Lion TCR Achieves Triple FDA Milestones with IND Clearance for Chronic Hepatitis B Following Earlier Fast Track and Orphan Drug Designations
SINGAPORE and GUANGZHOU, China, Sept. 15, 2025 /PRNewswire/ -- Lion TCR, a clinical-stage biotechnology company pioneering T-cell receptor (TCR)-based therapies, today announced that it has received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) to initiate phase1b/2 clinical trials for its proprietary TCR-T cell therapy, LioCyx-M004, in patients with chronic hepatitis B (CHB). This approval represents the third major regulatory milestone for the same product candidate, which previously received both Fast Track and Orphan Drug Designations for the treatment of hepatitis B virus-related hepatocellular carcinoma (HBV-HCC).
The IND clearance authorizes Lion TCR to commence clinical studies of LioCyx-M004 in CHB patients – a global population exceeding 290 million – and marks the first TCR-T therapy to enter clinical development for chronic hepatitis B.
"This triple recognition from the FDA underscores the transformative potential of LioCyx-M004 and validates our strategic approach targeting HBV-associated diseases through TCR-T cell therapy," said Dr. Xiaoming Peng, CEO of Lion TCR. "Having demonstrated promising anti-tumor and antiviral activity in liver cancer, we are now well-positioned to evaluate its impact on the underlying viral infection that drives cancer development."
The company's earlier regulatory achievements include Orphan Drug designation, which provides incentives for therapies targeting rare diseases such as HBV-HCC in the United States, and Fast Track status, designed to accelerate the development and review of treatments for serious conditions addressing unmet medical needs.
LioCyx-M004 represents an innovative autologous cell therapy engineered using mRNA to encode T-cell receptors that specifically target hepatitis B virus antigens. Preclinical and early clinical studies have demonstrated the therapy's ability to reduce viral antigen load and promote T-cell-mediated clearance of infected cells, while maintaining a favorable safety profile to date.
Dr. Tina Wang, Chief Medical Officer of Lion TCR, added, "Our ability to successfully redirect our lead candidate from oncology to virology demonstrates the remarkable versatility and broad therapeutic potential of our platform. We look forward to initiating the CHB trial and exploring its potential as a functional cure for this chronic infection."
The company's mRNA-encoded in vivo TCR-T therapy targeting HBV has gained validation from a prominent U.S. cancer research institute. Studies have demonstrated significant efficacy in eliminating HBV-positive cells in animal models, with therapeutic effects comparable to ex vivo engineered T cells. This validation provides a robust scientific foundation for the industrialization of in vivo TCR-T therapy targeting HBV.
Lion TCR is actively advancing this innovative approach, accelerating the development of in vivo TCR-T therapy. Compared to traditional ex vivo T cell therapies, the in vivo platform offers distinct advantages including shorter production cycles, reduced costs, and enhanced accessibility, potentially expanding treatment opportunities for patients worldwide.
Additionally, the company is enhancing its AI-powered TCR discovery platform with plans to expand its product pipeline to address additional solid tumor indications, including lung cancer, breast cancer, and gastrointestinal cancers.
About Lion TCR
Lion TCR is a clinical-stage biotechnology company that originated from the Agency for Science, Technology and Research (A*STAR) in Singapore. The company's lead product targets hepatitis B virus (HBV)-related diseases and represents the world's first HBV-specific TCR-T cell therapy to receive U.S. FDA approval for an international multi-center Phase II clinical trial. It has been granted FDA Fast Track designation and Orphan Drug status, in addition to obtaining approval for clinical trials from China's National Medical Products Administration (NMPA).
Utilizing an AI-powered TCR discovery platform and advanced RNA technology, Lion TCR is expanding its pipeline to address various solid tumors as well as infectious diseases. The company's diversified portfolio includes both autologous cell therapies and off-the-shelf treatments, with a dedicated focus on developing in vivo TCR-T therapies aimed at significantly reducing costs and improving treatment accessibility.
SOURCE Lion TCR
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