June 08, 2026 —
OriCell Therapeutics announced that China’s National Medical Products Administration has cleared Ori-C101, the company’s proprietary GPC3-targeted autologous CAR-T therapy, to proceed into a confirmatory Phase 2 clinical trial in patients with GPC3-positive advanced hepatocellular carcinoma, or HCC.
The study is designed as a prospective, randomized, open-label, multi-center registration trial evaluating the efficacy and safety of Ori-C101 in patients who have failed two or more prior lines of therapy. According to OriCell, this marks the first GPC3-directed immune cell therapy globally to enter a confirmatory trial and the first CAR-T product for liver cancer to reach a randomized Phase 2 study.
Hepatocellular carcinoma remains a major global cancer burden, with China accounting for a large proportion of new cases and deaths each year. For patients whose disease has progressed after both immune checkpoint inhibitors and tyrosine kinase inhibitors, treatment options are extremely limited, creating a significant unmet need in late-line advanced HCC.
The NMPA clearance follows Phase 1 results from the BEACON study, which were presented as an oral presentation at the 2026 American Society of Clinical Oncology Annual Meeting. All enrolled patients had received at least two prior lines of therapy, including both TKI and ICI treatment.
In the Phase 1 study, Ori-C101 showed an objective response rate of 50% across the evaluable population, 66.7% in the recommended Phase 2 dose cohort, and 100% in the highest dose cohort. Responses were also rapid, with 89% of responders achieving objective response by the first post-infusion assessment. Median overall survival reached 21.4 months in the overall population, with a 12-month survival rate of 69.3%.
Safety was described as manageable. Cytokine release syndrome was reported as manageable, with no immune effector cell-associated neurotoxicity syndrome, no off-target toxicity, and favorable tolerability across the study population. One notable patient achieved partial response at the first assessment, progressed to complete response by month four, and remained in remission at 24 months.
Ori-C101 targets GPC3, a tumor-associated antigen overexpressed in more than 70% of hepatocellular carcinomas and also found in several other cancers, including gastric cancer, lung squamous cell carcinoma, and ovarian clear cell carcinoma. The therapy is designed to identify and eliminate GPC3-positive tumor cells and is built using OriCell’s proprietary Ori®Ab AI-assisted antibody discovery and engineering platform and Ori®Armoring structure-enhanced cell platform.
The confirmatory Phase 2 trial will be co-led by Professor Fan Jia of Zhongshan Hospital, Fudan University, and Professor Qin Shukui of Nanjing Tianyinshan Hospital, China Pharmaceutical University. As the trial advances, OriCell aims to evaluate whether Ori-C101 can shift the treatment goal in advanced HCC from disease control toward durable remission in patients who have exhausted existing options.
The program also highlights broader momentum in solid tumor CAR-T development. While CAR-T therapies have transformed several hematologic malignancies, success in solid tumors has been more challenging due to antigen heterogeneity, trafficking barriers, and immunosuppressive tumor microenvironments. Ori-C101’s advancement into a randomized confirmatory trial represents an important clinical milestone for GPC3-directed CAR-T therapy and for cell therapy development in liver cancer.
