LioCyx-M004 for HBV-Related Hepatocellular Carcinoma Earns Fast Track Designation


According to Healio, the U.S. Food and Drug Administration (FDA) recently granted Fast Track designation to LioCyx-M004. This therapy, developed by T-cell immunotherapy company Lion TCR, is designed to treat patients with advanced hepatitis B (HBV)-associated hepatocellular carcinoma.

Hepatocellular Carcinoma

Although it is a rare cancer, hepatocellular carcinoma is also the most common form of primary liver cancer. This means that the cancer originates in the liver as opposed to metastasizing from other organs. While doctors do not know the exact cause of hepatocellular carcinoma, the cancer more often manifests in those with pre-existing or long-term liver diseases. Having hepatitis B or C, being obese, or consuming large amounts of alcohol also increase the risk. This cancer may not prompt symptoms in early stages. However, as hepatocellular carcinoma progresses, symptoms can include:

  • Unintended weight loss
  • Appetite loss
  • Jaundice (yellowing of the skin, eyes, and mucous membranes)
  • Abdominal bloating or inflammation
  • Fatigue or general malaise
  • Nausea and vomiting
  • Dark urine
  • Pale, chalky stool
  • Upper right abdominal pain, lumps, or feeling of fullness

Learn more about hepatocellular carcinoma here.

About LioCyx-M004

In a prior press release, Lion TCR described LioCyx-M004 as:

autologous T cells modified to express HBV-specific TCR. These modified T cells have been shown to be able to lyse HBV-infected HCC cells upon target recognition in in-vivo pre-clinical studies and in patients.

The treatment may help to promote immune-protective properties. LioCyx-M004 is given via infusions. In earlier studies, LioCyx-M004 was shown to be relatively safe and well-tolerated. It also helped patients with better disease control and management, and showed a sustained response. LioCyx-M004 earned Orphan Drug designation within this realm as well.

Currently, Lion TCR is enrolling patients in a Phase 1b/2 clinical trial to evaluate LioCyx-M004 in conjunction with lenvatinib for patients with HBV-related hepatocellular carcinoma. If this therapy is found to be safe, effective, and well-tolerated, it could greatly fulfill an unmet treatment need for patients.

Fast Track designation aims to expedite the review and development of therapies intended to fill an unmet need for serious conditions. This designation offers a variety of potential benefits, including:

  • Rolling Review for Biologic License Applications (BLAs) or New Drug Applications (NDAs)
  • More frequent meetings with and increased assistance from the FDA
  • Accelerated Approval and Priority Review eligibility

To learn more about Fast Track designation, check out this helpful guide from the FDA.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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