Breakthrough! LEPU BIO has completed the enrollment of the first patient in the Phase II clinical trial for hepatocellular carcinoma

According to the Zhitong Finance APP, recently, LEPU BIO (02157) has successfully completed the enrollment of the first patient in the Phase II clinical study of its self-developed GPC3-targeted antibody-drug conjugate (ADC) MRG006A for the indication of advanced hepatocellular carcinoma (HCC)! MRG006A is the world's first GPC3-targeted ADC drug to enter Phase II clinical trials. MRG006A (GPC3 ADC) is developed based on LEPU BIO's next-generation Hi-TOPi ADC technology platform, which precisely binds to GPC3-positive tumor cells and releases toxins within the tumor to achieve targeted killing of tumor cells. Hepatocellular carcinoma (HCC) is a highly prevalent malignant tumor globally, with most patients diagnosed at an advanced stage. After first-line treatment failure, options for subsequent therapies and their efficacy are limited. Against this backdrop, the innovative ADC drug MRG006A targeting the liver cancer-specific marker GPC3 has garnered significant attention. According to the People's Daily, at the "GPC3-ADC Liver Cancer Expert Advisory Meeting" on July 15, Dr. Zhao Hong, Chief Physician of the Hepatobiliary Surgery Department at the Cancer Hospital of the Chinese Academy of Medical Sciences, stated in an interview, "GPC3 is a cancer embryonic antigen involved in cell proliferation, differentiation, migration, and apoptosis. It is almost not expressed in normal tissues but is highly expressed in 70%-80% of hepatocellular carcinoma cases, making it a 'golden' target for precisely targeting liver cancer cells." He added, "As a GPC3-targeted ADC drug entering clinical stages, MRG006A's innovative value also lies in its technological breakthroughs." Dr. Zhao explained: MRG006A is developed based on the Hi-TOPi technology platform, akin to a "precision-guided missile"—with a monoclonal antibody targeting GPC3 serving as the "navigation system," accurately locating liver cancer cells, and a linker carrying a cytotoxic drug (topoisomerase I inhibitor) as the "warhead," achieving specific killing of tumor cells. This mechanism retains strong anti-tumor efficacy while reducing damage to normal cells due to its targeting, addressing the issue of excessive toxicity associated with traditional cytotoxic drugs. Clinical data indicate that its adverse reactions mainly focus on liver function abnormalities (elevated bilirubin and transaminases) and mild bone marrow suppression, which are overall controllable, further validating its clinical applicability. From the perspective of clinical progress, MRG006A has shown significant potential. Dr. Huang Zhen, Deputy Chief Physician of the Hepatobiliary Surgery Department at the Cancer Hospital of the Chinese Academy of Medical Sciences, mentioned, "In the Phase I clinical trial, the dose-escalation study achieved results beyond expectations. In the dose group, two GPC3-positive patients showed significant tumor shrinkage after enrollment, and the treatment effect is encouraging." The advancement of the Phase II clinical study of MRG006A not only represents an important breakthrough in the development of LEPU BIO's ADC pipeline but is also expected to fill the clinical gap in liver cancer treatment