Lion TCR Pte Ltd today announced that it has received Fast Track Designation from United States Food and Drug Administration (U.S. FDA) for LioCyx-M004, autologous T-cells transfected with mRNA encoding Hepatitis B surface antigen (HBsAg) specific TCR, for the treatment of HBV-related hepatocellular carcinoma (HBV-related HCC). This Fast Track Designation provides Lion TCR with an expedited path towards the regulatory approval for its leading investigational product, LioCyx-M004, which is being developed as a potential first-in-class drug for HBV-related HCC. This designation was granted based on that the efficacy of LioCyx-M004, as demonstrated by an improvement in the overall survival in patients with HBsAg-positive HCC relapsed or refractory to prior systemic treatment.

The FDA Fast Track Designation is designed for sponsors to gain access to expedited drug approval, via eligibility for Accelerated Approval and Priority Review, for medical conditions that are serious and potentially life-threatening, and where there is an unmet medical need through early and frequent meetings with the FDA to discuss drug development plans^[1]. The purpose is to get important new drugs to the patient earlier.

In earlier phase 1 study, LioCyx-M004 has showed the well-tolerated safety profile and promising prolonged overall survival. In September 2021, FDA Investigational New Drug (IND) Clearance for a Phase 1b/2 multi-center study has been obtained^[2]. This is the first ongoing clinical trial that uses HBV-specific TCR T cell therapy to target HBV-related HCC.

"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. 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. Efforts for patient recruitment for the Phase 1b/2 study in the U.S. and Asia are underway." said Dr Tina Tingting Wang, COO and CMO of Lion TCR.

"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." said Dr Peng Xiaoming, CEO of Lion TCR.