出血，医生一直找不到病因……直到她说出这个病！丨医起推理吧

药物引起的胃肠道出血，可要注意了！来源 | 医脉通作者 | 小满这是安安（化名）第二次因为胃肠道出血到医院就诊了。01无诱因的胃溃疡出血，到底怎么回事？安安，28岁，1个月前因为胃痛和胃肠道出血在当地医院就诊，医生给她开了泮托拉唑，嘱咐她每天服用40mg的剂量。她回家以后按照医生给的建议服用药物。但一个月后，她再次发生了胃肠道出血，被转诊至本院。为什么吃了“泮托拉唑”后，还是会发生胃肠道出血？因为之前安安就诊时并没有做详细检查，所以医生决定先给安安做一个详细的检查。很快，她的检查结果就出来了。内窥镜检查：发现胃内有多处溃疡（图1）。图1 内窥镜检查图像消化性溃疡是一种由胃酸和胃蛋白酶对消化道黏膜的自我消化作用引起的慢性疾病，主要发生于胃和十二指肠，其核心机制是胃液侵袭性增强与黏膜防御能力下降的失衡。其中非甾体抗炎药物（NSAIDs）的使用和幽门螺杆菌（H. pylori，Hp）感染胃溃疡和十二指肠溃疡的主要危险因素。医生被安安的内窥镜下巨大的溃疡吓到了，于是再次向她求证是否有胃溃疡史或服用胃肠道药物。安安仔细想了又想，说：“我以往没有得过任何胃肠道疾病或胃溃疡，而且我也没有服用过任何致溃疡的药物。”为了一探究竟，医生对安安溃疡处进行了多次取样，并做了进一步检查：幽门螺杆菌（Hp）的测试结果：阴性。腹部CT扫描结果：正常。血常规检查结果：血清钙、胃泌素和嗜铬粒蛋白A水平也均在正常范围内（表1）。表1 安安的实验室检查结果于是医生给安安继续开了泮托拉唑，继续每天40mg两次给药。然而，回家以后的安安在服用药物以后，并没有得到好转，她的腹痛在持续4周以后，仍不消退。于是医生考虑是顽固性溃疡，又在治疗方案里添加了法莫替丁，用法为每天40mg。然而4周后，安安的征状仍然没有得到改善。医生们陷入了困局，无诱因的胃溃疡出血，治疗又无效果，病因到底在哪里？02她说出的一种病史，难道就是病因？突然，安安向医生告知了她患有抑郁症，这难道是病因？医生沉思了一会，回复她：“心情会影响到消化器官，但药物治疗也会有作用的。”“药物，对，药物，安安，你有服用哪些药物？”“安非他酮。”“安非他酮？！”医生又陷入沉思。因为“安非他酮”是一种多巴胺再摄取抑制剂，是一种常用的抗抑郁药和戒烟辅助剂，其常规用量推荐是：从每天150mg的最小剂量开始，增加至维持剂量为每天300mg，最大给药剂量450mg。安非他酮在治疗剂量下耐受性良好，但其常见的副作用包括口干、恶心、激动和失眠。虽然没有证据表明安安的胃溃疡与“安非他酮”有关，但医生还是要求安安的精神科医生给她停用了安非他酮。万幸，在安非他酮停药两周后，安安的腹痛得到了明显的缓解。两个月后，安安再次接受内窥镜检查，结果显示，消化性溃疡减少到几乎初始大小的一半（图2）。图2 停药2月后内窥镜检查图像但可惜的是，安安拒绝了接受第三次内窥镜检查，因为她的腹痛完全消失了。03安非他酮，为何会引起胃溃疡？消化性溃疡多由胃酸损伤引发，常见于胃及十二指肠，主因幽门螺杆菌感染或NSAIDs使用。本文患者安安第一次入院开了泮托拉唑，但并未减轻痛苦，后入院通过检查，排除Hp等常规风险因素，又通过排查过往的所有服药等经历，锁定安非他酮，停药联合抑酸治疗显著改善症状。但在以往的安非他酮副作用病例报告中，并没有找到引起消化性溃疡的报告。而在安非他酮的说明书中，也只明确提到临床常见的不良事件有激越、口干、失眠、头痛/偏头痛、恶心/呕吐、便秘和震颤，偶见吞咽困难、肝损伤/黄疸、直肠疾病、结肠炎、胃肠道出血、肠穿孔以及胃溃疡，则提示安非他酮可能与消化性溃疡存在潜在关联，但还需进一步研究验证。目前临床上对于消化性溃疡，常用的治疗药物主要分为抑制胃酸分泌药物和黏膜保护治疗药物：1.抑制胃酸分泌药物。表2 消化性溃疡常用治疗药物（抑制胃酸分泌药物）一览表2.胃肠黏膜保护剂主要是弱碱性抗酸剂和铋剂，抗酸分泌治疗的基础上加用胃黏膜保护剂能快速缓解症状以及改善溃疡修复质量。弱碱性抗酸剂：包括铝碳酸镁、磷酸铝、硫糖铝、氢氧化铝凝胶等，其主要是通过黏附、覆盖在溃疡面、阻止胃酸及胃蛋白酶侵袭溃疡面和促进内源性PGs合成等，便秘或腹泻是其主要不良反应。铋剂：除具备弱碱性抗酸剂功效外，该药物还能抗Hp，多用于根除Hp联合治疗，短期服会有黑便，因防铋积蓄，少用于溃疡治疗。此外对于NSAIDs相关性溃疡：应尽可能暂停或减少NSAIDs剂量；如果病情需要继续服用，尽可能选用对胃肠道黏膜损害较小的NSAIDs或高选择性COX⁃2抑制剂，以减少不良反应；对计划长期服用NSAIDs的患者，如果Hp阳性推荐根除Hp治疗；停服NSAIDs后，可用常规治疗溃疡方案进行治疗；当未能中止NSAIDs治疗时，应选用抑酸剂进行溃疡治疗。通过该病例也是想提醒大家，对于难治性溃疡，建议转诊上级医院，排除未规律服药可能，需进一步内镜或病理检查。参考文献：[1] Kuna L,Jakab J,Smolic R,et al.Peptic Ulcer Disease:A Brief Review of Conventional Therapy and Herbal Treatment Options.J Clin Med.2019,8(2):179.Published 2019 Feb 3. doi:10.3390/jcm8020179[2] Zhang BB,Li Y,Liu XQ,et al.Association between vacA genotypes and the risk of duodenal ulcer: a meta-analysis. Mol Biol Rep. 2014;41(11):7241-7254. doi:10.1007/s11033-014-3610-y[3] Momayez Sanat Z,Mohammadi Ganjaroudi N,Pirsalehi A.Peptic Ulcer Disease Following Use of Bupropion:A Case Report. Clin Med Insights Case Rep.2023,16:11795476231153279. Published 2023 Feb 8.doi:10.1177/11795476231153279[4] Settle EC Jr.Bupropion sustained release:side effect profile.J Clin Psychiatry.1998,59 Suppl 4:32-36.[5] 消化性溃疡基层诊疗指南（2023年）[J].中华全科医师杂志,2023,22(11):1108-1115.责编｜亦一封面图来源｜视觉中国为“诱使”患者手术，医方“谎称”专家参与手术？患者死亡后，医院被加重处罚丨医眼看法幼儿肺炎后死亡，家属质疑系“打错针致死”，尸检发现黄豆……是过失，还是意外？医脉通是专业的在线医生平台，“感知世界医学脉搏，助力中国临床决策”是平台的使命。医脉通旗下拥有「临床指南」「用药参考」「医学文献王」「医知源」「e研通」「e脉播」等系列产品，全面满足医学工作者临床决策、获取新知及提升科研效率等方面的需求。☟戳这里，更有料！

2025-05-03

·药明康德

疾病控制率超90%的抗癌多肽偶联药物；诺华核药新适应症监管申请获受理…… | TIDES周报

近期，全球多肽和寡核苷酸（TIDES）领域迎来系列进展。Avacta Therapeutics公司公布其主打在研多肽偶联药物AVA6000（FAP-Dox）的1期临床试验积极数据。接受≥250 mg/m²剂量的唾液腺癌患者，疾病控制率达到91%。诺华（Novartis）的放射配体疗法（RLT）Pluvicto（177Lu vipivotide tetraxetan，镥[177Lu]特昔维匹肽注射液）的新适应症监管申请获得中国国家药监局药品审评中心（CDE）受理。此外，诺华达成合并协议收购Regulus Therapeutics。欢迎读者长按/扫描以下二维码，申请获取含有完整表格整理的《2025年5月第1期TIDES疗法进展盘点》。▲长按识别上方二维码，申请获取《2025年5月第1期TIDES疗法进展盘点》AVA6000：1期试验结果公布Avacta Therapeutics公司日前公布了其主打在研多肽偶联药物AVA6000的1期临床试验积极数据。AVA6000是一种在肿瘤微环境中特异性激活的多柔比星（doxorubicin）前药，旨在减少传统化疗的全身性副作用。在1a期剂量递增研究中，无论是每三周一次（Q3W）还是每两周一次（Q2W）的给药方案，AVA6000均显示出良好的耐受性。即使剂量升高至每三周385 mg/m²，也未达到最大耐受剂量（MTD）。在唾液腺癌患者（n=11）中，接受≥250 mg/m²剂量水平的AVA6000治疗后，多位患者获得确认缓解，疾病控制率达到91%。中位无进展生存期（PFS）尚未达到，当前中位随访时间已超过25周。Avacta Therapeutics公司目前正在1b期扩展队列中持续招募包括唾液腺癌、三阴性乳腺癌以及高级别软组织肉瘤的患者，相关数据预计将于2025年底公布。Pluvicto：新适应症监管申请获得受理日前，中国国家药监局药品审评中心官网公示，诺华的前列腺特异性膜抗原（PSMA）靶向放射配体疗法Pluvicto的新适应症监管申请获得受理，并被拟纳入优先审评，用于治疗既往接受过雄激素受体通路抑制剂（ARPI）治疗的PSMA阳性转移性去势抵抗性前列腺癌（mCRPC）成年患者。针对该项适应症，Pluvicto于今年3月获得了美国FDA批准。Pluvicto是通过静脉注射的放射配体疗法，由靶向配体与治疗性放射性核素镥结合而成。进入血液后，Pluvicto可靶向结合表达PSMA的前列腺癌细胞。结合后，放射性同位素释放的能量可破坏靶细胞，抑制其复制能力和/或引发肿瘤细胞死亡。图片来源：CDE官网今年3月，诺华宣布FDA批准Pluvicto用于接受过一种ARPI治疗且被认为适合延迟化疗的PSMA阳性的mCRPC患者。此适应症获批是基于3期PSMAfore临床研究的结果，将符合Pluvicto治疗条件的患者人群扩大约三倍。研究结果显示，与更换ARPI方案相比，Pluvicto将PSMA阳性mCRPC患者的影像学进展或死亡风险降低59%，Pluvicto组的中位影像学无进展生存期显著延长（9.3个月vs. 5.6个月，p<0.0001）。诺华达成协议收购Regulus TherapeuticsRegulus Therapeutics日前宣布，已与诺华达成一项合并协议。根据该协议，诺华将以8亿美元的初始付款收购Regulus。此外，Regulus也将在该公司的主打在研产品farabursen达成潜在监管里程碑时，获得额外9亿美元的款项，使交易总额最高可达约17亿美元。Regulus Therapeutics是一家专注于发现和开发靶向微RNA（microRNA）创新药物的生物医药公司。该公司充分发挥其在寡核苷酸药物发现和开发领域的专长，已建立了一系列在研药物管线。Regulus Therapeutics的主打在研疗法farabursen是一款靶向miR-17的新型、下一代寡核苷酸药物，用于治疗常染色体显性多囊肾病（ADPKD）。该药物近期已完成一项1b期多剂量递增临床试验。限于篇幅，本文仅针对部分重要进展做简单介绍。欢迎读者长按/扫描以下二维码，申请获取含有完整表格整理的《2025年5月第1期TIDES疗法进展盘点》。▲长按识别上方二维码，申请获取《2025年5月第1期TIDES疗法进展盘点》参考资料：[1] Avacta Therapeutics Presents Data from Lead pre|CISION® Candidate FAP-Dox (AVA6000) at the 2025 AACR Annual Meeting. Retrieved May 1, 2025 from https://www.globenewswire.com/news-release/2025/04/28/3069067/0/en/Avacta-Therapeutics-Presents-Data-from-Lead-pre-CISION-Candidate-FAP-Dox-AVA6000-at-the-2025-AACR-Annual-Meeting.html[2] 中国国家药监局药品审评中心（CDE）官网. Retrieved Apr 28，2025, From https://www.cde.org.cn/main/xxgk/listpage/4b5255eb0a84820cef4ca3e8b6bbe20c[3] FDA expands Pluvicto’s metastatic castration-resistant prostate cancer indication. Retrieved May 2, 2025 from https://www.fda.gov/drugs/resources-information-approved-drugs/fda-expands-pluvictos-metastatic-castration-resistant-prostate-cancer-indication[4] Regulus Therapeutics Enters into Agreement to be Acquired by Novartis AG. Retrieved April 30, 2025 from https://www.prnewswire.com/news-releases/regulus-therapeutics-enters-into-agreement-to-be-acquired-by-novartis-ag-302442023.html免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

放射疗法临床3期优先审批临床2期临床结果