Lion TCR Announces FDA Approval of IND Application for Lead Product LioCyx-M004 to Initiate Global Phase 1b/2 Trial in HBV-Related Hepatocellular Carcinoma

SINGAPORE, GUANGZHOU, CHINA and LOS ANGELES, Sept. 23, 2021 /PRNewswire/ -- Lion TCR Pte Ltd, a clinical-stage biotechnology company focused on novel TCR T-cell immunotherapy, is pleased to announce that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application for its lead product candidate, LioCyx-M004, to conduct a Phase 1b/2 international multicenter clinical trial (trial name: LIBERO).

This new drug clinical trial approval authorizes Guangzhou Laien Biological Medicine Co., Ltd. to initiate an Investigational New Drug (IND) clinical trial for its flagship product, LioCyx-M004, for the treatment of advanced hepatocellular carcinoma (HCC) associated with primary HBV infection. This marks the world's first clinical study utilizing a personalized TCR-T therapy for the treatment of HBV-related hepatocellular carcinoma.

In 2020, there were over 900,000 new cases of liver cancer globally, of which 90% were hepatocellular carcinoma (HCC). In Asia, hepatitis B virus (HBV) is a major etiological factor for HCC; in China, for example, 80%–90% of HCC cases are associated with HBV infection. Therefore, targeting HBV-associated antigens represents a highly promising therapeutic strategy. LioCyx-M004 is an autologous T-cell product that utilizes mRNA transfection to express T-cell receptors (TCRs) targeting HBV antigens, thereby endowing the T cells with the capacity to specifically recognize and eliminate HBV-associated HCC tumor cells. By effectively leveraging the transient expression profile of mRNA in combination with a multiple-infusion regimen, this approach effectively controls potential toxicity while ensuring therapeutic efficacy.

In the completed Phase I clinical trial, LioCyx-M004 demonstrated a favorable safety profile, with no occurrences of cytokine release syndrome (CRS) or neurotoxicity. Tumor responses were observed, with a duration of response lasting 27.7 months, and the median overall survival was significantly prolonged to 33.1 months. These findings provide a strong rationale for further exploring the efficacy of this cell therapy product. In the newly approved IND clinical study, LioCyx-M004 will primarily be evaluated as a monotherapy to further assess its efficacy, while the safety and efficacy of LioCyx-M004 in combination with lenvatinib will also be evaluated. As a standard first-line treatment for advanced hepatocellular carcinoma, lenvatinib has the potential to modulate the tumor immune microenvironment. The combination with LioCyx-M004 will yield complementary advantages, thereby further enhancing therapeutic efficacy.

Dr. Wang Tingting, Chief Operating Officer and Chief Medical Officer of Laien Bio, commented: “With its unique mechanism of action, favorable safety profile, and antitumor efficacy, LioCyx-M004 holds promise to become a first-in-class TCR-T cell therapy for the treatment of HCC. Robust scientific rationale further supports combining our TCR-T therapy with other modalities to further enhance efficacy and improve the prognosis of HCC patients. We are proud of the significant progress the company has achieved in recent years and look forward to rapidly initiating this clinical trial in the coming weeks to benefit patients with HCC. Patient enrollment will commence at City of Hope Comprehensive Cancer Center in the United States, a globally leading institution for cancer research and treatment. Additional clinical sites in China and Singapore are also undergoing concurrent regulatory review.”

Dr. Peng Xiaoming, Managing Director and Chief Executive Officer of Laien Biotech, stated: “The FDA’s approval of the Investigational New Drug (IND) application for clinical trials of Laien Biotech’s novel drug LioCyx-M004 further underscores its significance as an innovative therapy for liver cancer. Securing this IND approval marks a major milestone for Laien Biotech. At this important moment, I would like to express my gratitude to everyone who has supported and followed the development of Laien Biotech! Laien Biotech will continue its commitment to becoming a globally leading TCR-T cell therapy company, developing our core immunotherapy technology platforms, which encompass a TCR discovery platform for TCR-T therapies, autologous and allogeneic cell platforms, and gene editing technology platforms. By leveraging innovative TCR-T cell immunotherapy to benefit liver cancer patients, we aim to establish ourselves as a benchmark enterprise in the liver cancer treatment sector.”