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

Rhea-AI Summary

RemeGen (REGMY) announced significant positive results from its Phase 3 clinical trial of disitamab vedotin (DV) in combination with PD-1 inhibitor toripalimab. The study, evaluating the treatment as a first-line therapy for HER2-expressing locally advanced or metastatic urothelial carcinoma (la/mUC), successfully met its primary endpoints of progression-free survival (PFS) and overall survival (OS).

The trial, involving 484 patients across 74 sites in China, demonstrated statistically significant improvements in both PFS and OS compared to standard chemotherapy, regardless of cisplatin eligibility or HER2 expression levels. The treatment showed a manageable safety profile with tolerable adverse reactions. RemeGen plans to file a Biologic License Application (BLA) with China's NMPA, with detailed data to be presented at upcoming international conferences.

Positive

• Phase 3 trial successfully met both primary endpoints (PFS and OS)
• Treatment showed efficacy regardless of cisplatin eligibility or HER2 expression level
• Demonstrated manageable safety profile and tolerable adverse reactions
• Plans to file BLA with China's NMPA for regulatory approval

Negative

• None.

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'."

View original content to download multimedia:https://www.prnewswire.com/news-releases/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-302452193.html

SOURCE RemeGen Co., Ltd

FAQ

What were the primary endpoints achieved in RemeGen's (REGMY) Phase 3 trial for disitamab vedotin?

The Phase 3 trial met its two primary endpoints of progression-free survival (PFS) and overall survival (OS), showing statistically significant differences and clinically meaningful benefits compared to standard chemotherapy.

How many patients were enrolled in RemeGen's Phase 3 trial for urothelial carcinoma treatment?

The Phase 3 clinical trial enrolled 484 patients across 74 sites in China, starting in June 2022.

What is the combination therapy being tested in RemeGen's Phase 3 trial?

The trial tested disitamab vedotin (DV) in combination with PD-1 inhibitor toripalimab versus standard chemotherapy (gemcitabine with cisplatin/carboplatin) for HER2-expressing locally advanced or metastatic urothelial carcinoma.

What are RemeGen's next steps following the positive Phase 3 trial results?

RemeGen plans to present detailed trial data at major international academic conferences and file a Biologic License Application (BLA) with China's NMPA for this indication.

What patient population did the REGMY Phase 3 trial target?

The trial targeted systemic-treatment-naive patients with HER2-expressing (IHC 1+, 2+ or 3+) locally advanced or metastatic urothelial carcinoma (la/mUC).