SHANGHAI, China, June 8, 2026
OriCell Therapeutics has achieved a major regulatory and clinical milestone with the National Medical Products Administration (NMPA) clearance of its GPC3-targeted CAR-T therapy, Ori-C101, to advance into a confirmatory Phase II clinical trial for patients with GPC3-positive advanced hepatocellular carcinoma (HCC) who have failed two or more prior lines of treatment. The approval marks a significant step forward in the development of cellular immunotherapies for solid tumors and positions Ori-C101 as the first GPC3-directed immune cell therapy globally to enter a confirmatory clinical trial. It is also the first CAR-T therapy targeting liver cancer to reach a randomized Phase II study, underscoring growing momentum in the application of engineered cell therapies beyond hematologic malignancies. The trial will evaluate the efficacy and safety of Ori-C101 in a late-line HCC population with extremely limited treatment options, a setting where survival outcomes remain poor despite advances in immunotherapy and targeted therapies.
Phase I Data Demonstrate Encouraging Clinical Activity
The NMPA decision follows highly encouraging results from the Phase I BEACON study, which were recently presented in an oral session at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting. The study enrolled patients with advanced hepatocellular carcinoma who had previously failed treatment with both immune checkpoint inhibitors (ICIs) and tyrosine kinase inhibitors (TKIs), representing one of the most difficult-to-treat patient populations in oncology. Clinical findings demonstrated an impressive objective response rate (ORR) of 50% across evaluable patients, increasing to 66.7% in the recommended Phase II dose cohort and reaching 100% in the highest dose cohort.
Responses were rapid, with nearly 90% of responding patients achieving measurable tumor shrinkage at the first post-treatment assessment. Survival outcomes were equally encouraging, with a median overall survival of 21.4 months and a 12-month survival rate of 69.3%, substantially exceeding historical outcomes reported for currently available late-line therapies. Investigators also highlighted a notable case in which a patient progressed from partial response to complete remission and remained disease-free for 24 months following treatment.
Targeting GPC3 Opens New Opportunities in Solid Tumor CAR-T Therapy
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Ori-C101 is designed to target Glypican-3 (GPC3), a tumor-associated antigen highly expressed in more than 70% of hepatocellular carcinoma cases while demonstrating limited expression in normal tissues. This specificity makes GPC3 one of the most attractive targets for next-generation cancer immunotherapy. The therapy leverages OriCell’s proprietary Ori®Ab AI-assisted antibody discovery platform and Ori®Armoring structure-enhanced cell engineering technology, which were developed to improve tumor targeting and overcome the immunosuppressive microenvironment that has historically limited CAR-T success in solid tumors.
Researchers believe the armoured CAR-T approach may help address key challenges that have hindered cellular therapies in liver cancer and other solid malignancies. The favorable safety profile reported in the Phase I study further strengthens confidence in the platform, with manageable cytokine release syndrome (CRS), no observed immune effector cell-associated neurotoxicity syndrome (ICANS), and no off-target toxicity across treated patients.
Confirmatory Trial Could Redefine Treatment for Advanced HCC
Hepatocellular carcinoma remains one of the most common and deadly cancers worldwide, with China accounting for hundreds of thousands of new diagnoses and deaths annually. Patients who progress after treatment with both immunotherapy and targeted therapies currently face a severe lack of effective options. The newly approved confirmatory Phase II trial, a prospective, randomized, open-label, multi-center registration study, aims to determine whether Ori-C101 can establish a new treatment paradigm for these patients.
Beyond liver cancer, GPC3 is also expressed in several other solid tumors, including gastric cancer, lung squamous cell carcinoma, and ovarian clear cell carcinoma, creating opportunities for future expansion into additional indications. As OriCell advances this landmark study, Ori-C101 is emerging as one of the most promising CAR-T therapies in solid tumor oncology, with the potential to shift treatment goals from disease control toward durable remission and long-term survival in advanced liver cancer patients.
Source: OriCell Therapeutics press release
