#China #Shanghai #CAR-T therapy #Hepatocellular Carcinoma #OriCell

OriCell's GPC3 CAR-T Receives NMPA Clearance for Phase II Trial

OriCell Therapeutics Holdings Limited, a pioneering biopharmaceutical company, has made significant strides in cancer treatment with its GPC3-targeted CAR-T therapy, Ori-C101. Recently, the company announced that it has received clearance from the National Medical Products Administration (NMPA) in China to advance into a confirmatory Phase II clinical trial. This milestone is particularly noteworthy as it signifies the first GPC3-directed immune cell therapy to enter confirmatory trials globally.

Understanding Advanced Hepatocellular Carcinoma

Hepatocellular carcinoma (HCC) represents one of the most common cancers worldwide, with China alone accounting for approximately 410,000 new cases and 317,000 deaths annually. Unfortunately, patients dealing with advanced HCC have historically limited treatment options, particularly after failing initial therapies such as immune checkpoint inhibitors (ICI) and tyrosine kinase inhibitors (TKI). Once these standard treatments prove ineffective, patients often face an acute unmet medical need, with no approved subsequent therapies available.

Promising Phase I Results

The Phase I BEACON study results, which were presented at the American Society of Clinical Oncology (ASCO) Annual Meeting in 2026, showcased the strong potential of Ori-C101. Notably, 50% of evaluable patients with advanced HCC achieved an objective response rate (ORR). A staggering 66.7% from the recommended Phase II dose group and even 100% from the highest dose cohort experienced measurable tumor reduction.

Among the promising results:

• - Speed of Response: A remarkable 89% of patients who responded did so by their first assessment following infusion.
• - Overall Survival Rates: The median overall survival in the study population reached 21.4 months, with a 12-month survival rate of 69.3%. This is more than double the historical median of 10.6 months observed with available second-line agents.
• - Safety Profile: Importantly, the treatment displayed manageable cytokine release syndrome (CRS) with no occurrences of immune effector cell-associated neurotoxicity syndrome (ICANS) or significant off-target toxicity.

One specific case stands out, where a patient exhibited partial response initially, advanced to a complete response by the fourth month, and is still in remission after 24 months.

Innovative Mechanism of Action

GPC3 (Glypican-3) is a protein that is overexpressed in many cases of HCC and various other cancers. Ori-C101 utilizes unique platforms designed for precise targeting, utilizing both artificial intelligence-assisted antibody discovery and enhanced cell structures to effectively eliminate GPC3-positive tumor cells. This innovative approach not only sets the groundwork for maintaining treatment in earlier cancer stages but also suggests the possibility of expanding its use across multiple tumor types, hence addressing broader oncology needs.

Principal Investigator Insights

The Phase II trial is set to be led by esteemed professionals, including Professor Fan Jia from Zhongshan Hospital, Fudan University, and Professor Qin Shukui from Nanjing Tianyinshan Hospital. In a collective statement, the investigators emphasized the importance of the BEACON study and how it tackles barriers previously faced by GPC3-directed CAR-T therapies, thereby improving access for patients out of options. They expressed optimism regarding the ongoing Phase II trial, which aims to yield further evidence supporting this innovative cancer therapy.

Commitment to Innovation

OriCell's leadership, represented by co-founder and CEO Dr. Yang Huanfeng, expressed gratitude for the support received from the NMPA and highlighted the approval as a validation of the scientific integrity of their platform. With a clear regulatory pathway established, OriCell is poised to push forward, aiming to provide novel treatment alternatives for patients suffering from advanced HCC and ultimately contribute to China's ambitions in pioneering global innovative medications.

About OriCell Therapeutics

OriCell Therapeutics is a biopharmaceutical company dedicated to advancing cancer immunotherapies. Their strategic focus involves addressing pressing unmet medical requirements within oncology and immunology through a range of proprietary technology platforms. Recognized for its commitment to research and development, OriCell continues to build momentum toward global expansion while striving to offer hope to cancer patients around the world.