OriCell's GPC3 CAR-T Receives NMPA Clearance for Confirmatory Phase II Trial in Late-Line Advanced Hepatocellular Carcinoma

SHANGHAI, June 7, 2026 /PRNewswire/ -- OriCell Therapeutics Holdings Limited ( The "Company" or "Oricell") , announced 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 study is designed as a prospective, randomised, open-label, multi-centre registration trial evaluating the efficacy and safety of Ori-C101 in patients who have failed two or more prior lines of therapy.

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 — a milestone that underscores China's potential to lead the next wave of innovation in cell therapy for solid tumours.

HCC, a disease with few options at the late line

Hepatocellular carcinoma is among the most prevalent malignancies globally, with China alone accounting for roughly 410,000 new cases and 317,000 deaths each year. For patients whose disease has progressed through both immune checkpoint inhibitors (ICI) and tyrosine kinase inhibitors (TKI), options are essentially exhausted: existing second-line agents were developed for patients intolerant of, or progressing on, first-line sorafenib, and nothing has been approved beyond that point. The unmet need is acute.

Phase I data of Ori-C101: robust efficacy and manageable safety

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 wrapped up in Chicago, drawing significant attention from the oncology community. All enrolled patients had failed at least two prior lines of therapy, including both TKI and ICI treatment.

Key results:

Objective response rate (ORR): 50% across the evaluable population; 66.7% in the recommended Phase II dose (RP2D) cohort; 100% in the highest dose cohort (DL4)

Speed of response: 89% of responders had already achieved objective response by their first post-infusion assessment, with marked tumour shrinkage

Overall survival (OS): Median OS of 21.4 months in the overall population; 12-month survival rate of 69.3% — more than double the roughly 10.6-month historical median seen with available second-line agents, heralding a potential new era of long-term survival for patients with advanced HCC

Safety: Cytokine release syndrome (CRS) was manageable; no immune effector cell-associated neurotoxicity syndrome (ICANS) or off-target toxicity was observed, and tolerability was favourable across the study population

Notable case: One patient achieved partial response at first assessment, progressed to complete response by month four, and remains in remission at 24 months

Mechanism and broader potential

GPC3 (Glypican-3) is overexpressed in more than 70% of hepatocellular carcinomas, as well as in gastric cancer, lung squamous cell carcinoma, and ovarian clear cell carcinoma — making it a highly specific and compelling target for cancer therapy. Ori-C101 is engineered to identify and eliminate GPC3-positive tumour cells with precision, drawing on two proprietary platforms: Ori^®Ab (AI-assisted antibody discovery and engineering) and Ori^®Armoring (structure-enhanced cell platform).

Ori-C101's 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.

Principal investigators

The confirmatory Phase II study is a nation-wide, multi-centre trial co-led by Professor Fan Jia (Academician of the Chinese Academy of Sciences and Honorary President of Zhongshan Hospital, Fudan University) and Professor Qin Shukui (Qiantang Scholar and Professor at Nanjing Tianyinshan Hospital, China Pharmaceutical University).

In a joint statement, the two investigators said: "In our earlier work on late-line advanced HCC, the BEACON study delivered important progress. GPC3 is specifically overexpressed in HCC and represents an ideal therapeutic target, yet GPC3-directed CAR-T approaches have historically been constrained by the immunosuppressive tumour microenvironment. BEACON was designed precisely to break through that barrier and find a new, accessible path for patients who have exhausted their options. The preliminary results from this novel 'armoured' GPC3 CAR-T construct — showing strong objective response rates with a manageable safety profile — not only address an evidence gap in late-line refractory HCC but offer broader lessons for innovative immune cell therapies in solid tumours. We look forward to the Phase II randomised controlled study, and to obtaining the expected results under rigorous scientific design, comprehensive management, and strict quality control."

OriCell co-founder, Chairman and Chief Executive Officer Dr. Yang Huanfeng commented: "We are deeply grateful to the NMPA Centre for Drug Evaluation and relevant government authorities for their strong support of innovative drug development, and to Academician Fan, Professor Qin and their teams for their trust and active participation. This approval validates the scientific soundness and feasibility of our technology platform and marks a new stage of growth for OriCell. With a clear regulatory pathway now in place, we will push forward with Ori-C101's confirmatory clinical development, with the aim of bringing a genuinely new treatment option to patients as quickly as we can. We believe this progress will also underpin China's strategy for taking innovative medicines global and competing actively in international markets."

About OriCell Therapeutics

OriCell Therapeutics is a clinical-stage, globally oriented biotechnology company committed to pioneering cancer immunotherapy. Focused on addressing unmet medical needs in oncology and immunology, the company has built a robust pipeline spanning both solid and haematological tumours, powered by three proprietary technology engines: Ori^®AB (AI-assisted antibody discovery and engineering), Ori^®Armoring (structure-enhanced cell platform), and Ori^®OnGo (classic, rapid, and in vivo manufacturing platform). The company's clinical translation capabilities have earned broad international recognition, with multiple data readouts selected for oral presentations at leading conferences including ASCO and EHA, and published in top-tier journals such as The Lancet Haematology. OriCell is accelerating its global expansion, driven by a commitment to continuous innovation and the ambition to bring new hope to cancer patients worldwide. For more information, please visit www.oricell.com.

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SOURCE Oricell Therapeutics Holdings Limited