FDA Grants Fast Track Designation to LioCyx-M004 for HBV-related Hepatocellular Carcinoma

FDA fast track designation comes based on overall survival improvements from a phase 1 trial investigating LioCyx-M004 infusions in primary HBV-related hepatocellular carcinoma.

The FDA granted Fast Track Designation to a potential first-in-class therapy, LioCyx-M004, for the treatment of patients with Hepatitis B virus (HBV)–related hepatocellular carcinoma (HCC), according to a press release from the company responsible for the drug, Lion TCR Pte Ltd.1

The designation is a result of efficacy data, specifically focusing on overall survival (OS), related to LioCyx-M004 use in patients with Hepatitis B surface antigen (HBsAg)–positive HCC who relapsed or were refractory to prior systemic treatment.

“HBV-related HCC occurs in over 420,000 people every year worldwide and majority of advanced HCC patients relapse quickly after initial treatment. However, existing treatments are very limited especially on improving overall survival,” Tina Tingting Wang, COO and CMO of Lion TCR, said in a press release. “We believe that our innovative TCR-T therapy can fill this urgent and important unmet medical needs. With this Fast Track designation, we look forward to having more frequent communication with the Agency in the hope to attain a more expedited drug approval for our product for patient access.”

In previous research, a phase 1 study (NCT03634683) investigated LioCyx-M004 infusions in primary HBV-related HCC. There were no occurrences of cytokine release syndrome (CRS) among patients who received the therapy, as well as no signs of neurotoxicity. The disease control rate was 60% and duration of response was 27.7 months. Median OS in that population was 33.1 months.2

Clearance was obtained for a future phase 1b/2 multicenter study evaluating the safety and efficacy of LioCyx-M004 as a monotherapy and in combination with lenvatinib (Lenvima) for patients with HBV-related HCC.

“The field of T-cell therapy is highly dynamic and competitive. Innovative therapies are released faster than ever through expediated programs like Fast Track. Together with our Orphan Drug Designation obtained for the use of HBV-specific TCR T-cell therapy in HCC, we believe this Fast Track approval can drive forward the accelerated regulatory approval our proprietary first-in-class TCR T cell therapy.” Peng Xiaoming, CEO of Lion TCR, said in a press release.

Fast Track Designation is designed to grant accelerated access to approval for agents that can address potentially life-threatening conditions. The designation focuses on agents that can address unmet medical needs, expediting the approval process through early and frequent meetings with the FDA.


  1. Lion TCR Receives FDA Fast Track Designation for its HBV-specific TCR T Cell Therapy for Hepatocellular Carcinoma. News release. Lion TCR. December 22, 2021. Accessed December 23, 2021. https://tinyurl.com/yep3f9je
  2. Lion TCR Announces FDA IND Clearance for its LioCyx-M004 for Hepatocellular Carcinoma. News release. Lion TCR. September 23, 2021. Accessed December 23, 2021. https://tinyurl.com/2p9cpcev