en.Wedoany.com Reported - OriCell Therapeutics Holdings Limited ("OriCell") announced that its self-developed autologous CAR-T therapy targeting GPC3, Ori-C101, has received approval from China's National Medical Products Administration (NMPA) to conduct a confirmatory Phase II clinical trial for patients with GPC3-positive advanced hepatocellular carcinoma (HCC). This prospective, randomized, open-label, multicenter registration study aims to evaluate the efficacy and safety of Ori-C101 in patients who have progressed after at least two prior systemic therapies. According to the company, this is the world's first GPC3-targeted immune cell therapy to enter a confirmatory trial and the first CAR-T product for liver cancer to advance to a Phase II randomized controlled study.
Hepatocellular carcinoma is one of the most common malignant tumors globally, with approximately 410,000 new cases and 317,000 deaths annually in China. For patients who have progressed after both immune checkpoint inhibitor (ICI) and tyrosine kinase inhibitor (TKI) therapies, treatment options are essentially exhausted. Existing second-line drugs were developed for patients intolerant to or progressing after first-line sorafenib, with no approved therapies beyond that, leaving a significant unmet medical need.
Results from the Phase I BEACON study were presented as an oral report at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting. All enrolled patients had failed at least two prior therapies, including TKIs and ICIs. Key findings showed an objective response rate (ORR) of 50% in the evaluable population, 66.7% in the recommended Phase II dose (RP2D) cohort, and 100% in the highest dose cohort (DL4). 89% of responders achieved objective response at the first post-infusion assessment. The median overall survival (OS) in the overall population was 21.4 months, with a 12-month survival rate of 69.3%. In terms of safety, cytokine release syndrome (CRS) was manageable, with no observed immune effector cell-associated neurotoxicity syndrome (ICANS) or off-target toxicity. One patient achieved partial response at the first assessment, progressed to complete response by month 4, and remained in remission at 24 months.
GPC3 (glypican-3) is overexpressed in over 70% of hepatocellular carcinomas, as well as in gastric cancer, lung squamous cell carcinoma, and ovarian clear cell carcinoma. Ori-C101 is engineered to recognize and eliminate GPC3-positive tumor cells, based on two proprietary platforms: Ori®Ab (AI-assisted antibody discovery and engineering) and Ori®Armoring (structure-enhanced cell platform).
The confirmatory Phase II study is co-led by Professor Fan Jia (Academician of the Chinese Academy of Sciences, Honorary President of Zhongshan Hospital, Fudan University) and Professor Qin Shukui (Qiantang Scholar, Professor at Nanjing Tianshan Hospital, China Pharmaceutical University). Dr. Yang Huanfeng, co-founder, chairman, and CEO of OriCell, stated that this approval validates the feasibility of the company's technology platforms, and the company will accelerate the clinical development of Ori-C101 with the goal of bringing new treatment options to patients as soon as possible.
OriCell Therapeutics is a clinical-stage biotechnology company focused on cancer immunotherapy, with a pipeline of solid tumor and hematological malignancy therapies built on three technology engines: Ori®AB, Ori®Armoring, and Ori®OnGo. The company's data has been selected for oral presentations at conferences such as ASCO and the European Hematology Association (EHA), and published in journals including The Lancet Haematology.
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