Immuno-Oncology Resource Center

Immuno-Oncology Resource Center


Press Release

January 04, 2022
2 min read

FDA grants fast track designation to TCR T-cell therapy for hepatocellular carcinoma


Press Release

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

We were unable to process your request. Please try again later. If you continue to have this issue please contact

The FDA granted fast track designation to LioCyx-M004, a T-cell receptor T-cell therapy for treatment of adults with hepatitis B virus-related hepatocellular carcinoma.

LioCyx-M004 (Lion TCR) is a genetically modified autologous cell therapy derived from T cells that have been transfected with mRNA encoding to express a T-cell receptor (TCR) that recognizes the hepatitis B surface antigen on the surface of HBV-related cancer cells.

Photomicrograph of hepatocellular carcinoma (hepatoma), a malignant tumor often associated with chronic hepatitis
Source: Adobe Stock.

The FDA based the fast track designation on results of a phase 1, single-center study of patients with relapsed or refractory HBV-related HCC who underwent hepatectomy or radiofrequency ablation.

Results showed a disease control rate of 60%, median duration of response of 27.7 months and median OS of 33.1 months. No cases of cytokine release syndrome or neurotoxicity occurred, according to manufacturer-provided data.

The FDA previously granted orphan drug status to LioCyx-M004 for treatment of adults with HBV-related HCC.

“HBV-related HCC occurs in over 420,000 people every year worldwide, and the majority of [patients with] advanced HCC relapse quickly after initial treatment; however, existing treatments are very limited, especially for improving overall survival,” Tina Tingting Wang, MD, PhD, chief operating officer and chief medical officer of Lion TCR, said in a company-issued press release.

“We believe that our innovative [TCR T-cell] therapy can fill this urgent and important unmet medical need,” Wang added. “With this fast track designation, we look forward to having more frequent communication with the [FDA] in the hope to attain expedited drug approval for our product for patient access.”

The FDA previously provided clearance of Lion TCR’s investigational new drug application in September 2021.

Enrollment is underway for an international, multicenter phase 1b/phase 2 study that will assess the safety and efficacy of LioCyx-M004 as monotherapy or in combination with lenvatinib (Lenvima, Eisai) —a multiple receptor tyrosine kinase inhibitor — for the treatment of relapsed or refractory HBV-related HCC.

The study will be the first to use an HBV-specific TCR T-cell therapy for the treatment HBV-related HCC, according to the manufacturer.


Lion TCR announces FDA clearance of its IND application for its lead investigational product, LioCyx-M004, for hepatocellular carcinoma. Published Sept. 23, 2021. Accessed Jan. 3, 2022.