Aidixi (disitamab vedotin)

P3, N=574, Recruiting, RemeGen Co., Ltd. | Not yet recruiting --> Recruiting

HER2 expression in urothelial carcinoma was associated with a distinct immune microenvironmental profile characterized by Treg enrichment and relatively low immune checkpoint expression, as supported by our protein-level validation. These findings provide additional biological context for the potential therapeutic benefit of combining RC48 with ICIs. Given the exploratory nature of the HER2-high definition and the limited sample size of the validation cohorts, further prospective studies are warranted to confirm these observations.

Key advances include: (1) In bladder cancer, perioperative durvalumab (NIAGARA) and enfortumab vedotin plus pembrolizumab (KEYNOTE-905/EV-303) set new standards, while HER2-targeted disitamab vedotin plus toripalimab (RC48-C016) improved metastatic survival...(3) In prostate cancer, enzalutamide plus leuprolide improved survival in high-risk biochemical recurrence (EMBARK). Capivasertib plus abiraterone benefited PTEN-deficient metastatic hormone-sensitive disease (CAPItello-281). The PSMAddition trial demonstrated that adding [177Lu]Lu-PSMA-617 to standard therapy significantly improved radiographic PFS in PSMA-positive mHSPC. Docetaxel scheduling was optimized (ARASAFE), and an AI model (STAMPEDE) identified patients for AR inhibitor benefit. Novel agents like saruparib and pasritamig showed promise...The 2025 evidence establishes multiple new standards of care across GU cancers, emphasizing biomarker-driven strategies, immunotherapy integration, novel resistance mechanisms, and treatment optimization. This synthesis provides an evidence-based framework for updating guidelines and highlights the move toward more personalized management, while noting persistent challenges and future research needs.

First-line conversion therapy with oxaliplatin, fluoropyrimidine, sintilimab (a programmed death-1 inhibitor), and later trastuzumab yielded only transient stabilization followed by clear progression: After six cycles, AFP rose to 1 546.07 μg/L and target lymph nodes enlarged to 46 mm×31 mm on CT. Given treatment failure and persistent HER2 positivity, a second-line, biology-informed regimen was initiated: Disitamab vedotin (an HER2-targeted antibody-drug conjugate delivering monomethyl auristatin E), lenvatinib (a multi-targeted tyrosine kinase inhibitor blocking vascular endothelial growth factor receptor and other pro-angiogenic pathways), tislelizumab (a programmed death-1 inhibitor), and short-course capecitabine (discontinued after 7 days due to grade 3 thrombocytopenia)...This case underscores that in HER2-positive, chemotherapy-refractory HAS, a rationally designed, multimodal regimen integrating an HER2-directed antibody-drug conjugate, antiangiogenic agent, and immune checkpoint blockade can overcome therapeutic resistance, achieve meaningful downstaging, and enable long-term disease control. Early molecular characterization and aggressive, persona-lized intervention are essential for improving outcomes in this rare malignancy.

Given the exploratory nature of this analysis and the study's limitations, including the small sample size, single-center design, and lack of a control arm, these findings should be interpreted with caution. Larger prospective studies are warranted to validate these results.

Human epidermal growth factor receptor 2 (HER2) overexpression is a key therapeutic target for novel antibody-drug conjugates (ADCs) like disitamab vedotin (RC48) in bladder urothelial carcinoma (BLCA), but immunohistochemistry-based assessment is limited by intratumoral heterogeneity and sampling bias...The multimodal model significantly outperformed all single-modality models. A tri-phasic CT-based multimodal model enables noninvasive assessment of HER2 expression status in BLCA, providing a promising tool for selection of patients likely to benefit from ADC therapy.

P1/2, N=100, Recruiting, Pfizer

P2, N=29, Not yet recruiting, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology

P2, N=76, Not yet recruiting, Sun Yat-sen University

P2, N=39, Recruiting, Peking Union Medical College | Trial primary completion date: Jul 2026 --> Jul 2028

P2, N=192, Not yet recruiting, Peking University First Hospital

P=N/A, N=30, Not yet recruiting, West China Hospital