The U.S. Food and Drug Administration (FDA) has granted Tvardi's anti-cancer drug TTI-101 Fast Track designation for the treatment of unresectable liver cancer. TTI-101, an orally available STAT3 inhibitor, was granted Orphan Drug Designation last year for the treatment of hepatocellular carcinoma and idiopathic pulmonary fibrosis, and is currently in Phase 1 clinical trials in patients with solid tumors.

STAT3 is a transcription factor that is involved in cancer cell growth, angiogenesis, and metastasis, and is known to be activated in approximately 70% of all cancers. In HCC, STAT3 is highly expressed, making it a novel therapeutic target, and a new drug has been developed that can effectively inhibit it. TTI-101 is the first oral STAT3 inhibitor to be developed, and has demonstrated excellent antitumor effects in mice. Based on this, a clinical trial was planned to confirm its safety and therapeutic effectiveness when administered to humans.

Patients with solid tumors for which there are no more available treatments are currently enrolled in the TTI-101 clinical study, and it is expected that the study will prove its safety and therapeutic effectiveness so that it can be approved as a new liver cancer treatment.