The Food and Drug Administration (FDA) has granted Orphan Drug designation to TTI-101, an orally delivered, small molecule, STAT3 (signal transducer and activator of transcription 3) inhibitor, for the treatment of hepatocellular carcinoma (HCC).

Increased activity of the STAT3 protein has been shown to contribute to the development and growth of cancer. Direct inhibition of STAT3 signaling by TTI-101 is expected to lead to reduced tumor growth. In early studies, the investigational treatment was found to be well tolerated and demonstrated clinical activity across a broad range of tumors.

The designation for HCC was based on safety and efficacy data from an ongoing phase 1 study ( Identifier: NCT03195699), which included patients with histologically confirmed, locally advanced, inoperable, unresectable HCC who had failed prior therapies.

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“We are pleased to receive Orphan Drug designation for TTI-101 in HCC from the FDA,” said Imran Alibhai, PhD, CEO of Tvardi, “We look forward to the impending initiation of our phase 2 trial in HCC to test TTI-101 as monotherapy as well as in combination.”


Tvardi Therapeutics’ TTI-101 receives Orphan Drug designation for hepatocellular carcinoma. News release. April 21, 2022.

This article originally appeared on MPR