Poised to Reshape Treatment Landscape: The Phase 3 Clinical Trial of Disitamab Vedotin as a First-Line Therapy for HER2-Expressing Locally Advanced or Metastatic ‌Urothelial Carcinoma Reached its Primary Endpoints of PFS and OS

YANTAI, China, May 12, 2025 /PRNewswire/ -- On May 12th, 2025, RemeGen Co., Ltd. ("RemeGen", stock symbols: 688331.SH/09995.HK) announced that its phase 3 clinical trial (Study ID: RC48-C016) on disitamab vedotin (DV, brand name: 爱地希^®), the first domestically approved antibody-drug conjugate (ADC) in China, in combination with the PD-1 inhibitor toripalimab versus the standard chemotherapy as a first-line treatment for HER2-expressing locally advanced or metastatic urothelial carcinoma (la/mUC) has reached its two primary endpoints of progression-free survival (PFS) and overall survival (OS). Statistically significant differences and clinically meaningful benefits were observed from the strongly positive results of a prespecified interim analysis by Independent Data Monitoring Committee (IDMC). The major subgroup analyses showed that DV combined with toripalimab significantly improved PFS and OS compared with the standard chemotherapy treatment, irrespective of cisplatin eligibility or HER2 expression level. Furthermore, this regimen exhibited manageable safety profile and tolerable adverse reactions. Detailed data of this study are planned to be presented at major international academic conferences later this year. Subsequently, RemeGen plans to file Biologic License Application (BLA) for this indication to the Center of Drug Evaluation of National Medical Products Administration (NMPA) in China.

RC48-C016 is a randomized, active-controlled, multi-center phase 3 clinical trial evaluating efficacy and safety of DV plus toripalimab versus gemcitabine in combination with cisplatin/carboplatin in systemic-treatment-naive patients with HER2-expressing (defined as IHC 1+, 2+ or 3+) la/mUC. The study was initiated in June 2022 and conducted in 74 sites across China with 484 patients enrolled.

The principal researcher of this study, Professor Guo Jun from Peking University Cancer Hospital, said: "Extremely exciting! We, once again, jointly witnessed a strong positive result of DV combined with toripalimab in the first-line treatment of advanced urothelial carcinoma. Regardless of whether the patients are suitable for cisplatin treatment and regardless of patients' HER2 expression status, DV combined with toripalimab significantly improved PFS and OS. This outstanding efficacy proves the success of the 'HER2-ADC + immunotherapy' combination treatment concept, and is also a major breakthrough in the global treatment of urothelial carcinoma. We look forward to the excellent performance of DV in subsequent studies which should provide better decision-making basis for clinicians, bring more benefits to patients, and reshape the global treatment landscape of urothelial carcinoma with the 'Chinese approach'."

source: RemeGen Co., Ltd