Chinese biotechnology company OriCell Therapeutics Holdings Limited announced on Sunday that its proprietary GPC3-targeted autologous CAR-T therapy, Ori-C101, has received clearance from China's National Medical Products Administration (NMPA) to proceed into a confirmatory Phase II clinical trial in patients with GPC3-positive advanced hepatocellular carcinoma (HCC).

The prospective, randomised, open-label, multi-centre registration trial is designed to evaluate 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 anywhere in the world to enter a confirmatory trial, and the first CAR-T product for a liver cancer indication to reach a Phase II randomised controlled study.

Results from the Phase I BEACON study were featured as an oral presentation at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting, which recently took place in Chicago. All enrolled patients had failed at least two prior lines of therapy, including both TKI and ICI treatment.

The results showed an objective response rate of 50% across the evaluable population, 66.7% in the recommended Phase II dose cohort, and 100% in the highest dose cohort. As many as 89% of responders had already achieved objective response by their first post-infusion assessment, with marked tumour shrinkage. Median overall survival was 21.4 months in the overall population, with a 12-month survival rate of 69.3% -- more than double the roughly 10.6-month historical median seen with available second-line agents, Oricell said. One patient achieved partial response at first assessment, progressed to complete response by month four, and remains in remission at 24 months.

Ori-C101 is engineered to identify and eliminate GPC3-positive tumour cells with precision, drawing on two proprietary platforms: OriAb (AI-assisted antibody discovery and engineering) and OriArmoring (structure-enhanced cell platform). Its profile has the potential to support use in earlier lines of treatment, and GPC3's expression across multiple tumour types provides a strong scientific rationale for future indication expansion. As the confirmatory trial advances, the goal of treatment in advanced HCC has the potential to shift from controlling disease to achieving durable remission or even cure.